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Regulatory News | 06 June 2014 | By Alexander Gaffney, RAC
In March 2014, the European Medicines Agency (EMA) announced that it would soon launch a new pilot program intended to grant "adaptive licenses" to medicines, permitting patients with unmet needs to access the medicines far earlier than they would otherwise.
Now EMA has announced that the program is up and running, and that regulators have already accepted the first two medicines into the program, with another four waiting in the wings.
EMA's adaptive licensing pilot project 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."
A similar framework was launched in March 2014 by the UK's Medicines and Healthcare Products Regulatory Agency (MHRA).
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.
Payors, in particular, may prove to be one of the biggest stumbling blocks for the program. Drugs approved by EMA on a limited basis would still need to be paid for, either by patients, companies or government payors who often want evidence of the drug's value before agreeing to reimburse for a drug. Failure to obtain buy-in from payors might result in drugs that are technically available to patients, but realistically not.
Some industry analysts have also questioned how market exclusivity provisions might function under the program, noting that an approval under an adaptive license might trigger the start of a market exclusivity period typically only started when full approval is obtained. This might lessen incentives to participate in the program, some have postulated.
But concerns aside, EMA says it's had ample interest in the program. Twenty applications were sent in for the pilot program, of which nine were given in-depth consideration. Of those nine, two were selected for the program, with another four awaiting further consideration in the future.
The other 11 drugs not given in-depth consideration may also be considered in the future, EMA wrote.
The owners of the two drugs selected for the pilot program, however, are still under wraps. EMA said they are now in the process of contacting the two sponsors "to explore how adaptive licensing can be developed for the specific medicines."
"We are very pleased to have received this number of applications in a short period of time," said Eichler. “The projects submitted came from very early responders, and we know that more proposals are on their way.”
Tags: adaptive licensing, adaptive licenses, adaptive licensing pilot program