Continuing with our spotlight series, the European Biopharmaceutical Enterprises (EBE) is featuring its Advanced Therapies Working Group, a joint working group with EFPIA, which will be meeting today.
The Advanced Therapies Working Group is involved with some of the most exciting aspects of the biopharmaceutical industry from CAR-T cell therapies to gene therapies. Its goal is to advocate for a consistent and harmonised regulatory framework so that these cutting-edge advanced therapies can reach the patients who need them.
As part of this effort, the working group recently published a paper on hospital exemptions, which shows that while there is no need to change the legislative basis for advanced therapies, there is a need for tighter commission guidance on specific aspects of the legislation. This was recently echoed by Commissioner for Health and Food Safety Vytenis Andriukaitis who said that the current legislation provides a “clear and specific framework” for advanced therapies but he also noted, “At the same time, the application of the existing provisions can be optimized.”
In addition, the working group publishes important reports including a survey to identify the challenges that ATMP developers face from regulatory hurdles to clinical trial design and uncertainties related to market access. The results were recently published in an on-line journal called Molecular Therapy-Methods & Clinical Development. The study showed that patient access to these very innovative medicines remains a challenge in Europe. The next hurdle will be market access for ATMPs, and this is the topic of an abstract that has been accepted for the DIA conference next year. The working group will also soon publish a report on gene editing technologies and what they need for patient access to modern gene therapy products.
The group plays a large role in ensuring the voice of the biopharmaceutical industry is heard when it comes to regulations. For instance, it will be submitting a summary of the comments to the European Commission consultation on draft guidelines on Good Clinical Practice (GCPs) for ATMPs. It also sponsors a number of regulatory conferences on ATMPs, and will host one on 4 December in London, called “Advancing the delivery of ATMPs to patients.”
The Advanced Therapies working group is one of five EBE working groups, including Personalised Medicine, Innovation and Funding, Biotherapeutics and Biomanufacturing – don’t miss the next instalment in our spotlight series!