Health Technology Assessment and patients' rights: Assess versus access?
Patients’ organisations call for an assessment of the clinical added value of orphan drugs at the time of the marketing authorisation evaluation. The European Commission has commissioned Ernst & Young to explore the feasibility of a new Committee at the European Medicines Agency1. A new drug candidate faces a number of hurdles on its journey to reach the patient. Regulators are the first, and right after are the Health Technology Agencies and payers. Things can turn sour, from a political point of view, when that second scenario happens, when the regulators say “yes this drug is safe and effective” and the payers say “Oh well but we won’t reimburse it”. Regulators and health technology assessment experts have recently started to harmonise their respective methodologies to avoid some degree of redundancy and contradictions in their respective opinions. Patients are participating in the decision making at the European Medicines Agency, and work closely with some national regulatory agencies. More systematic involvement with health technology assessment agencies is only beginning. This paper illustrates some of the issues patients are discussing with health technology assessment bodies.
Quelle place pour les associations de malades dans le processus d'innovation médicamenteuse ?
Quelle place les associations de malades peuvent-elles occuper dans le processus d’innovation medicamenteux ? Pour repondre a cette question, nous avons choisi d’etudier la mobilisation des associations de maladies rares en France et en Europe. Nous observons dans un premier temps les prises de parole publiques des associations de maladies rares dans le debat bioethique en France pour constater que celles-ci, du fait des specificites de leurs pathologies et des espoirs qu’elles investissent legitimement dans le genie genetique, ont ouvert un nouveau front. Nous tentons ensuite de specifier le type d’expertise que ces associations privilegient afin d’interagir avec la communaute scientifique et les industriels ; une expertise issue de l’experience intime du malade avec sa maladie et le systeme de sante et de recherche dont nous montrerons qu’elle est d’autant mieux accueillie par les autres acteurs qu’elle respecte les juridictions de chacun. Enfin, nous rendons compte des modes d’intervention des associations sur le marche du medicament et des relations qu’elles ont nouees avec les autres acteurs du processus d’innovation medicamenteux pour souligner la dynamique partenariale et la cooperation de plus en plus etroite entre associations et industriels.
The struggle against neuromuscular diseases in France and the emergence of the "partnership model" of patient organisation.
The past few decades have witnessed the increasingly active participation of patient organisations in research activities concerning them. They contribute substantially to the funding of scientific and clinical research. More importantly, certain patient organisations take strategic decisions concerning that research, and contribute to the production of knowledge on their diseases. In France, the AFM (Association Française contre les Myopathies-French Muscular Dystrophy Organisation), is a striking illustration. The paper argues that the model of the AFM's engagement in research-the "partnership model"-is original insofar as it renews the power relations between patients and professionals found in two classic models: the "auxiliary model" and the "emancipatory model". Based on a long-term study of the French Muscular Dystrophy Organisation, this "partnership model" is characterised and its implications discussed in three respects: the possible generalisation of the mode of relations it establishes between patients and professionals; its effects on the steering of research; and its consequences for the dynamics of patient organisations movements.