New Research on VBP: Disease Severity and Unmet Need in the UK

The UK Department of Health’s value based pricing (VBP) Consultation Document proposes a process whereby higher prices would be granted to medicines that tackle diseases that produce the greatest burdens of illness – i.e. those diseases that are most severe or are associated with the greatest unmet need.

Koonal Shah

The UK Department of Health’s value based pricing (VBP) Consultation Document proposes a process whereby higher prices would be granted to medicines that tackle diseases that produce the greatest burdens of illness – i.e. those diseases that are most severe or are associated with the greatest unmet need.  This suggests combining severity of disease and unmet need into a single metric.  A literature review reveals that this approach has not been studied adequately (or perhaps at all) to date, either in the UK or elsewhere.

The Department of Health’s Policy Research Unit in Economic Evaluation of Health and Care Interventions (EEPRU) was asked by the Department to conduct an empirical study of society’s resource allocation preferences to obtain weights for burden of illness, therapeutic improvement and end-of-life.  The objective would be to apply these, under the coming VBP system, to assessments of both new technologies and displaced activities.

The EEPRU study defines burden of illness as distance from “normal” health; the measure combines health status with the effects of existing standard therapy on both length and quality of life.  The value of existing therapy, then, is in its contribution to the degree of burden.  Not considered explicitly in the EEPRU study are issues raised by the definition of unmet need implied in the VBP Consultation Document – i.e. that “unmet need” means that no treatment is available at all.

Data collection for the EEPRU study is being conducted online, using discrete choice experiments to elicit preferences.  Stakeholders have expressed concern about the limited scope of the EEPRU study in that it does not examine whether people have preferences about the availability of alternative treatments.

To address some of these issues, OHE currently is performing a small study, funded by an unrestricted research grant from the ABPI.  Based on interviews with members of the general public, it is intended to develop a more nuanced understanding of the extent of societal support for basing health care resource priorities on disease severity and unmet need.

The OHE survey will be administered in face-to-face interviews conducted by trained interviewers with experience in conducting health care preference studies.  Questions will be designed to identify respondents’ levels of support for policies that give greater weight to treatments that address disease severity and unmet medical need than to those that do not.  The research also will address whether, controlling for disease severity, unmet need per se affects societal preferences regarding priorities for treatment.  In this study, respondents also will be asked probing follow-up questions intended to elicit qualitative information about the thinking behind their choices.

This study will provide important, preliminary information about how disease severity and unmet need should be measured and how best to capture societal preferences.  The main points we expect the research to address include the following:

1. Whether members of the public would support a policy that uses combined disease severity and unmet need as a criterion for priority setting
2. What societal preferences about disease severity and unmet need can be observed
3. Whether preferences regarding unmet need depend at least in part on disease severity
4. The likely reasons for the preferences observed
5. The strength of the preferences observed

The OHE study, then, will both complement the EEPRU study by exploring additional possible attributes and provide additional evidence about whether preferences regarding unmet need depend on disease severity.  It can help define what further research is essential to produce valid measures.

The OHE research team for the project is Koonal Shah and Nancy Devlin.  Results are anticipated in summer 2012 and will be published as an OHE Research Paper.