August 19, 2014
The U.S. House of Representatives Energy and Commerce Committee has engaged in a flurry of activity since the May launch of the 21st Century Cures initiative that aims to accelerate the pace of cures and medical breakthroughs in the United States. The committee has held three roundtables and six Congressional hearings on various topics affecting the full arc of the drug development process – from discovery to development to delivery – as well as published four white papers.
Demonstrating the committee’s determination to hear from a broad range of stakeholders, here’s a summary thus far:
- 67 innovators, business executives, and government agencies have spoken at roundtables
- 35 leaders in biomedical R&D have provided testimony
- 9 healthcare innovation topics have been addressed
- 4 white papers have been authored by the committee
- 139 organizations have responded to the white papers:
- 45% patient advocacy groups/nonprofits
- 30% industry representatives
- 25% academics/professional societies
This is a bipartisan effort, co-chaired by Energy & Commerce Committee Chairman Fred Upton (R-MI) and Committee Member Diana Degette (D-CO). Rep. Degette stated at the launch of the initiative that “we can either work together, or we will fall behind.” Similarly, Chairman Upton explained the goal is to “cure the patients, and keep more jobs in the U.S. What family isn’t affected by this?”
With such high-level input for leaders across the biomedical research field, it is no wonder Rep. Joe Barton (R-TX) called 21st Century Cures “the most exciting thing the Committee is doing this year.” As the committee enjoys a brief recess before these meetings continue in September, here are five themes that have emerged from testimony and public submissions thus far:
1. Encourage and incentivize public-private partnerships that accelerate medical research.
Exemplified by the committee’s call for input from all stakeholders at the outset, the message that “we can’t do it alone” has been echoed throughout the sessions and public responses. Many organizations called out the Accelerating Medicines Partnership between the National Institutes of Health (NIH), 10 biopharmaceutical companies, and several nonprofit organizations to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets of disease. Witnesses called for more open communication between industry and regulators to expedite the drug development process. Sara Radcliffe, executive vice president of health, Biotechnology Industry Organization, testified that the U.S. Food and Drug Administration (FDA) and sponsors should be encouraged to “discuss potential surrogate or intermediate clinical endpoints earlier in drug development.”
2. Encourage new models for clinical trials that take advantage of today’s science.
Recognizing that clinical trials represent the biggest expense and one of the most difficult challenges in the R&D process, many stakeholders pointed to possible solutions to revamp the traditional model, including adaptive clinical trial designs and greater use of surrogate endpoints and patient-reported outcomes. The newly launched Lung Cancer Master Protocol (Lung-MAP) multi-drug, multi-arm, biomarker-driven clinical trial was highlighted as a possible model for future cooperative trials.
As an AP article on Lung-MAP explained it, “Instead of being tested for individual genes and trying to qualify for separate clinical trials testing single drugs, patients can enroll in this umbrella study, get full gene testing and have access to many options at once.” Richard Pazdur, M.D., director of FDA’s Office of Hematology and Oncology Products hopes this will “minimize the number of patients exposed to ineffective therapies.”
Jay Siegel, M.D., chief biotechnology officer at Johnson & Johnson, discussed the promise of adaptive clinical trials as one potential way for making clinical trials more efficient by reducing the time, costs, and patients needed. There are several efforts currently underway. One example is a consortium known as I-SPY 2, which uses biomarkers to match the types of breast cancer patients who are enrolled in a trial. As Siegel mentioned during his testimony, adaptive clinical trials present “the opportunity to reach success or failure faster, thereby reducing the cost of development.”
3. Leverage data collected to advance medical knowledge and improve patient care.
Electronic health records (EHRs) and patient registries have enormous potential to be better utilized in a learning healthcare system model that rapidly identifies new targets and new standards of care. Mark Blatt, M.D., worldwide medical director of Intel Corporation, stated that Congress has “set the conditions in the United States which are necessary but not sufficient to enable care to move into cyberspace.”
Jonathan Bush, chairman, CEO, and president of athenahealth, Inc., pointed out that there is little venture capital being spent on disruptive health information technology and that that the 21st Century Cures initiative needs to give a voice to the smaller players and entrepreneurs who can transform the system in the same way that they have changed other industries. Anne Wojcicki, CEO and co-founder of 23andMe, continued this point, stating “technology and healthcare are coming together, and consumers want to be a part of it. And you can see how it’s disrupted other industries and the rest of the world, and it is going to disrupt healthcare.”
4. Find the right incentives for encouraging the most innovative research.
At a roundtable/hearing focused on incentives, leaders debated the different ways to encourage more investment in medical innovation, including market exclusivity, expedited review for special medical uses and incentives for personalized medicine. As Jonathan Leff, partner, Deerfield Management, noted, “No one is happy it takes 10 to 15 years to develop drugs and that patients are waiting too long and that’s driving investment away.” The need for flexibility at FDA to manage the review of new models of drug development and surveillance was highlighted across several of the hearings. In addition, several groups have called for new incentives to create new antibiotics.
5. System needs a steady, predictable trajectory of support for medical research.
As the committee looks toward creative solutions to incentivize collaborations, the overall need for robust and predictable funding remains paramount for the federal agencies directly responsible for medical innovation, including the FDA and NIH. Multiple stakeholders pointed to the more than 20 percent drop in NIH’s purchasing power, including NIH Director Francis Collins, who stated that “what’s most desperately needed to continue what has been the most successful story of biomedical research that the world has ever seen is a steady, predictable trajectory of support.”
FDA would also benefit from robust, predictable funding. As FDA Center for Drug Evaluation and Research Director Janet Woodcock stated, the FDA strives to continue “successfully using a number of flexible and innovative approaches to expedite the development and review of drugs to the benefit of millions of Americans.”
FasterCures will continue to track this important initiative that seeks to ensure that the United States remains the leader in biomedical research that benefits our economy, improves lives, and speeds cures to patients.