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Access to High-Cost Medicines in Europe
Abstract
This chapter deals with challenges and possible solutions to ensure affordable patient access to high-cost medicines in Europe. In recent years, even high-income countries have been struggling to provide affordable access to new, high-cost medicines. Less affluent countries and small markets have been suffering from delayed launch and, in some cases, non-availability of these medicines. Prices are high and variable across European countries, and the differences do not necessarily reflect the economic situation of the countries. Payers in several European countries concluded confidential managed-entry agreements with the pharmaceutical companies. Authorities of different countries have established voluntary cooperation projects to gain improved access to information and to strengthen their purchasing power. In addition, there appears to be a trend towards evidence generation (e.g. through increased use of health technology assessment and pharmacoeconomic evaluations) and policies to be better prepared (horizon scanning). Some countries have developed appropriate policies to benefit from these medicines.
... However, their uptake in the current healthcare management model would be difficult due to their cost and complex development [8,9]. The growth in spending on diagnostics and therapies is driven by an increase in the volume of high-cost drugs (HCDs) in different countries [6,[10][11][12], including Spain [13]. Optimized management of these high-impact technologies would contribute to their full implementation and cost reduction. ...
... It focuses on the value of medicines, ensuring that they are clinically practical and cost effective, and reducing medicine wastage. In this regard, pharmaceutical spending on HCDs is a concern in the different national health systems [11,12]. There is no agreed definition of high-impact medicines (HIM) or HCDs. ...
... Several countries are concerned about sustainability and optimizing the use of these HCDs. Different strategies have been implemented, such as voluntary collaboration projects to obtain better access to information on these HCDs and centralized purchasing to optimize budgetary resources and increase access to the public health system [11,12,26]. However, our study is the first to quantitatively evaluate the effectiveness of online web tools in reducing waste and costs of perishable HCDs in five hospitals in the AHPS. ...
IntroductionDue to their impact on healthcare systems, the sustainability and optimization of high-cost drugs is an issue of concern for several countries. Different strategies have been implemented such as centralized purchasing to optimize budgetary resources. However, there is still a need for a mechanism to optimize these drugs further.Methods
We conducted this prospective multicenter intervention study in five hospitals in the Andalusian Public Health System of Cádiz (Spain) between July 2019 and September 2021. We developed an online website (Farmastock) and implemented it to determine the availability of high-cost, low-use, and near-expiry medicines in each hospital. We used a simple analysis using operational variables to assess the project intervention's savings impact on managing these high-cost drugs.ResultsThe implementation of Farmastock in Cádiz resulted in savings of 675,757.52 € for the Andalusian Public Health System, with 238 medicines transferred out of the 373 available. Of these medicines offered, the most considerable percentage were medicines used for pathologies with high clinical instability and accounted for nearly 80% of the medicines optimized by the tool.Conclusions
Farmastock allowed the Andalusian Public Health System to make substantial financial savings by not making new purchases of high-cost drugs available in other centers of this health network that were not being used. Therefore, this tool is a very efficient measure to contribute to the sustainability of the APHS and could be implemented in more hospitals soon.
... In Western European countries, there is a complex combination of the private and public sectors both in health financing and in health care deliv- 14,0 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 (European Commission, 2014;Vogler, 2018). It is the budget financing model (the Beveridge Model), according to which the public sector accounts for the majority of the cost of maintaining health care facilities. ...
In Ukraine, there is a steady increase in the mortality rate of the population, which is due to the low quality of medical provision. The main advantages and the efficiency factor of the health care system in the Western European countries are analyzed; the key factors regulating medical provision in Ukraine are systematized; the effectiveness of modern health care reform in Ukraine is evaluated; practical recommendations to improve the administrative and legal regulation of the system are developed. The analysis showed that the effectiveness of health care reform in Ukraine is currently about 30%, and the high mortality rate of the population in Ukraine is primarily due to the low quality of medical care and inadequate investment in the industry. The main health problems in Ukraine are failure to comply with legislation on the amount of funding and remuneration in the public health system, high levels of corruption and lack of control over the competence of doctors, inaccessibility of medical products for consumers due to the pharmaceutical market monopolization.
Resumen En la seguridad social en Centroamérica, Panamá y República Dominicana existe poca información que indique los requisitos y el procedimiento correcto que deben de seguir los oferentes para participar en los procesos de licitación. Entre dicha información, resulta más escasa aquella que se relaciona con la compra de medicamentos en el ámbito institucional. El objetivo de la presente investigación fue desarrollar una guía para la elaboración de dosieres para la oferta de medicamentos biológicos y biotecnológicos en las instituciones de seguridad social de Guatemala, El Salvador, Honduras, Costa Rica, Panamá, República Dominicana y el Consejo de Ministros de Salud de Centroamérica. Para ello, se recopilaron las normativas vigentes para el proceso de adquisición institucional de medicamentos biológicos y biotecnológicos de los
This report was produced as part of a series of six country case studies and forms part of a larger study on the impact of the financial crisis since 2008–2009 on health systems in the European Region. The countries studied in depth are Estonia, Greece, Ireland, Latvia, Lithuania and Portugal, which represent a selection of countries hit relatively hard by the global financial and economic crisis. In-depth analysis of individual countries, led by authors from the country concerned, adds to understanding of both the impact of a deteriorating fiscal position and the policy measures put in place as a result. These case studies complement a broader analysis which summarizes official data sources and the results of a survey of key informants in countries of the WHO European Region; they will also be published as part of a two volume study conducted jointly by the European Observatory on Health Systems and Policies and the WHO Regional Office for Europe.
This paper aims at investigating (i) differences and commonalities of drugs Price and Reimbursement (P&R) criteria between Italy and the other major EU countries and (ii) comparative advantages, pitfalls and possible reforms of the Italian model. In the main EU countries P&R is converging towards a contractual model where therapeutic added value and economic impact are playing a crucial role. However there are still important differences across countries in the way added value is measured, the comparators are chosen and the economic impact is integrated with clinical evidence. Compared to the other systems the main advantages of the Italian model are (i) the explicit adoption of a multiple criteria approach, which has allowed a higher flexibility in P&R negotiation, and (ii) that managed market entry contracts have been often used to manage uncertainty at market launch. However, the assessment process is not transparent, innovativeness and premium price have never been explicitly integrated and price negotiation is strongly influenced by a budget silos approach. The former two pitfalls can be faced in the short-term, whereas giving up spending caps on drugs would require a longer-term political consensus.
Background
To build capacity in medicines management, the Uganda Ministry of Health introduced a nationwide supervision, performance assessment and recognition strategy (SPARS) in 2012. Medicines management supervisors (MMS) assess performance using 25 indicators to identify problems, focus supervision, and monitor improvement in medicines stock and storage management, ordering and reporting, and prescribing and dispensing. Although the indicators are well-recognized and used internationally, little was known about the reliability of these indicators. An initial assessment of inter-rater reliability (IRR), which measures agreement among raters (i.e., MMS), showed poor IRR; subsequently, we implemented efforts to improve IRR. The aim of this study was to assess IRR for SPARS indicators at two subsequent time points to determine whether IRR increased following efforts to improve reproducibility.
Methods
IRR was assessed in 2011 and again after efforts to improve IRR in 2012 and 2013. Efforts included targeted training, providing detailed guidelines and job aids, and refining indicator definitions and response categories. In the assessments, teams of three MMS measured 24 SPARS indicators in 26 facilities. We calculated IRR as a team agreement score (i.e., percent of the MMS teams in which all three MMS had the same score). Two sample tests for proportions were used to compare IRR scores for each indicator, domain, and overall for the initial assessment and the following two assessments. We also compared the IRR scores for indicators classified as simple (binary) versus complex (multi-component). Logistic regression was used to identify supervisor group characteristics associated with domain-specific and overall IRR scores.
Results
Initially only five (21%) indicators had acceptable reproducibility, defined as an IRR score ≥ 75%. At the initial assessment, prescribing quality indicators had the lowest and stock management indicators had the highest IRR. By the third IRR assessment, 12 (50%) indicators had acceptable reproducibility, and the overall IRR score improved from 57% to 72%. The IRR of simple indicators was consistently higher than that of complex indicators in the three assessment periods. We found no correlation between IRR scores and MMS experience or professional background.
Conclusions
Assessments of indicator reproducibility are needed to improve IRR. Using simple indicators is recommended.
Electronic supplementary material
The online version of this article (10.1186/s40545-018-0137-y) contains supplementary material, which is available to authorized users.
In the context of pharmaceutical care, policy-makers repeatedly face the challenge of balancing patient access to effective medicines with affordability and rising costs. With the aim of guiding the health policy discourse towards questions that are important to actual and potential patients, this study investigates a broad range of regulatory measures, spanning marketing authorization to generic substitution and resulting price levels in a sample of 16 European health systems (Austria, Belgium, Denmark, England, Finland, France, Germany, Greece, Ireland, Italy, the Netherlands, Poland, Portugal, Scotland, Spain and Sweden). All countries employ a mix of regulatory mechanisms to contain pharmaceutical expenditure and ensure quality and efficiency in pharmaceutical care, albeit with varying configurations and rigour. This variation also influences the extent of publicly financed pharmaceutical costs. Overall, observed differences in pharmaceutical expenditure should be interpreted in conjunction with the differing volume and composition of consumption and price levels, as well as dispensation practices and their impact on measurement of pharmaceutical costs. No definitive evidence has yet been produced on the effects of different cost-containment measures on patient outcomes. Depending on the foremost policy concerns in each country, different levers will have to be used to enable the delivery of appropriate care at affordable prices. World Health Organization 2016 (acting as the host organization for, and secretariat of, the European Observatory on Health Systems and Policies).
Introduction
Improving health systems performance, especially in low-resource settings facing complex disease burdens, can improve population health. Specifically, the efficiency and effectiveness of supply chains and procurement processes for pharmaceuticals, vaccines and other health products has important implications for health system performance. Pharmaceuticals, vaccines and other health products make up a large share of total health expenditure in low-income and middle-income countries (LMICs), and they are critical for delivering health services. Therefore, programmes which achieve cost savings on these expenditures may help improve a health system's efficiency, whereas programmes that increase availability of health products may improve a health system's effectiveness. This systematic review investigates whether changes to supply chains and procurement processes can achieve cost savings and/or improve the availability of drugs in LMICs.
Methods
Using the PRISMA guidelines for systematic reviews, we searched PubMed, Embase, CINAHL and the Health Economic Evaluation Database to identify.
Results
We identified 1264 articles, of which 38 were included in our study. We found evidence that centralised procurement and tendering can achieve direct cost savings, while supply chain management programmes can reduce drug stock outs and increase drug availability for populations.
Conclusions
This research identifies a broad set of programmes which can improve the ways that health systems purchase and delivery health products. On the basis of this evidence, policymakers and programme managers should examine the root causes of inefficiencies in pharmaceutical supply chain and procurement processes in order to determine how best to improve health systems performance in their specific contexts.
Affordable patient access to medicines, particularly to new premium-priced medicines, is an issue, even in high-income countries. The pharmaceutical industry has argued in favour of price discrimination amongst countries based on confidential discounts as a solution to ensuring affordability and availability in less-resourced countries. In this editorial, we explain why we disagree, and we elaborate on why price transparency can contribute to affordable patient access to medicines.
Human epidermal growth factor receptor 2-positive (HER2+) breast cancer comprises approximately 15%–20% of all breast cancers and is associated with a poor prognosis. The introduction of anti-HER2 therapy has significantly improved clinical outcomes for patients with HER2+ breast cancer, and multiple HER2-directed agents (ie, trastuzumab, pertuzumab, lapatinib, and ado-trastuzumab emtansine [T-DM1]) are approved for clinical use in various settings. The treatment landscape for patients with HER2+ breast cancer is continuing to evolve. While novel agents and therapeutic strategies are emerging, biologic therapies, particularly trastuzumab, are likely to remain a mainstay of treatment. However, access issues create barriers to the use of biologics, and there is evidence for underuse of trastuzumab worldwide. A biosimilar is a biologic product that is highly similar to a licensed biologic in terms of product safety and effectiveness. Biosimilars of trastuzumab are in development and may soon become available. The introduction of biosimilars may improve access to anti-HER2 therapies by providing additional treatment options and lower-cost alternatives. Because HER2-targeted drugs may be administered for extended periods of time and in combination with other systemic therapies, biosimilars have the potential to result in significant savings for healthcare systems. Herein we review current and emerging treatment options for, and discuss the possible role of biosimilars in, treating patients with HER2+ breast cancer.
Biobetters are new drugs designed from existing peptide or protein-based therapeutics by improving their properties such as affinity and selectivity for the target epitope, and stability against degradation. Computational methods can play a key role in such design problems—by predicting the changes that are most likely to succeed, they can drastically reduce the number of experiments to be performed. Here we discuss the computational and experimental methods commonly used in drug design problems, focusing on the inverse relationship between the two, namely, the more accurate the computational predictions means the less experimental effort is needed for testing. Examples discussed include efforts to design selective analogs from toxin peptides targeting ion channels for treatment of autoimmune diseases and monoclonal antibodies which are the fastest growing class of therapeutic agents particularly for cancers and autoimmune diseases.
This article discusses pharmaceutical pricing and reimbursement policies in European countries with regard to their ability to ensure affordable access to medicines. A frequently applied pricing policy is external price referencing. While it provides some benchmark for policy-makers and has been shown to be able to generate savings, it may also contribute to delay in product launch in countries where medicine prices are low. Value-based pricing has been proposed as a policy that promotes access while rewarding useful innovation; however, implementing it has proven quite challenging. For high-priced medicines, managed-entry agreements are increasingly used. These agreements allow policy-makers to manage uncertainty and obtain lower prices. They can also facilitate earlier market access in case of limited evidence about added therapeutic value of the medicine. However, these agreements raise transparency concerns due to the confidentiality clause. Tendering as used in the hospital and offpatent outpatient sectors has been proven to reduce medicine prices but it requires a robust framework and appropriate design with clear strategic goals in order to prevent shortages. These pricing and reimbursement policies are supplemented by the widespread use of Health Technology Assessment to inform decision-making, and by strategies to improve the uptake of generics, and also biosimilars. While European countries have been implementing a set of policy options, there is a lack of thorough impact assessments of several pricing and reimbursement policies on affordable access. Increased cooperation between authorities, experience sharing and improving transparency on price information, including the disclosure of confidential discounts, are opportunities to address current challenges.
Objective
Zoledronic acid and denosumab were funded by the Australian government for the management of osteoporosis at an equivalent price to alendronate. The price of alendronate has declined by around 65 %, but the price of the other two therapies has remained stable. Using data published since the listing, this paper reports current estimates of the value of denosumab compared to alendronate from an Australian health system perspective. MethodsA cohort-based state transition model was developed that predicted changes in bone mineral density (BMD), and calibrated fracture probabilities as a function of BMD, age and previous fracture to estimate differences in costs and QALYs gained over a 10-year time horizon. ResultsThe base-case incremental cost per QALY gained for denosumab versus alendronate was 100,000 per QALY gained. If the price of denosumab was reduced by 50 %, the incremental cost per QALY gained falls to $50,068. DiscussionCurrent Australian legislation precludes price reviews when comparator therapies come off patent. The presented analysis illustrates a review process, incorporating clinical data collected since the original submission to inform a price at which denosumab would provide value for money.
Background:
In recent years, high-cost medicines have increasingly been challenging the public health budget in all countries including high-income economies. In this context, this study aims to survey, analyze and compare prices of medicines that likely contribute to high expenditure for the public payers in high-income countries.
Methods:
We chose the following 16 European countries: Austria, Belgium, Denmark, Finland, France, Germany, Greece, Hungary, Ireland, Italy, the Netherlands, Portugal, Sweden, Slovakia, Spain and United Kingdom. The ex-factory price data of 30 medicines in these countries were collected in national databases accessible through the Pharmaceutical Price Information (PPI) service of Gesundheit Österreich GmbH (Austrian Public Health Institute).
Results:
The ex-factory prices (median) per unit (e.g. per tablet, vial) ranged from 10.67 cent (levodopa + decarboxylase inhibitor) to 17,000 euro (ipilimumab). A total of 53% of the medicines surveyed had a unit ex-factory price (median) above 200 Euro. For two thirds of the medicines, price differences between the highest-priced country and lowest-priced country ranged between 25 and 100%; the remaining medicines, mainly low-priced medicines, had higher price differential, up to 251%. Medicines with unit prices of a few euros or less were medicines for the treatment of diseases in the nervous system (anti-depressants, medicines to treat Parkinson and for the management of neuropathic pain), of obstructive airway diseases and cardio-vascular medicines (lipid modifying agents). High-priced medicines were particularly cancer medicines.
Conclusion:
Medicine prices of Greece, Hungary, Slovakia and UK were frequently at the lower end, German and Swedish, as well as Danish and Irish prices at the upper end. For high-priced medicines, actual paid prices are likely to be lower due to confidential discounts and similar funding arrangements between industry and public payers. Pricing authorities refer to the higher undiscounted prices when they use price data from other countries for their pricing decisions.
Millions of people around the world do not have access to the
medicines they need to treat disease or alleviate suffering.
Strict patent regimes interfere with widespread access to
medicines by creating monopolies that maintain medicines
prices well beyond the reach of those who need them.
The magnitude of the AIDS crisis in the late nineties brought this to
the public’s attention when millions of people in developing countries
died from an illness for which medicines existed, but were not available
or affordable. Faced with an unprecedented health crisis — 8,000 people
dying daily — the public health community launched an unprecedented
global effort that eventually resulted in the large-scale availability of
quality generic HIV medicines and a steady scale-up in access to those
medicines. This has allowed nearly 13 million people to lead longer,
healthier lives. However, trends in international intellectual property law
could impact many of the policy tools used to scale up HIV treatment.
Developments in global health and specifically access to medicines
policies are now at an important juncture. Impressive progress has been
made in access to medicines for HIV and many lessons can be learned
from that experience. But it is important to examine whether those
lessons can be applied for other diseases. Today’s pharmaceutical patent
regime affects almost all medicines developed since 1995 in most
INTRODUCTION
2
INTRODUCTION
countries. The high prices of new medicines, such as for cancer,
tuberculosis and hepatitis C, cause huge access challenges globally, in
both developed and developing countries. These new global challenges
pose the question of whether the public health approaches to medicines
patents developed in response to the HIV/AIDS crisis are exclusive to HIV
or whether they can be applied more broadly.
This book provides a history of the parallel developments in global
public health and international patent laws: detailing the current
situation, how we got here, and how we can move forward to best protect
the future of medical innovation as well as the lives that will depend on it.
This book is an update of an earlier account that was published in 2009:
The Politics of Pharmaceutical Monopoly Power: Drug Patents, Access, Innovation and
the Application of the WTO Doha Declaration on TRIPS and Public Health.
Background
The management of cancer is predicated on the availability and affordability of anticancer therapies, which may be either curative or noncurative.
Aim
The primary aims of the study were to evaluate (i) the formulary availability of licensed antineoplastic medicines across Europe; (ii) patient out-of-pocket costs for the medications and (iii) the actual availability of the medication for a patient with a valid prescription.
Materials and methods
The survey tool was based on the previous ESMO studies that addressed the availability and accessibility of opioids for the management of cancer pain. A total of 185 field reporters from 49 countries were invited to participate. The preliminary set of data was posted on the ESMO website for open peer-review, and amendments have been incorporated into the final report.
Results
There are substantial differences in the formulary availability, out-of-pocket costs and actual availability for many anticancer medicines. The most profound lack of availability is in countries with lower levels of economic development, particularly in Eastern Europe, and these are largely related to the cost of targeted agents approved in the last 10 years. Discrepancies are less profound among medications on the WHO model essential medicines list (EML) for cancer and in curative settings. However, medicine shortages also affect WHO EML medicines, with relevant therapeutic implications for many patients.
Conclusions
The cost and affordability of anticancer treatments with recent market approval is the major factor contributing to inequity of access to anticancer medications. This is especially true with regards to new medications used in the management of EGFR- or ALK-mutated non-small-cell lung cancer, metastatic melanoma, metastatic renal cell cancer, RAS/RAF wild-type metastatic colorectal cancer, HER2 overexpressed breast cancer and castration-resistant metastatic prostate cancer.
Purpose
The article reflects on the findings of this special issue and discusses the future challenges for policy, research and society. The findings suggest that challenges emerge as a result of legitimacy deficits of both consensus and contestatory modes of public involvement in health priority setting.
Design/methodology/approach
The article draws on the discussions and findings presented in this special issue. It seeks to bring the country experiences and case studies together to draw conclusions for policy, research and society.
Findings
At least two recurring themes emerge. An underlying theme is the importance, but also the challenge, of establishing legitimacy in health priority setting. The country experiences suggest that we understand very little about the conditions under which representative, or authentic, participation generates legitimacy and under which it will be regarded as insufficient. A second observation is that public participation takes a variety of forms that depend on the opportunity structures in a given national context. Given this variety the conceptualization of public participation needs to be expanded to account for the many forms of public participation.
Originality/value
The article concludes that the challenges of public involvement are closely linked to the question of how legitimate processes and decisions can be generated in priority setting. This suggests that future research must focus more narrowly on conditions under which legitimacy are generated in order to expand our understanding of public involvement in health prioritization.
Reforms have been introduced across Europe to increase prescribing efficiency with existing drugs. These include measures to lower prices of generics as well as increase their prescribing versus originators and patented products in a class or related class. This is essential to maintain comprehensive health care in Europe given continued pressures. The alternative is insufficient funds for new innovative drugs and increasing drug volumes with ageing populations. OBJECTIVE: To review the influence of measures and initiatives to increase the prescribing and dispensing of generics at low prices on ambulatory care prescribing efficiency. In view of this, provide guidance as authorities strive to introduce further reforms to meet their goals. METHODOLOGY: A narrative review of published papers combined with case histories. RESULTS: The different supply- and demand-side measures have reduced generic prices to as low as 2% to 3% of pre-patent loss prices in some cases as well as appreciably enhanced their utilisation. As a result, prescribing efficiency has increased without compromising care. In some cases, the reforms have led to expenditure actually falling despite appreciably increased volumes. CONCLUSIONS: Increasing use of generics at low prices will help maintain the European ideals of comprehensive and equitable health care. However, countries will continually need to learn from each other.
Hepatitis C, hepatitis B, HIV, TB and malaria are the five major causes of infectious disease death worldwide. In a breakthrough that rivals the invention of penicillin, drugs that cure hepatitis C, with minimal side effects and high success rates, have reached the market, but, in what must be one of the greatest tragedies of modern times, these life-saving medications are not being deployed on a mass scale. Pharmaceutical patents are gifted to private corporations by governments for the dual purposes of protecting R&D expenditure and encouraging innovation. Unfortunately the monopoly pricing power these patents provision currently lacks adequate checks and balances, is open to abuse, and is quite clearly being abused. The sort of legislative changes required to deliver on the original goals of pharmaceutical patents will take years or even decades to eventuate. Parallel importation of generic medication offers hope to the millions of patients with HCV unable to afford access to vastly overpriced originator medications. Doctors prescribing and monitoring patients taking generics can take comfort from the fact that the REDEMPTION trial results show, like the HIV generics that came before them, that HCV generics deliver robust clinical results.
Background:
Uganda introduced a multipronged intervention, the supervision, performance assessment, and recognition strategy (SPARS), to improve medicines management (MM) in public and not-for-profit health facilities. This paper, the first in a series, describes the SPARS intervention and reports on the MM situation in Uganda before SPARS (baseline).
Methods:
To build MM capacity at health facilities, health workers were trained as MM supervisors to visit health facilities, assess MM performance, and use the findings to provide support and standardize MM practices. Performance is assessed based on 25 MM indicators covering five domains: dispensing quality (7 indicators), prescribing quality (5), stock management (4), storage management (5) and ordering and reporting (4). From the end of 2010 to 2013, MM supervisors assessed baseline MM performance of 1384 government (85 %) and private not-for-profit facilities at all levels of care in about half of Uganda's districts.
Results:
The overall MM baseline median score was 10.3 out of a maximum of 25 with inter-quartile range (IQR) of 8.7-11.7. Facility domain scores (out of a maximum of 5) were as follows: storage management, median score of 2.9 (IQR 2.3-3.4); stock management 2.3 (IQR 2.0-2.8), ordering and reporting 2.2 (IQR 1.3-2.5), and dispensing quality 2.1 (IQR 1.7-2.7). Performance in prescribing quality was 0.9 (IQR 0.4-1.4). Significant regional differences were found: overall scores were highest in the Northern region (10.7; IQR 9.2-12.4) and lowest in the Eastern region (9.6; (IQR 7.8-11.2) (p < 0.001). Overall scores did not differ by facility ownership; however, government facilities scored lower in dispensing and storage and higher in ordering and reporting. Hospitals scored higher overall and in domains other than prescribing and stock management. Districts classified a priori as having high capacity for implementing SPARS had higher scores at baseline compared to lower-capacity districts.
Conclusion:
Assessing and building national capacity in MM is needed in both private not-for-profit and government facilities at all levels of care. The indicator-based, multipronged SPARS assessment has been described here, while the strategy's impact has yet to be documented.
Introduction:
New hepatitis C virus (HCV) medicines have markedly improved treatment efficacy and regimen tolerability. However, their high prices have limited access, prompting wide debate about fair and affordable prices. This study systematically compared the price and affordability of sofosbuvir and ledipasvir/sofosbuvir across 30 countries to assess affordability to health systems and patients.
Methods and findings:
Published 2015 ex-factory prices for a 12-wk course of treatment were provided by the Pharma Price Information (PPI) service of the Austrian public health institute Gesundheit Österreich GmbH or were obtained from national government or drug reimbursement authorities and recent press releases, where necessary. Prices in Organisation for Economic Co-operation and Development (OECD) member countries and select low- and middle-income countries were converted to US dollars using period average exchange rates and were adjusted for purchasing power parity (PPP). We analysed prices compared to national economic performance and estimated market size and the cost of these drugs in terms of countries' annual total pharmaceutical expenditure (TPE) and in terms of the duration of time an individual would need to work to pay for treatment out of pocket. Patient affordability was calculated using 2014 OECD average annual wages, supplemented with International Labour Organization median wage data where necessary. All data were compiled between 17 July 2015 and 25 January 2016. For the base case analysis, we assumed a 23% rebate/discount on the published price in all countries, except for countries with special pricing arrangements or generic licensing agreements. The median nominal ex-factory price of a 12-wk course of sofosbuvir across 26 OECD countries was US37,729 in Japan to US101,063 and PPP118,754) were at least 1.09 and 1.63 times higher, respectively than in the US (PPP72,765). Based on PPP-adjusted TPE and without the cost of ribavirin and other treatment costs, treating the entire HCV viraemic population with these regimens at the PPP-adjusted prices with a 23% price reduction would amount to at least one-tenth of current TPE across the countries included in this study, ranging from 10.5% of TPE in the Netherlands to 190.5% of TPE in Poland. In 12 countries, the price of a course of sofosbuvir without other costs was equivalent to 1 y or more of the average annual wage of individuals, ranging from 0.21 y in Egypt to 5.28 y in Turkey. This analysis relies on the accuracy of price information and infection prevalence estimates. It does not include the costs of diagnostic testing, supplementary treatments, treatment for patients with reinfection or cirrhosis, or associated health service costs.
Conclusions:
Current prices of these medicines are variable and unaffordable globally. These prices threaten the sustainability of health systems in many countries and prevent large-scale provision of treatment. Stakeholders should implement a fairer pricing framework to deliver lower prices that take account of affordability. Without lower prices, countries are unlikely to be able to increase investment to minimise the burden of hepatitis C.
Despite the high level of spending on cancer medicines in Australia, consumer organisations and the pharmaceutical industry often make claims of delayed or lack of access to new cancer medicines-claims that are frequently supported by prominent coverage in the Australian media. These claims, while morally and psychologically compelling, tend to ignore the complexity of medicines funding decisions. In this commentary we summarise the current situation regarding the registration and funding of cancer medicines in Australia, elucidate the main challenges associated with access to cancer medicines in the Australian context, and describe some of the steps that have been taken to address these challenges.
In October 2015, the third international Pharmaceutical Pricing and Reimbursement Information (PPRI) Conference was held in Vienna to foster discussion on challenges in pricing and reimbursement policies for medicines. The research presented highlighted that commonly used pharmaceutical pricing and reimbursement policies are not sufficiently effective to address current challenges. Conference participants called for fundamental reforms to ensure access to medicines, particularly to new and potentially more effective and/or safe medicines, while safeguarding the financial sustainability of health systems and working towards universal health coverage.
A biosimilar is an officially regulated and approved copy of an originator biologic therapy. Improved affordability and consequent wider patient access compared with biologics are a significant appeal of biosimilars. Regulatory guidelines for biosimilar development and approval are rigorous and undergoing constant refinement. The process of licensing approval for all biosimilars requires demonstration of comparability in quality, efficacy, and safety between the biosimilar and reference (originator) product, which is undertaken in a stepwise procedure of nonclinical and clinical evaluation. The approval of >20 biosimilars in Europe in several drug classes, including the first monoclonal antibody biosimilar, bears testimony to the increasing regulatory acceptance of these agents. In contrast, the clinical application of biosimilars remains underrecognized by physicians across therapy areas. Therefore, this article aims to provide a comprehensive review of the biosimilar development process and to provide multidisciplinary guidance on the potential therapeutic utility of biosimilars in clinical practice. Specifically, experts discuss clinical developments in the introduction of biosimilars across the disciplines of gastroenterology, nephrology, oncology, and rheumatology, and from a payer perspective, and also highlight a common need for ongoing pharmacovigilance, robust head-to-head clinical studies, and real-world data to establish the long-term risk-benefit profile of biosimilars. In conclusion, significant potential exists for biosimilars to revolutionize biologic therapy by widening patient access across therapy areas.
European patients and citizens need access to safe, effective and affordable medicines while the health care system should be financially sustainable, and innovation should be encouraged. This is perhaps the key challenge for the national competent authorities and public payers as pharmaceutical pricing and reimbursement remains the competence of EU Member States. In the light of increasing financial pressure while further new high-priced medicines are expected to come to the market, new approaches to achieve the above-mentioned objectives might be required. Without disregarding the subsidiarity principle, possible benefits of cooperative approaches should be studied and discussed.
In this context, a consortium of Gesundheit Österreich Forschungs- und Planungs GmbH, SOGETI Luxembourg S.A. and the University for Health Sciences, Medical Informatics and Technology was commissioned by the European Commission (DG SANTÉ / Chafea) to explore the pharmaceutical pricing policies of external price referencing (EPR) and differential pricing (DP) with regard to their ability to achieve two of the three above-mentioned policy objectives: to improve patients’ access to medicines and to generate savings for public payers.
In particular, this ‘study on enhanced cross-country coordination in the area of pharmaceutical product pricing’ aimed to survey existing EPR schemes in European countries and to develop possible improvements to the current EPR practice, as well as to analyse how DP schemes could possibly be designed for European countries, including addressing identified constraints to DP in Europe. Furthermore, it should be explored how EU-level coordination mechanisms could support the improvement of EPR systems and the establishment of a DP scheme.
To achieve these research objectives, the authors relied upon a range of methods including a literature review, a survey of competent authorities for pharmaceutical pricing, interviews with procurement experts, price simulations, a legal analysis, research of cooperation models and SWOT (strengths, weaknesses, opportunities, and threats) analyses. Extensive reviews involving the services of the EC, stakeholders and academics (‘peers’) were performed to ensure the high quality of the report.
Objective To identify characteristics (factors) about health technology assessment (HTA) decisions that are important to the public in determining whether public engagement should be undertaken and the reasons for these choices.
Design Focus groups using a nominal group technique to identify and rank factors relevant to public engagement in HTA decision-making. Thematic analysis was also undertaken to describe reasons underpinning participants’ choices and rankings.
Setting Members of the Australian general public.
Participants 58 people, aged 19–71 years participated in 6 focus groups.
Results 24 factors were identified by participants that were considered important in determining whether public engagement should be undertaken. These factors were individually ranked and grouped into 4 themes to interpret preferences for engagement. Members of the public were more likely to think public engagement was needed when trade-offs between benefits and costs were required to determine ‘value’, uncertainties in the evidence were present, and family members and/or carers were impacted. The role of public engagement was also seen as important if the existent system lacked transparency and did not provide a voice for patients, particularly for conditions less known in the community.
Conclusions Members of the public considered value, impact, uncertainty, equity and transparency in determining when engagement should be undertaken. This indicates that the public's preferences on when to undertake engagement relate to both the content of the HTA itself as well as the processes in place to support HTA decision-making. By understanding these preferences, decision-makers can work towards more effective, meaningful public engagement by involving the public in issues that are important to them and/or improving the processes around decision-making.
Early access programs, (EAPs) are adopted by an increasing number of pharma companies due to several benefits offered by these programs. EAPs offer ethical, compliant, and controlled mechanisms of access to investigational drugs outside of the clinical trial space and before the commercial launch of the drug, to patients with life-threatening diseases having no treatment options available. In addition to the development of positive relationships with key opinion leaders (KOL), patients, advocacy groups and regulators, the data captured from the implementation of EAPs supports in the formulation of global commercialization strategies. This white paper outlines various circumstances to be considered for the implementation of EAPs named patient programs, the regulatory landscape, the benefits and challenges associated with implementing these programs and the key considerations for their successful implementation.
The term ‘biosimilar’ refers to an alternative similar
version of an off-patent innovative originator biotechnology product (the ‘reference product’). Several biosimilars have been approved in Europe, and a number of top-selling biological medicines have lost, or will lose, patent protection over the next 5 years. We look at the experience in Europe so far. The USA has finally implemented a regulatory route for biosimilar approval. We recommend that European and US governments and payers take a strategic approach to get value for money from the use of biosimilars by (1) supporting and incentivising generation of high-quality comprehensive outcomes data on the effectiveness and safety of biosimilars and originator products; and (2) ensuring that incentives are in place for budget holders to benefit from price competition. This may create greater willingness on the part of budget holders and clinicians to use biosimilar and originator products with comparable outcomes interchangeably, and may drive down prices. Other options, such as direct price cuts for originator products or substitution rules without outcomes data, are likely to discourage biosimilar entry. With such approaches, governments may achieve a one-off cut in originator prices but may put at risk the creation of a more competitive market that would, in time, produce much greater savings. It was the creation of competitive markets for chemical generic drugs—notably, in the USA, the UK and Germany—rather than price control, that enabled payers to achieve the high discounts now taken for granted.
O Valor Terapêutico Acrescido (VTA) de um medicamento significa maior eficácia, segurança e/ou conveniência em relação a um comparador, que deve ser a melhor alternativa terapêutica existente. O VTA também pode significar o preenchimento de uma lacuna terapêutica, numa indicação clínica onde as terapias existentes são insuficientes, devendo nesta situação o medicamento ter apenas uma relação benefício/risco favorável. A demonstração do VTA, que é uma verdadeira inovação terapêutica que não deve ser confundida com inovação comercial ou tecnológica, deve ser feita através de metodologia robusta, obedecendo às regras da Medicina Baseada na Evidência.
Market access is the fourth hurdle in the drug development process and the primary driver for global income of any new drug. Without a strategy in place for pricing, showing value for effectiveness and an understanding of the target purchasers’ needs, the drug will fail to reach its intended market value. Introduction to Market Access for Pharmaceuticals is based on an accredited course in this area, taken from the European Market Access University Diploma (EMAUD), and is affiliated with Aix Marseille University.
Medicines Access Programs (MAP) offer access to publicly unfunded medicines at the discretion of pharmaceutical companies. Limited literature is available on their extent and scope in Australia and New Zealand. This study aims to identify MAPs for cancer medicines that were operational in 2014-15 in Australia and New Zealand and describe their characteristics. A preliminary list of MAPs was sent to hospital pharmacists in Australia and New Zealand to validate and collect further information. Pharmaceutical companies were contacted directly to provide information regarding MAPs offered. Key stakeholders were interviewed to identify issues with MAPs. Fifty-one MAPs were identified covering a range of indications. The majority of MAPs were provided free of charge to the patient for medicines that were registered or in the process of being registered but were not funded. Variability in the number of MAPs across institutions and characteristics was observed. Australia offered more MAPs than New Zealand. Only two of 17 pharmaceutical companies contacted agreed to provide information on their MAPs. Eight stakeholder interviews were conducted. This identified that while MAPs are widely operational there is lack of clinical monitoring, inequity to access, operational issues and lack of transparency. Our results suggest a need for a standardised and mandated policy to mitigate issues with MAPs.
Enthusiasm for performance-based risk-sharing arrangements (PBRSAs) continues but at variable pace across countries. Our objective was to identify and characterize publicly available cases and related trends for these arrangements. We performed a review of PBRSAs from 1993 to 2016 using the University of Washington PBRSA Database. Arrangements were categorized according to a previously published taxonomy. Macro-level trends were identified related to the timing of adoption, countries involved, types of arrangements, and disease areas. Our search yielded 437 arrangements. Among these, 183 (41.9%) were categorized as currently active, while 58.1% have expired. Five main types of arrangements have been identified, namely coverage with evidence development (149 cases, 34.1%), performance-linked reimbursement (104 cases, 23.8%), conditional treatment continuation (78 cases, 17.8%), financial or utilization (71 cases, 16.2%), and hybrid schemes with multiple components (35 cases, 8.0%). The pace of adoption varies across countries but has renewed an upward trend after a lull in 2012/2013. Conditions in the USA may be changing toward a more favorable environment of PBRSAs. Interest in PBRSAs remains high, suggesting they are a viable coverage and reimbursement mechanism for a wide range of medical products.
Background:
The NHS Cancer Drugs Fund (CDF) was established in 2010 to reduce delays and improve access to cancer drugs, including those that had been previously appraised but not approved by NICE (National Institute for Health and Care Excellence). After 1.3 billion GBP expenditure, a UK parliamentary review in 2016 rationalized the CDF back into NICE.
Methods:
This paper analyses the potential value delivered by the CDF according to six value criteria. This includes validated clinical benefits scales, cost-effectiveness criteria as defined by NICE and an assessment of real-world data. The analysis focuses on 29 cancer drugs approved for 47 indications that could be prescribed through the CDF in January 2015.
Results:
Of the 47 CDF approved indications, only 18 (38%) reported a statistically significant OS benefit, with an overall median survival of 3.1 months (1.4-15.7 months). When assessed according to clinical benefit scales, only 23 (48%) and 9 (18%) of the 47 drug indications met ASCO and ESMO criteria, respectively. NICE had previously rejected 26 (55%) of the CDF approved indications because they did not meet cost-effectiveness thresholds. Four drugs-bevacizumab, cetuximab, everolimus and lapatinib-represented the bulk of CDF applications and were approved for a total of 18 separate indications. Thirteen of these indications were subsequently delisted by the CDF in January 2015 due to insufficient evidence for clinical benefit-data which were unchanged since their initial approval.
Conclusions:
We conclude the CDF has not delivered meaningful value to patients or society. There is no empirical evidence to support a 'drug only' ring fenced cancer fund relative to concomitant investments in other cancer domains such as surgery and radiotherapy, or other noncancer medicines. Reimbursement decisions for all drugs and interventions within cancer care should be made through appropriate health technology appraisal processes.
Purpose:
We provide a review of current knowledge on comparability between biosimilars and originator biologics in view of the continuous evolution occurring in this highly dynamic area.
Methods:
English-language literature indexed in MEDLINE was explored, without time limits, to July 31, 2016, using the terms biosimilar, biotechnologic drug, biologic drug, monoclonal antibody, fusion protein, and anti-tumor necrosis factor. The reference lists of identified articles were examined carefully for additional pertinent publications.
Findings:
Biological medicines are much more structurally complex and extremely sensitive to manufacturing conditions and therefore more difficult to characterize and produce than small molecule drugs. Even minor changes in manufacturing may lead to significant variations of the cellular systems used for biological production, as well as to differences in the structure, stability, or other quality aspects of the end product, all of which have the potential to affect tolerability and/or efficacy and increase the risk of immune responses. Owing to these issues, specific regulatory guidance on biosimilars is continuously evolving, and there is some disagreement on which studies need to be implemented to approve a biosimilar. According to current literature, the following points on biosimilars deserve consideration: biosimilar development is characterized by global harmonization, although several not fully answered questions remain regarding extrapolation of indications, switching or interchangeability, and tolerability; in patients with rheumatic diseases, the tolerability and efficacy of biosimilars in clinical practice remain to be established; several medical and patient associations have published position papers on biosimilars requesting that safety, efficacy, and traceability be carefully considered; long-term postmarketing studies should be implemented to allow physicians to gain confidence in biosimilars.
Implications:
On the basis of current knowledge, and taking into consideration both regulatory rules and medical society positions, it can be concluded that, although cost savings are highly desirable, the approval process for biosimilars needs to place tolerability and efficacy, supported by scientifically sound evidence, as the highest priority. Moreover, physicians must retain full authority regarding the decision about which biopharmaceutical to use for treating patients.
Crowdfunding is a financing method characterized by pooling together a large number of smaller contributions to support a specific initiative. The emergence of social media and numerous online platforms has allowed crowdfunding to flourish in popularity. Crowdfunding is an especially effective and popular tool in the realm of supporting charitable or ideological causes. Increasingly prevalent in this realm is crowdfunding for health care costs.
Objective:
The objective of this study was to survey pharmacists' knowledge and views of biosimilars in Quebec and France.
Methods:
An online and anonymous survey was conducted. The survey was divided into two parts including: (1) ten multiple choice questions on main characteristics that distinguish biosimilars from generic drugs; (2) fifteen statements on biosimilars key issues (interchangeability, immunogenicity risk management…). Pharmacists were asked to indicate their level of agreement to these statements using a 5-item Likert scale. A descriptive statistical analysis of the results was performed.
Results:
A total of 229 pharmacists answered the survey (141 in Quebec and 88 in France). Pharmacists know the main differences between generic drugs and biosimilars. Viewpoints of pharmacists on biosimilars key issues are alike: nomenclature of biosimilars is essential to avoid confusions with the reference drug; the creation of a list of biosimilar and interchangeable biologic drugs is necessary; responsibilities for immunogenicity risk management should be shared between pharmacists and physicians. However, viewpoints vary regarding the patient informed consent for biologic drugs substitution.
Conclusion:
Knowledge and views of pharmacists about biosimilars in Quebec and in France are alike. Pharmacists should be knowledgeable about the particularities and key issues of biosimilars because they will play a key role for their introduction in clinical practice. They should be aware of the evolution of the legal framework of biosimilars to ensure their safe and optimal use.
Background
Decisions made by the National Institute for Health and Care Excellence (NICE) exert an influence on the allocation of resources within ‘fixed’ National Health Service budgets. Yet guidance for different types of health interventions is handled via different ‘programmes’ within NICE, which follow different methods and processes. Objective
The objective of this research was to identify differences in the processes and methods of NICE health technology assessment programmes and to explore how these could impact on allocative efficiency within the National Health Service. Methods
Data were extracted from the NICE technology appraisal programme, medical technologies guidance, diagnostic assessment programme, highly specialised technologies programme, and clinical guidelines process and methods manuals to undertake a systematic comparison. Five qualitative interviews were carried out with NICE members of staff and committee members to explore the reasons for the differences found. ResultsThe main differences identified were in the required evidence review period, or lack thereof, mandatory funding status, the provision of a reference case for economic evaluation, the requirement for and the type of economic analysis undertaken, and the decision making criteria used for appraisal. Conclusion
Many of the differences found can be justified on grounds of practicality and relevance to the health technologies under assessment. Nevertheless, from a strict utilitarian view, there are several potential areas of inefficiency that could lead to the misallocation of resources within the National Health Service, although some of these might be eliminated or reduced if an egalitarian view is taken. The challenge is determining where society is willing to trade health gains between different people.
Background
In view of the current financial and demographic situation in Portugal, accessibility to health care may be affected, including the ability to adhere to medication. Objective To evaluate the perceived effects of the crisis on elderly patient’s access to medicines and medical care, and its implications on medicine-taking behaviour. Setting Community pharmacy.
Method:
A cross-sectional study was undertaken during April 2013, where elderly patients answered a self-administered questionnaire based on their health-related experiences in the current and previous year. Binary logistic regression was used to ascertain the effects of potential predictors on the likelihood of adherence. Main outcome measures self-reported adherence.
Results
A total of 1231 questionnaires were collected. 27.3% of patients had stopped using treatments or health services in the previous year for financial motives; mostly private medical appointments, followed by dentist appointments. Almost 30% of patients stopped purchasing prescribed medicines. Over 20% of patients reduced their use of public services. Out-of-pocket expenses with medicines were considered higher in the current year by 40.1% of patients. The most common strategy developed to cope with increasing costs of medicines was generic substitution, but around 15% of patients also stopped taking their medication or started saving by increasing the interdose interval.
Conclusion
Reports of decreasing costs with medicines was associated with a decreased likelihood of adherence (OR 0.42; 95% CI 0.27–0.65). Lower perceived health status and having 3 or more co-morbidities were associated with lower odds of adhering, whilst less frequent medical appointments was associated with a higher likelihood of exhibiting adherence.
Published in: Int J Clin Pharm. 2017;39:104-12.
Crowdfunding involves raising money from large groups of individuals, often through the use of websites dedicated to this purpose. Crowdfunding campaigns aimed at raising money to pay for expenses related to receiving medical treatment are receiving increased media attention and there is evidence that medical crowdfunding websites are heavily used. Nonetheless, virtually no scholarly attention has been paid to these medical crowdfunding campaigns and there is no systematic evidence about how widely they are used and for what reasons, and what effects they have on the provision of medical care and individuals' relationships to their health systems. Ethical concerns have been raised in relation to these campaigns, focusing on issues for campaigners and donors such as exposure to fraudulent campaigns, loss of privacy, and fairness in how medical crowdfunding funds are distributed. Medical crowdfunding websites themselves have not been systematically studied, despite their significant influence on how these campaigns are developed and promoted. In this paper, we identify three very broad and pressing ethical questions regarding medical crowdfunding for social scientists to address and offer some preliminary insights into key issues informing future answers to each: Who benefits the most from medical crowdfunding and how does medical crowdfunding affect access to medical care; How does medical crowdfunding affect our understanding of the causes of inadequate access to medical care; and How are campaigner and donor privacy affected by website design? Our observations indicate the need for increased scholarly attention to the ethical and practical effects of medical crowdfunding for campaigners, recipients, donors, and the health system as a whole.
Objectives:
Health Technology Assessment (HTA) of Medical devices (MDs) and MD-based procedures can be challenging due to the unique features and particularities of this group of technologies, such as device-operator interaction. The aim of this study was to (1) clarify, and supplement earlier findings on European HTA institutions' structural, procedural and methodological characteristics with regard to the assessment of MDs and to (2) capture the institutions' perceptions regarding challenges and future trends.
Methods:
Semi-structured telephone interviews with 16 representatives from leading European HTA institutions were performed between April and July 2015. Summative and directed content analysis was used for the analysis, which is reported according to the COREQ checklist.
Results:
Findings from the analysis of the interviews were manifold and partially confirmed what has been noted in the literature (e.g. scarce evidence; identifying relevant studies challenging due to more incremental innovations). Additional themes emerged that can be important for future considerations by HTA institutions and policy-makers alike (e.g. areas for future research; need for specific tools).
Conclusions:
The collective opinion of 16 European HTA institutions on MD evaluation could provide ideas to ameliorate the current regulatory situation beyond the modified EU regulation and start broader, more in-depth methodological discussions around the issue. Interviewed experts seem to agree that new approaches such as coverage with evidence development as some countries already introduced could help to overcome the problem with scarce evidence.
The effects of the UK’s prospective exit from the European Union are in hot dispute. Arguments for and against continue to be keenly contested except, that is, when the conversation turns to science and medicine. Here, the loudest voices and most prominent arguments suggest that the decision taken by the public will cause harm.
Biologics embrace a wide range of substances synthesized by cells or living organisms by means of different biological processes, including recombinant DNA technology, controlled gene expression, or antibody technologies. A biosimilar establishes similarity to the reference medicinal product in terms of quality characteristics, biological activity, safety, and efficacy based on a comprehensive comparability exercise. Minimizing development costs and accelerating their market access create a convergence of interests between health services, worried about sustainability, and generic manufacturers. While the demonstration of bioequivalence is sufficient for small synthetic molecules, this approach is not scientifically applicable to a copy of biological drug constituted by large and complex molecules, which are similar but not identical to the originator and are also subject to different post-translational processes. Internists should be confident that the development process of biosimilars ensures a comparable risk-to-benefit balance with the originators. On the basis of available evidence and pharmacovigilance network, there are no grounds to believe that the use of a biosimilar carries more risks for the patient than the use of an originator. Since the first biosimilar was authorized in Europe in 2006, no clinical alerts have raised red flags about the established EMA biosimilar pathway. In this article, we discuss some of the most frequent concerns raised by clinicians about biosimilars and try to explains the scientific principles underlying the biosimilar concept established in the EU in order to license biosimilar drugs.
Patients with cancers of differing histologies that express certain biomarkers are likely to benefit from treatment with targeted therapies. However, targets can be present in malignancies other than those indicated by a drug's label, and as a result, affected patients will have no access to those potentially useful drugs. To tackle this issue, the French National Cancer Institute developed the AcSé Programme in 2013. This programme is designed to make treatment decisions or recommendations on the basis of the presence of relevant biomarkers for malignancies with no targeted therapies available and also aims to improve safety, and evaluate the efficacy of targeted drugs used outside of their approved indications. Patients across France have access to molecular testing in 28 molecular genetics centres and to targeted therapies within phase II trials provided no other trials exist in which they could reasonably be included. Trials include patients below the age of 18 if safe dosing data are available. As of January 2016, 183 French clinical sites and over 7,000 patients are participating in AcSé led trials. Proof of concept is being demonstrated through trials designed to investigate the effectiveness of crizotinib and vemurafenib in a wide variety of cancers.
The science, regulatory, and pharmacovigilance processes for biosimilars are evolving. The Biologics Price Competition and Innovation Act created an abridged approval pathway for biosimilars. The first biosimilar was Food and Drug Administration approved in 2015 with others currently pending approval. As biosimilars become marketed, nurse practitioners will be faced with many challenges, including patient questions regarding them. Nurse practitioners have an important role in the identification of possible safety profile differences between biosimilars and innovator biologicals, especially for immunogenicity. A number of practical considerations need to be addressed including substitutability; off-label prescribing; patient-specific record keeping; and computerized prescriber order entry, education, and reimbursement.
Health Technology Assessment (HTA) is used to assess the value of new technologies and by producing coverage recommendations it indirectly controls the uptake of new technologies in many European countries. While HTA generally relies on a robust assessment of the clinical cost-effectiveness of a new technology, the clinical and economic evidence required for this purpose is often not available for Orphan Drugs (ODs), partly because of challenges related to the recruitment of patients to participate in clinical trials. A number of European HTA agencies have started to implement specific policies to address the challenges related to evidence requirements for the case of ODs. In this study, we map out the policies currently in place in eight European countries regarding HTA and its application to the case of ODs and explore the implications these policies have for coverage decisions.
Between 2013 and 2014, spending on specialty drugs, including biologics, increased 32.4%, while spending on small-molecule drugs increased just 6.8%. By 2016, 8 of the 10 top-selling drugs are expected to be biologics. While many biologics will be going off patent, there will likely be multiple prospective manufacturers of biosimilars, and a growing emphasis on regulatory guidelines to ensure their efficacy and safety, in the very near future. A strong factor and assumption surrounding biosimilar development and use is the potential for healthcare cost savings; the introduction of biosimilars is expected to reduce drug costs, although to a lesser degree than seen with small-molecule generic drugs. Managed care clinicians and providers must carefully consider the economic implications and potential cost-effectiveness of uptake of biosimilars for therapy in clinical practice.
The financial sustainability of cancer services as part of national health systems is a major challenge;1 oncology consumes up to 30% of total hospital expenditure and the amount spent on expensive cancer drugs is rising fast.2 In view of the pipeline of new drugs, these costs are likely to continue to grow.3 Apart from the risk of unequal access between European countries, burdening health systems with fast-growing costs for these drugs means that the sustainability of cancer care could be compromised.
One element increasingly under scrutiny is the pricing policy of pharmaceutical companies. Both in the recent American Society of Clinical Oncology (ASCO) meeting and in various publications,4, 5 and 6 attention has been drawn to the lack of transparency on the pricing of various drugs. Different models to judge the appropriateness of prescribing drugs have been proposed; the European Society for Medical Oncology (ESMO) and ASCO models try to balance cost and effectiveness. However, a wholehearted decision to include cost effectiveness as a criterion does not yet seem possible.
An overview of actual prices in European countries does, to our knowledge, not exist, and anecdotal evidence has suggested that differences in price levels might be high. We surveyed the prices for several cancer drugs in European countries through the membership of the European Organization of Cancer Institutes (OECI) and Cancer Core Europe.7 A word-based survey was emailed to all full members of the OECI (n=51), both European Union (EU) members and non-EU members, and to the non-OECI member of Cancer Core Europe. The survey was sent to the board of the centre; for most centres the leading pharmacist responded, together with an oncologist. We asked centres to provide list or official and actual prices, corrected for VAT differences, and asked for information about central or government coordinated purchasing. The actual price was defined as the net price—ie, as price per one dose (eg, one 100 mg vial or one capsule of 12·5 mg) to allow for a comparison in case of different pack sizes. Any type of discount was taken into account because we asked the centres to provide us with the price they actually pay. We received a response from 21 centres from 15 countries. Most responses were received in June and July 2015. For some countries we received more than one response and from these we present an average.