Background
Real-world data (RWD) and real-world evidence (RWE) play an increasing role in health care decisions across different disease areas. These types of data have the potential to optimize clinical trial design, inform regulatory decision making, and allow for the overarching medical community to address questions previously considered unanswerable.
The Translating Real-World Data into Real-World Evidence Project brings together the Liver, PSC, and Rare Diseases Forums to explore the opportunities and challenges associated with application of RWD and RWE across a collective yet inherently nuanced medical landscape.
Subject matter experts from industry, regulatory agencies, insurers, academia, and patient organizations will consider foundational areas and key uncertainties pertinent to the operationalization of RWD throughout the scientific community, including though not limited to the following: 1) whether RWD are fit-for-use in generating RWE; 2) data quality, curation, and continuity; and 3) reliability and validity of innovative analytic tools to address questions of interest such as causality and improve the efficiency of clinical studies for mitigation of existing knowledge gaps and patient outcomes.
More information on each of the participating disease-specific Forums can be found below:
The Liver Forum aims to advance the regulatory sciences for the treatment of NAFLD, NASH, and liver fibrosis. For conditions with a long asymptomatic natural history such as NASH, the time to reach hard endpoints such as cirrhosis, hepatocellular carcinoma and death can be a significant barrier for clinical trials. The potential for accelerated approval, requiring identification and validation of surrogate markers, is hampered by the heterogeneity of disease presentation, underlying disease mechanisms, and progression. The field also lacks ideal standards for assessing and staging fibrosis, NAFLD, NASH, and cirrhosis. Effective therapeutic strategies will likely require combination therapies, personalized according to patient characteristics. However, the regulatory pathway for combination therapies can be complex, full of uncertainties, and burdensome for patients, clinical researchers, regulators, and the industry. The Liver Forum provides a platform for ongoing multi-stakeholder dialogue to address these issues.
The Primary Sclerosing Cholangitis (PSC) Forum, which operates under the auspices of the Liver Forum, aims to advance the regulatory sciences for the treatment of PSC and its associated morbidities. For conditions, such as PSC, studies of medical treatment are complicated by asymptomatic presentation, poorly understood natural history, and a long time to reach hard endpoints, such as liver transplantation or death. The potential for accelerated approval, requiring identification and validation of surrogate markers, is hampered by the low incidence of the disease, numerous phenotypes, variable disease progression, and multiple clinical outcomes. As treatments continue to evolve, this rapidly moving and dynamic field will require collaborative bridges and coordinated efforts between many parties.
The Rare Diseases Forum brings together stakeholders to carefully deliberate on the necessary steps to transform clinical research by modernizing clinical trials to advance the development of new therapeutics for people with rare diseases. The Rare Diseases Forum aims to facilitate the development of new therapeutics for rare diseases by 1) building scientific knowledge and developing a common understanding of issues and potential solutions, 2) maximizing efficiency through collaborations, and 3) making use of innovation and use of novel/innovative analytics, and efficient/rigorous evaluation of novel biotechnology.
Action Plan
The project will consist of a series of webinars to explore the following areas:
I. Translation of real-world data from various sources to fit-for-purpose evidence
- Identification of biomarkers associated with disease progression
- Determination of efficacy for investigational treatments through the use of external controls
II. Acceptable and innovative statistical approaches for the analysis of real-world data
III. Challenges
- Maintenance of data collection
- Continuity across age groups
Expected Outcomes
Expected outcomes include the formation of new working groups to carry on the work of the overarching project by addressing priority areas identified.
Working groups will communicate and collaborate through conference calls, email, webinars, and web-meetings as needed. There is no time requirement for participation, but working group members are strongly encouraged to actively contribute.
Project Launch
This project launched with Webinar 1 on July 20, 2021. The first webinar featured patient, industry, academic, and regulatory experts from the US and Europe. Topics discussed include: 1) overarching principles for fit-for-purpose data; 2) data quality and effective standards for the regulatory process; 3) practical application of RWE; 4) lessons learned from use of RWD as external controls; 5) the role of data at different stages of drug development: from Proof of Concept to confirmatory; and 6) translation of RWD from various sources to fit-for-purpose evidence. Webinar 2 took place on Monday, November 8, 2021 from 9:00 am EST - 11:00 am EST. Topics discussed include: (1) a successful real-world evidence case-study, (2) exploring innovative statistical approaches for the analysis of real-world data and (3) the challenges of maintaining data collection and continuity across age groups.
