In the review article, published recently in the Journal of the American College of Cardiology, the authors focus on the cardiovascular disease-related recommendations, providing insights in five key areas: 1) global cardiovascular health; 2) screening for early detection and treatment of cardiovascular disease; 3) catalyzing innovation, including accelerating drug development, addressing research and development capacity and leveraging digital health; 4) smart financing strategies; and 5) global health leadership.

"These NASEM recommendations and this manuscript are among the most important efforts of my career, because if they are adopted by the U.S. government, they have the potential to enact true change for global health."

“The Committee has highlighted priority areas that demand continued investment and underscored the imperative to change the way the United States approaches global health, to a more proactive and integrated strategy,” the authors write. “One targeted area is CVD, as part of general expansion of the global health vision to encompass chronic NCDs. The outcome of investment in global health, now shifting to include NCDs, will be resilient countries with positive health outcomes, robust trade partners, safer travel destinations, and more active collaborators in preventing and controlling global health problems that affect citizens of every country, at every income level.”

Should the U.S. choose to prioritize cardiovascular disease and other NCD programs within its global health strategy, the authors stress the country could benefit in the following three ways: 1) adoption of a universal purpose; 2) economic prosperity and trade benefits; and 3) the safeguarding of U.S. global health investments.

“These NASEM recommendations and this manuscript are among the most important efforts of my career, because if they are adopted by the U.S. government, they have the potential to enact true change for global health,” says corresponding author Valentin Fuster, MD, PhD, MACC. “The next step for the committee is to present these recommendations to the U.S. Senate and to President Donald J. Trump.”

Fuster V, Frazer J, Snair M, et al. J Am Coll Cardiol 2017;Nov 30:[Epub ahead of print].

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“Earlier and predictable patient access to effective new cardiovascular therapies is the primary goal underpinning proposals to collaboratively engage regulatory and payer representatives in pre-approval drug development decisions,” the authors write. They also suggest that transparency and accountability can be shown by defining requirements for reimbursement before regulatory approval. They explain that implementing strong scientific standards for cost effectiveness or other analyses used in reimbursement decision-making can help achieve greater transparency and acceptance of reimbursement decisions.

While consistency and quality of payer interactions are questionable, steps are still being taken to promote earlier interaction among regulators and payers in North America, Europe and other parts of the world. For example, although it is limited, the U.S. allows both the Federal Drug Administration and Centers for Medicare and Medicaid Services to concurrently review data about medical devices to incorporate both perspectives into a single clinical trial. Europe has also taken steps to advance progress toward early interaction by engaging a variety of tools to handle uncertainty from lack of information about budget impact, cost effectiveness, use in real life and access.

Additionally, the authors explain that greater resource allocation is needed for updating data on efficacy and safety, and revoking either regulatory or reimbursement approvals should not be underestimated.

“There is no easy solution, but collaborative engagement of regulators, payers, health technology assessment bodies, pharmaceutical companies, academic leaders, investigators, patients, physicians, professional organizations, and other health care providers represents an important step to developing a system that reflects the views and needs of all stakeholders,” the authors write.

Zannad F, Alonso Garcia MLA, Borer JS, et al. J Am Coll Cardiol 2017;70:2822-30.

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Although survival rates to adulthood for patients with congenital heart disease (CHD) is close to 90 percent, survival rates to a similar age for patients with severe CHD, including those with single-ventricle anatomy, is much lower at 56 percent. In addition, long-term follow up of patients who had the Fontan procedure suggests 59 percent freedom from adverse cardiovascular events five years post operation, but only 29 percent freedom after 15 years. This suggests an “important emerging medical crisis,” the authors write.

According to the report, an accurate definition of FALD that is widely recognized is necessary for investigators to conduct research that will lead to improved understanding of FALD epidemiology, causation, pathophysiology, prevention and treatment. In addition, research into cardiac and extracardiac mechanisms that contribute to patients’ risk for FALD could help identify those at higher risk of the disease and enable earlier screening. A longitudinal registry of patients who have had the Fontan procedure could help advance these research efforts, according to the report.

The report committee recommends several practices for prevention, screening and treatment of FALD. For prevention, the authors advise that providers focus on optimizing underlying Fontan physiology and preventing liver injury before and after a Fontan operation. Screening for FALD should, at a minimum, include baseline liver function tests five years post Fontan operation for children, or sooner if a liver injury or Fontan failure is suspected. Adults and adolescents who have had the Fontan procedure should have liver function tests every one to three years. Routine physicals should include laboratory studies; evaluation of Hepatitis A, B and C exposure; and liver imaging. In addition, the report outlines considerations for Fontan patients undergoing evaluation for heart or liver transplantation or combined heart and liver transplantation.

Collaboration between health care societies and organizations, research institutions and subspecialists involved in treatment of Fontan patients will be critical to mobilizing resources necessary to improve understanding and treatment of FALD. The authors conclude that moving forward, “We need an investment toward research and technologies post-Fontan operation that matches our past and current commitment to pre-Fontan patients.”

Daniels CJ, Bradley EA, Landzberg MJ, et al. J Am Coll Cardiol 2017;70:3173-94.

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