With over 1,000 currently ongoing clinical development programs for cell and gene therapies and tissue engineering worldwide, the biopharmaceutical industry looks to address the challenges associated with the clinical development of advanced therapy medicinal products (ATMPs).
During this year’s UK Bioscience Forum that took place in London, Annie Hubert, senior director of European Public Policy for the Alliance for Regenerative Medicine, spoke on a panel titled ‘Overcoming the barriers to cell and gene therapies’ about successfully bringing ATMPs to markets and healthcare systems that have not yet fully adapted to the newly-established therapies.
Speaking to Outsourcing-Pharma during the event, the executive said that the main strategic recommendation coming from the Alliance for ATMPs developers is early dialogue and data aggregation, and when asked to specify ‘how early’, she replied:
“As soon as developers have an encouraging proof of concept for a product, even before the first clinical data, they can start engaging with patient organizations…it’s never too early.”
According to the executive, developers should initiate engagement with patients at the point that they hold a valuable proof of concept, in order to understand the ‘natural history’ of the disease and the current state of treatment, as well as the main hurdles for the patients.
“Acquiring and integrating this knowledge will help the developer understand the potential value for the product and how to build a suitable clinical development program according to patients’ needs,” Hubert explained.
Moreover, early dialogue will allow developers to get a better understanding of the outcomes that are important to regulators and investors, and the sooner they have this information, “the sooner they can design a development program that will encapsulate all these different aspects and requirements, and address them,” she added.
Researchers that do not integrate these aspects in their clinical development programs, according to Hubert, risk approaching a marketing authorization process with ‘data gaps’, which are very difficult to address in late development stages.
“Early engagement is one of the critical things that ATMP research companies can do to overcome or avoid obstacles that might occur at their attempt to enter the market,” the executive concluded.
