Eurordis - Patient involvement in Protocol Assistance

Patient involvement in Protocol Assistance
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Why it is so important for you

Protocol Assistance to pharmaceutical companies developing new drugs is mentioned in the European Regulation on Orphan Medicinal Products. The regulation states that these companies can ask for free-of-charge help in developing protocols. This is one of the benefits provided for developing orphan drugs. How does it work?




After having received the European Commission’s decision on the designation of orphan drug status (based on the opinion of the COMP1 at EMEA2), the sponsor of an orphan drug is entitled to request Protocol Assistance prior to the submission of an application for Marketing Authorisation. Assistance can be provided at any time of drug development, during the initial development phase or during the post-authorisation phase, and it can be provided several times; follow-up assistance is also available. The advice is given by the Scientific Advice Working Party (SAWP) at EMEA and is not in any way binding with regard to any future Marketing Authorisation application of the product concerned. A company request for assistance contains prospective questions concerning quality, preclinical and clinical aspects relating to the proposed future development of the orphan drug.


The involvement of patients in Protocol Assistance for orphan drugs was triggered by Eurordis. Advice given is the result of collegial work from SAWP, its experts, COMP, other interested parties, and since 2004/2005, patients! The pharmaceutical company is asked to send a list of questions beforehand to EMEA. Eurordis analyses the questions and decides whether patients need be involved or not. If that is the case, Eurordis contacts patients affected by the condition to ask them to come to a meeting at EMEA to represent all European patients with the condition. Eurordis provides patients with a preliminary briefing by phone. Patient representatives are then cleared by EMEA for potential conflict of interest, and a confidentiality agreement is signed. They then read the file on the drug concerned, and answer a number of questions during the meeting. Examples of questions asked are:
Is there anything you believe the patient information leaflet should insist on?
Are there side effects of the proposed medication of particular concern to you?
Do you think the drug would be easy to take?
Do you think there were important stakeholders not involved in the clinical trials?

Since 2005, quite a few patients have been involved. Was the experience a good one or not? The following testimonies should shed some light on this issue.
- Ulrica Sterky (Riksförbundet Cystisk Fibros, Sweden)
‘It was a great experience. There was a lot to read and it was not always easy to understand; but I think it is important to think of potential risks involved with the drugs, and also practical matters, such as how the drug is going to be administered. The proposals I made were highly appreciated.’


- Sophie Ravilly (Vaincre la Mucoviscidose, France)
‘The presentation made by the pharmaceutical company was very interesting. However, I had the feeling that we, patient representatives, were not listened to enough, especially when we talked about increase of quality of life and the number of times per day to administer the medication.’
- Birgit Dembski (Mukoviszidose e.V., Germany)
‘The patient representatives were given ample opportunity to participate in the debate and voice their opinion, but because I lack the necessary scientific background, I wasn’t able to take part on discussions on content. The fact that patient representatives are involved in protocol assistance is a very important step forward.’

‘Not all pharmaceutical companies request Protocol Assistance,’ says François Houÿez, Health Policy Officer at Eurordis, ‘but when they do, the latest report from the COMP shows that a positive outcome at the time of Marketing Authorisation for their product is higher.’ EMEA, and presently the Scientific Advice Working Party, are very supportive of this important step forward. Another sign of the growing involvement of rare disease patients in the European regulatory process, something Eurordis has always fought for.

Eurordis is always on the lookout for patient representatives interested in being involved in Protocol Assistance. Do not hesitate to contact us if you are interested (Anja Helm).

For more information:
Protocol Assistance process and dates (EMEA)
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1Committee for Orphan Medicinal Products
2European Medicines Agency
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This article was previously published in the June 2006 issue of our newsletter.
Author: Jerome Parisse-Brassens
Photos : meeting © Flavio Minelli; pills © www.freeimages.co.uk ; Sophie Ravilly © Virades de l’Espoir

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