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Translating Research into Health Policy: The Citizen Petition Experience with the Food and Drug Administration

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Abstract

Translating research findings into policy is important. Health policy researchers often testify before Congressional subcommittees and provide background on health policy issues. A rarely used, but important, tool for facilitating translation of research into policy is via filing a Citizen Petition.

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The United States Constitution protects the right of citizens to petition the government for “a redress of grievances.” This right has important implications for citizens desiring to advance the public health by petitioning administrative agencies, such as the Food and Drug Administration, to take safety actions. We examined a total of 1,915 petitions filed between 2001 and 2013 to investigate the outcomes of citizen petitions that address public health concerns. We found that most petitions were filed by manufacturers against other manufacturers. Only 346 (18%) of all petitions were submitted by individuals and non-profit organizations, and 178 (87.3%) of these petitions with a final response were denied. On average, these petitions required 2.85 years for a final agency decision, and many decisions remain pending 10–13 years after their initial submission. The great majority of the approved requests included some form of risk communication, such as labeling changes, boxed warnings or placement of a drug into a Risk Evaluation and Mitigation Strategy. As a policy instrument to improve the safety of medical and food products, the citizen petition process requires sophisticated legal and scientific expertise, and may not represent a viable route for ordinary citizens to petition the FDA to “redress grievances.”
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Brand-name drug companies have been filing citizen petitions with the Food and Drug Administration expressing concerns about generic drugs under review, in last-ditch efforts to hold off competition. A promising solution lies in erecting procedural blocks.
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The First Amendment to the US Constitution guarantees “the right of the people...to petition the Government for a redress of grievances.” When it comes to the regulation of drugs and protection of public health, individuals have the right to address their concerns by directly petitioning the US Food and Drug Administration (FDA). Any person (including a non-US citizen) can request that the FDA “issue, amend, or revoke a regulation or order or take or refrain from taking any other form of administrative action.” Although healthcare professionals rarely submit such petitions, they can exert a powerful impact on the labeling requirements for drugs. Metformin is one such example. Metformin is widely accepted as the first-line drug for the treatment of type 2 diabetes mellitus. It effectively lowers hemoglobin A1c levels by 1% to 2% and is weight neutral, safe, and inexpensive. Moreover, one trial demonstrated that it reduces cardiovascular disease complications in patients with newly diagnosed type 2 diabetes mellitus. When metformin was first approved in 1994, it was contraindicated in patients with heart failure and in those with elevated creatinine levels because of concerns about lactic acidosis. This restriction on drug use usually necessitated a switch from metformin to a glucose-lowering agent in a different category, one that frequently carried other risks (such as hypoglycemia), appreciably increased cost, or both. In 2006, the FDA eliminated the heart failure contraindication in response to 2 observational studies.1 These studies suggested that metformin is safe and may actually confer mortality benefits in patients with heart failure.1 However, the contraindication in patients with elevated creatinine levels remained unchanged. Since then, concerns over lactic acidosis have been examined and found to be largely unfounded unless kidney disease is advanced. On the basis of the available data, metformin can be used safely …
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In recent years, brand-name drug companies have engaged in an array of conduct that has delayed generic competition. While some of the activity — such as settlements between brand and generic firms and “product hopping” from one drug version to another — has received attention, another behavior has, until now, flown under the radar. This Article examines the activity of “citizen petitions.” A citizen petition is a request for the U.S. Food and Drug Administration (FDA) to take an action such as evaluating a drug’s safety or effectiveness. When used appropriately, it could raise awareness of legitimate concerns with a drug. But when used inappropriately, it could extend the brand firm’s monopoly by delaying FDA approval of generic drugs. This delay could result in literally millions of dollars a day being transferred from consumers to drug companies. Despite their delaying potential, citizen petitions have not been examined in significant detail. This Article offers the first empirical study of citizen petitions, reviewing every petition filed with the FDA between 2001 and 2010. It finds that petitions have increased in the past decade and that 68% of petitions are filed by brand companies, with more than 75% of brand petitions targeting generic drugs. The study concludes that the FDA has granted 19% of citizen petitions and has denied 81%. It finds that generics’ petitions are more successful, with 28% granted and 72% denied, as compared to brands’ petitions, of which 19% are granted and 81% denied. To reduce delays from petitions, Congress enacted legislation in 2007 that required the FDA to rule on certain petitions within 180 days. This study finds that this legislation has not been successful in reducing the number of petitions. After passage of the legislation, the average number of filings per year increased from 27 to 34. Brand petitions against generics increased from 9 to 16 per year. And the grant rate for brands’ petitions against generics declined from 20% to 19%. Building on the empirical study, the Article highlights the incentives brand firms have to file petitions, along with the role petitions play in the toolbox of delaying conduct. It also introduces examples that demonstrate the problem, such as the petitions delaying (1) depression drug Wellbutrin for 133 days at a cost to consumers of $600 million and (2) insomnia drug Ambien for 1225 days at a cost of $3.1 billion. The landscape in the pharmaceutical industry today is ripe for petitions, with an impending “patent cliff,” declining drug-company profits, and increased use of related conduct such as brand-generic settlements and product hopping. As a tool that is being used more frequently and that has evaded attempts to limit its abusive potential, citizen petitions warrant scrutiny.
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To the Editor: The immunomodulatory drugs thalidomide and lenalidomide recently received US Food and Drug Administration approval for the treatment of multiple myeloma.¹- 4 Venous thromboembolism (VTE) is a potentially serious complication associated with their use.⁵- 8 We conducted a systematic review of thromboembolism occurring with immunomodulatory drug treatment of cancer.
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Thalidomide- and lenalidomide-associated thromboembolism among patients with cancer
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