Two EURORDIS representatives appointed to the CAT

Two EURORDIS representatives appointed to the Committee for Advanced Therapies at the European Medicines Agency

test tubesThe much-awaited Committee for Advanced Therapies (CAT) is now a reality. The creation of this scientific committee at the EMEA is the last step towards implementation of the EU Regulation on Advanced Therapies, which was adopted in 2007. This regulation, and the Committee that has been created as a result, has the potential to stimulate the development of novel and emerging therapies and improve access to them by patients.

Advanced therapy medicinal products are new medical products based on genes (gene therapy), cells (cell therapy) and tissues (tissue engineering). Due to the enormous amount of advanced therapy medicinal products that are now being developed, a specialised committee had become necessary. These advanced therapies could help treat a number of rare diseases such as ADA-SCID (Severe Combined Immunodeficiency), muscular dystrophy, leucodystrophy, haemophilia, thalassemia and cystic fibrosis. They have interesting potential for patients and industry.

‘The promises that advanced therapies hold for the successful treatment of certain rare diseases are now closer to becoming a reality’, argues Michele Lipuci Di Paola, recently appointed as the EURORDIS representative to the Committee. ‘The creation of the CAT is the most important part of the process, because without it advanced therapy medicinal products could never reach the market’.

It is important to underline that the CAT has an advisory role to another Committee at the EMEA, the Committee for Medicinal Products for Human Use (CHMP), which makes recommendations to the European Commission regarding a logo of the EMAproduct’s marketing authorisation. Nevertheless, the CAT is extremely important because it is responsible for assessing advanced therapy products, preparing a draft opinion on each application and submitting this to the CHMP prior to their final recommendation. The members of the committee study the applications of companies seeking to develop advanced therapies and draft an opinion as to whether these products can be designated as advanced therapies and meet safety standards on the market. They also give expert advice on non-clinical data, which can help small- and medium-sized companies developing these products to obtain a certification that will take them a step further along the way to market authorisation.

The CAT is composed of five members of the CHMP, plus one representative from each Member State, as well as two healthcare professionals and two patient representatives. EURORDIS was selected as one of the two patient representative members of the Committee. ‘We are proud of the nomination of Dr. Fabrizia Bignami, as member and Dr. Michele Lipucci Di Paola as alternate. They both have put their scientific knowledge and personal commitment to the service of rare disease patients,’ says Yann Le Cam. ‘It is crucial that rare diseases be well represented in a body that seeks to regulate advanced therapies since most rare diseases are genetic and could greatly benefit from advances in this field.’

Fabrizia BignamiFabrizia Bignami was appointed as member based on her eight years of experience representing patients’ interests in relation to the development of advanced therapies. Her commitment to helping those affected by rare diseases initially led her to the French Muscular Dystrophy Association (AFM) and later, to EURORDIS as Therapeutic Development Director. As Permanent Observer at the EMEA Committee for Orphan Medicinal Products (COMP), for the past six years, she has observed the increasing number of orphan drugs falling under the definition of advanced therapy medicinal products. She holds a PhD in Cellular and Developmental Biology from the University of Rome. Her scientific background is particularly suitable as it is focussed on cell and gene therapy. Fabrizia is Italian, has lived most of her adult life in France and is fluent in English. She brings to the CAT a unique mix of scientific and advocacy work with a true international background.

Michele Lipucci Di PaolaMichele Lipucci Di Paola was appointed as an alternate member. He brings to the CAT his PhD and work experience in cell biology as well as his personal experience on thalassaemia. Thalassaemia is a rare genetic blood disorder that results in poor or non production of functional haemoglobin. As a patient advocate, intimately familiar with thalassaemia, he understands the importance of identifying new clinical protocols and innovative therapeutic approaches such as gene therapy and molecular reactivation of the production of foetal haemoglobin. Michele has been active in several patient groups and has been a board member of EURORDIS.

The fact that patient representatives are involved in this Committee is important because expertise on the evaluation of these products is scarce for the moment; and aspects such as long-term follow-up of patients and risk management strategies for the post-authorisation phase are crucial. Patients also have unique experienced-based expertise when assessing clinical trials, which are key to obtaining market authorisation. ‘As patients we have first-hand experience in clinical trials and can give an informed opinion about how to organise them, make them transparent, ethically acceptable and how to transmit the results to the outside world.’ explains Michele Lipucci Di Paola. ‘To this effect, EURORDIS organises training sessions to educate patients about the drug development process and has produced a Charter on Clinical Trials.’

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This article first appeared in the April 2009 edition of the Eurordis Newsletter.

Author: Paloma Tejada
Photo credits:© EMEA & EURORDIS & European Community, 2009


Page created: 15/03/2010
Page last updated: 16/12/2010
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