The exceptional or rare diseases are expected to affect 30 million people in the European Union and approximately the same number in the United States, each disease independently concerns a limited number of patients.
The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have new collaboration on exceptional diseases to share experiences and best practices on each other’s regulatory approach to the development of medicines for these diseases.
The international alliance in this area is mainly important to make sure that the limited number of studies that can be conducted to benefit all patients.
The agencies will exchange information on various aspects of the development and scientific assessment of medicines for rare diseases such as:
- Plan of clinical trials in small populations and the use of statistical analysis methods;
- Substantiation of trial endpoints such as outcomes of a trial
- Preclinical proof to carry development programs
- EMA’s conditional marketing authorization and FDA’s accelerated approval
- Risk management strategies for long-term safety issues with medicines for rare diseases.
The existing EMA/FDA alliance discusses issues like:
- Patient engagement
- Bio similar
- Orphan medicines
- Medicines to treat cancer
- Medicines for children
- Pharmaco care
The first meeting of the rare diseases alliance took place by teleconference on 23 September 2016. The alliance will at first meet once a month via teleconference and will be chaired jointly by FDA and EMA.
Date: 26/09/2016