We discussed in previous posts the Commission’s announcement at the end of 2020 of its new pharmaceutical strategy for the EU. One topic identified as in need of revision was the unmet medical needs in areas currently not within the scope of the legislation governing rare diseases and paediatric medicines.  We have previously discussed recent consultations and stakeholder engagements by the Commission to explore possible changes to the legislative regime in these areas, including the possibility of reducing the ten-year market exclusivity period for orphan medicines and changing the criteria for determining the rarity of a disease.

Following the responses to these consultations, in May 2021, the Commission launched its latest public Consultation on the proposed revisions to the legislation.  The Commission’s statement accompanying the launch asserts that its evaluation “revealed shortcomings in the current system concerning in particular the development of medicines in areas of high unmet need for patients and their accessibility to all EU patients across the Member States.” Industry bodies representing the innovative pharmaceutical industry have now published their responses to the Consultation, as summarised below.


The European Federation of Pharmaceutical Industries and Associations (EFPIA) published its response on 29 July 2021, accompanied by a report by Dolon (a strategic pricing and market access consultancy specialising in rare diseases) and supporting illustrative case studies.  EFPIA considers those living with rare diseases have greatly benefited from the progress achieved since the regulations were introduced, and that the current incentives under the two Regulations are working well.

Commenting on the Commission’s proposed changes, EFPIA states:

“aiming to reduce the period of market exclusivity, narrow development incentives, promote faster generic competition and cap the aggregate number of patients that can benefit from a designated orphan product, is likely to drive research investment away from meeting the needs of patients and weaken a sector at the very heart of EU competitiveness and service to citizens.”

In terms of boosting the development of medicines in these areas, EFPIA considers that assistance with R&D (where medicines under development can benefit from national and/or EU funding), assistance with authorisation procedures and additional post-authorisation incentives would all be effective.  When considering what other measures may help boost drug development, it considers there to be three main factors when deciding whether to invest in a disease area, as follows:

  • Scientific/clinical factors (e.g., disease knowledge, infrastructure, company expertise);
  • Economic factors (e.g., size of patient population, competitive landscape, time to market); and
  • Policy factors (e.g., IP incentives, regulatory, P&R frameworks).

EFPIA objects to any changes to the orphan medicine designation criteria, and did not agree with any of the Commission’s suggestions to help provide patients, including children, with better access to medicines and treatment for rare diseases, stating that the proposals “are ill-conceived and might be counterproductive as they do not take into consideration the spectrum of drivers that effectively impact patient access.”  


The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has also published a response to the consultation, citing two position papers it has published on the regulations. EUCOPE states that it was concerned that the Commission’s consultation presents “a narrow view of the process of pharmaceutical innovation and its benefits to patients…” and noted, with particular concern, the suggestion of linking incentives to product availability.  This, it considers, would “affect disproportionately small- to mid-sized companies and restrict[…] both designation and incentives for rare diseases medicines development.”

In relation to the Orphan Regulation, EUCOPE suggests that the R&D ecosystem needs to be strengthened by focusing on the support for pre-clinical and clinical research.  It calls for a broad, criteria-based approach to designation (rather than the Commission’s proposal of defining unmet need) with the current prevalence threshold remaining the main criterion, and criteria for incentives that go beyond the absence of any approved therapeutic option.  Concurring with EFPIA’s response, EUCOPE considers that the concept of “significant benefit” should remain a cornerstone of the Regulation, and that rewards, in addition to market exclusivity, should be carefully designed to incentivise developers.

With regard to the Paediatric Regulation, EUCOPE supports steps to actively stimulate paediatric R&D and calls upon the Commission to consider different ways to improve PIPs within the current legal framework, for example suggesting that the PIP should be a high-level plan with only some basic elements agreed upfront. It considers a broad, criteria-based approach to designation is needed (again, rather than defining unmet need in the Regulation) taking into account the differences across the spectrum of paediatric needs. In the same vein as EFPIA’s opinion, EUCOPE considers that the current incentive system has “significantly contributed to fostering the development of paediatric therapies”, citing the revision as “an opportunity to enhance the predictability and attractiveness of the incentive’s framework for children’s medicines.”

Other Responses

The responses to the Consultation have not yet been published. However, it has been reported that The Alliance of Regenerative Medicines (ARM) agrees that a definition of unmet medical needs need not be included in the Regulations, stating that “Disease severity, burden of illness, impact on quality of life of patient/family are also important factors”.

The Commission is due to publish stakeholders’ responses to the Consultation on its website shortly and intends to publish its proposal for new legislation in the first quarter of 2022.