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Patient Advocacy: EMA’s Launch Of Patient Involvement Pilot Program

October 2, 2014

This is the seventh Blog Post in a series that examines the influence and power of patients and patient advocacy groups in helping and influencing the different stages of drug development.

The European Medicines Agency (EMA) announces September 26th, the launch of a pilot program to involve patients in the benefit-risk evaluation  of medicines at the regulatory agency’s Committee for Medicinal Products for Human Use (CHMP). CHMP is the EMA committee responsible for preparing the agency’s opinions on questions concerning human medicines – it conducts the initial benefit-risk analysis of medicines. The pilot project will run for at least one year, with a report to be presented to CHMP summarizing the project along with recommendations for implementation of the project. A document identifying the steps and principles of the pilot project is available on the EMA website.

Guido Race, EMA Executive Director says:

“As patients live with their condition on a day-to-day basis, their views on the therapeutic effect of a medicine and its impact on their quality of life – particularly when these are balanced against the risks – may differ from those of other stakeholders. Involving patients in CHMP discussions brings the patients’ voice into the decision-making process ….”

The first medicine in EMA’s pilot project is the orphan drug Afamelanotide. The medicine’s indication is for the rare disease Erythropoietic Protoporphyria (EPP). EPP is a rare inherited metabolic disorder with an enzyme deficiency that results in hypersensitivity of the skin to sunlight and artificial light. About 5,000 – 10,000 people worldwide have EPP. There is currently no approved treatment.

At the September 2014 CHMP meeting, 2 EPP patients presented their personal experiences living with this condition and also answered questions.

The objective of EMA’s pilot program is similar to a pilot program launched in Canada this past August – getting patients involved with the regulatory agency’s review of orphan drugs. Rona Ambrose, Canada’s Minister of Health, announces the launch of a pilot project “ … targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs …. The Pilot Project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect.” Two drug manufacturers agreed to participate in the Pilot Project with their drug review submissions :

•   Hoffmann-La Roche Limited’s drug Gazyva (Obinutuzumab) for Chronic Lymphocytic Leukemia (CLL)

•   Hyperion Therapeutics’ drug Ravicti (Glycerol Phenylbutyrate) for Urea Cycle Disorders (UCDs).

EMA and Health Canada are just two international regulatory agencies that are now increasing patient involvement in the drug regulatory process.

Please Note: “Kid Hugging a Rainbow” by Marendo Müller, artwork (Own work) [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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