Advanced therapy medicinal products (ATMPs) bear game changing potential for the treatment of severe conditions for which we have no appropriate therapies today. Though an EU regulatory framework is in place since 2008 and more than 900 ATMPs are in clinical trials, the number of authorized ATMPs with currently less than 10 is very low. The “success rate” of approximately 50% at the time of authorization is disappointing and significantly lower compared to other new therapeutic entities (80 to 90%). Diane Seimetz, founding partner of Biopharma Excellence, has addressed in her recently published article on ATMPs:
- what the challenges are at different stages during development
- how the challenges can be prevented or successfully mastered, and
- how the regulatory environment can foster the success rate.
We have long-term experience with ATMP development and regulatory strategy and procedures. Please do not hesitate to ask us if you need support in
- advice on design of the development program
- preparation for agency interactions including writing briefing books
- writing dossiers for the certification of ATMPs by EMA
- writing IMPDs/IBs,
- solving critical issuesduring development.
Regulators have realized the need for measures to facilitate the development of cell based therapies. On 15-16 November 2016, a workshop on the scientific and regulatory challenges of innovative cancer immunotherapy medicines based on genetically modified T-cells took place at the European Medicines Agency (EMA). The workshop was organized by the Agency’s Committee for Advanced Therapies (CAT), and aimed to facilitate dialogue between the CAT and medicine developers from industry and academia on current scientific developments, regulatory requirements for development. During the workshop medicine developers shared their experience in quality, nonclinical and clinical development, discussed challenges arising from the regulatory environment in the EU and the US and initial thoughts on counter measures with regulators.
For more see HERE