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News Archive


Drugs for Ultra-Rare Disorders: Cost Drivers and Budget Impact

In this new paper, the Institute reports on cost drivers and budgetary impact of drugs for rare and ultra-rare disorders in Europe. In a systematic search, a total of seven studies with specific estimates of the budget impact of drugs for rare diseases for a total of nine countries were identified. Annual per-capita spending for orphan drugs ranged from €0.48 in Russia to €16 in France. Only one study on URDs was identified. In Germany, annual treatment costs per patient for drugs for non-oncological URDs vary between €1,175 and €726,890. In a regression analysis, a significant inverse relationship between availability of alternative treatments for the same indication and annual treatment costs was found. In addition, log prevalence was found to have a significant inverse relationship with log annual treatment cost.

Thus, an inverse relationship between prevalence and annual treatment costs was found specifically for drugs for non-oncological URDs. Despite annual treatment costs in the range of several hundreds to thousands of euros for some of the URD drugs, per-capita spending for URD drugs was found to be relatively small.

Also see Discussion Papers.

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HTA Stakeholders Call for More Efforts into the Further Development and Testing of Social Cost Value Analysis as an Alternative Approach to Health Economic Evaluation

A new position paper authored by an international group of health economists, and experts in clinical pharmacology, Health Technology Assessment (HTA), and medical ethics calls for more efforts into the further development and testing of alternative health economic evaluation paradigms, notably social cost value analysis (SCVA).

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Vocatus Atque Non Vocatus Deus Aderit.

Du kannst Tränen vergießen,
Weil er gegangen ist.
Oder du kannst lächeln, weil er gelebt hat.
Du kannst die Augen schließen und beten,
dass er wiederkehrt.
Oder du kannst die Augen öffnen
Und all das sehen, was er hinterlassen hat

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Invitation to Stakeholders concerned with Economic Evaluation and Health Technology Assessments (HTAs)

International implementation of Health Technology Assessments (HTAs) has varied greatly, largely driven by differences in understanding and capturing the "value" offered by health technologies, such as new pharmaceutical products, diagnostic procedures, and medical devices. Given the relevance of this development, please allow us to take this opportunity to draw your attention to this year's Heidelberg Health Economics Summer School.

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