EU Adaptive Licensing Program Moves Forward, With More Clarity for Sponsors

The European Medicines Agency (EMA) wants to make it easier for companies to use its adaptive licensing pilot program, a new effort to grant accelerated access to medicines intended for limited populations of patients with serious unmet medical needs.

Background

The adaptive licensing pilot program was first announced in March 2014, and is an attempt to move beyond clear-cut yes-or-no approval dichotomies, and to instead allow some drugs to be approved for limited populations before being approved for wider use.

The theory is that because populations suffering from conditions or diseases with unmet needs have a much higher tolerance for risk, these populations should be allowed earlier access to therapies that have not yet been fully proven. Those early approvals would in turn support subsequent and broader approvals by allowing sponsors to collect real-world use data, EMA postulated.

"With the adaptive licensing pilot project, we intend to explore with real medicines in development a progressive licensing approach that would allow timely access for patients to new medicines that address serious conditions with unmet medical needs," explains Hans-Georg Eichler, senior medical officer at EMA, in a statement. "The approach seeks to maximize the positive impact of new medicines on public health by balancing timely access for patients with the need to provide adequate evolving information on their benefits and risks."

As EMA explained, adaptive licensing is only for a narrow subset of prospective therapies. Drugs must be intended to meet an unmet need, must be supported by evidence in well-defined subpopulations, and must commit to further studies after licensing is granted. Companies wishing to participate in the pilot program were also asked to "outline a vision" of how payors, regulators and other stakeholders would work together throughout the product lifecycle.

In June 2014, EMA confirmed that it has received more than 20 applications for consideration, of which two were selected for inclusion in the pilot program.

New Q&A Guidance

Now, two months later, EMA says it has obtained enough "initial experience" from the program to draft and release a new "question and answer" document intended to "clarify the terms of engagement and expected outputs for prospective applications."

In particular, EMA explained, the document clarifies:

• the criteria used by the Agency to select suitable candidates for the pilot project
• the concrete steps involved in the process and expected outcomes
• the context and aim of the exploratory discussions between the Agency and selected candidates

For example, the five-page guidance explains it wants products which stand to benefit from an "iterative development pathway" and the collection of real-world monitoring data. The document also explains that it is meant to benefit early-stage development programs the most. Drugs that have already reached Phase III testing are "less likely" to be suitable for, or benefit from, the program.

The selection (or lack thereof) of a drug for the pilot program has no bearing on its chances for approval, EMA added.

EMA also indicated that it "continues to welcome applications from interested companies" for the program, "as it intends to include as many programs as necessary in the pilot to gather sufficient knowledge and experience." EMA is similarly interested in addressing scientific and technical questions and refining the program itself, it said.

Program findings will be formally evaluated at the end of 2014.

Q&A Document

EMA Statement