The value conversation for a drug, especially from a patient’s perspective, extends well beyond merely drug cost. In addition to utilization of cost-effectiveness analyses, decision makers need access to all relevant data to assist informed decisions around suitable and appropriate care. Maintaining transparency and easy access to relevant data is one of the keys to continued progress in this field. Value assessment tools would similarly benefit from incorporation of patient-reported outcomes. Transparent, collaborative, and iterative approaches will be essential to create and define the success of value assessment tools.
As the drug price debate persists—and Congressional committees continue to probe and question pharmaceutical manufacturers on their pricing strategies1—transparency and data sharing will remain key if the US health care industry is ever to agree on the definition of value.
While difficult to discern from the current debate, the value conversation for a drug, especially from a patient’s perspective, extends well beyond simply the drug’s cost. So, when contemplating these diverse expectations, value assessment tools should be able to accommodate payers, employers, providers, and policymakers, along with patients who need to be active participants in their treatment decisions.
This is particularly true for chronic diseases such as rheumatoid arthritis or certain cancers that require extended treatment. A payer, for example, would be concerned with the overall treatment cost and associated “downstream costs” such as keeping the patient out of the emergency room (ER). A provider would see value in good clinical outcomes for his patient, with minimal hospitalization and few ER visits. And an employer may judge value based on whether an employee can maintain a productive work schedule through a treatment. A patient, on the other hand, might value ease of administration, symptom control, and low financial stress.
Therefore, in addition to the current gold standard of value measurement—cost-effectiveness analysis (CEA)2—decision makers need access to all relevant data to assist informed decisions around suitable and appropriate care.
CEA, represented as the cost per quality-adjusted life year, helps patients and providers make rational choices after weighing the benefits and trade-offs of an intervention within the financial context. Coverage determinations by federal programs like Medicare are independent of CEA, but critics remain concerned with the methods used or underlying intentions of those who conduct CEA—including formulary restrictions and reduced access to services.3 This has seen a gradual change as organizations such as the American Heart Association, American Society for Clinical Oncology, and the National Comprehensive Cancer Network have initiated value conversations that consider CEA along with clinical benefit and quality of life.4
Maintaining transparency and easy access to relevant data is one of the keys to continued forward progress. With the above concerns in mind, the Innovation and Value Initiative (IVI)’s Open-Source Value Platform (OSVP) makes transparency and access to data a priority. OSVP’s approach incorporates diverse stakeholder perspectives along with patient variables (eg, age, disease stage, personal preferences, and response to treatment) that bear on the value proposition of a treatment. Importantly, patient perspectives and values—gained via patient focus groups and input from patient partners—adds the unique personal (patient) touch on elements of a treatment pathway that may be overlooked by care providers.
By allowing patient-level simulation, instead of an “average patient” simulation, OSVP can evolve into a personalized value assessment for the individual patient. IVI’s new tool for modeling treatment decisions in non–small cell lung cancer (NSCLC), for example, allows users to input specific demographic and financial information.5 This is important because drug acquisition and administration costs vary greatly by the site of care (ie, community clinic vs a hospital) and where the drug is sourced from. Also, when comparing two drugs for NSCLC, different treatment-related adverse events (AEs) can impact both the risk and financial calculations. Users can assign preferred weight to attributes such as progression-free survival, total health care costs, oral administration, and specific AEs via the software’s multicriteria decision analysis option.
Embracing patient-reported outcomes (PROs) would further benefit OSVP as multiple studies6,7 have shown that PROs impact clinical care at various levels. Remote between-visit PRO documentation ensures continued patient care, especially in chronic conditions such as chronic obstructive pulmonary disease (COPD) or cancer, to allow prompt interventions that can avert ER visits.
The University of Maryland’s Patient-Driven Values in Healthcare Evaluation (PAVE) program is working along similar lines to engage stakeholders, educate patients and researchers, and disseminate the methods and principles of patient-driven assessments for real-world implementation of the findings.8 One of PAVE’s projects is incorporating the benefits and risks of COPD interventions in an economic model by evaluating different methods for patient-driven value assessments of health care services.9
As policymakers work to find common ground in the health care cost debate, focusing on eliminating low-value care from the patient, provider, and payer perspective—whether it is a drug that has a marginal impact on the desired outcome or a health care service (eg, an unnecessary imaging test) that may not impact outcome—will go a long way in reducing health care costs. Transparent, collaborative, and iterative approaches will be essential to define the success of these ventures.
1. Lovelave B Jr. House democrats launch drug-pricing probe into a dozen major health-care companies. CNBC. January 14, 2019. https://www.cnbc.com/2019/01/14/house-democrats-launch-drug-pricing-probe-on-a-dozen-companies.html. Accessed July 30, 2019.
2. Baumgardner JR, Neumann PJ. Balancing the use of cost-effectiveness analysis across all types of health care innovations [blog]. Health Affairs. April 14, 2017. doi:10.1377/hblog20170414.059610
3. Weintraub WS, Cohen DJ. The limits of cost-effectiveness analysis. Circ Cardiovasc Qual Outcomes. 2009;2(1):55-58. doi:10.1161/CIRCOUTCOMES.108.812321
4. Kimberly Westrich; National Pharmaceutical Council. Current landscape: value assessment rameworks. https://www.npcnow.org/system/files/research/download/npc-current-landscape-value-assessment-frameworks-final.pdf. Published 016.
Accessed July 30, 2019.
5. Innovation and Value Initiative. IVI-NSCLC Value Model. thevalueinitiative.org website. https://www.thevalueinitiative.org/ivi-nsclc-value-model/. Accessed July 30, 2019.
6. Basch E, Deal AM, Kris MG, et al. Symptom monitoring with patient-reported outcomes during routine cancer treatment: a randomized controlled trial. J Clin Oncol. 2016;34(6):557-565. doi:10.1200/JCO.2015.63.0830
7. Svedsater H, Jones R, Bosanquet N, et al. Patient-reported outcomes with initiation of fluticasone furoate/vilanterol versus continuing usual care in the Asthma Salford Lung Study. Respir Med. 2018;141:198-206. doi:10.1016/j.rmed.2018.06.003
8. Basch E, Barbea L, Kerrigan CL, Velikova G. Implementation of patient-reported outcomes in routine medical care. Am Soc Clin Oncol Edu Book. 2018;38:122-134. doi:10.1200/EDBK_200383
9. University of Maryland School of Pharmacy. Patient-Driven Values in Healthcare Evaluation (PAVE). pharmacy.umaryland.edu website. https://www.pharmacy.umaryland.edu/centers/patient-driven-values-healthcare-evaluation-pave/. Accessed July 30, 2019.
10. University of Maryland School of Pharmacy. Patient-Driven Values in Healthcare Evaluation (PAVE), research. pharmacy.umaryland.edu website. https://www.pharmacy.umaryland.edu/centers/patient-driven-values-healthcare-evaluation-pave/research/. Accessed July 30, 2019.