European project for flexible drug approvals moves up a gear

* EMA adaptive pathways pilot scheme enters second stage

* First face-to-face meeting to discuss a new drug April 7

* Aim is to speed initial access in small patient groups

LONDON, April 1 (Reuters) - A European project to streamline drug approvals moves up a gear next week when regulators sit down with the first company to have an experimental product assessed under a new flexible pilot scheme.

Patients are impatient to access novel treatments and regulators on both sides of Atlantic are trying to evaluate promising medicines more swiftly than in the past.

In the United States, the Food and Drug Administration has introduced a successful “breakthrough” designation, which has speeded up approval of innovative drugs for cancer and other serious diseases.

The European Medicines Agency (EMA) is going a step further with its “adaptive pathways” initiative, which foresees early approval of drugs for restricted patient populations based on small initial clinical trials.

Approval would then be expanded progressively, based on additional studies. Alternatively, the medicine could be pulled from the market if new data throws up unexpected problems.

Proponents of the scheme acknowledge there will be concerns about lowering evidence standards, but they argue the alternative is to ignore progress in medical science and deprive patients of the early benefits of new medicines.

“I think this is a way of working that is applicable to many drugs,” said Tomas Salmonson, chairman of the EMA’s influential Committee for Medicinal Products for Human Use (CHMP).


Fifty eight experimental drugs have been submitted as candidates for the EMA’s adaptive pathways pilot project, first unveiled a year ago, and eight have been selected for the second stage.

This involves face-to-face meetings to examine the evidence needed for individual projects and includes input from health technology assessment bodies, such as Britain’s National Institute for Health and Care Excellence (NICE), which decide if new drugs offer value for money.

The EMA declined to identify specific drugs or companies involved but an official said the first stage II meeting on April 7 would concern an advanced therapy product, meaning it is a medicine based on genes, cells or tissue engineering.

Other drugs selected to go forward include ones for cancer and rare diseases, developed by small and large firms.

At its heart, the new adaptive system tries to strike a balance between speed and safety, while recognising that evidence develops continually as clinical data builds up.

“Regulators, payers and society at large will have to become more sanguine with levels of uncertainty over an initial time period,” EMA senior medical officer Hans-Georg Eichler and other scientists wrote in the journal Clinical Pharmacology and Therapeutics last month.

“However, acceptance of uncertainty must be counterbalanced by a realistic, transparent and pre-agreed pathway for continued evidence generation.” (Editing by David Clarke)