The Heart Failure Collaboratory is an academic consortium of stakeholders committed to improving the eco-system of heart failure care, which originated from a Think -Tank meeting held on March 31, 2017 (reference; JACC VOL.71, NO.4, 2018). Working groups were formed through volunteer efforts from participants of the meeting.
To advance scientific discussion, efficiency of clinical trials and evidence generation in heart failure and foster development of therapies by creating a collaborative environment in which FDA, government agencies including NIH and CMS, and the heart failure community can interact to optimize innovation and advancement of new therapeutic products.
The current state of heart failure therapeutic development in the United States is in critical need of attention. Despite the clinical burden to heart failure patients and the associated health care costs, investment in heart failure drug and device development programs has decreased relative to other clinical conditions. Although large randomized controlled trials allow robust evaluation of treatment strategies, the current clinical trial environment is burdened by increasing complexity and cost, regulatory requirements, competing demands on stakeholders, and implementation challenges. Particularly concerning is the decreasing patient and investigator participation in clinical trials in the United States. These concerning trends has led leaders in the field, including regulators and other government entities, industry, third-party payers, and academic investigators to examine mechanisms to stimulate heart failure drug and device development. The clinical research infrastructure for other disease states suggest some feasible ways in which the heart failure ecosystem can be reformed. Improved patient engagement, rejuvenated physician commitment, promotion of a clinical research culture, collaboration among the stakeholders during trial conceptualization and performance, centrally organized and vetted research networks, novel methods of evidence generation and trial recruitment, and implementation of new therapies may help reverse the deleterious trend threatening cardiovascular therapeutic innovation. Nevertheless, establishment of a functional multifaceted heart failure clinical trial apparatus will require more than a simple recapitulation of the successes realized by others, and novel methodologies will be needed in addition to active teamwork among the community. These ideas are being used as a launch pad for ongoing endeavors through a multi-stakeholder effort entitled the “Heart Failure Collaboratory.” Engaged stakeholders include patients, investigators, academic leaders, pharmaceutical and device industry representatives, society representatives, third party payers, and government representatives from the United States Food and Drug Administration, National Institutes of Health, and Centers for Medicare and Medicaid Services.
This new effort has established five working groups to tackle individual issues with short- and long-term goals for improving efficiency in clinical trials and fostering the development of effective heart failure therapies: 1) Digital Health and Technologies 2) Regulatory Policy and Implementation Science in Drug Development 3) Regulatory Policy and Implementation Science in Device Development 4) Research Networks and the Role of Societies and 5) Representative Populations.
Digital Health, Electronic Health Records, and Social Media
- Develop a process for utilizing social media to enhance trial enrollment
- Develop a proposal for utilizing/changing EMR’s into a research friendly application
- Encourage policies to change the current system to enhance evidence generation using digital tools and EMR’s
Regulatory Policy: Drugs & Devices
- Identify potential solutions to reducing regulatory burden in drug/device development, including how to make trials more efficient
- Identify methods of communication to ensure industry receives information in a timely and interpretable fashion
- Develop methods to unify the eco-system of regulatory approval and therapy utilization post-approval
Research Network and Societies
- Develop and operationalize site networks for streamlining processes (contracting, IRB, etc)
- Develop a method to utilize society networks to enhance research
- Work with resources (such as PCORnet) to develop patient research networks/foundation or equivalent
- Develop standards of representative populations for trials based on epidemiology by HF phenotype (% of blacks, elderly, HFpEF, women, comorbidities, etc)
- Develop novel methods for recruitment of these patient cohorts in trials
- Develop methods to test generalizability and assist with implementation of therapies in these populations post-approval (special population registries, etc)