Position Paper / European Policy
CF Statement to
European Commision, DG Enterprise Cystic Fibrosis greatly welcome the EC - consultation
document from DG Enterprise on “Better medicines for children” and the
invitation for comments.
Seventy million children at risk There are seventy-five million children in the European
Union all under the age of sixteen, who are at risk when taking medicines. Fifty to ninety
(in neonates) percent of the available medicines have not been tested for safe use in
children and consequently lack information on safe and effective use. “Children do at least deserve as much attention and
carefulness as do adults” (Byrne) Children represent a truly vulnerable population with
developmental, metabolic, physiological and psychological differences from adults. There is a high level of concern among Patient Organisations in
the Europe Union about the safety and efficacy issues, the limited amount of
age-appropriate research and the lack of regulation accorded to medications that are
currently being used in the treatment of children. This situation results in significant and unnecessary risks, lack of efficacy,
unavailability of therapeutic advances, unexpected adverse effects and even death among
this age group. The importance of the subject “children and medicines” will considerably grow in the near future because of increasing insights from genetic and molecular research and the possibilities for early detection of disease. Consequently medicines will play a more vital role for children in prevention, suppression and/or delay of disease expression.
A large joint and coherent effort A large cohesive effort of all parties - clinical,
research, industry, regulatory and financial community and last but not least the patient
organisations - is necessary to address the present unacceptable situation for existing
medicines as well as new medicines. At present, there are no appropriate regulations in place
in Europe for the testing of new drugs for children Cystic Fibrosis greatly welcome the EC - consultation
document from DG Enterprise on “Better medicines for children” and the
invitation for comments. Cystic Fibrosis is supportive of resolutions, statements
and activities of those various stakeholders in this proposal - the Council of the
European Union, the European Federation of Pharmaceutical Industries (EFPIA), the European
Forum for Good Clinical Practice (EFGCP) and the Confederation of European Specialists in
Paediatrics. SPECIAL INITIATIVES Patient Organisations would like to see specific
initiatives developed in the areas of: Rare Genetic
Disorders that require a special and separate supportive approach and have been
adopted as a special field of interest by Patient Organisations. New technologies using
molecular diagnostic tests will be used more and more to detect genetic mutations linked
to various diseases. Sixty five percent of all of the opinions of the Committee for Orphan
Medicinal Products (COMP) concern those medical conditions (usually Rare Diseases)
affecting children. In respect to rare disorders special attention is asked for the
communication on the consultation paper by the European Organisation for Rare Disorders
(EURORDIS) which we fully support.
Collaboration Beyond Europe:
Improvements in the
ethical and scientific requirements for conducting research in children (also
including clinical trials with children on pharmacogenetic properties of new medicines). A
strong and adequate European framework specialised in the ethical aspects of trials with
children is necessary in order to protect the dignity and the rights of children. The
European Forum for Good Clinical Practice (EFGCP) is working, and since recently in
partnership with patient organisations, on
these issues also to see that paediatric patients are not overexposed to research. Increased awareness
and education concerning the required activities for the availability and prescription of safe and
effective medicines for children. Dialogue on
the benefits and risks of paediatric research for individual children participating in
trials as well as for public health in general, is important. Awareness of the great potential pressure and
burden which is laid on parents and children participating in trials. Government officials, researchers and the
pharmaceutical industry must be motivated towards a synergistic approach with investments
at national levels and the European level. Priority for (preventive) treatment of serious and life threatening diseases The active role
that Patient Organisations play and could play
in this field. Many Patient Organisations have excellent relationships with paediatric
specialist groups at both national and European levels. The involvement of patient
organisations on the European policy and regulatory level as well on the projectbound and
individual level should be recognised by making available the required financial
resources. CYSTIC FIBROSIS FAVOUR
Cystic Fibroisis Denmark is worried about the
considerable backlog and the gap between the present and required situation. Many existing medicines have to be re-tested which means a large
research investment. Regulation and incentives to test new medicines are lacking. Therefore, it is essential that measures be taken in the
short term to address this urgent and
unacceptable situation. Europe’s children will greatly benefit from a well
co-ordinated, well supported, and a well-funded approach. 29 April 2002 |