Background on Cures 2.0
On June 22, 2021, Congresswoman Diana DeGette (D-CO-01) and Congressman Fred Upton (RMI-06) released draft legislation called Cures 2.0 to build on the success of their previous legislation called the 21st Century Cures Act, which was signed into law on December 13, 2016, after securing overwhelming bipartisan support. The preceding legislation was focused on improving how new drugs and medical innovation are researched and developed in the U.S. and included the Cancer Moonshot Program led by then Vice President Biden. The new Cures 2.0 proposal seeks to improve how those new therapies and innovations are delivered to patients. Over the past year, the DeGette-Upton effort produced another version of Cures 2.0 focused on COVID response that was released on April 27, 2020, but was scrapped with the incoming Biden administration. The proposal will continue to be developed and revised as it moves through Congress and likely, will be folded into a larger package.
CURRENT VERSION OF CURES 2.0
This latest version of Cures 2.0 would authorize President Biden’s Advanced Research Projects Agency for Health (ARPA-H) initiative and address key gaps impacting how innovative health care reaches or fails to reach patients. It would start with FDA and focus on increasing diversity in clinical trials to ensure efficacy and safety data is collected on all populations that ensures proper drug labeling and avoids adverse drug reactions while also clarifying breakthrough designations that coordinate better with CMS. Cures 2.0 would also facilitate Medicare coverage of these breakthrough drugs, regenerative medicine, and devices once FDA makes the designation. Medicare coverage would be provided to genetic testing and much of the telehealth services that became the norm during the COVID-19 pandemic. Finally, it would focus on NIH and CDC by authorizing significant new funding resources for initiatives to enhance health literacy, promote vaccinations and pandemic planning, accelerate bringing critical need antimicrobial drugs to market, advance caregiver skill development and boost translational research and development.
21ST CENTURY CURES ACT SUMMARY
The 21st Century Cures Act was a landmark piece of legislation designed to accelerate medical treatments, devices, research, and diagnostic breakthroughs. A core component of the 21st Century Cures Act was to streamline drug and device approval pathways at the FDA to support innovation, particularly in drugs, combination products, antimicrobials, orphan drugs, rare disease therapeutics, and regenerative medicine. The law established the Next Generation of
Researchers Initiative and reauthorized the rare pediatric disease drug voucher program. It also included provisions to promote electronic health record (EHR) technology and provide savings for Medicare and Medicaid and the Prevention and Public Health Fund. It provided billions of dollars in funding for medical research initiatives, including:
- $4.8 billion over 10 years for NIH: $1.45 billion for the Precision Medicine Initiative, $1.51 billion for the Brain Research Through Advancing Innovative Neurotechnologies Initiative (BRAIN), $1.8 billion for the Cancer Moonshot Initiative and cancer research, and $30 million for regenerative medicine using adult stem cells
- $500 million to the FDA to move drugs and medical devices to patients more quickly
- $1 billion over two years for grants to states for opioid abuse prevention and treatment
- $14 million for fiscal years 2018-20 for National Mental Health and Substance Use Policy Laboratory (NMHSUPL) grants within SAMHSA
Cures 2.0 Overview
Cures 2.0 would build upon its predecessor, the 21st Century Cures Act, by improving the delivery of and accessibility to medical innovations for patients. While ARPA-H is drawing significant attention, there are other important changes proposed that would accelerate bringing medical innovations to market and broad adoption throughout health care systems.
It would authorize $6.5 billion to create ARPA-H, which would be based off the Department of Defense’s Defense Advanced Research Projects Agency (DARPA), a key resource for developing technological advancements for the U.S. military. ARPA-H would follow DARPA’s model by providing experts with the resources to advance medical breakthroughs for Alzheimer’s disease, cancers, diabetes, and other conditions. A potential ARPA-H project envisioned by the Biden Administration is to develop an MRNA vaccine technology to prevent most cancers.
KEY COMPONENTS OF CURES 2.0
The Cures 2.0 would address several areas impacting the delivery of medical innovations to patients with the following actions:
- Require pandemic planning and assessment of COVID patients called long haulers, who suffer from symptoms long after being infected
- Expand Medicare coverage by making innovative new health care technologies
accessible to beneficiaries
- Increase diversity in clinical trials to ensure that new drugs and treatments are useful for all populations
- Require the FDA to improve upon the collection and use of real-world evidence when developing new patient-focused treatments
- Expand caregiver training programs to ensure patients receive a higher quality of care Improve accessibility and availability of health information for patients so they better understand their illness and treatment options
- Make telehealth services more accessible for Medicare, Medicaid and Children’s Health Insurance Program (CHIP) beneficiaries
SECTION 1: PUBLIC HEALTH AND COVID-19 RESPONSE
Cures 2.0 contains several provisions that would establish collaborative
practices based on lessons learned during the COVID-19 pandemic. The legislation would require the secretary of Health and Human Services (HHS) to conduct a nationwide study to further understand the implications of long COVID, a condition that causes patients to experience a range of symptoms related to COVID-19 for weeks, and even months, after contracting the virus. It also would require the secretary to develop a nationwide testing and vaccine distribution strategy, including consideration for how to help patients with rare diseases mitigate public health emergencies, to be used in future pandemics following a series of national meetings with key stakeholders. It would also propose $25 million annually for the CDC to fund a public education campaign on the importance of vaccines for fiscal years 2022-24.
It would require HHS to establish a Committee on Critical Need Antimicrobials made up of representatives from NIAID, CDC, BARDA, FDA, CMS, VA and DoD that would develop regulations for approving “critical need antimicrobial drugs” – a new FDA designation – as well as other recommendations and include education strategies for providers and patients on appropriate use of antimicrobial drugs.
SECTION 2: PATIENTS AND CAREGIVERS
Cures 2.0 authorizes grants for caregiver education and training programs to improve the skills of the home care team to better complement clinical visits.
It would require HHS to work with CMS to improve health literacy to ensure patients from multicultural and multilingual backgrounds can understand their illness, covered benefits and the treatment options at their disposal.
Diversity in Clinical Trials
Cures 2.0 has several provisions to address transparency and barriers currently inhibiting diversity in clinical trials that include:
- HHS Secretary Report: It would require HHS to provide a report to Congress and the FDA containing the prevalence of demographic subgroups (sex, age, race and ethnicity) in clinical trials and whether subgroup-specific safety and effectiveness measures have been implemented. The report must be made available to the public and include an action plan with HHS recommendations to improve the representation of demographic subgroups in clinical trials.
- Government Accountability Office (GAO) Report: It would direct GAO to review how HHS currently addresses participation barriers faced by populations underrepresented when conducting or supporting clinical trials.
- Improving Recruitment Efforts in Clinical Trials: The bill would require HHS to implement a public awareness campaign to improve the recruitment of minorities in clinical trials. The campaign must increase awareness and understanding of upcoming and current clinical trials and how to enroll in the trials as well as increase the availability of enrollment resources.
SECTION 3: FOOD AND DRUG ADMINISTRATION
Communication and Digital Health
With the goal of having common regulatory standards across agencies on the integration of digital technologies for improved health care, it would establish an automatic communication requirement between FDA and CMS for products granted BTD to ensure timely access to innovative products. HHS would be required to submit a report to Congress on how to improve interagency collaboration and alignment on digital health technologies.
Novel Trial Design Grants
It would authorize a new $25 million grant program for incorporating novel clinical trial designs into clinical protocols and applications for drugs, prioritizing the use of digital health technologies and real-world evidence in drug development.
Cell and Gene Therapy
HHS would have to inform Congress about the current state of cell and gene therapy regulations and potential regulatory challenges for the FDA in the future.
Real World Evidence
It would create the Real World Evidence Task Force to coordinate HHS programs and provide recommendations on ways to encourage patient engagement in using this evidence.
Cures 2.0 would create at least two new institutes within FDA with one focusing on the most burdensome diseases that represent a leading cause of mortality or disability or affecting at least 50 million Americans and the other focusing on diseases impacting fewer than 200,000 people in the U.S. or for which over 90% of such diseases have no therapy approved by FDA or over 50% of patients are children.
It would clarify when sponsors of drugs and regenerative medicine can be granted investigational drugs and apply for BTD during the FDA approval process. The FDA would also be required to publish guidance to provide clarity on the development and submission of chemistry, manufacturing, and controls (CMC) data relating to breakthrough therapies, fast track products, accelerated approval, and regenerative advanced therapies. Finally, other evidence—real world evidence—is allowed to be used and can derive from clinical evidence or patient registries to fulfill post-approval study requirements to confirm the predicted clinical benefit of a therapy.
SECTION 4: CENTERS FOR MEDICARE AND MEDICAID SERVICE
Telehealth, Medicare Restrictions and Coverage
The bill includes the Telehealth Improvement for Kids’ Essential Services (TIKES) Act, which would assist states in effectively integrating telehealth into their Medicaid program and CHIP. It also includes the Telehealth Modernization Act, which would permanently remove Medicare's geographic and originating site restrictions and extend the waivers implemented during the pandemic. This means a patient must no longer live in a rural area and be physically in a doctor's office or clinic to use telehealth services. It would also allow HHS to permanently expand the types of services that can be reimbursed under Medicare and the types of health care providers that can offer telehealth services. In addition, HHS must provide a report on the current capabilities and deficiencies of CMS’s computer systems.
Cures 2.0 would require that when FDA designates a breakthrough device, during the transitional coverage period (that starts on the date of approval and ends after four years) a breakthrough device is deemed to be reasonable and necessary and approved for pass-through payment. Within three months of this FDA approval, HHS must assign a unique temporary or permanent code for coverage. After the four year period, HHS must establish a process for the coverage of these breakthrough devices. HHS must provide a report to Congress on this program.
Cures 2.0 would increase access to genetic screening and counseling by covering genomic precision medicine consultation but only when provided by a qualified licensed pharmacist with appropriate training. It would create a pediatric DNA sequencing clinical services demonstration project spanning up to five states (with preference for states that do not cover DNA sequencing clinical services) to provide genomic testing for children with rare diseases. Project participants must provide information and data on how such services improve the diagnosing of rare diseases, explanation of how and the extent to which coverage of genomic testing may improve clinical outcomes, and a report to the Health Resources and Services Administration (HRSA) on the extent to which DNA sequencing reduces health disparities. Further, a pilot grant program would be created within the CMS Innovation Center to test the delivery of personalized-medicine consultations. Finally, it prohibits the use of geographic tracking features and biometrics within Medicare electronic visit verification (EVV) system.
SECTION 5: RESEARCH
It would establish President Biden’s ARPA-H initiative with $6.5 billion. Its mission would be to speed transformational innovation in health research and application and advance the implementation of health breakthroughs. The new ARPA-H would be largely modeled after the military’s Defense Advanced Research Projects Agency, or DARPA. ARPA-H would aim to accelerate new medical breakthroughs with the intent to cure cancer, Alzheimer’s and other devastating diseases. Specifically, ARPA-H would:
- Take on work that requires large scale, sustained coordination
- Expand capacity in fields such as technologies, data resources, and disease models
- Establish new paradigms by supporting high risk endeavors
- Use solutions, such as financial incentives, to overcome market failures
- Complement research undertaken by the NIH and the private sector.
To support additional research, it would provide $25 billion for universities, research institutions, and public laboratories throughout the country to continue their work on thousands of federally funded research projects.
Cures 2.0 would prioritize the delivery of medical innovations to patients by increasing diversity in clinical trials to ensure efficacy and safety data is collected on all populations that ensures proper drug labeling and avoids adverse drug reactions; enabling faster Medicare coverage of breakthrough drugs, regenerative medicine, devices and genetic testing; expanding telehealth access and coverage; and providing funding for initiatives to enhance health literacy, promote vaccinations and pandemic planning, accelerate bringing critical need antimicrobial drugs to market, advance caregiver skill development and boost translational research and development.