Quantifying the persisting orphan-drug shortage public health crisis in the United States

Szymon Jarosławski,Chiraz Azaiez,Daria Korchagina,Mondher Toumi

doi:10.1080/20016689.2017.1269473

Szymon Jarosławski, Chiraz Azaiez + Show 2 more

Open Access

https://doi.org/10.1080/20016689.2017.1269473

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Abstract

ABSTRACTBackground: Orphan drugs (ODs) are pharmaceuticals manufactured for rare conditions that affect less than 200,000 people in the US. ODs are therefore produced in small quantities to meet sparse demand. Since 2010, OD shortages have become frequent, but no comprehensive, quantitative studies exist.Objective: The objective of this study is to assess the rates of OD shortages per therapeutic class and their trends over time in the United States.Study design: OD approvals were collected from publicly available information on the US Food and Drug Administration (FDA) website on 13 June 2016. Data on OD shortages were collected from the FDA and the American Society of Health-System Pharmacists (ASHP) websites. We reviewed the number of shortages per year and per therapeutic area. Multiple indications for the same drug were counted individually.Results: Of 569 ODs approved, 50% were approved in the decade ending in 2015. Oncology was found to be the most represented therapeutic area (34% of all OD approvals), followed by endocrinology (11%). Shortage data were available from 2008. In total, there were 66 (12%) OD shortages, with an average shortage duration of 455.5 days. Shortages were observed mainly for oncology products (19 cases, 13% of oncology ODs) and endocrinology products (14 cases, 22% of endocrinology ODs)Conclusion: Despite the FDA strategic plan for preventing and mitigating drug shortages (October 2013), remaining OD shortages still pose an enduring challenge to patient care, with a median shortage duration of almost 15 months. In many instances, ODs are the only available therapy for rare diseases, and OD shortages can lead to serious health deterioration and death. More research is needed to elucidate the causes of shortages and their impact on patients’ health.

Highlights

The US Food and Drug Administration (FDA) defines a drug shortage as a situation in which the total supply of all clinically interchangeable versions of an FDA-regulated drug is inadequate to meet the current or projected demand at the patient level [1]
Orphan drug (OD) shortages are of special concern, since they are used to treat rare diseases affecting less than 200,000 people in the US [4]
Five hundred and sixty-nine ODs were approved by the FDA since the provision of the Orphan Drugs Act in 1983

Summary

Introduction

The US Food and Drug Administration (FDA) defines a drug shortage as a situation in which the total supply of all clinically interchangeable versions of an FDA-regulated drug is inadequate to meet the current or projected demand at the patient level [1]. New and ongoing drug shortages are a serious public health issue in the US. Orphan drug (OD) shortages are of special concern, since they are used to treat rare diseases affecting less than 200,000 people in the US [4]. Due to a special period of market exclusivity granted to OD manufactures by the FDA, one company typically produces the drug’s entire supply. Manufacturing interruptions or discontinuances of ODs result in patients being deprived of the only available treatment. When the company had to close its manufacturing plants due to quality issues, patients relying on the company’s OD suffered serious health consequences, including death [5]. Orphan drugs (ODs) are pharmaceuticals manufactured for rare conditions that affect less than 200,000 people in the US. Since 2010, OD shortages have become frequent, but no comprehensive, quantitative studies exist

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