The European Medicines Agency (EMA) released a new draft guideline on 24 January assessing Advanced Therapy Medicinal Products (ATMPs) using a risk-based approach.
ATMPs are biological medicinal products such as gene therapy, somatic cell therapy or tissue-engineered products. According to EMA, ATMPs contain an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence. Further, an ATMP's therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence. Vaccines against infectious diseases are not ATMPs.
ATMP products may be associated with a number of risks depending on the quality, classification, biological activity and application of the ATMP. As a result, an optional risk-based approach was added to Directive 2001/83/EC (Annex 1, part IV) to allow applicants to determine the "extend of quality, non-clinical and clinical data to be included in the Marketing Authorisation Application (MAA).
The Draft Guideline on the risk-based approach according to Annex I, part IV of Directive 2001/EC applied to Advanced Therapy Medicinal Products intends to detail what a risk-based approach entails and how to establish a specific profile for each risk factor.
The draft guideline notes that risks are specific to each product and subject to a plethora of factors. Examples of factors contributing to risk include the biological characteristics of the product, the manufacturing process and the specific therapeutic use of the ATMP. Each risk factor must be identified and analyzed by the Marketing Authorisation Holder (MAH) and a specific profile for each risk must be generated.
This analysis should begin as early in the development process as possible and continue throughout development of the ATMP product. Risk analysis should mature along with the product.
Risk analysis should "determine the extent of quality, non-clinical and clinical data to be included in the MAA in accordance with the scientific guidelines relating to the quality, safety and efficacy of medicinal products."
Risk profiling is a four-step process that involves identifying the risks to patients and third parties, identifying the relevant risk factors that may contribute to risks to patient and third parties, mapping the relevant data for the identified risk factors against the risks, and the conclusion of the risk factor/risk analysis relationships.
There is no prescribed template for generating a risk-based approach presentation, and applicants are encouraged to present the information in a way that is both logical and meaningful to their product and the risks associated with it.
Comments on the draft document are due on 30 June 2012.