A rational Medicines Policy for Europe

1. A rational Medicines Policy for Europe Danielle Bardelay Medicines in Europe Forum HAI Seminar - The Hague 18 November2004

2. Therapeutic advance ? • « The practices of the pharmaceutical industry and regulators blurr the distinction between genuine therapeutic advance and mere innovation » • (ISDB - International Society of Drug Bulletins - Declaration on therapeutic advance in the use of medicines Paris 15 -16 November 2001) -

3. newly marketed substance, or indication, or formulation, etc. industrial innovation (chemistry, biotechnology) therapeutic advance : « a new treatment that benefits the patient when compared to existing options » (ISDB Declaration) - A clear difference between:

4. 23 years of independent ratingof 2871 new drugs(by La revuePrescrire) • Bravo : 0,24 % • A real advance : 2,68 % • Offers an advantage : 7,56 % • Possibly helpful : 15,85 % • Nothing new : 66,63 % • Not acceptable : 2,79 % • Judgement reserved : 4,25 %

5. Convergent analysis • US National Institute for Health Care Management : 76 % of new drugs classified as non-priority for review by the FDA • Canadian Patented Medicine Prices Review Board : just 6 % breakthrough medications • The content of reviews on new drugs published by La revue Prescrire and the Swedish Medical Product Agency bulletin is broadly similar(Int J Risk & Safety in Medicine 2004)

6. Concerns with cost • prices of 12 new anticancer drugs approved by the European medicines Agency between 1995 and 2000 (Garattini S British Medical Journal 2002): • 3 to 350 times as much as reference drug • for « few or non substantial advantages over existing preparations »

7. Concerns with safety • triazolam story • fenfluramin - phentermin story • amineptine story • cerivastatin story • rofecoxib story (+ valdecoxib, parecoxib, etc.) • SSRI antidepressants story • next ?… new antidiabetics, or anticoagulants, or immunosuppressants, or Ritaline°... ?

8. Dramatic lack of : • adequate comparative evaluation : • no idea of the added therapeutic value • transparent pharmacovigilance : • no real prevention of side effects • prevention of medication errors : • no proper use of drugs • primary prevention to limit risk factors : • little success in disease limitation

9. Priority to relevant evaluation • pre-marketing evaluation • because post-marketing means uncertain (FDA statistics on fulfilment of conditions) • comparative evaluation • versus best available option (at right dose, etc.) (WHO report on Priority Medicines) • focused on benefits and on risks

10. Areas for priorities • rare diseases : the Orphan Drug Regulation is already here. A survey on fairness of its implementation is needed. • pediatric diseases : the coming Regulation may help for small groups of children. But other groups already have too many drugs. • elderly, pregnant women, patients with renal failure, etc. require specific evaluation

11. Areas for priorities(continued) • cancer, multiple sclerosis, Alzheimer disease, severe viral infections : their severity is not an excuse for poor evaluation. • acute and chronic pain : also not an excuse for the extremely poor current evaluation. • groups of old useful drugs : require additional evaluation (for ex : on doses) but companies are not interested (for ex : diuretics). • drug utilization studies.

12. Priority to pharmacovigilance • transparency of agencies, access to data, visibility of decision making • attention paid to patients direct reporting • visible implementation of decisions (warnings, cautions, etc.)

13. Priority to prevention of medication errors • not all errors are linked to dangerous professional practices • safer packaging, relevant and understandable patient leaflets are needed (Council of Europe working group) • promotional biased patient « information » pushed by companies is not needed

14. Priority to primary prevention • diabetes, obesity, cardiovascular diseases, lung (and other) cancers in Europe will not disappear with help of more new drugs • nutritional, environmental, infectious, primary prevention is the priority (WHO Strategy 2004) • cost of prevention campaigns and cost of drug reimbursement should be compared

15. Restoring confidence • more and more patients and health professionals consider drug agencies and health authorities as unreliable • the Dutch Presidency initiative is an opportunity for restoring confidence : • choice of research priorities • attribution of financial support • follow up of supported research • should be fully transparent

16. Medicines in Europe Forum • will closely follow up this initiative on priority medicines • hopes that patients needs will be seriously considered • hopes that neglected diseases more frequent outside Europe will really be considered • and thanks HAI warmly for this seminar which raises patient oriented priorities.