Through 4 January, the European Commission is seeking feedback on the extent to which legislation related to medicines for children and rare diseases is fit for purpose.
In particular, the EC is seeking feedback on whether the incentives associated with this legislation are sufficient for promoting the research, development and marketing of rare disease and pediatric medicines.
The purpose of the evaluation is twofold, the commission says: to assess the strengths and weaknesses of the two legal instruments separately and combined, and to give insight on how the various incentives that are related to the legislation have been used and the financial consequences.
“The evaluation will give a sound evidence base about the functioning of the two legal instruments from a public health and a socio-economic perspective that will be used to consider the possible need for any future changes,” the commission said, noting that it’s asking private citizens and healthcare professionals to share their experiences with and perspectives on access to orphan medicines in general, and on the role the EU orphan regulation plays in the development of orphan medicines.
Last October, the commission published a report on 10 years
of the Pediatric Regulation being in effect, noting that that the number of completed pediatric investigation plans (PIPs) is clearly trending upward, and the proportion of clinical trials that include children also increased.
Drugmaker Shire noted in its comments that the Orphan Medicinal Products (OMP) Regulation has attracted investment in rare diseases as new therapies have increased from eight, before the adoption of the OMP Regulation, to 143. In addition, the EU Pediatric Regulation, based on a set of obligations and rewards, has proven beneficial, with the authorization of over 230 new pediatric medicines since its inception.
“However, patient access to orphan drugs, which is dependent on the individual national pricing and reimbursement mechanisms, remains a challenge,” Shire said. “These patient access barriers at national level are complex and vary from country to country. For example, the average time to get funding is two years in England versus seven months in Germany. In addition, only about half of approved EU medicines have been funded in England - 68 OMPs have been reimbursed in the NHS, compared to 116 in France and 133 in Germany.”
Industry groups EFPIA and EuropaBio sought additional information on the EC’s intentions with this consultation, adding: “We would like to emphasise the vital importance of maintaining a favourable regulatory environment to maintain the momentum and continue to achieve progress in the fields of paediatric and orphan medicines.”
German industry group VFA also said that the legislation on medicines for children and rare diseases has been successful and, “There is no need to change these regulations.”