European Commission evaluation of the medicines for rare diseases and children legislation
The European Commission (EC) made public its first comprehensive evaluation of two regulations, the orphan and paediatric legislation, respectively adopted in 2000 and 2006. The two evaluations were conducted under the Commission’s Better Regulation Guidelines, regarding transparency in designing and evaluating European Union (EU) policies and laws, taking into account the views of citizens and stakeholders. The evaluation of the mentioned regulations was carried out from a public health and socio-economic perspective, and included the publication of a roadmap, recent studies on paediatric medicines and on orphan diseases, and consultation of stakeholders. The two regulations were evaluated together as the majority of rare disease may have an onset in childhood, and many childhood diseases are rare.
The EC recalled in its evaluation the level of awareness in the 1980s and 1990s regarding the absence of access to treatments for rare disease patients and children in Europe. At that time, the pharmaceutical industry did not sufficiently invest in orphan drugs. In 2000, more than 100 million European children affected by a rare disease had no access to treatment, or the treatment was unavailable. Doctors had to treat rare disease paediatric patients with rare disease medicines for adults, with this leading to adverse reactions in some cases. The evaluation of the two regulations, Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products focused on weaknesses and strengths of the two regulations, and is based on five criteria: effectiveness, efficiency, relevance, coherence, and EU added value to the two regulations.
According to the evaluation, the two regulations have been beneficial in promoting the development and availability of medicinal products for patients with rare diseases and for children. This has been achieved by channeling investment into a previously neglected field through the known incentives, obligations and rewards set out in the legislations, enabling a strategy that could not have been achieved at Member State level considering the small number of patients affected by rare conditions. The number of available medicines for RD patients and children have increased and are more readily available. In addition, more clinical trials in children are conducted, meaning less ‘off-label’ use of adult medicines; the evaluation deems that Europe now has a ‘paediatric research environment’ that makes this possible.
The evaluation does note that the legislation has not yet been able to foster development in the areas where the need for medicines is most keenly felt, with a concentration of products in more ‘profitable’ therapeutic areas. It was also questioned whether the prevalence threshold of not more than 5 in 10000 patients is the adequate tool to identify rare diseases needing support in the development of medicines: consideration of personalised medicine may complexify the issue further. It was also highlighted that development of new medicines for children are still a result of adult’s needs, and specific measures to counter this are justified.
The results of the EC evaluation will help guide discussions on the legislative level, as well as the development of an affordable Pharmaceutical Strategy for Europe, making rare diseases medicines more accessible, with innovation support within the EU pharmaceutical sector. The evaluation noted that although incentives are still necessary to help bring medicines for RD to market, this market is, for some RD more and more similar to the market for non-orphan products. The need for specific provisions for orphan drugs, such as extended market exclusivity, may need to be reassessed in the future. The evaluation also found that the regulations have led to increased healthcare systems costs, although this is balanced or indeed outweighed by improved quality of life. It was also highlighted that not all patients in Member States benefit equally from the advances made by the two regulations, largely due to launch decisions, and the variation in Member States’ pricing and reimbursement systems. It is hoped that the findings, and a more global reflection on the pharmaceutical sector, will be help facilitate the key objectives of the Pharmaceutical strategy for Europe, that aims to put into place a ‘future-proof’ regulatory framework.
