Protocol assistance is the special form of scientific advice available for companies developing designated orphan medicines for rare diseases.

Protocol assistance in the EU

Protocol assistance is the special form of scientific advice available for companies developing designated orphan medicines for rare diseases. The European Medicines Agency can give scientific advice and protocol assistance to companies involved in developing medicines. For human medicines, scientific advice and protocol assistance are given by the Committee for Medicinal Products for Human Use (CHMP) on the recommendation of the Scientific Advice Working Party (SAWP).In addition to scientific advice, companies developing an orphan medicinal product can receive answers to questions relating to the criteria for authorisation of an orphan medicine. These include:

  • the demonstration of significant benefit within the scope of the designated orphan indication;
  • similarity or clinical superiority over other medicines. This is relevant if other orphan medicinal products exist that might be similar to the product concerned and which have market exclusivity in the same indication

The Agency also offers parallel scientific advice with health-technology-assessment (HTA) bodies. The aim of this is to allow medicine developers to gain feedback from from regulators and HTA bodies at the same time, early in the development of a medicine.

Protocol assistance in the US

Protocol assistance is given as a part of Orphan Drug Program for the development of drugs for rare diseases and disorders.

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