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THE NUMBER AND PRICE OF ORPHAN DRUGSMikko Seppänen, MD, PhD, Associate Professor

Specialist in Internal Medicine and Infectious Diseases

Clinical ImmunologistHead, 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 policymakers 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

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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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