EMA ramps up adaptive pathways pilot
pharmafile | December 23, 2014 | News story | Manufacturing and Production, Research and Development, Sales and Marketing | EMA, adaptive pathways, drugs, regulator
Six medicines have been selected to move to the next stage of the EMA’s adaptive pathways pilot project, to explore new ways to give patients faster access to new drugs.
The EMA launched the trial in March, to identify candidate drugs and manufacturers to take through an adaptive pathway to licensing. The ultimate aim is to find drugs that treat serious conditions with an unmet medical need, and reduce the time to a medicine’s approval.
The Agency received 34 applications from companies to include their medicines in the adaptive pathways pilot project: six for advanced-therapy medicinal products, 12 for orphan medicines, and 14 for cancer drugs.
The successful companies will now develop their proposals, including setting out their plans for health technology assessments and acquiring real-world data on their drugs. The EMA’s report on the first phase of the pilot acknowledges that the drugs “are all cases where the development pathway and the value decisions present difficult questions”.
The EMA is also working on ways to “discuss general issues such as opportunities and hurdles to the implementation of the adaptive pathways paradigm in the different public bodies”. In the report, the Agency identifies issues including patient input and earlier involvement with health technology assessment bodies as important lessons learned that need to be addressed in the second phase.
Earlier in December a research paper published jointly by researchers from the EMA, industry the MHRA and patient groups concluded that adaptive pathways could be ‘transformative’ for the industry, patients and healthcare systems, but only if there is substantial pressure and the political will to overcome regulatory barriers.
The pilot aims to help develop an understanding of how future adaptive pathways might work for different drugs. So far the EMA says there are two scenarios under which adaptive pathways may work: granting approval for a well-defined, high medical need subgroup, and then subsequently widening the indication to include a larger patient population; or granting an early or conditional approval – for example on the basis of surrogate endpoints – and then collecting data post-approval and upgrading to ‘full’ approval once more data are available.
Lilian Anekwe
Related Content
Rethinking oncology trial endpoints with generalised pairwise comparisons
For decades, oncology trials have been anchored to a familiar set of endpoints. Overall survival …
NICE issues positive final guidance for treatment of Duchenne Muscular Dystrophy from Santhera
Santhera Pharmaceuticals has received positive final guidance from the National Institute for Care and Excellence …
Eplontersen recommended for EU approval by CHMP for treatment of hereditary transthyretin-mediated amyloidosis
Wainzua (eplotersen), produced by AstraZeneca and Ionis, has been recommended for approval in the European …
