Core research objectives
- First, to understand whether rarity has an impact on the assessment of the incremental cost per QALY
of orphan drugs compared with drugs for more common diseases and whether rarity impacts mainly on the incremental costs or on the incremental QALYs.
- Second, given the difficulties of conducting randomized clinical trials and the progressive nature of many rare diseases, to identify alternative robust sources of data to accumulate knowledge on the effectiveness and societal value of orphan treatments which can be used for policy decisions.
- Third, to determine in what way(s) do current processes for assessing and appraising drugs need to be
adapted to make them suitable for orphan drugs, and whether all elements of societal value can be adequately reflected in existing decision-making procedures.
- Fourth, to develop and validate a framework to support decision-making relating to orphan drugs for rare diseases, by deploying a discrete choice experiment survey.
During the first two years of the project, WP3 has completed the following tasks:
- Systematic comparison of HTA decision-making processes for a sample of 34 orphan drug-indication pairs using the analytical framework developed in WP2. The study researched :
- agency-specific preferences in the type of evidence appraised
- risk and value preferences in agencies’ assessment of the same evidence, and
- how these influenced the decisions in each country, partly explaining some of the reasons for different HTA outcomes across countries.
The study identified differences at each stage of the process (e.g. in the evidence appraised, the interpretation of this evidence, and how it influenced the final outcome), further explaining the reasons for different HTA outcomes
- Literature review suggesting that DCE surveys could provide useful insights regarding benefit valuation of various health care interventions’ characteristics, which would be ignored in clinical outcomes or QALYs, and might be useful as additional information in a medical-decision process about health care interventions or services. Therefore, DCE outcomes may be more appropriate to show the relative importance of the attributes of a health care intervention to predict real choice
- DCE survey pilot for decision makers and for Italian patients with rare diseases.
During third and final year of the project, WP3 has completed the following tasks:
- Interviewed HTA body representatives to ensure the accurate interpretation of HTA processes and decisions analysed through the framework, and simultaneously get further insights on the drivers of these decisions in each agency
- Fully implemented the DCE survey for decision makers in five countries (Spain, UK, France, Germany, Italy) and for Italian patients with rare diseases
Assessing the Societal Value in Rare Diseases by Decision Makers and Patients
Principal Investigator: Dr Domenica Taruscio