The challenges of orphan drugs and associated solutions proposed by Nordic HTA agencies
Translated title
Udfordringer med orphan lægemidler and de associerede løsninger forslået af nordiske MTV-agenturer
Author
Saravanabavananthan, Sahana
Term
4. term
Publication year
2020
Submitted on
2020-05-29
Pages
51
Abstract
Orphan drugs for rare diseases expand treatment options but place pressure on publicly funded health systems due to very high prices and limited evidence. This thesis aimed to identify the challenges faced by Nordic health technology assessment (HTA) agencies in evaluating and adopting orphan drugs and to explore potential solutions. Seven individual interviews were conducted with HTA representatives in Denmark, Finland, Norway, and Sweden, and a collaborative process design was developed to outline steps toward effective cross-border collaboration. The interviews highlighted three prominent challenges: high prices, uncertainty or paucity of clinical evidence, and imbalanced negotiating power that disadvantages small purchasers. Agencies use a variety of measures to mitigate these issues, and enhanced Nordic collaboration is proposed as a possible way forward; however, a preliminary guideline indicates that many components must be agreed among countries before collaboration can work effectively. The thesis concludes that further work is needed and that inclusive engagement of all stakeholders is essential to devise adequate, sustainable solutions.
Orphan-lægemidler til behandling af sjældne sygdomme giver nye behandlingsmuligheder, men udfordrer offentligt finansierede sundhedssystemer på grund af meget høje priser og begrænset evidens. Dette speciale havde til formål at identificere de udfordringer, som nordiske sundhedsteknologivurderingsmyndigheder (HTA) møder ved vurdering og ibrugtagning af orphan-lægemidler, samt at belyse mulige løsninger. Der blev gennemført syv individuelle interviews med HTA-repræsentanter i Danmark, Finland, Norge og Sverige, og der blev udviklet et samarbejdsproces-design, som skitserer trin mod effektivt grænseoverskridende samarbejde. Interviewene pegede på tre gennemgående udfordringer: meget høje priser, usikker eller sparsomt klinisk evidensgrundlag og asymmetrisk forhandlingsstyrke til ugunst for små købere. Myndighederne anvender forskellige tiltag for at håndtere dette, og styrket nordisk samarbejde fremhæves som en mulig vej frem; en foreløbig retningslinje viser dog, at en række elementer skal afklares mellem landene, før et samarbejde kan blive effektivt. Konklusionen er, at der fortsat er behov for yderligere arbejde og bred inddragelse af alle relevante interessenter for at udvikle holdbare løsninger.
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