Scientists in academic or other institutions use the unique insights patients provide to inform their research. Pharmaceutical companies oftentimes look for customer input primarily in late development stages. So far however, their main focus has been on physicians. Working with all stakeholders, including patients, early in the R&D process can help make it more productive and lower development costs by reducing the failure rate.
Off to the PROM
Once a drug is on the market, one way of assessing treatment outcomes are patient-reported outcome measures (PROM). PROM development can be broken down into three phases:
- Determination of measurable health outcomes; framework development
- Item development (may overlap with step 1)
- Testing for comprehensibility (e.g. Is the questionnaire easily understood?)
Patients contribute to PROM development through questionnaires, interviews, focus groups or other means. Patients were involved in under 75% of PROM development studies published between 1980 and 2014 and oftentimes only in selected aspects. Notably, the choice of the type of outcome to be measured (step 1) was left to others in almost 90% of those studies. The variability of patient inclusion between studies calls for better guidelines and common standards in this process.
Organisations like the UK’s National Institute for Health Research (NIHR) provide funding specifically to enable patient and other public involvement. However, researchers still experience budget constraints that hinder appropriate patient involvement by academic or public bodies.
Patients’ Fair Market Value
Even though funding conditions are rarely ideal, various stakeholders agree that patients should be compensated for their contribution. According to surveys, ca. two-third of organisations provide financial compensation to patients or patient advocates. Ideally, this would be done through a standardised approach that reflects their Fair Market Value (FMV). However, the details of such an FMV are still unclear. Reimbursement of necessary expenses, e.g. caregiver, appear to find acceptance. In contrast, more fanciful compensation, like trips or meals, are less on payers’ radars. Payment for travel time is also uncertain, and willingness may depend on the associated burden, e.g. travel despite unfavourable physical condition. Overall, it appears that expense reimbursements would be easily incorporated in the FMV but anything beyond poses a bigger challenge. Unsurprisingly, other crucial factors for the FMV are the extent of patients’ experience and knowledge of the disease and relevant systems. FMV guidance should also consider the possibility for patients to decline payment offers.
Patients are consultants
It is an unquestioned business reality that professional consultancies monetise their data and expertise. By sharing their insights with researchers and drug developers, patients and their advocates are effectively consultants. Yet, it does not seem a natural conclusion to pay them as such. It could be argued that patients will benefit from the resulting advances in research and development, which is their return on the invested time and effort. However, this is a long-term benefit, and for numerous diseases it may not happen during the lifetime of the study participant.
- Finn, C. 28/08/2019. Minister commits to paying patient advocates for their time and work. https://www-thejournal-ie.cdn.ampproject.org/c/s/www.thejournal.ie/paying-patient-advocates-4784624-Aug2019/?amp=1 (accessed 29/08/2019)
- Finnegan, G. 23/07/2019. Fair Market Value: Survey reveals new insights on patient compensation. https://patientfocusedmedicine.org/fair-market-value-survey-reveals-new-insights-on-patient-compensation/ (accessed 21/08/2019)
- NIHR. How we involve patients, carers and the public. https://www.nihr.ac.uk/about-us/our-contribution-to-research/how-we-involve-patients-carers-and-the-public.htm (accessed 21/08/2019)
- O’HAGAN, P. and FARKAS, C., 2009. Bringing pharma R&D back to health. Boston, Massachusetts, USA: Bain & Company.
- WIERING, B., DE BOER, D. and DELNOIJ, D., 2017. Patient involvement in the development of patient-reported outcome measures: The developers’ perspective. BMC Health Services Research, 17(635), pp. 1-10.
- WIERING, B., DE BOER, D. and DELNOIJ, D., 2016. Patient involvement in the development of patient-reported outcome measures: a scoping review. Health Expectations : an international journal of public participation in health care and health policy, 20(1), pp. 11-23.