The current environment for heart failure (HF) clinical trials and the development and implementation of novel HF therapeutics is strained by cost, regulatory requirements, decreasing patient and investigator participation and other barriers, according to a state-of-the-art review paper published Jan. 22 in the Journal of the American College of Cardiology.

In March of 2017, various stakeholders and representatives from the U.S. Food and Drug Administration, National Institutes of Health and Centers for Medicare and Medicaid Services convened to begin the process of developing strategies to address these challenges. Christopher M. O'Connor, MD, FACC, et al., outline the current obstacles, actionable opportunities and targeted recommendations discussed at this meeting, including the formation of an organized Heart Failure Collaboratory.

The Heart Failure Collaboratory has established five working groups to tackle individual issues with short- and long-term goals for improving the current environment by producing, distributing and enacting guidance and recommendations: Electronic or Digital Health; Regulatory Policy and Implementation Science in Drug Development; Regulatory Policy and Implementation Science in Device Development; Research Networks; and the Role of Societies and Representative Populations.

Though randomized clinical trials are a primary resource for clinical evidence generation, the authors write that trials have become slow, cumbersome and costly due to myriad causes. Poor patient engagement, limited clinician participation and poor return on investment were three key causes discussed by stakeholders and representatives.

They explain that poor patient enrollment, due to inaccessible trial information, absence of support from members in the HF community and limited time for discussion during clinical visits, lead to slow and costly clinical trials. Low rates of clinicians participating as investigators in HF trials is the result of poor incentivization, the required time and trial bureaucracy, difficulty navigating onerous eligibility criteria, lack of institutional support and a lack of financial or professional compensation. Developing a clinically effective and commercially viable product is fraught with regulatory and payer uncertainty, and the return on investment can be minimal. Following phase 1 trials, only 10 percent of drugs for cardiovascular disease eventually achieve regulatory approval. These issues, and others, combine to create the current strained clinical research infrastructure.

"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 a focus on implementation of new therapies may help reverse the deleterious trend threatening cardiovascular therapeutic innovation," write O'Connor, et al.  "Establishment of a functional, multifaceted HF 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."

Keywords: ACC Advocacy, Research Personnel, Investments, Pharmaceutical Preparations, United States Food and Drug Administration, Medicare, Medicaid, National Institutes of Health (U.S.), Research, Heart Failure, Cardiomyopathies, Cardiovascular Diseases

< Back to Listings