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Presentation

Session: Drug Development
Room: Main Hall
Time: Fri 10:15-11:30

Presenter: Kathrin Roll (Helmholtz Zentrum Munich. Institute for Health Economics and Health Care Management)

Abstract

Background: This paper analyses schemes to promote authorization and reimbursement of orphan drugs.

Methods: Eight countries – Australia, Canada, Germany, Great Britain, France, Netherlands, Switzerland and the USA – were studied to compare specific regulations for orphan drugs regarding drug admission, Health Technology Assessment (HTA), decision-making for reimbursement, and off-label- and compassionate-use. Information was obtained by reviewing published and grey literature. Expert interviews were also conducted.

Results: In all analyzed countries (except Canada) specific legislation has been enacted to encourage the development of orphan drugs. However, Canada provides other incentives, e.g. for life threatening diseases or for small patient populations, for which orphan drugs generally qualify. The comparison of orphan drug laws reveals that the EU and the US offer the greatest incentives for the development of orphan drugs, whereas there is a tendency for Australia and Switzerland to profit from incentives in other countries. Most healthcare systems assess cost-effectiveness of drugs for reimbursement decisions. Although not explicitly stated, economic evaluation of orphan drugs takes the special circumstances for orphan drugs into account. Almost all countries reveal deviations from common assessment methods. If high-priced orphan drugs are not subject to common reimbursement practices, special schemes or programs for the reimbursement exist in all countries that were analysed. Therefore access to orphan drugs seems to be warranted. However, due to co-payments of 5% for orphan drugs, the US may represent an exception. Access to drugs prescribed off-label- or for compassionate-use is warranted in all countries. However, regulations for reimbursement of orphan drugs in the case of off-label or compassionate-use are heterogeneous. Currently, the analysed health care systems do not seem to offer an adequate solution to this problem.

Conclusion: In the near future, personalized medicine will lead to a subdivision of patient population into smaller groups. This might increase the numbers of authorized orphan drugs that qualify for the analysed schemes to promote authorization and reimbursement of orphan drugs. On the one hand, the use of special criteria for drug admission, HTA, and reimbursement promotes R&D for orphan drugs. On the other hand, high opportunity costs arise, because huge efforts are made for a minority of patients. A solution for this moral dilemma may be the application of the ’Rule of Rescue’ as moral imperative to rescue individuals in immediate peril, regardless of cost. Also, the use of ’No Cure, no Pay’ initiatives seem to be a promising method to introduce value based pricing in order to deal with rising drug costs.

Key Terms

orphan drugs, rare diseases, economic evaluation, reimbursement, decision-making

Authors:

Kathrin Roll (Helmholtz Zentrum Munich. Institute for Health Economics and Health Care Management) , Tom Stargardt (Munich University. Health Service Management) and Jonas Schreyoegg (Munich University. Health Service Management)

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