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

Abstract

Background: 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.

Price increase negotiations to address drug shortages in South Korea’s national health insurance

“…When categorized by disease characteristics, non-EDs and non-ODs were more prevalent. While previous studies have emphasized the importance of drug shortages in acute, severe, and rare diseases ( Jarosławski et al, 2017 ), the findings of this study confirm that South Korea is addressing shortages for less severe and rare diseases through public measures. Furthermore, the application of the system and rate of price increase differed according to the therapeutic class (ATC code).…”

Characteristics and treatment of elemental mercury intoxication: A case series

“…The challenges in locating and obtaining chelators such as DMSA may be related to increases in prescription drug shortages in the United States. These drug shortages, which often affect the supply of orphan drugs used to treat rare diseases, have increased in recent years and represent a significant public health concern in the United States 35 . In this case series, DMSA was able to be obtained from local chain pharmacies.…”

“…These drug shortages, which often affect the supply of orphan drugs used to treat rare diseases, have increased in recent years and represent a significant public health concern in the United States. 35 In this case series, DMSA was able to be obtained from local chain pharmacies. The pharmacies did not carry it in their normal stock and had to order it as a "drop ship" from the manufacturer; this resulted in a 1-to 2-week delay in delivery to the patients.…”

Compounded medication for patients with rare diseases

“…Drug shortages for orphan (Pedea) and non-orphan drugs (Ammonaps) did appear [ 28 ] and compounding was necessary then to continue the life-saving treatment. For other drug shortages (Cerezyme, Fabrazyme) compounding was not possible because the active ingredient was not available and patients didn’t get their proper treatment as long as the shortage took.…”