Global pharma manufacturers of Advanced Therapy Medicinal Products (ATMPs) face multiple & complex regulatory hassles. These products are used for gene therapy, somatic cell therapy and tissue engineering.
The issue is that these products contain exclusively non-viable cells or tissues and could be also vaccines against infectious diseases, said Florence Bine-Scheck M.D. Senior Medical Director, Voisin Consulting Life Sciences.
Dr. Bine-Scheck was speaking at the Topra India 2017 conference on ‘Building Regulatory Excellence' held in Bengaluru recently. She indicated that first the manufacturers need to confirm if the product is ATMP. Then they need to adopt a regulatory strategy based on its classification.
Addressing on the topic Specificities and Challenges in development strategy: Focus on Clinical and Regulatory aspects of EU regulatory framework on ATMP, she said that at the EMA quality of data needed to be assessed and certified. The process required 90 days. Some of the products approved between 2014 and 2016 are tissue engineering products for graft versus host disease, limbal stem cell deficiency and melanoma to name a few.
The first challenges is the complex regulatory framework for ATMP. The second is manufacturing because these are developed in academic or hospital setting with no Good Manufacturing Practise (GMP) norms in early phase. There is also limited availability of samples. The third challenge is the Nonclinical ATMP development ADME studies not relevant or feasible. The disease animal model is not always available. The fourth challenge is the clinical ATMP development. Here the long term safety data is vital. The frequency of follow-up needs to be planned on a case by case basis, she said.
Therefore, there is a need for a GMP guide specific to ATMPs. It also needs to be considered if some studies can be done concurrent with clinical trials. In this case, the in-vitro models could be an alternative. There is also a need to test various human tissues for potential safety issues on the basis of a risk-based approach, noted the Voisin Consulting medical director.
In this regard, the EMA guideline on the safety and efficacy follow-up on risk management of advanced therapy medicinal products needs to be adhered. If companies do not follow the same, then the possibility of serious and fatal immune response is imminent, said Dr. Bine-Scheck .
There is need to address the safety challenges for ATMPs. From long term safety data, watching for delayed immune response or ADRs, companies will need to be ready for the unexpected. They should record product traceability, ensure routine pharmacovigilance activities, plan post-marketing clinical studies to ensure long term follow-up for safety and efficacy, she pointed out.
This is where the importance of early discussions with regulatory agencies is mandated to overcome the challenges during development. It would also reduce the time to marketing application and reduce the cost of development, stated Dr. Bine-Scheck.