Patient-Centered Outcomes Challenge Award

The Value Assessment Initiative Challenge Awards began in 2017.The 

Application Deadline    March 1, 2022 @ 11:59 PM EST

KEY DETAILS

As a part of its overall Value Assessment Initiative, the PhRMA Foundation has established the Value Assessment Challenge Awards to help promote leadership and innovative approaches to determining value in health care.

A key challenge in accounting for patient-centered outcomes and impacts in value assessment is the lack of existing data (e.g., clinical trials, observational data sources) and data collection tools (e.g., prospective EMR data collection) to measure outcomes important to patients.

We invite submissions based on bold and creative ideas to advance methods and process associated with value assessment (and/or value elements). The PhRMA Foundation seeks papers that describe solutions to one or both of the following questions. (The total response must not exceed 3,000 words excluding title, figures and bibliography.)

1.  What are potential solutions for more rapidly prioritizing and closing evidence gaps to measure treatment effects on patient-centered outcomes and impacts (e.g., using RWE and electronic health data) in clinical or economic evaluations? Include case studies and consider the role of key research organizations (e.g., PCORI, AHRQ).
2.  What templates or techniques can be employed to reliably demonstrate important gaps in meaningful patient-centered outcomes and impacts?

BACKGROUND

In recent years, a variety of “value frameworks” have emerged—largely in reaction to rising health care costs and other perceived health system inefficiencies—with the aim of better assessing the value of health care interventions and particularly new medicines. Many of these frameworks seek to provide evidence and/or a recommendation for payers in making decisions about specific medical products and services.

At the same time, many researchers and thought-leaders are recognizing that, for value and value assessment to play a more meaningful role in health care decision-making in the U.S., better definitions and methods for assessing value must be developed. Appropriately defining and measuring the value of a health care intervention can significantly alter individual perceptions of value.

The value of an intervention depends on the outcomes it produces in a specific disease and the characteristics of the patient. As such, one important aspect of value assessment is the identification and measurement of outcomes that matter to patients, but that often are not captured in pre-registration clinical trials or lack meaningful patient engagement in their development and use. Patient-centered outcomes (PCOs) can be defined as the outcomes important to patients in the way they experience a disease or a treatment for that disease. PCOs may include a range of measures: clinical (mortality, biomarkers), patient-reported outcomes (symptoms, function, preferences), treatment-related attributes (mode of administration), resource availability and use (hospitalizations), and/or societal impacts (productivity, caregiver burden). Further, individual patients will often have preferences specific to their circumstances and inclinations. Important PCOs are often omitted from traditional approaches to value assessment methods and processes, which tend to focus on a selected subset of clinical outcomes simply because these are the endpoints studied in trials. Incorporating patient input throughout the medical product development life-cycle, post-market real-world data collection efforts, and value assessment processes can inform the development and inclusion of PCOs.i

As the field of value assessment evolves, it will be critical to invest in better methods and data infrastructure to identify, capture, and quantitatively incorporate PCOs in value assessment of a medical product and ensure that these analyses reflect what matters most to patients. While significant progress has been made in the field of PCO research (PCOR), there remains a need to better incorporate PCOs in the value assessment process. Initial steps to identify which PCOs are core to specific patient communities can also guide prioritization for data generation efforts.

In the shift toward a value-driven healthcare system, there appears to be growing consensus on the need to apply a concept of value that is reflective of the patient perspective and based on active patient participation that incorporates the patient voice throughout the medical product development life-cycle. As advances in the life-sciences industry continue to result in novel medical discoveries and promising cures for patients affected by acute, chronic, and rare diseases, it is more important than ever to ensure the field of value assessment advances with it. Until significant progress is made to better align value assessment with PCOs and the ability to capture, measure and operationalize them, defining the true value of a health care intervention will remain a challenging endeavor. Better capture of PCOs in value assessment can also pave the way for their consideration in other decision contexts, such as innovative contracting arrangement and quality measurement.

Examples may include, but are not limited to:

• Propose a conceptual approach that allows a population-based value assessment to be tailored to individual preferences at the point of care decision-making
• Develop an approach to define a comprehensive set of PCOs to capture health care value that can be integrated into a value assessment framework
• Design a framework for identifying PCO measures, including relevant data sources, such as real-world data or qualitative research with patients
• Identify solutions to data limitations and barriers related to operationalization of PCOs, such as leveraging patient registries
• Design a value assessment methodology that accounts for patient-centered outcomes
• Propose a mechanism to collect or disseminate patient-reported data to incorporate in value assessment
• Explore how emerging innovative contracting arrangements can play a role in developing or advancing adoption of PCOs and measures

i See, FDA Patient Focused Drug Development (PFDD). “Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.” https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development