By Maxine Bookbinder 

February 23, 2015 | In an effort to heal the ailing American health care system, the 114th Congress is expected to consider several bipartisan healthcare bills this year, ranging from more efficient discovery and development processes to patient input expansion. 

“This is the golden age of opportunity for new technology, basic science, the maturity of the human genome project, and for potential cures for disease,” says Kim McCleary, director of strategic initiatives at FasterCures, a think tank of the Milken Institute determined to remove barriers to medical progress. McCleary notes that the “15 year, $2 billion” therapy may finally become history. There is a universal hunger to make health advances more democratized, to improve economic impact and quality of life, for a better system of development and delivery.”

For example, McCleary notes a patient-centered focus. “We are now in a new age of patient empowerment. The model of healthcare used to be paternalistic. Doctors decided what was important and beneficial. Now, the patient is a more active participant. Patients are driving decisions.  Patient organizations are shaping conversations in a way that they didn’t have an opportunity to before. Patients are no longer just consulted as tokens. Regardless of what happens with individual pieces of legislation, this is the legacy of the movement.”

Current congressional action is mixed with a dose of patriotic altruism. “There is an interest in other countries in ramping up investments in science and progress,” says McCleary. “We don’t want to fall behind.”

Mary Woolley, president and CEO of Research!America, another advocacy group working to promote health research, agrees. “The time is now to discard the complacency that has left us with stagnant funding or worse for agencies like The National Institutes of Health (NIH) and provide at least a 10% increase in 2016. It’s up to Congress to assure our nation’s preeminence in science and innovation remains firmly in place by taking bold, swift action to help improve the health of Americans.

“The tsunami of Congressional proposals in support for research is a welcome change that the nation has been waiting for, in terms of finding answers to health challenges that only research can provide,” says Woolley. “Policymakers are stepping up at last to make research a high priority again, after nearly a decade of budget cuts and inaction on policies that drive private sector innovation. Many Americans say that patients with various diseases and their families, and scientists who conduct the research to improve health should have more influence on how the government funds for research to improve health are spent, according to our polls.”

One highly-anticipated bill is the 21st Century Cures Initiative, which emphasizes “discovery, development, and delivery cycle of new cures and treatments.” It was introduced by Congressman Fred Upton (R-MI), chairman of the Energy and Commerce Committee, and Congresswoman Diana DeGette (D-CO), and aims to close the gap between science and regulation. Specific measures include  modernizing and increasing access to clinical trials through simplified paperwork, reduced costs, and completion time; investing in advancing research; incentivizing the development of new drugs, including antibiotic drug development and dormant therapies and devices for unmet medical needs; and facilitating data sharing.

In addition, the bill addresses streamlining regulatory processes and equipping federal agencies, including the FDA, CDC, NIH, and CMS, with tools to maintain pace with innovation and technology. It also calls for simplifying patient recruitment, encouraging new public-private partnerships, encouraging modern manufacturing technology in the U.S. and updating medical device regulations.

The initiative comprises a 21st Century Consortium to foster continuing development in health information technology, such as apps to modernize discovery, treatment, and patient input, and to encourage young scientists and entrepreneurs to stay in the U.S.

Last year, members of Congress collected ideas from patients, medical professionals, innovators, and researchers. The public is invited to submit suggestions on improving the legislation at cures@mail.house.gov.   Supporters hope the initiative, expected to be 300 – 400 pages, will be introduced by Memorial Day.  

The National Organization of Rare Disorders (NORD) hopes Congress addresses several problems, including data collaboration. According to NORD, 80% of rare diseases are genetic; comparing data would benefit diagnoses and therapies.  “Understanding the progression of rare diseases is of the utmost importance to the National Organization for Rare Disorders,” says Diane Dorman, vice-president of public policy at NORD. “So little is known about the natural histories of most of them. By building registries that can speak with one another, it is hoped that in the long-term, possible discoveries could lead to a proper diagnosis and aid in the development of promising therapies. Whether this can be resolved legislatively is the question that must be answered by the scientific and medical communities,” adds Dorman, who hopes for a “meeting of the minds” between the House and Senate to ultimately pass the bill.

President Obama’s Precision Medicine Initiative, mentioned in his 2015 State of the Union speech and more fully outlined at the end of January, proposes a $215 million investment for the NIH, the FDA, and the Office of the National Coordinator for Health Information Technology (ONC).  Objectives include the creation of a “cancer knowledge network” and acceleration of cancer trials and treatment; development of a national research cohort including at least one million volunteers; regulatory modernization; public-private partnerships; and a renewed, multi-stakeholder commitment to privacy protection.

Another proposed bill is the Ensuring Access to Clinical Trials Act, which would allow people receiving Supplemental Security Income (SSI) and Medicaid to participate in rare disease clinical trials without losing up to $2,000 in benefits. Introduced by Senate Finance Committee Ranking Member, Ron Wyden (D-Ore.), Finance Committee Chairman Orrin Hatch (R-Utah), and Senators Edward Markey (D-Mass) and Sherrod Brown (D-Ohio) in January,  this bill will make permanent the Improving Access to Clinical Trials Act of 2009, due to expire in October. The Government Accountability Office (GAO) studied it and did not report any negative findings.

The original bill mandated that clinical trial compensation not be considered income for recipients of SSI and Medicaid.

“We all know what’s at stake and how it’s often difficult to find participants for rare disease clinical trials,” Wyden said in a press release. “This law has helped increase the number of people who can participate and, hopefully, be a part of the effort to improve treatments and find cures.”  

Since 2009, 36 people with rare diseases participated in clinical trials who otherwise would not have been able to. Despite the small number, “this is a big deal,” says one senate staffer, “because it is hard to find people for these trials.”

No timeline has been set for debate or vote. “The law is definitely helping people,” says the staffer. “We hope for more sponsors.”

In January, Sen. Dick Durbin (D-IL), reintroduced the American Cures Act to increase funding for biomedical research at the NIH, CDC, Department of Defense Health Program (DHP), and the Veterans Medical and Prosthetics Research Program by the rate of 5% above GDP-indexed inflation yearly.

According to Durbin’s office, 53% of all funding for basic research came from the federal government in 2012. Today, the federal government spends two-thirds less on research and development than it did in 1965. The American Cures Act would reverse this. “Maintaining significant funding for scientific research is the smartest investment we can make, and it will pay for itself,” Durbin said in a press release.

Also in January, Rep. Rosa DeLauro (D-CT) and Rep. Brian Higgins (D-NY) re-introduced The Accelerating Biomedical Research Act, designed to create a new BCA cap adjustment for the NIH to allow funding in excess of $29.4 billion and increase NIH funding by 10 percent for the first two years and about six percent yearly through 2021. 

“One of my proudest accomplishments as a member of Congress is helping to double NIH’s funding,” DeLauro said in a press release. “Work supported by the NIH has saved the lives of countless Americans. Failure to invest in health research and disease prevention results in huge costs to our health, society, economy and knowledge itself.  Whether it is cancer, Ebola, or the flu, the benefits of medical research are obvious. Congress must stop forcing the NIH to do more with less.”

Later this year, Ohio Senator Sherrod Brown (D) plans to reintroduce the Strategies to Address Antimicrobial Resistance (STAAR) Act, intended to strengthen the federal response to antibiotic resistance by promoting prevention and control through partnerships at the CDC and local health departments; tracking drug-resistant bacteria through improved data collection and mandated reporting; supporting enhanced research efforts; reauthorizing a task force and coordination agency efforts; and directing the NIH to collaborate with other agencies and experts to address the problem.

If passed, Brown’s bill will establish an Office of Antimicrobial Resistance (OAR) at the Department of Health and Human Services (HHS) to provide greater leadership, coordination, and accountability involved in combating drug resistance.

A few of these bills failed previous passage and were reintroduced; whether they reach debate and a vote is unknown. However, progress has already been made. “There is the potential for things to grow even if not legislated,” says Faster Cure’s McCleary. “People are saying, ‘This is what we want.’”