Actions tailored to support OD development process, based on other international experience and ideas to trigger collaboration between academia, patients, industry and regulators in Europe.
In comparison to the US, the OMP development landscape in Europe seems rather small. Although the number of available OMPs has increased significantly since the advent of the EU OMP Regulation, the number of OMP designations have decreased in Europe over the past four years, indicating a contraction in OMP development. In the US, this decline is not as severe.
The current pipeline for OMP products in the US is twice the size of the one in the European pipeline. Moreover, estimations predict that 67% of the worldwide orphan drug pipeline sales will originate from the US by 2024, with Europe and the rest of the world are lagging behind.
The differences in the level of OMP development activity between the US and Europe can be explained by differing price and market conditions, but may also result from a different incentive environment (tax credits and subsidies for clinical trials) and less stringent OMP designation criteria. For instance, in the EU, the designation criteria include lack of alternative treatments and demonstration of significant benefit, which are not requirements for the designation in the US.
While it is important to recognize the different economic and market conditions in the US and EU, it is worth investigating whether the EU can learn from the US in terms of OMP incentives overall and whether some measures in place in the US could help direct more R&D into areas of high unmet medical need.
The Focus Group reflects about measures to support the Orphan Drug development process. It works on proposals of further developing the European Orphan Drug Ecosystem to enhance patient access, increase EU competitiveness and create growth, including a more streamlined approach with the US.
The Focus Groups furthermore creates additional ideas to foster cooperation between academia, patients, industry and regulators in Europe.