[Ip-health] Politico articles on European Commission pharma incentives for pediatric, orphan drugs

Dimitri Eynikel Dimitri.Eynikel at msf.org
Thu Aug 13 08:23:01 PDT 2020

Dear all,

i hope everyone is enjoying a much needed summer break.
meanwhile the European Commission has published its evaluation of the orphan drug and pediatric regulation.

the commission has released
an external study https://www.politico.eu/wp-content/uploads/2020/08/final-report_orphan-regulation-study_en.pdf

A staff working document with the Commissions own conclusions https://ec.europa.eu/health/sites/health/files/files/paediatrics/docs/orphan-regulation_eval_swd_2020-163_part-1.pdf

-Announcement of an ex ante impact assesement for a possible legislative change to be published this fall

Below some politico articles on the topic.

best regards

Commission questions pharma incentives for pediatric, orphan drugs
Evaluation says market for some drugs has started to resemble standard medicines.
by Carlo Martuscelli
Aug 11, 2020, 8:34 PM
The European Commission on Tuesday opened the door to changing EU legislation on pharma incentives, a move that would potentially affect drugmakers’ profits on some blockbuster drugs if enacted.

In a staff working document, the Commission presented its first major evaluation of the rules governing drugs for children and for patients with rare diseases. While the assessment did not elaborate on any policy changes to come, it recorded a mixed verdict over the efficacy of the existing legislation.

“Both regulations have not adequately managed to support development in areas where the need for medicines is greatest,” the EU executive concluded, while noting that more of the medicines covered are reaching patients since the rules were passed.

The Commission said the market for some rare disease drugs has started to resemble standard medicines, putting into question whether the current 10 years of market exclusivity — and its associated costs to the public purse — are necessary.

“Any future response to the shortcomings and future challenges identified in this evaluation should strike a balance between incentives for innovation on the one hand, and availability and patient access (for orphan and pediatric patients) on the other,” the text read.

The EU’s orphan drug regulation was introduced two decades ago to foster the development of medicines for rare diseases, which drugmakers might otherwise ignore due to the small patient pool, through the use of exclusivity periods. These shield the manufacturing company from competition from generic medicines longer than normal.

The pediatric legislation dates back to 2007 and also provides for some special market protection.

Among the shortcomings identified, the Commission questioned whether the metric used to identify a rare disease — one affecting fewer than five in 10,000 people — is still working. It highlighted personalized medicine, which involves the identification of subgroups of patients using biological flags called biomarkers, as a complicating factor.

“While such developments may hold great potential for optimal tailoring of treatments to diseases, they should not lead to unnecessary multiplications of rare diseases out of common diseases, neither proliferation of exclusivity periods,” the Commission said.

In its announcement, the Commission said the result of the evaluation would inform any future legislative change, and that the next step would be to release an impact assessment in the fall.

Industry pushback
Many of the best-selling orphan drugs, like Imbruvica for lymphoplasmacytic lymphoma or Darzalex for multiple myeloma, treat rare cancers. Medicines such as these could see some of their special benefits curtailed if the EU changed its rules.
Lobbyists for branded drugmakers have already signaled their resistance to alterations in legislation.

Nathalie Moll, director general of the European Federation of Pharmaceutical Industries and Associations, said in a statement ahead of the evaluation’s release that the Commission should focus on non-legislative options when it comes to modifying the rules around drugs to treat rare diseases.

Responding to an external report by consultancies Technopolis Group and Ecorys, which fed into the Commission’s evaluation, Moll said that reopening the orphan drugs regulation “is not likely to address the core challenges regarding unequal access and availability of orphan medicines within the European Union.”

The European Confederation of Pharmaceutical Entrepreneurs, which represents smaller pharma companies, said that incentives for companies help “make it possible to develop new technologies to treat more rare diseases more effectively.”

However, Yannis Natsis, policy manager for access and affordability at the nonprofit European Public Health Alliance, said in an interview that there was a real problem with the current state of affairs.

“Most of these [drugs] don’t make it in most of the EU member countries, and that’s a huge problem,” he said. “We need to look at factors why … and also look at the effect of these pieces of legislation on the competition landscape.”

In a statement, the Commission said that it will publish a preliminary impact assessment on the topic in the fall.

Commission publishes external review of orphan drug legislation
by Carlo Martuscelli
Aug 10, 2020, 6:42 PM
The European Commission quietly released the final version of an external review of the EU’s orphan drug legislation, intended to inform its upcoming evaluation.

A draft version reported by POLITICO in July highlighted inefficiencies in the rules for rare disease drugs but stopped short of providing forward-looking policy recommendations.

The 400-page external report, written by consultancies Technopolis Group and Ecorys, is meant to inform a Commission staff working document on orphan and paediatric drugs, which was slated for release by early August.

The report was uploaded to an incomplete webpage on which the Commission is expected to publish the outcome of its own evaluation into the two regulations. A prior announcement, dated August 6, links to the page where the external review can be found but without the Commission’s own assessment.

According to the text, the Commission is set to conclude that “both Regulations fostered the development and availability of medicines for patients with rare diseases and for children,” but that access to these medicines is unequal across the bloc.

“Regulations have not adequately managed to support development in all areas of rare and paediatric diseases where the need for medicines is greatest,” it reads.

The Commission said it will examine its policy options to update the legislation. A first step will be drafting a preliminary impact assessment due in the fall.

“Any future solution will aim to address any identified shortcomings and foster patient-centred innovation as well as ensuring the availability of, and access to, medicines for patients with rare diseases and for children,” it said.


Dimitri Eynikel | Access Campaign EU Policy & Advocacy Advisor

Médecins Sans Frontières | Doctors Without Borders

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