Supporting academic and non-for-profit champions to repurpose medicines
28.10.21
EFPIA, together with other industry trade associations, patient groups and regulators from across Europe, led by the Expert Group of the European Commission on the Safe and Timely Access to Medicines for Patients (STAMP), has developed a proposal for a framework to support not-for-profit organisations and academia in the repurposing of authorised medicines.
The chair of EFPIA’s Regulatory Strategy Committee Alan Morrison (MSD) commented the launch of the repurposing pilot as follows; “This pilot launch comes at a timely moment to test whether a streamlined and more transparent regulatory pathway for repurposing of off-patent established products increases the chances of including existing scientific evidence into regulatory assessment. One of the goals of the pilot is to raise awareness regarding the standards required for regulatory-ready evidence on the road to further increase availability of authorised therapeutic use.”
To test this framework, EMA and several National Competent Authorities in EU Member States are launching a pilot project on 28 October 2021. EFPIA has been part of designing the pilot framework and is part of STAMP’s Repurposing Observatory Group.
“Repurposing” is the process of identifying new uses for existing drugs/active substances in indications outside the scope of the original indication. A new use will usually be supported by clinical data and this evidence may or may not be ready for submission to authorities. In cases where the data is submitted to a competent regulatory authority and meets the standards for efficacy, safety and quality, the medicine will be granted a new indication for that use following the normal regulatory process initiated by the Marketing Authorisation Holder through a variation procedure.
Normally, a Market Authorisation Holder (MAH) initiates variations, line extensions or submits a new medicinal product application with the repurposed indication. However, in the case of repurposing, there are often little or no commercial incentives to carry out further research. A not-for-profit party may be more oriented to researching the products that are off-patent and out of regulatory data protection. However, although involved in generating data, not-for-profit organisations may lack the expertise and resources to navigate regulatory routes and requirements, including finding the relevant MAH of the finished product with whom to collaborate in order to bring the indication “on label”. The pilot helps in this regard, but it will still be MAH’s responsibility to take the last step and submit the variation application for a new use. There may be ways to streamline this step in the future.
The goal of the pilot project proposal is to facilitate the regulatory recognition of new indications. It applies to well-established, authorised medicines that are adequately supported by a data set (e.g. preclinical data, safety data, clinical efficacy data, real world data). The pilot project proposal pertains to medicines that are out of the data protection periods and in cases where the MAH is not taking the lead in applying for a new therapeutic use for the medicine.
The innovative industry looks forward to participating in this pilot and to assessing, together with other stakeholders, the outcome once the report has been published, with a mind to increasing availability of authorised therapeutic use for patients who need it.
Further information is available on the EMA’s website.