Five steps to approving advanced therapy Parkinson’s drugs

PD in Practice

Author: Dr Kieran BreenPublished: 19 November 2015

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Ever wondered how new and experimental Parkinson’s medicines make it to market? Dr Kieran Breen – of the European Medicines Agency Committee for Advanced Therapies – gives us the lowdown on the rigorous approval process

All advanced therapy medicinal products (ATMPs) are regulated in Europe by the Committee for Advanced Therapies of the European Medicines Agency (EMA CAT).

This specialised group was established in 2009 and contains experts from all of the EU member states in addition to two representatives nominated by patient organisations and healthcare professional bodies.

I was nominated by the European Parkinson’s Disease Association (Parkinson’s Europe) as one of the patient organisation representatives on the CAT.

1. Crucial criteria

The CAT is closely involved in all stages of ATMP regulation. Firstly, we decide whether a new product fulfils the criteria required for it to be classified as an ATMP. We usually assess up to 50 of these requests per year.

2. Specialised science

The second – and probably most important – role is the provision of scientific advice to product developers. Because of their specialised nature, ATMPs need to be considered in detail on a case-by-case basis. Companies are encouraged to contact the CAT to discuss the manufacturing process, the development of pre-clinical studies and the design of clinical trials. The CAT receives up to 40 of these per year. However, carrying out the appropriate studies does not guarantee that the product will be approved.

3. Under scrutiny

Once the final marketing authorisation application for the product is submitted, it is assessed in great detail by a number of the CAT members with support from external expert scrutineers. Specific patient groups may also be invited to comment at this stage. In particular, the ratio between the benefits (efficacy) and risk (safety) of the medicine is considered in order to determine whether it would be appropriate to use the product within the clinic. In addition to my scientific contribution to the CAT, my role is also to assess these questions from a patient’s perspective.

4. The right recommendation

The CAT recommendation is forwarded to the EMA Committee for Human Medicinal Products, which makes the final decision prior to endorsement by the European Commission.

5. National approval

Once approved, the drug must undergo a detailed economic assessment in individual countries, as for all new drugs, to decide whether it is cost-effective and should be made available to patients. In the UK, for instance, this is determined by the National Institute for Health and Clinical Excellence (NICE).

For more info on ATMPs, read Dr Breen’s first article here

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