On the eve of the 10th anniversary of Europe’s Orphan Drug Regulation, the EMA undergoes a study of efficiency and efficacy
The European Medicines Agency (EMA) underwent a year-long evaluation process, conducted by one of the Big Four global auditors, Ernst and Young. Designed to assess the effectiveness and efficiency of the EMA, the evaluation, consisting of interviews, surveys, observations and case studies, shined a light into every corner of the EMA, examining the centralised and decentralised procedures of the agency. Working closely with EMA staff, National Competent Agencies (NCA), experts, industry, patient organisations and external stakeholders, recommendations to optimise the agency’s operations and strategies emerged around eight main topics: the organisation of the various EMA committees; NCA involvement in EMA work; the role of the EMA Secretariat; Procedures; Communication; Industry fees; Telematics; and Future challenges.
According to the audit, the EMA "appears to be a learning organisation that shows a permanent willingness to develop an ongoing improvement process. However the higher complexity and enlarged scope of responsibility and activities reveal some weaknesses associated with their specific risks. The system is progressively attaining its maximum capacity."
The Committee for Orphan Medicinal Products is singled out for praise...
The audit singles out the Committee for Orphan Medicinal Products (COMP) as a success: "Both the industry and other stakeholders tend to agree that the creation of COMP and related incentives have had a positive impact on research and development for specific products for orphan diseases. The procedure showed immediate success, with 83 submissions in 2001. This number has increased until 2005, when it stabilized around 120 submissions per year (with the exception of 2006). This coincided with a global increase and stabilisation in the number of authorised medicinal products for orphan diseases, with an average of 12.5 new medicinal products/year receiving approval from the CHMP for orphan diseases during the 2001-2008 period (range: 7-18, vs. only 2 in 2000)".
...although some words of warning are dispensed
The report observes that "... the careful consideration of whether a population can be considered as an orphan population may become a more complex issue in the future. Indeed, the trend towards the development of targeted therapies and personalised medicine could lead to more and more segmentation of patient populations into sub-populations. The rationale for such segmentation should be carefully monitored, as these subgroups may end up meeting the criteria for orphan status, while being sub-indication of a non-orphan disease. More applications of this type may lead to an increase of COMP’s workload in the near-future". Furthermore, the COMP’s "sustainability may be put at stake both because the system may not appropriately compensate NCAs for their involvement ... Although the current orphan products policy is unanimously recognized as having very positive outcomes, most stakeholders have expressed their concern over two subjects. First, some interviewees doubt the sustainability of a system that does not allow directly Rapporteurs and Co-Rapporteurs’ compensation and which budget has significantly increased in recent years. ... Second, although orphan medicines do reach the market more easily than they used to, their reimbursement is a raising issue at the national level. While this matter does not strictly enter the scope of the EMA, the unwillingness of national reimbursement bodies to pay for medicines that end up being very expensive and treating a very small population may on the long run undermine EMA efforts to provide all patients with new and accessible medicines".
Fostering access to authorised medicinal products in all Member States
While the EMA has contributed to the harmonisation of the EU internal market for medicines, the audit reports that "many stakeholders regret that medicines’ distribution falls out of the EMA scope. However, the industry provides already the EMA with some data about the distribution of authorised products according to the so-called "Sunset clause" (requirement for centrally authorised products to be placed on the European market within three years of the authorisation being granted). Monitoring such data with a look on the availability of authorised products in each Member State may allow the EMA to identify main weaknesses of the system. As pointed out in the first objective of the EC Communication on the future of the pharmaceutical sector, adopted on December 10th, 2008, this challenge may require political actions both at EU and Member States level: options to improve the availability of medicinal products for patients in need, with a particular focus on smaller markets should be developed in close cooperation with Member States by 2010".
The tenth anniversary of the European Orphan Drug Regulation was celebrated earlier this month in a two-day event that drew 120 participants from industry, research, government and patient groups - including delegates from the USA and Canada. (A full report of the 10th anniversary events will appear in the next issue of OrphaNews Europe). The audit report by Ernst and Young provides crucial information on how to shape the next ten years of orphan drug policies and practices and highlights the areas in need of particular attention.
Consult the Ernst and Young report