Patient-centered evidence is playing an increasingly important role in health care decision-making. Regulators, health technology assessment (HTA) bodies, payers and employers are seeking a more complete understanding of how disease and treatment affect patients' lives, alongside traditional measures of clinical benefit.
This shift is evident across the health care ecosystem.
The FDA's Patient-Focused Drug Development initiative and Benefit-Risk Framework encourage regulators to consider patient experience data when they make decisions about new drugs. HTA organizations around the world increasingly include patient evidence and input in value assessments. Payers and employers are also looking beyond traditional clinical endpoints to better understand treatment burden, quality of life and outcomes that matter most to patients.
Why it matters:
The challenge is that most organizations still generate patient-centered evidence on a study-by-study basis. While individual studies provide valuable insights, they rarely provide a full picture of what is known, what evidence is missing or what will be needed to support future decisions.
Zooming in:
We use a Patient Experience Dossier (PE Dossier) framework to address this challenge.
A Patient Experience Dossier organizes patient-centered evidence around the aspects of disease and treatment that matter most to patients and are most relevant to health care decision-making. This may include burden of disease and unmet need, treatment experiences, patient preferences, meaningful outcomes and barriers to access and care.
By bringing these domains together in a single, decision-oriented resource, organizations can identify where more evidence is needed across each step of patient journey, in different markets and on key patient experience topics. It can then map those evidence gaps to the decisions they are intended to inform throughout the product lifecycle.
Rather than creating a separate workstream, the dossier becomes the foundation for both evidence planning and evidence generation.
Evidence Planning & Strategy. The first step is to assess the patient-centered evidence that is already available and identify evidence gaps.
- These gaps may be tied to a stage in the patient journey, market or specific patient experience topic.
- The next step is to connect those gaps to the decisions they need to support. This may include internal product strategy and Target Product Profile discussions, anticipated regulatory and HTA evidence expectations and environment-shaping activities that support patient access and adoption.
- This process helps organizations determine where new studies are needed, where existing studies can be strengthened and how patient-centered evidence can support multiple decisions throughout the product lifecycle.
Evidence Generation. The next step is to fill important evidence gaps.
- Sometimes this requires new studies, generated through qualitative, quantitative, or mixed methods approaches. In other cases, small enhancements – such as adding patient-centered questions to planned qualitative research or expanding baseline surveys to better capture patient and caregiver burden – can substantially strengthen the evidence package.
Bottom Line:
A Patient Experience Dossier is more than a summary of existing evidence or a traditional evidence gap assessment. It is a strategic planning framework that organizes patient-centered evidence around the aspects of disease and treatment that matter most to patients.
It also connects evidence gaps to the decisions the evidence is meant to support. This enables organizations to prioritize research investments, avoid unnecessary duplication and ensure patient-centered evidence is available when regulators, HTA bodies, payers, providers and other decision-makers need it.
Go deeper:
We introduced the concept of Patient Experience Dossiers in a Health Affairs Forefront article, describing how these dossiers can help organizations organize patient-centered evidence, identify evidence gaps and support decision-making throughout the product lifecycle.
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