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OD Expert Group presents its policy recommendations for the revision of the OMP Regulation

On October 13, during the Rare Conversations Conference, our Experts presented the OD Expert Group’s policy proposals for a sustainable and resilient healthcare environment for rare diseases. These proposals are the result of intense collaboration among different stakeholders.

We were delighted to share with the rest of the rare disease community our prolonged and committed effort to elaborate a policy framework in light of the upcoming revision of the EU OMP Regulation. The framework elaborated by our Experts is aimed at improving the development of treatments for rare and ultra-rare diseases by creating a favorable incentive ecosystem to deliver therapies where there are none, but also to support innovative and improved treatments where treatments already exist.

By thoroughly analyzing the current European framework, the OD Expert Group realized the urgent need to improve the R&D ecosystem and the existing incentives, while ensuring more regulatory predictability. It is in this context that the OD Expert Group developed a set of policy proposals for a sustainable rare disease ecosystem, and presented them during the Rare Conversations Conference 2022. These recommendations elaborate on the 14 recommendations, previously issued in 2021, specifically:

  • Modulation of market exclusivity;
  • Strengthening EMA’s role in advising OMP developers through the OMP pathway;
  • Increasing legal certainty around the concept of Significant Benefit.

The principles on which these policy proposals have been developed are essential to ensure that efforts of modulating incentives for OMP development ultimately benefit rare disease patients. This operational framework for modulation includes:

  • The maintenance of broad Orphan Drug Designation;
  • A modulation of incentives in accordance with the differences of the investment case;
  • The avoidance of the use of unmet need definition to modulate incentives;
  • The key importance of legal certainty and predictability;
  • The need that incentives include and go beyond modulation of Market Exclusivity.

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Posted on 14. October 2022