The OD Expert Group welcomes the objectives of the revision of the OMP Regulation

On 26 April 2023, the Europe Commission published the EU Pharmaceutical Package proposal, revising the OMP Regulation as part of the revision of the EU pharmaceutical legislation. The revision aims to improve the existing legal framework to ensure access to affordable medicines in the EU, foster innovation in areas of unmet medical need, and enhance supply security while adapting to new scientific and technological developments and reducing regulatory burden.

We welcome the objectives and the extensive efforts of the European Commission to the revision, as it represents a unique opportunity for improving the lives of people with rare diseases and supporting rare disease R&D and innovation in the EU. While several provisions represent a step in the right direction, other proposals risk not reaching their objective to address the existing gap of 95% of rare diseases without a treatment option.

We  suggest some recommendations on modulation of rare disease incentives to improve the revision of the OMP Regulation, based on our policy proposals:

  1. Modulation should be based on a combination of criteria

Modulation should not be based solely on a restrictive and static definition of (high) unmet medical need. The interpretation of what constitutes “(high) unmet medical needs” varies in content and has distinct meanings to different stakeholders. Rare diseases have complex and inconsistent clinical manifestations, which can change over time and between patients. Similarly, the needs of patients also evolve over time. Additionally, most rare diseases lack disease-modifying and curative treatments today. 

  1. Modulation of market exclusivity (ME)

Modulating market exclusivity (ME) refers to the graduation of the ME incentive to different groups of OMPs. We propose the following modulation of ME for the three archetypes we identified, differing in terms of their investment case:

  • Archetype 1: First authorised product for the treatment, diagnosis or prevention of a given rare condition. We propose an increase of the ME period to twelve (12) years. Two additional years of ME compensate for the high level of regulatory and market access uncertainty.
  • Archetype 2: Product based on new technology/mechanism of action for a given rare condition or addressing a population subset/clinical manifestation of a rare condition not sufficiently served by existing authorised products. We propose maintenance of the current ME period of ten (10) years. Maintaining the current period of ME will ensure continued innovation of OMPs that rely on ME as a key incentive, therefore avoiding under-incentivising development projects that would be beneficial for rare disease patients.
  • Archetype 3: Product based on an existing technology/mechanism of action for a given rare condition or addressing a rare condition with (for instance) 3 or more authorised treatments overall. We propose a reduction of the ME period to eight (8) years. A reduction of no more than two years will likely continue to drive the level of innovation that we see today, without under-incentivising development projects that would be beneficial for rare disease patients. 
  1. Legal certainty and predictability are key

Any modulation must provide legal certainty and predictability. This means that investors must be able to know before taking the investment decision which types of incentives a project will be eligible to and under which conditions. In addition, the innovation model of pharma companies is based on lifecycle management, in which multi-indication product innovation is a central element to decrease risk and a key facilitator of wider investment in OMP development. Thus, the OMP Regulation should continue to see subsequent indications as unique development projects, facing additional costs and risks.

  1. Modulate according to differences in the investment case

Modulation should be based on the investment case for launching research and development in a specific rare disease or disease area. The investment case approach enables identification of diseases areas where the investment case is either particularly weak or particularly strong given current incentive levels. condition/class of products, are main drivers of the investment case for OMPs. 

  1. Refining the criteria for and assessment of significant benefit (SB) 

In order to reduce uncertainty and improve predictability around significant benefit (SB), it is important that concepts and the scientific contents of the criteria for and assessment of SB are refined. Such refinement shall include:  the EMA and EUnetHTA to devise a standard method and criteria for indirect assessment for the demonstration of SB; recognition and acceptance of SB in the market access relative effectiveness assessments.

To find all the details on our proposals, you can read our full report: “Modulating Incentives for OMP Development: Modulation framework and policy proposals”.

Next steps

Following the Ordinary Legislative Procedure (OLP), the proposal will now be sent to, and discussed by, the European Parliament and the Council.

Download our Press Release

Posted on 11. May 2023