United Mitochondrial Disease Foundation
2022 Congressional Outlook
As it reconvenes, Congress faces a packed agenda in a legislative year that will be shortened by the upcoming midterm elections and remains buffeted by the persistent coronavirus pandemic. The list of healthcare priorities is notably lengthy, and how much of this crowded agenda will move forward this year through the Build Back Better Act (BBBA) or other means remains to be seen. The following discusses issues that UMDF and others in the rare disease community are watching as we begin 2022.
Build Back Better Act: At the forefront of the agenda is the stalled BBBA, which contains several ambitious healthcare initiatives and a wide assortment of other healthcare funding and policy changes. As of this writing, there has been no progress in reaching an agreement on a bill that would gain the support of Sen. Joe Manchin (D-W.Va.). Negotiations are being described as “paused.”
President Joe Biden and congressional Democrats are expected to continue their efforts to reach an agreement on a scaled-down bill that might contain child care funding, 12-month coverage for children in Medicaid and the Children’s Health Insurance Program (CHIP), funding to address social determinants of health issues, and several provisions to control the cost of high drug prices. It remains to be seen whether this effort will be successful or if negotiations will falter and lose momentum.
Fiscal Year (FY) 2022 Appropriations Outlook: FY 2022 federal appropriations are currently pending, with the government currently funded on a short-term basis through Feb. 18. Democrats and Republicans have been at a standoff over conflicting policy and funding priorities since the new fiscal year began on Oct. 1, 2021. Democrats would like to secure significant increases for domestic priorities, but Republicans insist on equal treatment for defense funding and no changes in certain longstanding policy riders (such as the Hyde Amendment related to abortion). Ultimately, both sides have reasons to cooperate on resolving the appropriations standoff, hopefully in March. If so, significant funding increases for many healthcare programs – such as the National Institutes of Health (NIH), substance abuse programs, and public health and workforce investments – can be expected.
UMDF has successfully advocated for the inclusion of language in the House and Senate FY 2022 Labor-Health and Human Services (HHS)-Education appropriations report that urges the NIH to invest additional dollars in primary mitochondrial disease research, as well as language that would ensure inclusion of mitochondrial disease as eligible for research funding for the Department of Defense (DOD)’s Peer-Reviewed Medical Research Program, which has provided over $20 million to mitochondrial disease research efforts since 2016.
Meanwhile, President Biden will need to put forward a proposed budget for FY 2023, followed by the House and Senate adopting a budget resolution with topline spending figures. The House would then turn its attention to annual appropriations bills for FY 2023, with a goal of passing them all before the August recess. However, Senate Republicans will have little incentive to get those bills through the Senate before election day. If control of either the House or Senate changes hands, the bills are likely to be further delayed into 2023.
As it has done over the past several years, UMDF will advocate for the inclusion of language and funding related to mitochondrial disease research at the NIH, DOD, and potentially other agencies, and will continue to educate Congressional appropriators about the importance of increased funding for mitochondrial disease research. In the coming months, we will reach out to you to engage your lawmakers and ask that they support our FY 2023 funding requests.
Cures 2.0: In November, Reps. Diana DeGette (D-Colo.) and Fred Upton (R-Mich.) introduced the highly anticipated Cures 2.0 Act (H.R. 6000). Among other provisions, this bipartisan bill would accelerate medical research, increase patient access to novel therapeutics, remove current barriers to telehealth services, and codify a recent regulation that would accelerate Medicare coverage of breakthrough devices. This bill would also create a semi-autonomous Advanced Research Projects Agency for Health (ARPA-H) agency within NIH to generate cutting-edge research across numerous fields and agencies.
Notably for UMDF, the bill also includes a UMDF-supported measure sponsored by Reps. Scott Peters (D-CA) and Eric Swalwell (D-CA) that would create a demonstration program in Medicaid for covering whole genome, whole exome, and gene panel sequencing for the detection of disease-causing genetic variants that may lead to faster rare disease diagnosis.
Additional discussions with stakeholders are expected to take place in 2022. Although the bill is popular among House members committed to biomedical research and its sponsors are pressing for passage, the bill has an uncertain future given the lack of a corresponding Senate bill. Some or all of the bill could be considered as part of the user fee amendment reauthorization process (see below).
Drug Pricing and User Fee Agreements: Drug pricing reform continues to be a hotly debated topic yet Democrats find it difficult to agree on the scope of provisions. For now, it looks like more comprehensive approaches, such as allowing Medicare to negotiate drug prices or overhauling pharmacy benefit manager rebates, appear to be out of reach. However, if a narrower BBBA prevails, there is the potential for the inclusion of several drug pricing provisions, such as restructuring of the Part D benefit and a new cap on monthly insulin out-of-pocket expenses. If the BBBA does not advance, these provisions could move as part of another legislative vehicle, or possibly on their own.
In addition, legislation implementing user fee agreements for most medical products – including drugs and medical devices– must be reauthorized prior to their expiration on Oct. 1, 2022. Without this reauthorization, medical product review divisions cannot collect user fees attached to new product applications, significantly reducing funding and preventing or considerably delaying approvals for new products. Seen as a must-pass piece of legislation, other healthcare provisions in the biomedical research and health technology spaces, among others, may be joined into the final package before passage. This is seen as a potential vehicle for certain provisions of the aforementioned Cures 2.0 should the stand-alone bill not pass.
COVID-19 Public Health Emergency (PHE)/Telehealth: As the pandemic wears on, the healthcare industry has found itself in an unprecedented state of flux. HHS extended the federal public health emergency (PHE) through Jan. 13, 2022 and various states have their own PHEs with differing end dates. By law, the federal PHE must be renewed every 90 days. It is currently scheduled to go through April 16 and most expect at least one additional extension until mid-August, but after that is less clear. HHS has committed to provide states at least 60 days’ notice prior to the expiration of the PHE.
The PHE declaration gives HHS the authority to provide important billing and oversight flexibilities for services like telehealth. Telehealth waivers have enabled a significant increase in the volume of these services being provided during the pandemic. Among the key PHE-related flexibilities, telehealth services may be furnished via telephone. Audio-only communications are also allowed for certain services. Geographic restrictions that barred services to patients in non-rural and non-health professional shortage areas (HPSAs) are temporarily no longer in effect, and patients are allowed to receive services in their homes or other non-clinical locations, whereas previously, they would need to be in an office setting. Additionally, state licensure requirements for professionals enrolled in Medicare with an equivalent license from another state have been waived. States can similarly waive licensing requirements with respect to Medicaid.
Telehealth is critical for mitochondrial patients that are immunocompromised and at greater risk from complications from COVID-19. Telehealth limits this exposure by allowing their treatment to take place in their home. Additionally, telehealth flexibilities have significant impacts on the rare disease community, especially rare disease patients located in rural areas whose access to medical specialists may be limited. Many mitochondrial patients have to travel significant distances to see specialists in the field. The burden of travel, in some instances, has been remedied by the telehealth flexibilities afforded by the PHE waivers, which allows telehealth services to be rendered by out-of-state specialists.
When the PHE ends, the authority for these waivers will expire. However, there is bipartisan congressional support to extend these flexibilities, at least temporarily to gather more data to resolve concerns about quality, effectiveness, cost, and fraud.
Health Equity/Improving Data Collection: Equity in health outcomes continues to be a top priority for the Biden Administration and Congress, driven in part by overwhelming evidence of how the pandemic has disproportionately impacted historically marginalized and low-income groups. Accordingly, there is a continued push for comprehensive data, including data on social determinants of health, which are critical to understanding disparities and helping to inform planned interventions .
Such data collection may help address unmet needs for rare disease patients, including rare diseases that disproportionately affect underserved populations. Patients with rare diseases who also are people of color often face extra barriers to care as they are less likely to receive timely and accurate diagnoses, access to educational and support services, and participate in clinical trials. Demographic data will also be important as Congress and the FDA consider changes to clinical trial enrollment practices to address unconscious biases and create more diverse and representative samples, which will help to ensure treatments are more effective for all patients including minority, disabled, and other underserved and often underrepresented populations.
Several bills addressing health equity are currently under consideration in Congress. The BBBA presently includes a series of proposals to address maternal mortality and morbidity. Known collectively as the “Momnibus,” these provision promote outreach and culturally competent services, greater diversity in the maternal and child workforce, and grants to improve maternal health and reduce maternal mortality, especially among groups who are the most severely affected by adverse outcomes before, during, and after the delivery of a child. If enacted, these changes would complement Medicaid reforms including a new 12-month postpartum coverage requirement. These policies would be a high priority for Congress to pass in the event that the BBBA does not end up passing.
Also, the Center for Medicare and Medicaid Innovation (CMMI), which is charged with creating new Alternative Payment Models, continues to make equitable health outcomes a central pillar of its strategy refresh. CMMI has expressed a great interest in models that can identify at-risk beneficiaries and provide targeted outreach and interventions to vulnerable patient communities.
Public Health Legislation: Only a short time after the onset of COVID-19 in early 2020, discussions began on Capitol Hill regarding how to shore up the nation’s public health system to more effectively respond to future pandemics. These discussions have involved topics such as a more stable permanent funding source to help strengthen our public health infrastructure and how to ensure a more nimble response to future public health threats. Significant funding has been poured into federal, state and local public health agencies as part of the various COVID-19 relief bills passed by Congress. This includes substantial funds for public health data modernization efforts.
Several public health-related bills have been introduced in the Senate on a wide range of topics, including proposals to create a Commission on the COVID-19 pandemic, increase our domestic manufacturing capacity to prevent future supply chain issues, and establish new manufacturing policies that would boost the nation’s supply of generic medicines and active pharmaceutical ingredients that are chronically in shortage. This package could also serve as a possible alternative vehicle for public health provisions currently contained in the BBBA should it be trimmed down or not pass altogether.