Advanced Therapy Medicinal Products (ATMPs) are a heterogeneous group of drugs that must meet special regulatory requirements due to their innovative and complex nature. In a new scientific article, experts from the Paul-Ehrlich-Institut examine scientific and regulatory aspects involved in the ATMP marketing authorisation procedure using the example of gene therapeutics. The experts also describe experiences gathered while attempting to balance both the extensive data requirements for the marketing authorisation evaluation and the unmet medical need from the patient's point of view. The article was published on 18 August 2022 in issue 5/2022 of Pharmakon.
Since the ATMP legislation entered into force at the end of 2008, 21 Advanced Therapy Medicinal Products have received marketing authorisation in the European Union. These include:
ATMPs must undergo a centralised marketing authorisation procedure at the European Medicines Agency (EMA). The assessment of an ATMP is carried out by experts from the Paul-Ehrlich-Institut and the other national medicines authorities of the EU Member States in various European committees, such as the Committee for Advanced Therapies (CAT), which was created specifically for this product group. The CAT leads the scientific assessment of quality, efficacy and safety, including the environmental impact of ATMPs. Germany is represented on the CAT by experts from the Paul-Ehrlich-Institut, the Federal Institute for Vaccines and Biomedicines. The recommendation regarding approval or refusal of marketing authorisation is prepared by the CAT and reviewed and confirmed by the Committee for Medicinal Products for Human Use (CHMP) at the EMA. The CHMP recommends that the EMA either approves or refuses the marketing authorisation. Following a positive CAT/CHMP assessment, the European Commission will grant a European-wide marketing authorisation for an ATMP.
ATMPs’ special mode of action allow them to offer new treatment options for patients who have often exhausted "conventional" therapies aimed at the causes of disease. They also open up treatment options for patients, predominantly those with rare diseases for which there are currently no possibilities for treatment. Due to these circumstances, usually only limited data from non-randomised, non-comparative clinical trials with a limited number of patients is available for the marketing authorisation procedure.
The question therefore arises as to how experts responsible for assessing quality, safety and efficacy in the national medicines authorities and in the CAT can strike a balance between the authorisation of urgently needed medicines and the demand for good evidence. Should more data and a longer follow-up period be required and the authorisation be granted significantly later? How does an authorisation based on limited data affect reimbursement and access for patients to these innovative medicines in the EU Member States?
In their Pharmakon article, experts at the Paul-Ehrlich-Institut examine these questions and various aspects of ATMP authorisation from a scientific and regulatory perspective using the example of gene therapeutics. Experiences gathered in connection with the collection of clinical data under everyday conditions in the post-authorisation phase are also presented, and opportunities for improvement are highlighted.
Updated: 22.09.2022
