Pfizer Takes Issue with European Commission’s Plans to Regulate ATMPs
According to a summary of the 63 stakeholder responses compiled by the Commission and released Wednesday, a significant number of respondents focused on the ATMP quality requirements and said the authorizations of ATMPs “were not sufficiently adapted to the special characteristics of these products.”
Pfizer noted that the proposed requirements place the regulatory burden on companies at the end of the product’s development process. The company says that because early development work is often conducted by research organizations or small- and medium-sized enterprises, there should be “a more appropriate balance that would also encourage earlier interaction with regulatory authorities.”
Other stakeholders’ opinions on what should be specifically altered ranged from requests for additional flexibility to requests that data requirements be determined according to the disease, target patient and type of product. Some also requested GMPs (good manufacturing practice) guidelines to be established for the development of ATMPs.
Despite their differences, there was a consensus among stakeholders that hospitals be exempt under certain circumstances to allow patients to access new treatments for unmet medical needs.
Active Substances
Due the variability of the living materials used to construct ATMPs, a few stakeholders in their comments “referred to difficulties in the application of the concept of [an] ‘active substance.’”
In order to deal with this variability, Pfizer recommends a certification system of the active ingredient of ATMPs that “may be analogous to the mechanisms already available for drug master files, plasma master files, vaccine antigen master files or perhaps the European Pharmacopoeia certificate of suitability.” These types of systems may allow for products to be endorsed by a regulator earlier in the development process and prior to the start of a clinical trial, Pfizer said.
Other respondents noted the “impossibility” of conducting large preclinical and clinical trials, especially since pre-clinical data is not always suitable to evaluate the safety of cell-based and gene therapy medicinal products.
International Harmony
Pfizer’s comments on the consultation also specifically requested that the European Commission organize its revisions with other international regulators and discuss potentially conflicting requirements. “One potential area for investigation would be the applicability of the US limitations on cell and tissue products sourced in Europe to allogenic stem cell therapies,” Pfizer said.
In addition, the UK’s MHRA (Medicines and Healthcare Products Regulatory Agency) noted that because only two products have been authorized via the centralized procedure, the Commission should coordinate with European national regulators.
The entirety of the stakeholder responses to the regulation can be viewed here.
