Societal preferences for funding orphan drugs
Research type
Research Study
Full title
Societal preferences for funding orphan drugs
IRAS ID
167841
Contact name
Siobhan Bourke
Contact email
Duration of Study in the UK
0 years, 3 months, 19 days
Research summary
Orphan drugs is the term applied to drugs meeting regulatory approval for the treatment of rare conditions. These drugs may be expensive to develop relative to the small numbers of patients who receive these treatments. As such, these therapies tend to be high-cost and rarely meet existing cost effectiveness criteria. Unless there are special conditions for exceeding the cost-effectiveness threshold, orphan drugs would struggle to get reimbursed on the NHS. Policies have already been developed to incentivise pharmaceuticals companies to produce orphan drugs internationally, and many HTA organisations recommend orphan drugs for reimbursement despite their apparent cost-ineffectiveness. However, there remains controversy concerning whether or not orphan drugs should adhere to the rigor of traditional cost effectiveness standards, or whether or not these drugs should have an alternate process for funding within national health care systems. Many believe that society’s preferences for funding orphan drugs is essential in determining any health care systems policy for funding orphan drugs.
Previous studies of society’s value of rare diseases have focused mainly on the characteristic of rarity. However, this is not the only characteristic of rare diseases that society may value. Rare diseases are usually serious and life threatening conditions, for which there are limited, or no alternative treatments available. While this is true also of some common diseases, it may be the case that society values the equity of access to treatment for rare diseases. However the range of attributes relating to societies value of orphan drugs have yet to be fully explored.
This study will identify the significant characteristics of orphan drugs by gaining opinions from 3 different stakeholder groups: patients with rare disease and /or their care givers, expert clinicians and policy makers. Three techniques to measure preferences will be used. Discrete choice experiment will estimate preferences by allowing a number of attributes to be traded against each other and a Person trade off technique which will measure the different values of health states in society.REC name
Wales REC 5
REC reference
15/WA/0181
Date of REC Opinion
22 May 2015
REC opinion
Favourable Opinion