The pharmaceutical and biotechnology industries reacted with enthusiasm and relief last week as the European Parliament approved at first reading a compromise package on the Advanced Therapy Medicinal Products Regulation, which aims to harmonise EU rules for somatic cell therapy, gene therapy and tissue regulation.

Early adoption of the regulation by the European Council now looks like a real possibility. The Commission regards the compromise package as “fully acceptable”, while the Council shares the Commission’s view that the proposal should not venture beyond the technicalities of harmonising the regulatory framework for advanced therapies across the European Union.

Barring a change of heart by EU ministers, the regulation should now come into force around the middle of this year. There would then be a year’s grace before the new provisions actually applied, giving the European Medicines Agency time to establish the multidisciplinary expert committee – the Committee for Advanced Therapies – foreseen in the Commission’s proposal and to fill the current gap in technical requirements for tissue engineered products.

What has calmed the frazzled nerves of the biopharmaceutical sector is that the compromise drafted by an alliance of the Parliament’s Socialist, Liberal and European United Left–Nordic Green Left groups, and adopted by a clear majority of MEPs on 25 April, overrode a late attempt to insert into the text ethical amendments drafted by the Parliament’s Legal Affairs Committee.

These had stipulated that the regulation should apply only to “products made of cells for which marketing is feasible in the near future and which do not raise major controversies”. Specifically, they would have excluded from the new rules, on ethical grounds, “advanced therapy medicinal products that contain or are derived from human embryonic or foetal cells, primordial germ cells or cells derived from those cells”.

A level playing field proposed

The intention of the Commission and Council was that any ethical qualms about issues such as deriving advanced therapies from human embryonic stem cells should fall under the subsidiarity principle and be left to the discretion of the member states. This was the position backed by industry associations and patient organisations eager to see a level playing field for advanced therapies in the EU.

But a last-minute reversal in late March threatened to scupper hopes of a relatively uncontroversial regulation going through Parliament on a first reading. Miroslav Mikolášik, the main parliamentary rapporteur for the regulation, announced he was terminating informal discussions between the Commission, the Parliament and the Council on reaching a first-reading agreement as he insisted on the JURI Committee’s amendments being part of the negotiations.

The European Association for Bioindustries, EuropaBio, warned at the time that including these amendments in the compromise package would “cause certain products and technologies to remain unregulated, posing potential health hazards to European patients and disadvantaging Europe’s emerging Advanced Therapies sector”. Fundamental disagreement over ethical safeguards demanded by centre-right and Green MEPs had led to Parliament’s Environment Committee rejecting a first draft of Mikolášik’s report on the regulation in September 2006.

Long haul towards second reading avoided

In the event, Parliament’s adoption of the compromise package put forward by the PES-ALDE-GUE/NGL alliance on 18 April has put these ethical issues back in the hands of the member states and avoided what industry feared could be a long haul toward a second reading.

The relief felt by biopharmaceutical companies was summed up by EuropaBio, which hailed Parliament’s voted as “a big day for European biotech and for European patients. The Advanced Therapies Regulation will put an end to the bewildering patchwork of guidelines, regulations and procedures that exist today, where some countries have no specific framework at all”.

The main provisions of the Advanced Therapies Regulation as it stands are:

- A centralised marketing authorisation procedure for advanced therapies through the EMEA.

- A Committee for Advanced Therapies at the EMEA to assess data relating to these products and track scientific developments in the field.

- Tailor-made technical requirements for advanced therapies.

- Tougher requirements for risk management and traceability.

- Special incentives for small and medium-sized enterprises

These incentives would include early evaluation by the EMEA of quality and non-clinical safety data for SMEs developing advanced therapy products, independent of any marketing authorisation application.