Szymon Jarosławski

Design of patient access schemes in the UK

BackgroundPatient Access Schemes (PAS) are alternative market access agreements between the UK Department of Health and drug manufacturers. They are implemented to enable the UK National Institute for Health and Clinical Excellence (NICE) to recommend expensive medicines for use in the UK NHS.ObjectiveWe aimed to analyse the extent to which NICE drug appraisals influence the construction of PAS and what rationale underlies the variety of approaches to their design.MethodsWe analysed publicly available documentation on PAS developed as a part of the NICE Health Technology Assessment process.ResultsWe demonstrate how the design of PAS is determined by the kind of evidence that is available to model cost effectiveness of a drug and by the incremental cost-effectiveness ratio that is deemed acceptable in a given patient population. PAS aimed to reduce drug cost to the NHS by means of various discounts or rebates on a per-patient basis rather than by lowering the list price of drugs. While almost all schemes were proposed by the industry in reply to negative draft recommendations by NICE, motivations of the stakeholders to implement PAS are not disclosed in the publicly available documentation.ConclusionA more transparent process might be necessary to protect against a perverse impact of PAS on international reference pricing that uses list prices rather than the real cost of purchasing medicines that the NHS incurs.

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.

Non-profit Drug Research and Development at a Crossroads

In wealthy nations, non-profit drug R&D has been proposed to reduce the prices of medicines. We sought to review the ethical and economic issues concerning non-profit drug R&D companies, and the possible impact that their pricing strategy may have on the innovation efforts from for-profit companies targeting the same segment of the pharmaceutical market. There are two possible approaches to pricing drugs developed by non-profit R&D programs: pricing that maximises profits and “affordable” pricing that reflects the cost of manufacturing and distribution, plus a margin that ensures sustainability of the drug supply. Overall, the non-profits face ethical challenges - due to the lack of resources, they are unable to independently commercialize their products on a large scale; however, the antitrust law does not permit them to impose prices on potential licensees. Also, reduced prices for the innovative products may result in drying the for-profit R&D in the area.

Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration

ABSTRACT Background: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks. Objective: We sought to assess the usage of patient-reported outcome (PRO) claims in a comprehensive set of US FDA orphan drug approvals dated between 1/1/2012 and 31/12/2016, and characterize them. Study design: Orphan drug approval documentation was obtained from the US FDA website. Drug Package Inserts (PI) were analyzed to extract information on PRO-related language. Results: Among 178 drugs that met inclusion criteria, 16 (9%) products approved for 16 orphan indications contained PRO language in the Clinical Studies section of the PI. All PRO instruments concerned disease symptoms, and two also referred to patient functioning. The most common PRO instrument was a bleed-specific rating scale for four products approved for the treatment or prevention of bleeding episodes in patients with genetic bleeding disorders. Conclusions: There is a need to implement public incentives for academic development of PRO instruments for rare conditions and for regulatory policies that mandate the collection of PRO endpoints in pivotal trials of orphan drugs.