Tuesday, June 7, 2016

Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers

T. Morel, S. Aymé, D. Cassiman, S. Simoens, M. Morgan and M. Vandebroek. Orphanet Journal of Rare Diseases 2016 DOI: 10.1186/s13023-016-0444-9

This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context.




Our study data confirmed that patients and their caregivers were willing to accept greater risk or side effects associated with a new medicine, for instance, in the hope for some extra chance in drug response or greater health improvement potential. Attitudes about benefit-risk may change over time with disease progression or context of care.

In that context, we believe that patients and caregivers should be increasingly involved as active research partners in the development of clinical outcomes assessments – including patient-reported outcomes (PRO) measures – that directly evaluate how the patient feels, functions or survives. If a treatment effect is not meaningful to the patient, it is not a benefit to the patient.

Open Access
This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://​creativecommons.​org/​licenses/​by/​4.​0/​)

Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers