Title: $15,000 a dose: A patient-centered study of drug development and research

Rationale: The United States spends $100 billion annually on cancer care, with the majority of costs connected to drug development and technological advances.¹ Several researchers have raised concern that the cost of cancer care will continue to rise until it becomes unsustainable.^1,2 One strategy for addressing the rising treatment costs is to consider cost when planning clinical trials. Value-of-information theory (VOI), commonly used in economic and decision science settings, is one example of a method to address this issue. Applied to clinical trials, VOI theory could help determine which trials should be funded by considering a combination of factors such as drug price, cost of the trial, and duration of treatment.³ Using this theory, a drug that is projected to offer only similar clinical benefit – such as progression-free survival – as another but is more costly would not be selected for trial funding.

While VOI highlights how consideration of cost is needed at the development phase, we believe that patients have a role to play in this phase as well.⁴ Currently, no studies to date assess patients’ perspectives about the escalating cost of treatment and the implications for drug development, giving rise to a challenge in the design and conduct of clinical research. High treatment costs have resulted in increased cost-sharing with patients through higher premiums, co-pays, and deductibles. Since patients bear more of the burden of paying for these cancer treatments, it is critical that their perspective is taken into account early in the drug development phase. For example, it is important to know how patients feel about the development of highly expensive drugs that would 1) contribute to the societal burden of increasing health care costs, and 2) likely be too expensive for the majority of patients in the era of increased cost-sharing. It has been suggested that what patients really want from health care (i.e. timeliness, hope, and certainty with no interest in the real cost of treatment or the percent GNP devoted to health care) is often “irrational and unrealistic.”⁵ We feel that patients must first be presented with information about actual treatment costs and the process of drug pricing, and only then can they be expected to consider the trade-offs of health care decisions as informed, rationale consumers.

Plan: We will conduct a pilot project that will survey and interview patients at the UCSF Breast Care Center about their perspectives on the cost of cancer care and drug development in relation to clinical research. The aim of the study will be to design a patient-centered framework for approaching costs within the context of clinical research. We will recruit a diverse group of participants, including women who are and are not currently enrolled in clinical trials, women with and without a prior cancer diagnosis, and women in diverse socioeconomic, age, and ethnic groups. Participants will be presented with simple scenarios that illustrate the critical issues about drug costs and insurance coverage rates, and how clinical research can influences these issues. These scenarios will be designed with the advice of Celia Kaplan, DrPH, an expert in qualitative methods, using language and terms understandable to the average patient. Participants will be surveyed to understand patient perspectives on the following: 1) trends in cancer treatment costs, 2) implications of cost-sharing (co-pays, high deductibles) on drug development and research, 3) the process of drug pricing, and 4) opinion of using VOI methodology in the early phase of drug development. We plan to frame some of our analysis according to participants’ responses to certain ethical questions such as who should determine how money is spent in health care and how “irrational and unrealistic” preferences should be considered.

This study is “shovel-ready” – the investigative team has worked together on similar projects in this clinical setting and these participant groups, and has patient surveys and IRB protocols ready for submission once funding is secured.

Criteria and Metrics for Success: From the responses and data gathered from this study, a framework for approaching costs within the context of clinical research will be developed.  Specifically, the pilot study will be a success if the study can determine the following: 1) patient views regarding VOI methodology for use in drug development, and 2) guiding principles around patient treatments costs and clinical research.

The broader implications of the study will guide more selective funding of clinical trials (such as through VOI) or a reassessment of these selection methods that considers patients’ perspectives on the cost of cancer care and drug development. The investigators have the unique opportunity to implement the findings of this study in the UCSF Athena Breast Health Network, an innovative collaboration across the 5 University of California medical centers that is integrating clinical care and research to drive innovation in prevention, screening, treatment and management of breast cancer. Our study will develop the knowledge base required to establish a scalable and sustainable approach to the conduct of research in a novel clinical care and research cohort such as Athena. Elissa Ozanne, the study PI, is the Director of Risk Assessment and Decision Science for Athena, and Laura Esserman, a study collaborator, is the PI for the UC-wide Athena Network. Together, these study investigators will design the study such that results can be directly applied to ongoing and future Athena research efforts.

Cost and justification: The anticipated costs are $25,100 to support 1) 5% effort ($10,800) for the PI (Elissa Ozanne, PhD), 2) a clinical trial coordinator (Rebecca Howe) at 20% of her effort ($9,800), 3) patient recruitment and survey costs ($2,800), 4) data analysis costs ($1,200), and 5) supplies ($500).

Collaborators: Elissa Ozanne, PhD (UCSF – Department of Surgery, Institute for Health Policy Studies), Michael Hassett, MD (Harvard Medical School), Rebecca Howe (UCSF – Department of Surgery, Institute for Health Policy Studies), Lisa Bero, PhD (UCSF – Department of Clinical Pharmacy), Michael Alvarado, MD (UCSF – Department of Surgery), Laura Esserman, MD, MBA (UCSF – Department of Surgery, PI of Athena), Celia Kaplan, DrPH (UCSF – Department of Medicine)

References

1. Meropol NJ, Schrag D, Smith TJ, et al: American Society of Clinical Oncology guidance statement: The cost of cancer care. J Clin Oncol 27:2868-3874, 2009
2. McFarlane J, Riggins J, Smith TJ: SPIKE$: A six-step protocol for delivering bad news about the cost of medical care. J Clin Oncol 26:4200-4204, 2008
3. Schmidt C: Researchers Consider Value-of-Information Theory for Selecting Trials. J Natl Cancer Inst 102(3):144-146, 2010
4. Fleck LM: The costs of caring: Who pays? Who profits? Who panders? Hastings Cent Rep 36(3):13-17, 2006
5. Detsky AS: What patients really want from health care. JAMA 306(22):2500-2501, 2011