Treatment of HF in an Era of Multiple Therapies: Statement From the HF Collaboratory

Statement From the HF Collaboratory

Ankeet S. Bhatt, MD, MBA; William T. Abraham, MD; JoAnn Lindenfeld, MD; Michael Bristow, MD; Peter E. Carson, MD; G. Michael Felker, MD, MHS; Gregg C. Fonarow, MD; Stephen J. Greene, MD; Mitchell A. Psotka, MD, PHD; Scott D. Solomon, MD; Norman Stockbridge, MD, PHD; John R. Teerlink, MD; Muthiah Vaduganathan, MD, MPH; Janet Wittes, PHD; Mona Fiuzat, PHARMD; Christopher M. O'Connor, MD; Javed Butler, MD, MPH, MBA


JACC Heart Fail. 2021;9(1):1-12. 

In This Article

Developing an Implementation Science Framework

Developing a framework that is robust, durable, and malleable to the changing landscape of discovery science in HF will require renewed effort prioritizing implementation science across all major stakeholders (Central Illustration). Trialists, while continuing to evaluate novel HF therapeutics in large prospective efficacy studies, must also develop trials focused on implementation of therapies. These trials may need to borrow expertise from alternative disciplines, including behavioral science, sociology, and health economics, among others. These implementation trials may use traditional or novel strategies (e.g., randomization within registries, cluster randomization across sites). Implementation trials should also focus on uptitration and evaluating combinations of therapeutics as well as health system–based strategies such as engagement with non-physician personnel to aid in optimizing therapy. "Lean" shared case report forms[50] can standardize data collection across trials, facilitating data-pooling efforts. An implementation committee may be proposed that plans for the study of post-trial implementation of results. Relationships will need to deepen across community providers to enroll patients into implementation and sequencing trials. Funding agencies, in collaboration with health systems, will have to prioritize trials to answer questions regarding how to implement therapies.

Central Illustration.

Developing an Implementation Science Framework in Heart Failure
This figure captures the key stakeholders and tenants needed to build an implementation science framework across heart failure (HF) with reduced ejection fraction care. Key stakeholders will include clinical researchers, academia, regulatory agencies, sponsors, and patients, among others. An implementation framework should leverage novel methods of analyses and emerging tools to allow for implementation science to keep pace with discovery science in heart failure with reduced ejection fraction.

Payers will be central in ensuring coverage mechanisms that limit out-of-pocket patient cost and accelerate access. Ideally, standardization across different payers will avoid situations in which out-of-pocket cost and inadequate coverage limit use of disease-modifying therapies. Robust cost-effectiveness evaluation will be needed to promote broad coverage.[51,52] As payment models shift toward value-based payment structures in which population health management will be crucial, limiting costs and accelerating access will be important in curtailing costs.[53] Payers will need to continue to fund claims-based analyses that might provide insights into how to provide the most effective and fiscally responsible care for patients with HFrEF. Regional differences in covered populations will also need to be addressed. Regulatory frameworks to ensure expedited review and approval of beneficial therapies may allow for earlier integration into clinical practice and earlier initiation of strategy and implementation trials.

Patient representation in trial design, recruitment, and management will be important as implementation and strategy trials are effectively designed. In addition, focus groups with patients will allow researchers to understand how many therapies that typical patients will be willing to take or at which point issues with adherence and/or side effects may become barriers to effective drug therapy. Furthermore, engaging patients to understand which behavioral economic strategies may be most likely to succeed may improve the design and potential success of strategy and implementation trials.