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The Number and Price of Orphan Drugs

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Mikko Seppänen, MD, PhD, Associate Professor
Specialist in Internal Medicine and Infectious
Clinical Immunologist
Head, Rare Disease Center of HUS

Publicada em: Saúde e medicina
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The Number and Price of Orphan Drugs

  1. 1. 4/15/2016 1 THE NUMBER AND PRICE OF ORPHAN DRUGS Mikko Seppänen, MD, PhD, Associate Professor Specialist in Internal Medicine and Infectious Diseases Clinical Immunologist Head, Rare Disease Center of HUS ORPHAN DRUGS • Before 1983: 38 orphan drugs for rare diseases • US Orphan Drug Act 1983; Japanese 1993; EMA 2000 • Criteria – for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease – prevalence of condition in EU ≤5:10,000 or unlikely that marketing would generate sufficient returns to justify the investment needed for its development • US: <6.37:10,000, Japan <4:10,000 – no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized,or, if exists, the medicine must be of significant benefit to those affected by the condition. • Applications evaluated by EMA Committee for Orphan Medicinal Products (COMP) – 10 years of marketing exclusivity from approval (US: “7 years, 50% tax credit, R&D funding) – by far less strict premarketing research criteria OD - FINANCIAL FIGURES • 12% annual growth (other drugs 6%) • In 2020 predicted that ODs will cover – 20% of total drug market – 178 billion USD = 178,000,000,000 USD • Advantages for pharmaceutical industry in OD development – lack of alternatives for patients – lower R&D costs – easily defined patient populations – prices that the drugs are able to command • So far orphan drug developers have managed to defend the cost of these life-changing drugs due to – relatively small patient populations they serve – continued paucity of options for sufferers www.evaluategroup.com/orphandrug2015 OD DESIGNATIONS • Orphan drug designations (n) increased in 2014 – Europe: to n=201 (62% ↑) – US: to n=291 (12% ↑) – currently >15-20 new drugs into market yearly • will it become harder to justify prices? http://ec.europa.eu/health/documents/community-register/html/alforphreg.htm OD DESIGNATIONS • Cumulative numbers of designations rarely known by policy makers or by treating physicians • Exclusivity = freedom from competition – if medically superior – does not increase competitive bidding & pricing OD PRICING • Strensiq® /primary hypophosphatasia: >2 million €/y /adult 80 kg patient http://apps.who.int/medicinedocs/documents/s21793en/s21793en.pdf
  2. 2. 4/15/2016 2 OD PRICING VS DEVELOPMENT COST Development cost OD PRICING AND PROBLEMS • High pricing will drain an increasing amount of invaluable resources in health care? – how will we afford all these drugs? – prioritization, based on what? • expected/proven gain in survival/ QALY? • ”the acceptable cost of human life year???” England: acceptable cost per QALY: 360,000-1,360,000€ Ivacaftor/CF:335,00 -1,274,000 GBP/year • is survival >> reduced complications w/o shown survival benefit (yet?)? – prioritization should not be done by the treating physician • Ethical questions – politicians and policy makers (understandably) reluctant to consider – either way … if politically can or cannot be afforded • Will EMA and FDA be able to…???? markets are global… – create more transparency – define acceptable profit margins THANKS • 19,4% (296/1525) against diseases with prevalence <0.2:10 000
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