ObjectivesThe research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.This work aims at identifying orphan medicinal products that failed the developmental process and investigating reasons for and possible factors influencing failures.DesignDrugs designated in Europe under Regulation (European Commission) 141/2000 in the period 2000–2012 were investigated in terms of the following failures: (1) marketing authorisation failures (refused or withdrawn) and (2) drugs abandoned by sponsors during development.Possible risk factors for failure were analysed using statistically validated methods.ResultsThis study points out that 437 out of 788 designations are still under development, while 219 failed the developmental process. Among the latter, 34 failed the marketing authorisation process and 185 were abandoned during the developmental process. In the first group of drugs (marketing authorisation failures), 50% reached phase II, 47% reached phase III and 3% reached phase I, while in the second group (abandoned drugs), the majority of orphan medicinal products apparently never started the development process, since no data on 48.1% of them were published and the 3.2% did not progress beyond the non-clinical stage.The reasons for failures of marketing authorisation were: efficacy/safety issues (26), insufficient data (12), quality issues (7), regulatory issues on trials (4) and commercial reasons (1). The main causes for abandoned drugs were efficacy/safety issues (reported in 54 cases), inactive companies (25.4%), change of company strategy (8.1%) and drug competition (10.8%). No information concerning reasons for failure was available for 23.2% of the analysed products.ConclusionsThis analysis shows that failures occurred in 27.8% of all designations granted in Europe, the main reasons being safety and efficacy issues. Moreover, the stage of development reached by drugs represents a specific risk factor for failures.
Many tools have been developed in Europe to accelerate the access to and the availability of medicines. However, this is currently governed by the national Member State procedures for pricing and reimbursement. In many cases, this leads to procedures that often take many months or even years to be completed. This paper explores ways that would allow a more accelerated approach and thus enable a more efficient administrative procedure to be adopted. Therefore, this would favor the timely availability of medicines for severe diseases when an unmet medical need is present.
To promote, as well as protect, the public health is a key charge of the EMEA (European Medicines Agency). One tool to achieve the former is to provide scientific advice. The scientific advice and protocol assistance procedures have recently undergone revision, with new procedures coming into effect from July 2006; these are intended to allow the EMEA to handle the increasing workload effectively.The Scientific Advice Working Party (SAWP) has expanded to 26 members, and the procedure timeframe has been made shorter (offering 40-or 70-day procedures). The SAWP also now takes external expert input at an earlier stage. Parallel (i.e. co-ordinated) scientific advice from both the EMEA and the US Food and Drug Administration is also now possible and the initial experience with this is presented involving Reparixin (INN; Dompé pha.r.ma SpA).
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