When it was signed into law in December of 2016, the bipartisan 21st Century Cures Act aimed to expedite the U.S. drug approval process, streamline clinical trial processes, funnel increased money into precision medicine, and enhance the use and sharing of electronic health information.
As previously reported in Oncology Times, the act's funding for National Institutes of Health (NIH) innovation projects included $1.8 billion for the Cancer Moonshot initiative, which was led by former Vice President Joe Biden.
The National Comprehensive Cancer Network (NCCN) held an online summit with speakers and panelists to examine the effects of the Cures Act on accelerating cancer research and to explore what successor legislation to this act-referred to as Cures 2.0-should contain. The summit was moderated by Clifford Goodman, PhD, Senior Vice President of the Lewin Group, and included references to how the Cures Act has helped shape the response to the COVID-19 pandemic.
"We are in a much better spot today" with the pandemic because of the Cures Act, said Rep. Fred Upton (R-MI), a key sponsor of the 2016 act along with Rep. Diana DeGette (D-CO). At the summit, Upton and Degette announced that they have released a concept paper on what Cures 2.0 should look like, and said they may release new 2.0 draft legislation this summer.
21st Century Cures Act Advances
The 21st Century Cures Act contributed to significant advances, including establishment of the Oncology Center of Excellence (OCE) at the FDA, innovative treatments such as CAR-T, a framework for using real-world evidence at the FDA, and advances in digital health such as increased data interoperability, said Clay Alspach, JD, a principal with Leavitt Partners who helped draft the Cures Act legislation when he worked on Capitol Hill. He noted that the OCE is valuable because it brought together different silos at the FDA, while the law's rule on data interoperability was "a huge advance."
At the virtual summit, policy recommendations from an NCCN working group were presented by Jeff Allen, PhD, President & CEO of the Friends of Cancer Research, and breast medical oncologist Ronald S. Walters, MD, MBA, MHA, MS, the NCCN Board Chair and Associate Head for the Institute for Cancer Care Innovation at the University of Texas MD Anderson Cancer Center. The policy recommendations identify strategies to address gaps and opportunities related to cancer research and health information technology (HIT). The policy recommendations include the following:
1. Congress must provide robust, sustained, and predictable annual funding increases to guarantee continued clinical advances and clinical trial success.
2. The U.S. Department of Health and Human Services (HHS) must encourage increased interagency collaboration and communication to enhance the oncology product approval process and benefit future coverage processes.
3. The FDA should establish parameters for the use of real-world data (drawn from non-trial sources such as electronic health records and claims data, for example), including patient-reported outcomes, post-market analysis, and the representation of diverse population groups to leverage real-world evidence.
4. Future federal legislation and HHS regulations should encourage and give incentives to ensure widespread patient participation in clinical trials to increase diverse study populations.
5. HHS should build upon regulations of the Office of the National Coordinator for HIT to expand available standardized data elements in a manner that is actionable to the patient and the health provider.
6. Congress and payers should promote policies and reimbursement mechanisms that support interoperability and encourage the aggregation of patient data that will promote shared decision-making and increase understanding among patients, providers, and payers.
7. The Centers for Medicare & Medicaid Services (CMS) and private and commercial payers should maintain and build on advancements made during the COVID-19 pandemic to enhance patient access to telehealth services and increase access to tumor boards to improve patient care.
Referring to the third recommendation, the 21st Century Cures Act "set the stage for accelerating the use of real-world data" in drug development, said medical oncologist Amy Abernethy, MD, PhD, FDA's Principal Deputy Commissioner of Food and Drugs.
Now, she said, "I think COVID-19 has forced us to think about the role of real-world data writ large." She noted that oncology was already "fertile ground" to put real-world data into use in the process of drug development because of the availability of high-quality data sets. Abernethy added that oncology advances have been made in combining multiple real-world data sets from EHRs, claims information, registries, patient reporting, and patient sensors with biological datasets.
Abernethy noted that the abrupt impact of COVID-19 on clinical trials has led the agency to consider the swiftly increased use of telehealth and remote monitoring and the need for regulatory flexibility. For example, she said, FDA staffers are thinking about using real-world data to fill in gaps in clinical trials caused by the pandemic-participants who missed clinic appointments, for example. The pandemic has also led to a "big push for master protocols" for clinical trials, she added.
The fifth recommendation focusing on improved use of standard data elements to foster interoperability in HIT is a work in progress, said speakers. Ideally, now that the HIT Cures Act rule has been finalized, this rule "will give patients unprecedented access and control over their medical records," said Don Rucker, MD, MBA, MS, National Coordinator for HIT in the Office of the National Coordinator for Health IT.
"For patients and caregivers affected by cancer, the rule, when fully implemented, will improve care coordination by allowing patients to aggregate and share records across clinicians, increasing a patient's [access] to take their records and seek further opinion. The rule will also allow new opportunities for patients to participate in clinical trials and contribute data to researchers working on new treatments and cures."
But significant challenges remain. "I think patients don't yet totally understand how to use their data...Today it takes an extraordinary effort for patients to access their records at multiple facilities in order to share it so it is correlative with other patients and relevant for research," said Jenny Ahlstrom, a multiple myeloma patient and founder of HealthTree, the first online portal for multiple myeloma patients to find their best treatment options. "Many forget that patients are not just data sets...HealthTree was designed to empower patients to make the most educated decision at every stage of their disease journey, all while moving the research community closer to a cure."
Noting that COVID-19 has "created enormous challenges for the cancer community," Ahlstrom said that HealthTree, a division of the patient-driven nonprofit CrowdCare Foundation, has recruited more than 1,000 multiple myeloma patients for an observational study on the pandemic's impact on them.
Agreeing with Ahlstrom that there is considerable room for improvement in HIT was Anobel Odisho, MD, MPH, urologist and Clinical Informatics Lead at the University of California (San Francisco) Center for Digital Health Innovations and the UCSF Helen Diller Family Comprehensive Cancer Center.
"The dramatic and rapid uptake of telehealth has shown great promise for patient care, but also exposed deficiencies in the existing software solutions for providing remote care," said Odisho. "Providers have been able to transition to video visits and telehealth, but the field is immature and there are significant opportunities to improve the quality of care and efficiency of care delivery...I think for us the big issue is data interoperability." He noted that telehealth visits at his cancer center have mushroomed from 8 percent to 70 percent of total patient visits since the pandemic.
Odisho added, "Now that the Cures Act rule has been finalized, allowing patients to access their own data, provisions against information blocking, and clear timelines, HIT developers are empowered to develop solutions for improving remote patient care-such as video visits, automated workflows, and asynchronous virtual care. We will have to keep a close eye on utilization of information blocking exemptions to ensure they are having the intended effect and are not being exploited outside of the intended scope."
DeGette noted that the 21st Century Cures Act which she co-sponsored "gave a lot of support to the FDA." This support, she said, "allows them to be more nimble with clinical trials," which should be helpful as the country battles the COVID-19 pandemic with its many unknowns.
The concept paper developed by DeGette and Upton for potential Cures 2.0 legislation has received more than 500 comments, and includes the following goals and recommendations:
1. Improvement of public health and pandemic preparedness.
2. Better caregiver integration into health care, for example by training through educational skills programs.
3. Enhanced patient engagement in health care decision-making leading to increased patient empowerment.
4. Increased population diversity representation in clinical trials.
5. Further modernization of FDA, which might include grants for innovative clinical trial design, increased emphasis on patient-focused drug development, and use of real-world data and real-world evidence.
6. CMS modernization to allow the agency to keep up with new technology and scientific advances.
In a joint statement, DeGette and Upton said, "Thanks to [the Cures Act], we're better able to prevent and screen cancer, we have a better understanding of the human brain, and we're improving the field of stem cell science...But despite these successes, there's still much more work to be done."
Peggy Eastman is a contributing writer.