EFPIA, others question EC's proposed orphan and pediatric drug development updates

Regulatory NewsRegulatory News | 04 August 2021 |  By 

An initiative by the European Commission (EC) to boost the development of pediatric drugs and medicines for people with rare diseases has received stakeholder feedback that in many cases expresses concern with the proposed upending of the status quo.
A 60-day period of public consultation for the initiative’s roadmap closed at the end of July; in all, 112 comments were received from individuals, non-governmental organizations (NGOs), public authorities, medical societies, pharmaceutical trade associations and individual firms. About a quarter of the responses were from NGOs, while nearly half came from individual companies or business organizations, or from trade associations.
Existing EU rules for incentivizing drug development for children and for rare diseases are nearly 20 years old, notes the EC on its webpage for the initiative. The proposed revised rules modernize regulation of and incentives for drug development for these populations with three main principles to guide the roadmap to new regulations.
Fundamentally, according to the EC, the revisions to EU pediatric and orphan medicinal products regulations aim to ensure that “products addressing the specific needs of children and patients with rare diseases are developed.” The new rules will also help ensure that these groups can have access to medicines in a timely fashion, and that procedures for assessing and authorizing new medicines are efficient.
The European Federation of Pharmaceutical Industries and Associations (EFPIA) was one of the groups that responded during the consultation period. In its comments, EFPIA dubbed the current incentive framework “a great success,” advocating for the retention of current incentives.
The association expressed concern that reducing periods of market exclusivity and speeding generic approvals, some of the changes proposed in the roadmap, would be “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.”
EFPIA also emphasized the “complexity of the research ecosystem” in the EU; the group funded a report and worked up several case studies in support of its position that “broad incentives” should be maintained while adding targeted incentives that get at sticking points impeding drug development in certain areas. “Economic incentives are not the only solution to tackle the challenges in underserved areas for children and people living with rare diseases,” wrote EFPIA in its comment; the association advocated for a holistic approach that also looks at removing other regulatory and economic hurdles to drug development.
During an earlier period of consultation that closed in January 2021, EuropaBio’s comments stressed that any revamped EU legislation should have “the goal to build on the current success of the existing legislation and to encourage further investment in rare diseases in order to increase and accelerate innovation.” This association also argued against placing further limits on orphan designation or chipping away at current market exclusivity provisions. Neither should the EC make rewards conditional on market launch requirement, said EuropaBio.
Rather, suggested EuropaBio, the EC could consider an expansion of EMA’s PRIME designation to orphans and a boost in regulatory flexibility, in addition to easing early consultation and dialogue with EMA during the development process.
Even the generics manufacturer Teva wrote in January that it is “alarmed” over some of the options discussed in the roadmap, “which seem to risk destabilising the whole ecosystem rather than looking at how it can be improved.” Teva made clear that any “proposed modulation of market exclusivity under the Orphan Regulation,” in particular, could introduce so much uncertainty that research and development efforts might be driven away from the EU.
Teva made the case for tailored incentives for generic and biosimilar products, since those companies also must weave through a complex regulatory landscape when seeking marketing authorization for orphan and pediatric medicines.
The EC is expected to adopt the roadmap by the first quarter of 2022. However, some firms, including Teva, expressed the opinion that proceeding apace with that schedule would be inadvisable; instead, Teva called for a structured dialogue under the format of a High Level Forum.
EC initiative


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