Various types of regulators play vital roles in all stages of gene and cell therapy (GCT) development. The medicines agencies such as the European Medicines Agency (EMA) in the European Union (EU) and The Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom (UK) are heavily involved in facilitating the development and patient access to GCTs and other medicines.
The EMA is a decentralised agency of the European Union (EU), and is responsible for the scientific evaluation, supervision and safety monitoring of medicines. EMA protects public and animal health in EU/EEA by ensuring that all medicines available on the EU market are safe, effective and of high quality. Based on EMA's recommendation, the European Commission (EC) makes a legally binding decision for all the centrally authorised medicinal products. If granted by the EC, the centralised marketing authorisation for the medicine is valid across the entire EU/EEA.
While many of the GTCs, especially those that are classified as Advanced Therapy Medicinal Products (ATMPs), are regulated centrally by the EMA, some may be regulated by national medicines agencies and through different regulatory pathways. The term “regulators” in fact, has a very wide meaning – it includes any competent authorities, including but not limited to the Medicines Agencies, National Competent Authorities, Health Technology Assessment (HTA) Bodies, Ethics Committees, Payers, and more. To learn about who they are and what other types of regulators are involved in medicines development process, please see EuroGCT’s Actors and Networks database.
Which regulator determines the prices of medicines?
The EMA does not make decisions on the pricing, reimbursement and the accessibility of medicines.
Decisions about pricing and reimbursement take place at member states and regional level, taking into account the health system of the respective country or region and through a series of processes. In the EU, regional and national Health Technology Assessment (HTA) bodies evaluate the new medicine by assessing its relative effectiveness compared to the existing medicines. The result is then used to inform reimbursement decisions made by national bodies and/or payers. The EU healthcare payers look at the medicine’s cost effectiveness, its impact on the country or region’s healthcare budgets and the seriousness of the disease, and use this information to decide whether they cover a medicine and under what conditions. The payers also negotiate the price of medicines with manufacturers.