Innovation by Patients with Rare Diseases and Chronic Needs

Abstract

We provide the first empirical exploration of disease-related innovation by patients and their caregivers. Our aims were to measure the frequency of innovation by these patients and their caregivers, and the improvement in well-being they experienced from using what they have developed. In addition, we explored the diffusion of their innovations to others, and factors associated with both patient innovation development, and patient innovation sharing.

We administered a questionnaire via telephone interviewing to a sample of 500 rare disease patients and caregivers. Subjects were selected from a list of individuals who contacted the helpline of a major patient association serving rare diseases patients from 2009 to 2012. The solutions reported by individuals who agreed to participate were validated for their novelty by two medical professionals. Logistic regression models were used to test the relationships between our key variables and patient innovation and solution sharing.

263 (52.6%) of our survey respondents reported developing and using a solution to improve management of their diseases which they thought to be novel. Of these, 40 (8%) were evaluated as indeed being novel by two expert medical evaluators. The remaining 44.6% were already known to medicine, although thought novel by the patients who recreated them. The likelihood of patient innovation increased as education level increased (OR 2.1, p<0.05), and as their perception of limitations imposed by their disease increased (OR 1.3, p<0.05). 84 individuals diffused their solutions to some degree, with 74 of these sharing via direct diffusion to other patients. There is a positive relationship between the impact of a solution on the respondents’ overall quality of life, and likelihood of solution sharing, and an inverted U relationship between age and solution sharing.

Given that there are hundreds of millions of people world-wide estimated to be afflicted by rare diseases, patient and their caregivers may be a tremendous potential source of innovations and information to improve management and care for many who are similarly afflicted. Our findings suggest that many patients could be greatly assisted by improved diffusion of known best practices to and among patients and their caregivers.

Keywords: patient innovation, rare diseases, diffusion

JEL Classification: O31