Regulatory pathways

Finding ways to optimize and accelerate regulatory pathways for orphan drugs.

The OMP Regulation has incentivised investment in research and development of treatments for rare diseases. However, a large number of development projects are still abandoned along the development path.

Around 17 percent of orphan development projects reach market approval and even fewer succeed in pricing and reimbursement negotiations. This high attrition rate is due to both failures at different stages of the development process and abandonments.

Abandonments may in part be linked to the fact that regulatory pathways are slow, cumbersome and not flexible enough to accommodate all challenges of orphan drug development. This is particularly relevant for small companies with fewer funds and experience in gaining market access, for innovative therapies and treatments for ultra-rare diseases.

Simplifying and accelerating regulatory pathways for Orphan Drugs can contribute to reducing the perceived up-front risk of OMP development and decrease the share of abandoned or failed projects ultimately increasing the availability of treatments for rare diseases. This can be particularly beneficial to promote and facilitate the development of treatments for the 95 % of rare diseases without an approved treatment.

The Focus Group analyses the strengths and weaknesses of the current system and suggest measures to improve flexibility, predictability and speed of the regulatory pathways to incentivize development and access to OMPs for rare disease patients. It reviews how regulatory pathways can be optimized to accommodate the needs of the rare disease environment.

This includes the application of novel biomarkers in assessing drug efficacy, acceptance of new clinical trial designs and leveraging of the generation of Real World Evidence (RWE). Furthermore, it touches upon requirements in proving significant benefit, harmonization of national market access procedures as well as simultaneous granting of marketing authorization for orphan and non-orphan indications.