Regulatory pathway

Need 3: Increase the flexibility and predictability of the OMP regulatory pathway

The regulatory pathway comprises the set of steps required for the regulatory approval of OMPs. Policymakers need to turn their attention to the unnecessary uncertainties and hurdles associated with the OMP regulatory pathway, which increase costs and time to market and discourage investment into important and demanded rare disease treatments.

Uncertainties in the regulatory pathway could also be a key causal factor behind the high attrition rate of OMPs at this stage of the development path.

Having a flexible regulatory pathway, which accommodates for the unique challenges of developing OMPs (e.g. lack of comprehensive evidence pre-authorisation), is important to ensure that innovative rare disease treatments can reach patients in a timely manner.

A predictable regulatory (and corresponding access) pathway, in which guidelines, requirements and expectations are clear and aligned, is important to maximise the benefits of the incentives provided by the OMP Regulation.

A lack of predictability over certain aspects of the regulatory pathway increases the risk of developing OMPs and thereby discourage investment.

The current regulatory pathway is not optimally designed to incentivise (fast) OMP development. Policy solutions need to include closer and more frequent collaboration between the EMA and OMP developers, clearer guidelines and higher certainty concerning the regulatory provisions and more flexible requirements for sub-groups

 The recommendations

  • Strengthen EMA’s role in advising OMP developers through the OMP pathway
  • Adopt guidelines on the use of alternative treatments (e.g. off-label use and pharmacy preparations) in the presence of approved OMPs
  • Increase the legal certainty around the concept of Significant Benefit
  • Adapt the regulatory pathway to the specificities of OMP groups with additional challenges