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The European Commission's plans to evaluate the EU pediatric and orphan drugs regulation in tandem has prompted questions from the pharmaceutical industry on how exactly this critical exercise would proceed and what its ultimate objective would be.


The EU pharmaceutical industry is looking for more clarity on the European Commission's roadmap for the joint evaluation of the EU pediatric and orphan drug regulations; the evaluation exercise aims to assess why the two pieces of legislation have together failed to improve the availability of medicines for rare pediatric diseases.


Clarity is needed not just on the "Commission's intentions regarding the ultimate objective of this joint evaluation," but also on the "methodology and on the [data] sources that will be used in line with the stated objective [of the roadmap]," the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Association for Bioindustries (EuropaBio) said in their joint written response to the roadmap.


The commission's joint evaluation of the EU Paediatric Regulation (No 1901/2006) and the Orphan Drugs Regulation (No 141/2000) is slated to commence in the first quarter of 2018 and end in the third quarter of 2019. The roadmap for the project was published late last year. (Also see "Combined Effect Of EU Pediatrics And Orphan Drugs Legislation Under EU Commission Scrutiny" - Pink Sheet, 14 Dec, 2017.) Industry has long regarded the Paediatric Regulation as burdensome and overly prescriptive; once the results of the joint evaluation are available, the commission will decide on whether or not to propose any legislative amendments to this legislation. (Also see "‘Not What We Expected,’ R&D Industry Says Of ‘Onerous’ EU Paediatric Regulation" - Pink Sheet, 12 Jul, 2017.)


EFPIA and EuropaBio have cautioned against "too much generalization" and said that both regulations had successfully stimulated the development of medicines for special populations, with each addressing specific development challenges. The groups emphasized the "vital importance of maintaining a favourable regulatory environment" to maintain the momentum and for continued progress in the fields of pediatric and orphan medicines.


Specific Issues

In terms of specifics, the two industry groups want the final objective of the joint evaluation to be more precisely stated in the roadmap.


EFPIA and EuropaBio are also seeking further explanation on the commission's plan to undertake another study on the orphan drugs legislation in 2018. Specifically, industry wants to know the anticipated scope of this additional study, whether a separate consultation process is foreseen on its scope, what remaining research questions it would address, and what methodology and sources for data gathering would be used for the study. In addition, they want clarity on how the joint evaluation exercise will fit in with commission's existing study on the "Economic impact of the Paediatric Regulation, including its rewards and incentives," which was published last year.


Regarding the commission's plan to hold targeted consultation with EU member states and specific interest groups, industry wants to know which aspects of the joint evaluation exercise would be explored in more depth. To fully capture the picture of enablers and barriers to addressing unmet needs in special populations, EFPIA and EuropaBio recommend that the commission should also consult with financial investors in the industry.


With respect to the financial and cost-benefit aspects of the joint evaluation, the groups say it is important to understand the overlap with the work already done by the commission for its 10-year report on the Paediatric Regulation published last year. (Also see "No Amendments Any Time Soon For EU Paediatric Regulation, Says Hotly Awaited Report" - Pink Sheet, 26 Oct, 2017.) In particular, EFPIA and EuropaBio are interested in knowing how data will be gathered in an appropriately objective way, and duplication of pediatric-related work would be avoided.


The commission has said the two legal instruments were being jointly evaluated because they were both intended to redirect investment in previously neglected therapeutic areas through a scheme of incentives, rewards and obligations.

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