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.
Study on essential drug use status and its influencing factors among cerebral infarction inpatients in county level hospitals of Anhui Province, China
Background and purpose Drug costs is one of the main components of hospitalization expenditure for cerebral infarction inpatients. In China, the National Essential Medicine System (NEMS) was created to relieve the heavy drug-cost burden for patients. The objective of this study was to investigate essential drug-use status and its influencing factors among cerebral infarction inpatients in county-level hospitals of Anhui province, China. Methods Three county-level hospitals were selected through a multi-stage cluster random sampling method. The hospitalization cost data of cerebral infarction inpatients in the three hospitals were extracted from the Anhui provincial information platform of the New Rural Cooperative Insurance System (NCMS), and whether the proportion of essential drug cost in the total drug cost reached the median value of 33.05% which was set as the evaluation index for essential drug-use status. Questionnaires for hospitals and physicians were designed and given to them to assess influencing factors. Results We retrieved the cost data of 2,189 inpatients from the NCMS platform and investigated 51 corresponding physicians in total. The drug costs accounted for 52.6% of the total hospitalization cost, and essential drug costs alone accounted for 37.0% of the total drug costs. The essential drug-cost proportion was high among physicians with a higher recognition degree on NEMS, older age, lower final academic degree, longer work experience and lower professional title. Married physicians and those with tight organizational affiliation also prescribed more essential drugs. Conclusions Increasing the proportion of essential drugs was an effective way to reduce the disease burden for cerebral infarction patients. Perfecting the NEMS, increasing government investment, reinforcing education and propaganda, and formulating relevant incentive and restrictive mechanisms were all effective ways to promote and increase the number of essential drug prescriptions written by physicians.