viernes, 28 de junio de 2019

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy | Orphanet Journal of Rare Diseases | Full Text

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy | Orphanet Journal of Rare Diseases | Full Text



Orphanet Journal of Rare Diseases

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy

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Orphanet Journal of Rare Diseases201914:154
  • Received: 20 March 2019
  • Accepted: 11 June 2019
  • Published: 

Abstract

Background

Decision makers have huge problems when attempting to attribute social value to the improvements achieved by new drugs, especially when considering the use of orphan drugs for rare diseases. We present the results of a pilot study aimed to investigate patient preferences regarding public funding for drugs used to treat rare diseases.

Methods

An online questionnaire was used as a discrete choice experiment (DCE) survey to explore the preferences of patients with cystic fibrosis and haemophilia in Italy. The questionnaire focused on relevant issues that were defined in a review of the literature. A conditional logistic model showed preferences for specific attributes.

Results

A total of 54 questionnaires (20% response rate) were completed. The issues that received the greatest attention were improvement in health, treatment cost and value for money. However, disease severity and the availability of other treatments were important social values that could not be ignored.

Conclusions

The findings presented here provide evidence as to what patients with cystic fibrosis or haemophilia think are the most important considerations on which to base decisions in health technology scenarios, and regarding the priorities for funding.

Keywords

  • Discrete choice experiment
  • Cystic fibrosis
  • Haemophilia
  • Decision making
  • Rare disease
  • Orphan drugs
  • Italy

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