Sarah Elizabeth Garner

• PhD BPharm
• World Health Organization WHO

Introduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is ver...

To ensure equitable access to medicines and vaccines, organizational efforts and purchase volumes have been pooled in joint procurements and negotiations for decades in some regions of the world, as well as globally through supranational procurement mechanisms. In Europe, countries started to collaborate on procurement and negotiations recently whe...

[This corrects the article DOI: 10.3389/fphar.2021.639207.].

Background: Surveillance of antimicrobial consumption (AMC) is important to address inappropriate use. AMC data for countries in the European Union (EU) and European Economic Area (EEA) and Eastern European and Central Asian countries were compared to provide future guidance. Methods: Analyses of 2014–2018 data from 30 EU/EEA countries of the Europ...

Introduction Junior doctors are responsible for a substantial number of prescribing errors, and final-year medical students lack sufficient prescribing knowledge and skills just before they graduate. Various national and international projects have been initiated to reform the teaching of clinical pharmacology and therapeutics (CP&T) during undergr...

The UHC key concepts and relevant UN resolutions recommending transparency, including in relation to HTA and pricing. The role of innovation and intellectual property frameworks will be discussed, including financing and co-ordination of R&D for health technologies. Examples on the importance information exchange can play to improve negotiations wi...

This report assesses five government-led, voluntary cross-country collaborations in the WHO European Region that aim to improve access to essential medicines through joint activities: the Baltic Procurement Initiative, Beneluxa Initiative, Fair and Affordable Pricing, Nordic Pharmaceutical Forum and Valletta Declaration. These cover activities incl...

Introduction: Twenty-five years ago, the World Health Organization (WHO) published the Guide to Good Prescribing (GGP), followed by the accompanying Teacher's Guide to Good Prescribing (TGGP). The GGP is based on a normative 6-step model for therapeutic reasoning and prescribing, and provides a six-step guide for students to the process of rationa...

Fewer than half of new drugs have data on their comparative benefits and harms against existing treatment options at the time of regulatory approval in Europe and the USA. Even when active-comparator trials exist, they might not produce meaningful data to inform decisions in clinical practice and health policy. The uncertainty associated with the p...

The World Health Organization Global Patient Safety Challenge, Medication Without Harm, aims to reduce serious, avoidable medication-related harm by 50% in 5 years, globally. Three areas have been identified for early priority action. This technical report addresses Medication Safety in Transitions of Care; why it is a priority, what has been done...

Objectives: Value assessment frameworks have gained prominence recently in the context of U.S. healthcare. Such frameworks set out a series of factors that are considered in funding decisions. The UK's National Institute of Health and Care Excellence (NICE) is an established health technology assessment (HTA) agency. We present a novel application...

As per the EMA definition, adaptive pathways is a scientific concept for the development of medicines which seeks to facilitate patient access to promising medicines addressing high unmet need through a prospectively planned approach in a sustainable way. This review reports the findings of activities undertaken by the ADAPT-SMART consortium to ide...

Background The National Institute for Health and Care Excellence (NICE) was established in 1999 and provides national guidance and advice to improve health and social care. Several steps in the research cycle have been identified that can support the reduction of waste that occurs in biomedical research. The first step in the process is ensuring ap...

Introduction: Registries collect clinical data to evaluate specified outcomes for a population defined by a particular disease, condition or exposure. They can be used to describe the natural history of a condition or disease, determine the effectiveness or cost-effectiveness of products, measure or monitor safety and harm, and measure quality of c...

In press

In press

Background: Rheumatoid arthritis (RA) is a systemic auto-immune disorder, involving persistent joint inflammation. NSAIDs are used to control the symptoms of RA, but are associated with significant gastro-intestinal toxicity, including a risk of potentially life threatening gastroduodenal perforations, ulcers and bleeds. The NSAIDs known as the se...

Objectives: Some countries make considerable effort to involve patients and patient groups in their health technology assessment (HTA) processes; others are only just considering or are yet to consider patient involvement in HTA. Methods: This commentary offers four arguments why patient involvement should be prioritized by those HTA agencies th...

Clin. Pharmacol. Ther. 100, 685–698 (2016); doi:10.1002/cpt.509 The authors note that the graphic for Figure 6 was inadvertently duplicated as Figure 8. The correct graphic for Figure 8 is shown below. (Figure presented.) Knowledge matrix for adaptive biomedical innovation (ABI). This approach improves knowledge quality and quantity over all four d...

The current system of biomedical innovation is unable to keep pace with scientific advancements. We propose to address this gap by re-engineering innovation processes to accelerate reliable delivery of products that address unmet medical needs. Adaptive Biomedical Innovation (ABI) provides an integrative, strategic approach for process innovation....

Analyses of healthcare database (claims, EHR) are useful supplements to clinical trials for generating evidence on the effectiveness, harm, use and value of medical products in routine care. A constant stream of data from the routine operation of modern healthcare systems, which can be analyzed in rapid cycles enables incremental evidence developme...

Regulatory decisions are often based on multiple clinical end points, but the perspectives used to judge the relative importance of those end points are predominantly those of expert decision makers rather than of the patient. However, there is a growing awareness that active patient and public participation can improve decision making, increase ac...

Adaptive pathways for medicines have gained momentum and, in Europe, adaptive pathways have recently been introduced into the European Medicines Agency (EMA) processes after a successful 2-year pilot. Although the concept, as initially proposed, contained several elements that would have required regulatory reforms, the adaptive pathways program ha...

Table S1. Topical clindamycin compared with placebo for hidradenitis suppurativa. Table S2. Oral tetracycline compared with topical clindamycin for hidradenitis suppurativa. Table S3. Ethinylestradiol and cyproterone acetate compared with ethinylestradiol and norgestrel for hidradenitis suppurativa. Table S4. Etanercept compared with placebo for...

More than 50 interventions have been used to treat hidradenitis suppurativa (HS) and so therapy decisions can be challenging. To summarise and appraise randomised controlled trial (RCT) evidence for HS interventions in adults. Searches were conducted in MEDLINE, EMBASE, CENTRAL, LILACS, five trials registers and abstracts from eight dermatology con...

After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Although it is a significant achieveme...

Objectives: To elicit a comprehensive stakeholder view on the acceptability of using real-world evidence (RWE) for informing trial design and establishing relative effectiveness (RE) of new medicines in regulatory and HTA assessments. MethOds: IMIGetReal (www.imi-getreal.eu) is facilitating stakeholder dialogue on the role of RWE through case studi...

Background: Hidradenitis suppurativa (HS) is a chronic inflammatory skin condition characterised by recurrent painful boils in flexural sites, such as the axillae and groin, that affects about 1% of the population, with onset in early adulthood. Objectives: To assess the effects of interventions for HS in people of all ages. Search methods: We...

Pharmaceutical expenditure has increased rapidly across many Organisation for Economic Cooperation and Development (OECD) countries over the past three decades. This growth is an increasing concern for governments and other third-party payers seeking to provide equitable and comprehensive healthcare within sustainable budgets. In order to create he...

Objectives: Colloquial evidence (CE) has been described as the informal evidence that helps provide context to other forms of evidence in guidance development. Despite challenges around quality, and the potential biases, the use of CE is becoming increasingly important in assessments where scientific literature is sparse and to also capture the exp...

The recent launch of a pilot scheme for 'adaptive licensing' by the European Medicines Agency could spell a new era in drug development. Rather than focusing on the purely regulatory aspect of the system, the proposals challenge several aspects of existing development processes, including the conventional roles of sponsor, regulatory and reimbursem...

The concept of adaptive licensing (AL) has met with considerable interest. Yet, some remain sceptical about its feasibility. Others argue that the focus and name of AL should be broadened. Against this background of on-going debate, we examine the environmental changes that will likely make adaptive pathways the preferred approach in future. The ke...

Background Practical solutions are needed to support the appropriate use of available health system resources as countries are continually pressured to 'do more with less' in health care. Increasingly, health systems and organizations are exploring the reassessment of possibly obsolete, inefficient, or ineffective health system resources and potent...

Successfully advancing Adaptive Licensing requires coordinated planning and action across multiple stakeholders. The Janus Initiative combines a safe haven multi-stakeholder process with an integrated, best-of-breed quantitative simulation toolset to facilitate biomedical eco-system innovation discussions grounded in case-based realities. The proce...

It is very fitting that the strap line for the article on reassessment (1) is derived from the quote “politics is the art of the possible” attributed to Otto von Bismarck. Bismarck was the first chancellor of the unified German Empire that preserved peace in Europe until 1914. Politically deft, he persuaded the southern German states to join with h...

Digital technologies, along with advancements in data analytics, genomic and diagnostic technologies, and access to real-world patient data, are making “adaptive” fast-track access for precision and more personalised medicines a real possibility. For companies holding the rights to potential breakthrough drugs, these new, more interactive and fl ex...

Evidence-based policy making calls for the use of the best available evidence to support decision-making. Traditional hierarchies of evidence interpret this as focus on RCTs with most placing systematic reviews at the top. The proliferation of systematic reviews has now led to ‘tertiary analysis’ – reviews of multiple sources of secondary analysis....

Objectives: To examine the challenges to using systematic review evidence to develop guidance for decommissioning ineffective health services, and the problems experienced by clinicians and commissioners when they attempt to implement the evidence from this guidance. Methods: Interviews with 23 clinicians and 15 commissioners from nine commissio...

Background Selective outcome and analysis reporting (SOR and SAR) occur when only a subset of outcomes measured and analyzed in a study is fully reported, and are an important source of potential bias. Key methodological issuesWe describe what is known about the prevalence and effects of SOR and SAR in both randomized controlled trials (RCTs) and n...

Despite international agreement that stopping low value practices will increase efficiency, identifying them is difficult and controversial. Opponents of centralized lists of low value practices stress that the actual problem is inappropriate low value use, and better targeting and implementation of treatment thresholds is needed. Our objective was...

Background: Systematic reviews provide a transparent and robust summary of existing research. However, health system managers, national and local policy makers and healthcare professionals can face several obstacles when attempting to utilise this evidence. These include constraints operating within the health system, dealing with a large volume o...

Minocycline is an oral antibiotic used for acne vulgaris. Its use has lessened due to safety concerns (including potentially irreversible pigmentation), a relatively high cost, and no evidence of any greater benefit than other acne treatments. A modified-release version of minocycline is being promoted as having fewer side-effects. To assess new ev...

Traditional drug licensing approaches are based on binary decisions. At the moment of licensing, an experimental therapy is presumptively transformed into a fully vetted, safe, efficacious therapy. By contrast, adaptive licensing (AL) approaches are based on stepwise learning under conditions of acknowledged uncertainty, with iterative phases of da...

The demand for real-world research (RWR) from many stakeholders has resulted in an increasing need for well-trained professionals who understand how to perform and evaluate RWR and to interpret the results of such data. The Drug Information Association (DIA) Real World Outcomes Task Force (RWOTF) developed a survey to explore North American workfor...

This article maps out adaptive licensing to help inform a vital debate on the future of pharmaceutical development and licensing. There are various interpretations of this development concept and therefore the implications of any changes may vary, but it has been identified that the concept has arisen from the rapidly changing pharmaceutical and ec...

These are commentaries of a Cochrane review, published in this issue of EBCH, first published as: Garner SE, Eady A, Popescu CM, Newton J, Li Wan Po A. Minocycline for acne vulgaris: efficacy and safety. Cochrane Database of Systematic Reviews 2003, Issue 1. Art. No.: CD002086. DOI: 10.1002/14651858.CD002086. Further information for this Cochrane r...

The dwindling supply of new antibiotics largely reflects regulatory and commercial challenges, but also a failure of discovery. In the 1990s the pharmaceutical industry abandoned its classical ways of seeking antibiotics and instead adopted a strategy that combined genomics with high-throughput screening of existing compound libraries. Too much emp...

Acne is a chronic inflammatory disease of the pilosebaceous unit resulting from androgen-induced increased sebum production, altered keratinisation, inflammation, and bacterial colonisation of hair follicles on the face, neck, chest, and back by Propionibacterium acnes. Although early colonisation with P acnes and family history might have importan...

Over the past 10 years NICE has identified over 800 clinical interventions for potential disinvestment. But Sarah Garner and Peter Littlejohns report that although disinvestment will increase efficiency and quality, the opportunity for cash saving is unlikely to meet the necessary targets

No country can afford all the health care interventions that might benefit patients. Demand will always outstrip available resources, so priorities have to be agreed upon. Such decisions are controversial, making it vital that they are underpinned by robust transparent processes and methods. In the United Kingdom, this is the responsibility of the...

Although randomized clinical trials (RCTs) are the bedrock for establishing which interventions are efficacious, there is increasing recognition that they cannot address all needs, especially the need to determine, in a timely manner, the safety and effectiveness of different interventions used in the diverse array of patients and settings that mak...

The concept of using public funds to pay for healthcare interventions only when provided in the context of ongoing research is receiving increasing attention worldwide. Nevertheless, these decisions are often controversial and implementation can be problematic. The aim of this study was to investigate the views of United Kingdom stakeholders on the...

The National Institute for Health and Clinical Excellence (NICE) will have existed for 10 years on April 1, 2009. Over the past decade, the institute's methodological approach to the development of guidance and assessment of the value of health-care interventions has received international interest and acclaim. Furthermore, individual decisions, in...

Antimicrobials should be a special case because of the risk of antimicrobial resistance

Background: Acne is a common skin disorder among women. Although no uniform approach to the management of acne exists, combination oral contraceptives (COCs), which contain an estrogen and a progestin, often are prescribed for women. Objectives: To determine the effectiveness of combined oral contraceptives (COCs) for the treatment of facial acn...

All European countries are facing common challenges for delivering appropriate, evidence-based care to patients with cancer. Despite tangible improvements in diagnosis and treatment, marked differences in cancer survival exist throughout Europe. The reliable translation of new research evidence into consistent patient-oriented strategies is a key e...

There is growing concern about the quality and safety of prescribing in the UK. Added to the increasing prevalence of antimicrobial resistance, this makes a persuasive case for improving education about antimicrobials within a broader programme of education about prescribing. Moreover, the need for education is not confined to the professionals who...

Pityriasis rosea is a scaly rash that mainly affects young adults. It can be very itchy but most people recover within 2 to 12 weeks. To assess the effects of interventions for pityriasis rosea. We searched the Cochrane Skin Group Specialised Register (December 2004), the Cochrane Central Register of Controlled Clinical Trials in The Cochrane Libra...

Background: Acne is a common skin disorder among women. Although no uniform approach to the management of acne exists, combination oral contraceptives (COCs), which contain an estrogen and a progestin, often are prescribed for women. Objectives: To determine the effectiveness of combined oral contraceptives (COCs) for the treatment of facial acn...

Editor's note: The anti-inflammatory drug rofecoxib (Vioxx) was withdrawn from the market at the end of September 2004 after it was shown that long-term use (greater than 18 months) could increase the risk of heart attack and stroke. Further information is available at www.vioxx.com. Rheumatoid arthritis (RA) is a systemic auto-immune disorder, in...

Editor's note: The anti-inflammatory drug rofecoxib (Vioxx) was withdrawn from the market at the end of September 2004 after it was shown that long-term use (greater than 18 months) could increase the risk of heart attack and stroke. Further information is available at www.vioxx.com. Osteoarthritis is a chronic disease of the joints, characterised...

Background: Acne is a common skin disorder among women. Although no uniform approach to the management of acne exists, combination oral contraceptives (COCs), which contain an estrogen and a progestin, often are prescribed for women. Objectives: To determine the effectiveness of COCs for the treatment of facial acne compared to placebo or other...

EDITOR—Although the review and commentary by Webster et al on the management of acne contain good sense, the opinions presented do not fully reflect the evidence base or the risks associated with antibiotics.1 Webster promotes the use of antibiotics in the routine management of even mild acne when there is considerable global effort to limit antim...

Rheumatoid arthritis (RA) is a systemic auto-immune disorder, involving persistent joint inflammation. NSAIDs are used to control the symptoms of RA, but are associated with significant gastro-intestinal toxicity, including a risk of potentially life threatening gastroduodenal perforations, ulcers and bleeds. The NSAIDs known as the selective Cox I...

Rheumatoid arthritis (RA) is a systemic auto-immune disorder, in which the synovial lining of many joints and tendon sheaths are persistently inflamed. To assess the efficacy and toxicity of rofecoxib for treating RA. We searched the following electronic databases up to December 2000: MEDLINE, EMBASE, Cochrane Database of Systematic Reviews, Cochra...

Samenvatting Minocycline wordt veelvuldig gebruikt ter behandeling van ernstige acne vulgaris. Het middel zou vele voordelen hebben ten opzichte van de eerstegeneratie-tetracyclinen, maar het is duurder en berichten over het overlijden van twee patiënten die minocycline gebruikten, hebben grote onrust veroorzaakt. Onder dermatologen bestaat geen co...