EU regulator wants more advanced therapies

Europe’s regulator is looking for ways to stimulate the development of advanced therapies, after only a handful of such products reached the market in eight years since the introduction of specialist regulations.

Rules on developing advanced therapy medicinal products (ATMPs) – including gene therapies, tissue engineered products and individually tailored cell therapies – have been in place since 2008, but only five such products are currently authorised.

One ATMP has been withdrawn and another has had its EU licence suspended, although GlaxoSmithKline's Strimvelis for a very rare genetic immune disorder, ADA-SCID, is likely to receive a European marketing authorisation soon following backing from regulators in April.

The European Medicines Agency said that industry representatives, regulators, academics and doctors met to discuss ways to encourage innovation while safeguarding patients and ensuring access to new therapies.

A patient representative, a doctor and a representative of NICE also attended the meeting in late May.

The stakeholders discussed how to encourage R&D, optimise regulation, and how to move products from “hospital exemption” status, where they are used without a licence in some member states, to full marketing authorisation.

Delegates also discussed ways to improve funding, investment and patient access, according to a report summarising proceedings.

Recurring themes included the need for early interaction and guidance from regulators and the need to tackle inequalities in patient access.

A filing for one ATMP product was withdrawn in 2013 – a knee cartilage repair product from Anika Therapeutics.

The marketing authorisation for another knee cartilage repair ATMP, MACI, has been suspended because manufacturer Aastrom closed its production facility in 2014 for commercial reasons.

EMA executive director, Guido Rasi, said: “We have organised this meeting with all relevant stakeholders to discuss concrete proposals on how we can nurture a regulatory environment that encourages development of ATMPs, safeguards public health and, ultimately, facilitates timely access for patients to much needed treatments.”

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