Yet Another European Approach to Early Drug Access

Rising concerns among Europe's healthcare payers about the additional costs of innovative medicines are doing nothing to stem the tide of initiatives to speed new products to the market.

The latest new scheme is the European Medicines Agency's ‘PRIME’ (short for 'PRIority Medicines'), launched on March 7 "to strengthen support to accelerate medicines that target an unmet medical need." It will offer early advice to medicine developers so that they have the best chance of producing robust data on benefits and risks, and allow more rapid assessment. Improved clinical trial designs should ease data generation and the evaluation of applications for marketing authorization, and early dialogue will also serve to boost patient participation in trials and make best use of limited resources, says EMA in its announcement.

The scheme focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options-medicines formally considered priority medicines within the European Union. It builds on existing EU regulatory tools, and will take advantage of the shorter timeframe envisaged for decisions on medicines for unmet needs that have been evaluated under an accelerated assessment procedure.

“Our goal is to foster better planning of medicine development," said Guido Rasi, EMA’s Executive Director, so that patients can "benefit from scientific progress and cutting edge medicines as soon as possible.” His theme was echoed by Tomas Salmonson, Chair of the EMA's Committee for Medicinal Products for Human Use, who expects progress from closer collaboration among the (EMA's) scientific committees, and "by gaining and sharing knowledge on the medicine throughout its development."

To qualify for the scheme, potential must be demonstrated by early clinical data, and once selected, a medicine will receive attention from an expert appointed by the EMA who will help build knowledge ahead of a marketing authorization application, organize meetings with relevant EMA committees and working parties, as well as with health technology assessment bodies, and will mentor throughout the development process.

This arrangement is made in agreement with the European Commission, which is legally responsible for marketing authorization decisions in Europe's tangled drug control system. Vytenis Andriukaitis, European Union Commissioner for Health, gave his blessing to PRIME as "a major step forward for patients and their families that have long been hoping for earlier access to safe treatments for their unmet medical needs."

In fact Andriukaitis is viewing it as something of a panacea for many current ills. He is looking forward to the enhanced scientific support of PRIME to help "accelerate the development and authorization of new classes of antibiotics or their alternatives in an era of increasing antimicrobial resistance." He also sees it as "a potential godsend for those suffering from diseases for which there are currently no treatment options"-and particularly rare cancers, Alzheimer’s disease and other dementias.

And in line with the technocratic strategy that dominates current thinking in Jean-Claude Juncker's Commission, PRIME also wins points from Andriukaitis because "it optimizes the use of the current regulatory framework that can contribute to the European Commission's priorities in terms of boosting innovation, jobs, growth and competitiveness.”

What sort of welcome the scheme will obtain from hard-pressed paying agencies around the EU's member states remains to be seen.