CMS decision harms current and future treatment options for Alzheimer's and other chronic disease populations April 8, 2022 (Washington, D.C.) The Partnership to Fight Chronic Disease (PFCD) released the following statement from Chair Ken Thorpe regarding the Centers for Medicare and Medicaid Services (CMS) National Coverage Determination requiring Coverage with Evidence Development (NCD) for monoclonal antibodies (mABs) targeting amyloid for the treatment of Alzheimer’s disease.  “We have serious concerns about how the NCD severely limits access to an entire category of new treatments for Alzheimer’s disease and will exacerbate health disparities. The unparalleled precedent this week’s decision makes has compounded those concerns across the chronic disease community. CMS’s decision not only sets back access and hope for the well more than six million Americans living with Alzheimer’s disease, their families, and caregivers, it sets a dangerous precedent for others living with serious or life-threatening illnesses with limited to no treatment options. By singling out drugs approved through the FDA’s accelerated approval pathway, the decision sends shockwaves far beyond the Alzheimer’s community, impacting millions more living with serious chronic conditions including cancers and rare diseases. “This decision threatens access for all Alzheimer’s treatments within the class identified as well as any new therapies for which Medicare deems FDA approval and associated clinical data provide insufficient evidence of clinical benefit to Medicare beneficiaries. The clinical trial criteria outlined will exacerbate known health disparities by raising barriers to access. These barriers disproportionately affect people living in rural areas and people of color already underrepresented in clinical research. “Further, it puts at risk future innovations seeking the FDA’s accelerated approval pathway which has been delivering breakthrough treatments for three decades now and transforming the lives of some of our nation’s most vulnerable patients. “People living with chronic diseases, in consultation with their care partners and treating physicians, should have the option to use FDA-approved therapies and CMS should not undercut those treatment options by compromising access and coverage. By setting an unprecedented new standard for coverage of FDA-approved therapies and significantly undermining FDA’s authority on the safety and effectiveness of therapeutics, this decision will have a devastating effect. “We have waited decades for disease-modifying treatments for Alzheimer’s disease. A thousand people a day progress from mild Alzheimer’s disease to moderate disease and, accordingly, become ineligible for these treatments. CMS’s decision affects an entire promising class of new, disease-modifying treatments by making what is in essence a non-coverage decision with limited exceptions. This decision will set back innovation for Alzheimer’s disease significantly.”

Lack of cures combined with minimal viable treatments compounds increasing health and caregiving costs   May 27, 2021 (WASHINGTON, D.C.)   The Partnership to Fight Chronic Disease (PFCD)  today released a literary review and infographic data on Alzheimer’s disease and related dementias (ADRD), Parkinson’s disease and motor neuron diseases, specifically amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). The data review examines specific disease burden, economic burden, individual costs, and caregiver burden for these neurodegenerative diseases. The annual cost for these conditions in the U.S. was $655 billion in 2020, including direct medical and non-medical costs and indirect costs from lost productivity and uncompensated caregiving hours. Neurodegenerative disease arises when nerve cells in the brain or other parts of the nervous system lose function and eventually die. While there are some viable treatments currently available that may relieve some of the associated symptoms, only SMA has disease-modifying treatments available. Collectively, these diseases affected 4.7 to 6.0 million individuals in the U.S. between 2016-2017, were responsible for more than 272,000 deaths and more than 3 million disability adjusted life years in 2016. “The enormous burden of these illnesses is felt not just by the patient but also their families and caregivers,” emphasized Ken Thorpe, PFCD Chairman. “The vulnerability of people living with these conditions, the increasing demands of their illness and the prevalence projections we are seeing all point to a dire need for reforms to support those diagnosed and their caregivers and to encourage and prioritize research and innovation that leads to new treatments and cures.”  The data review notably highlights: Ethnic, demographic and education metrics, Direct and indirect medical costs, Specific health care cost components, Progressive productivity losses, and Unpaid caregiving costs. “The very nature of neurodegenerative disease commands a greater understanding of the growing numbers, the increasing needs and related costs for people living with these types of health conditions as research continues for urgently needed treatments,” said Dr. Allan Levey of the Department of Neurology and Goizueta Alzheimer’s Disease. For the full literature review and infographic overview on related issues, please visit: www.fightchronicdisease.org/resources/us-burden-neurodegenerative-disease .   ###  The Partnership to Fight Chronic Disease (PFCD ) is an international coalition of patient, provider, community, business and labor groups, and health policy experts, committed to raising awareness of the number one cause of death, disability, and rising health care costs: chronic disease.

Data support preserving access to accelerated approval drugs for the seriously ill   Commentary in the  American Journal of Managed Care  warns that restricting access to accelerated approval therapies has minimal budget implications, but poses significant harm to patients   March 30, 2021 (WASHINGTON, D.C.)  The Partnership to Fight Chronic Disease (PFCD)  today released, “ Quantifying Impact of Accelerated Approval Drugs on Medicaid Spending: De Minimus Impact, Maximum Attention ," an economic analysis examining the impact of the Food and Drug Administration’s accelerated approval pathway on Medicaid spending. A commentary based on the analysis was simultaneously published in the American Journal of Managed Care, " Limiting Access to Accelerated Approval Drugs: Costs and Consequences ." The analysis, authored by PFCD Chairman and Chair of the Department of Health Policy & Management for the Rollins School of Public Health at Emory University Kenneth Thorpe and American Action Forum President Douglas Holtz-Eakin , found that drugs approved through the FDA’s accelerated approval pathway accounted for less than one percent of annual Medicaid spending between 2007 and 2018. “Understandably, states are looking for effective means to manage their health care budgets. This was true before the COVID-19 pandemic and it is even more true today,” said Holtz-Eakin. “Through this analysis of Medicaid spending, we found that policies aimed at restricting access to therapies approved under the FDA’s accelerated approval pathway are not effective levers to address budget shortfalls. Given the unequivocally positive impact of these treatments on people living with diseases where there are no other treatment options, it is unacceptable for state and federal official to continue to pursue these ineffective policies.”  The accelerated approval pathway was initiated in 1992 to address unmet medical needs of oncology and HIV/AIDS patients. The pathway allows drugs for serious conditions to be approved by the FDA based on a surrogate endpoint — a measure that is reasonably likely to predict clinical benefit– with a post-marketing confirmatory study requirement to verify the predicted clinical benefit. In 2012, Congress modernized and enhanced the pathway to expand its use for rare diseases. The pathway has been credited with significant advances in the treatment of life-threatening diseases where patients have limited or no treatment options.  “The FDA accelerated approval pathway has given hope to countless patients struggling with life-threatening diseases, including cancer, HIV/AIDS and thousands of rare conditions,” said Annie Kennedy, chief of policy and advocacy at the EveryLife Foundation . “The burden of these diseases is so massive – from the individual patient and family burden to the broader societal and economic impact. It’s unthinkable to deprive patient communities the life-saving and life-altering benefits of these therapies that have potential to significantly alleviate those burdens. The findings published today demonstrate that therapies that meet the rigor of FDA approval through the accelerated approval pathway should not be candidates for Medicaid budget cuts.”  Medicines approved via accelerated approval meet the same standards for safety and efficacy as all FDA-approved medicines. Despite this, the accelerated approval pathway has come under scrutiny by public and private payers for its use of surrogate endpoints as the means to determine whether a drug works and because accelerated approval drugs are perceived as drivers of health care costs. Over the years, state Medicaid programs have inappropriately limited access to accelerated approval drugs and proposed excluding them from formularies, despite federal law requiring Medicaid to cover any FDA-approved treatment that meets the definition of a covered outpatient drug as defined in federal statute. For example, Massachusetts and Tennessee have formally requested waivers from the Centers for Medicare & Medicaid Services that would exempt them from this longstanding federal requirement.  Thorpe and Holtz-Eakin’s analysis yielded the following key findings:   Spending on drugs approved through the accelerated approval pathway accounted for less than one percent of annual Medicaid spending between 2007 and 2018. Spending on accelerated approval drugs remained steady at 0.6% to 0.8% beginning one year after the 2012 passage of the Food and Drug Safety and Innovation Act, which expanded use of the pathway to rare diseases. Hospital spending consumed the largest share of Medicaid spending year-over-year (34.5%), followed by physician and clinical services (11.7%) and all prescription drugs (8.9%). Contributors to the growth in overall Medicare spending from 2007 to 2018 included hospital spending (29.2%), all prescription drugs (16.7%), and physician and clinical services (16.2%). Accelerated approval drugs accounted for 1.3% of Medicaid spending growth from 2007 to 2018. “These data clearly support preserving access to accelerated approval drugs for seriously ill patients within Medicaid programs,” stated Thorpe. “State schemes to avoid coverage requirements directly undermine Congress’s intent in establishing, and later enhancing, the accelerated approval pathway and raise serious concerns that certain Medicaid patients are not being offered equal access to life-saving medicines.”  For the full analysis of Medicaid spending on accelerated approval treatments and more information and resources on the issue, please visit: www.fightchronicdisease.org/resources/acceleratedapproval . ###  The Partnership to Fight Chronic Disease (PFCD) is an international coalition of patient, provider, community, business and labor groups, and health policy experts, committed to raising awareness of the number one cause of death, disability, and rising health care costs: chronic disease.   Media Contact: Jennifer Burke Jennifer.Burke@fightchronicdisease.org

Findings Show Upper Payment Limits (UPLs) Could Increase Costs and Restrict Access to Critical Medicines March 31, 2025 (Washington, D.C.)  The Partnership to Fight Chronic Disease (PFCD) today released new research demonstrating that state Prescription Drug Affordability Boards (PDABs) and associated Upper Payment Limits (UPLs) may have unintended consequences that could negatively impact people living in with chronic diseases.  Interviews and a survey of health insurers confirm concerns many have raised: UPLs will likely increase patient costs and hinder access to prescribed medicines. These findings directly contradict assertions that state PDABs will improve affordability and access. A survey of regional and national health plan payers with PDAB-related experience and covering hundreds of millions of lives revealed significant concerns regarding the implementation of UPLs: 77% of health plan payers  believe UPLs would disrupt patient access to prescription drugs due to coverage changes, tiering adjustments, increased cost-sharing, or supply chain complications, including pharmacies potentially refusing to stock medicines with UPLs. 67% of payers  anticipate that patient cost-sharing for UPL-designated drugs will either increase (50%)  or remain the same (17%), while 70%  expect out-of-pocket (OOP) costs for drugs in the same class to either rise   (53%)  or stay unchanged (17%). 57% of payers  foresee increases in health insurance premiums if a UPL is enforced. 50% of surveyed payers  indicated that utilization management restrictions  on UPL-designated drugs would likely increase . 73% of respondents  expressed concerns that UPLs could lead to shortages  of critical medicines, with 60%  believing pharmacies might be unable to stock these drugs. 57% of payers  agreed that UPLs could result in providers receiving lower reimbursements for administering affected drugs, potentially reducing availability for patients. State lawmakers have introduced PDABs and UPLs in an effort to control health care costs and lower patient spending on prescription drugs. As of March 2025, eight states—Colorado, Maine, Maryland, Minnesota, New Hampshire, New Jersey, Oregon, and Washington—have enacted PDAB legislation, with four (Colorado, Maryland, Minnesota, and Washington) authorized to establish UPLs for drugs deemed “unaffordable.” However, definitions of “unaffordable” vary by state, and at least one PDAB has struggled to define this concept. While the intention behind PDABs and UPLs is to alleviate financial burdens on patients, the findings suggest that these policies may instead create significant disruptions, leading to higher patient OOP costs, premium increases, restricted pharmacy reimbursement, and reduced medicine availability. “People living with chronic diseases face constant challenges when it comes to managing their health, and prescription drug affordability boards are not a solution. Rather than improving affordability, UPLs could have the opposite effect—leading to increased costs for patients and limiting access to the medications they rely on to manage chronic conditions,” said Ken Thorpe, Chair of the Partnership to Fight Chronic Disease. “Policymakers should carefully consider the unintended consequences of these measures before implementing policies that may do more harm than good for the millions of people across the U.S. living with one or more chronic conditions.”  The full white paper can be viewed HERE .  An Avalere Insights piece can be viewed HERE .   ###

April 4, 2025 (Washington, D.C.)  – The Partnership to Fight Chronic Disease (PFCD) released the following statement in response to the announcement of the Contract Year (CY) 2026 MA and Part D final rule that will not include coverage of anti-obesity medications (AOMs). “There are many factors that contribute to America’s obesity epidemic. There are also many pathways to address it. Today’s decision blocks a major avenue for millions of Medicare and Medicaid patients who, without plan coverage, will not be able to access anti-obesity medications. “Patients and doctors deserve to have the latest scientific innovation on the table when making medical decisions. We are hopeful that, given the Trump administration’s mission to combat chronic disease, an alternative path will soon emerge, one that expands access to this important class of medications and eases obesity’s economic toll on our health care system.” For more information, please visit www.fightchronicdisease.org/obesity . ###