APME Position on Orphan and Peadiatric Regulations

The European Commission continues the evaluation of medicines for children and rare diseases. The Public Consultation can be found here:


APME (Association of Pharmaceutical Manufactures in Estonia) Position on Orphan and Peadiatric Regulations

Association of Pharmaceutical Manufactures in Estonia (APME) is committed through its’ national and international members to support the improvement of the regulatory environment in Europe and welcomes the opportunity to contribute to the revision of the pediatric and rare diseases legislation.

We share, in line with EFPIA statement, the Commission’s objectives to foster further biopharmaceutical development for children and patients suffering from rare disease, and to ensure faster and better accessibility of medicines across Member States.

We believe that the revision should not only address current bottlenecks but more importantly, should make the legislation future-proof and consider also future treatment options and modalities. We support the development of an environment which ensures that innovation is rewarded and leads to more effective treatment options for patients with rare diseases and children.

The important impact for patients with rare diseases as well for children is based on a few critical success factors in the Regulation that should not to be altered:

  • The current system of obligations and rewards in the Paediatric Regulation and of market exclusivity incentives in the Orphan Regulation have proven effective and need to be maintained. Above mentioned obligations and rewards have given manufacturers sufficient long-term predictability, to support the risk-based investment decisions for the development of new medicines, and align with the goals of the industrial strategy to boost Europe’s innovative ecosystem on the global stage. Attaching additional conditions to orphan or paediatric incentives would risk stifling the development of new treatments by removing the stability and predictability required by companies at the point of investment.
  • The current orphan designation criteria are predictable and have been effective to encourage the development of products for those diseases. Lowering these prevalence criteria risks taking away the incentives to develop treatments for those diseases and undermines research programmes where continuous research leads to new therapies for patients.

There are a number of policy measures that the European legislator can take to provide further therapeutic choices for patients:

  • Maintaining an appropriate incentive framework centered around IP protections, such as SPC and market exclusivity, complemented by specific additional incentives for areas of unmet need and rare diseases. Consider novel incentives such as a transferrable exclusivity voucher, to foster discovery and development of new, currently underserved therapy areas.
  • Allow for the company’s determination of the Paediatric Investigation Plan (PIP) condition to be based either on the (adult) condition, as currently, or another paediatric condition, when it is scientifically justified on the product’s mechanism of action. This would ensure that products for specific unmet paediatric needs can be developed with more efficacious ways.
  •  Improved efficiency of the regulatory framework for rare and paediatric diseases is welcome. This can be achieved by different mechanisms, as:
    • Reducing the number of review cycles for orphan designated drugs and the granularity of initial PIP commitments
    • Ensuring integrated regulatory processes by early transatlantic dialogue and supporting streamlined communications between the scientific committees and Member States via better connecting the Paediatric Committee (PDCO) and the Committee for Orphan Medicinal Products (COMP) to the Committee for Medicinal Products for Human Use (CHMP). This can ensure more predictability for companies developing orphan products, and help designing better paediatric developments plans alongside the adult development.

Hurdles to patients’ access to innovation cannot be solved solely through the review of the Paediatric and Orphan Regulations. Once new treatments are approved, we strive to make them available to patients, wherever they live.

Access is dependent on a number of mostly national and health-system-related factors and market dynamics that cannot be foreseen at the time of R&D investments. These include a complex interplay of factors, out of which many are outside of a company’s control, such as the level of regulatory requirements, differences in medical practices, the speed of pricing and reimbursement negotiations, etc[1].

Targeted access solutions will require a close collaboration of national healthcare systems (eg HTA and P&R bodies) on issues such as pan-European evidence generation for small and dispersed patient populations, meaningful joint clinical assessments, and novel pricing and payment models. Progress will require increased solidarity between MS.

[1] https://www.efpia.eu/media/554527/root-causes-unvailability-delay-cra-final-300620.pdf