Rare Disease Patients represented at the EMA Paediatric Committee

In the aftermath of the adoption of the EC Regulation for Paediatric Drugs, the European Medicines Agency (EMA) has created a specific committee for paediatric issues. The objectives of the paediatric regulation include improving the development and availability of medicines for children, ensuring that these medicines are of high quality, and that they are ethically researched and appropriately authorised.

The Paediatric Committee (PDCO) is instrumental in this process. Its main responsibility is to decide on the content of paediatric investigation plans (PIP) and adopt opinions on the quality, safety or efficacy of any medicine for use in the paediatric population. 'The PIP is important because children are not small adults,' explains Dr. Maria Mavris, Drug Development Programme Manager at EURORDIS. 'Drugs with a paediatric indication need to be developed specifically for children, taking into account special dosage and other parameters.'

The PDCO is composed of five members of the Committee for Medicinal Products for Human Use (CHMP), plus one representative from each Member State, as well as 3 healthcare professionals and 3 patient representatives.

EURORDIS was selected as one out of three patient representatives sitting on the Committee. 'This is a great victory for the rare disease community,' says Yann Le Cam, CEO of EURORDIS. 'Approximately 75% of rare diseases affect children. This is why we pushed for a specific paediatric EC Regulation and this is why we wanted rare disease representatives to take an active part in the PDCO.'

Even though the creation of a PDCO at the EMA was one of the measures included in the Regulation, which entered into force at the beginning of 2007, the formation of a new committee is a long and difficult process. 'We first responded to the expression of interest in August of 2007,' explains Ariane Weinman of EURORDIS. 'We worked very hard to put together a good application and sent two excellent CVs.' EURORDIS' efforts finally paid off when the candidatures of Tsveta Schyns and Karen Aiach were accepted in July last year.

Tsveta Schyns was selected based on her experience of rare paediatric genetic disorders. She also brings to the position, a degree in molecular science and genetic research, and a good knowledge of ethics and regulatory affairs. The mother of a child affected by Alternating Hemiplegia of Childhood, a very rare neurological chronic disease, Tsveta combines the patient's perspective with a solid scientific background and experience in networking in the field of paediatric neurology.

Karen Aiach was appointed as alternate and brings to the PDCO her personal experience with paediatric clinical trials and a solid knowledge of the drug development process. As the mother of a child affected by Mucopolysaccharidosis type III (also known as Sanfilippo Syndrome), she founded the Alliance Sanfilippo in 2005 in order to promote paediatric drug investigation for this disease.

'We all know that rare diseases are at the forefront of drug research, and as such, it is important that rare disease patient representatives are actively involved in the regulatory bodies overseeing drug investigation,' argues Tsveta Schyns, EURORDIS representative at the PDCO. 'This is even more important when dealing with drugs that will be used by children, for which additional ethical considerations apply.'

The PDCO is especially important for providing expert advice to pharmaceutical companies developing paediatric drugs and for avoiding unnecessary clinical studies on children. Over the years, it has been increasingly evident that many drugs are used off-label in paediatric populations (that is without sufficient studies for their efficacy and safety) and should be subject to stringent evaluations. Therefore paediatric investigation plans should be developed to ensure the safety and legitimacy of the medicines.

Although the PDCO was created in July 2007, patient representatives joined a year later. 'We have joined the already fully functioning PDCO and we need to learn fast,' explains Tsveta Schyns. 'At the beginning, it was a bit overwhelming, even managing the flow of correspondence delivered every day at the door! However, it is all very interesting and we have received great support from EURORDIS and other members of the Committee. This is a team effort - we bring the patient's perspective.' The PDCO has now entered its second year of existence and meets monthly at the EMA headquarters in London.

EURORDIS representatives are also present on other EMA Committees and Working Parties: the Committee for Orphan Medicinal Products (COMP), since 2001; Patients' and Consumers' Working Party (PCWP), since 2005; the Paediatric Committee (PDCO), since 2008 and, most recently, the Committee for Advanced Therapies (CAT).

For more information:
EMEA

This article was previously published in the March 2009 issue of our newsletter.

Author: Paloma Tejada
Photo credits: All photos © EURORDIS; EMA