Drugs For Rare Diseases: How To Fund Them?

Toronto, ON, Canada– Enzyme replacement therapies for rare inherited metabolic diseases are among the most expensive drugs in the world and have created a dilemma for public drug payers.

Often the cost effectiveness of a new drug is used to guide public drug funding, decisions, and this is usually estimated from the efficacy of the new treatment in randomized clinical trials.

However, randomized trials may be difficult or impossible to complete for truly rare conditions. A recent report published in Value in Health, “An Evaluation Framework for Funding Drugs for Rare Diseases” describes a novel approach to evaluating such drugs based on an understanding of the disease and the available evidence.

The study was co-authored by members of the Ontario Public Drug Programs Drugs for Rare Diseases Working Group.

Lead author, Dr. Eric Winquist says, “Our evaluation framework attempts to systematically address the unique challenges raised when considering funding new drugs for rare diseases, which are often expensive, with an evidence-based publicly funded drug program.”


Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision makers, and researchers worldwide).

International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health.

For more information: www.ispor.org

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