Policies to Accelerate Life-Changing Breakthroughs to Cure, Prevent and Treat Type 1 Diabetes and its Complications
Type 1 diabetes (T1D) is an autoimmune disease that requires management 24 hours a day, 7 days a week, 365 days a year. Even with intensive management, people with T1D spend a significant portion of time with high or low blood-sugar levels, putting them at risk for medical emergencies and devastating complications.
But thanks to recent advances, we are improving lives, today and tomorrow, and helping people stay healthy until we have a cure. Life-changing technologies such as continuous glucose monitors (CGMs) and first-generation artificial pancreas (AP) systems are now a reality. Next-generation therapies such as fully automated AP systems, beta cell replacement, immune therapies, and prevention therapies are now in human clinical trials, while groundbreaking research continues toward ultimate cures. Through both research and advocacy, JDRF is accelerating scientific discovery, commercial development, regulatory approval and healthcare access for a wide range of T1D breakthroughs.
JDRF advocacy can play an important role in accelerating innovation and ensuring access for all Americans. To that end, JDRF’s advocacy priorities are:
- Invest in Innovation: Advocate for continued funding for the Special Diabetes Program (SDP), which provides $150 million a year in T1D research.
- Ensure Regulatory Frameworks That Promote Innovation: Ensure United States regulatory policies provide clear and reasonable pathways for scientific research and therapy approvals.
- Promote Access for Life-Changing T1D Breakthroughs: Advocate for coverage, affordability and choice of T1D therapies in the health care system and for national policies that protect those with pre-existing conditions like T1D.
JDRF is the leading global organization funding type 1 diabetes (T1D) research, with a mission to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2 billion in research funding since our inception, and spurred billions more from public and private sector partners to generate a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D.
Investment in Innovation
Multi-year, continuous funding for the Special Diabetes Program (SDP), which provides $150 million a year in T1D research through the National Institutes of Health (NIH), is a top advocacy priority for JDRF.
The SDP has accelerated the development of artificial pancreas systems, which use sophisticated computer algorithms and continuous glucose monitors to provide the right amount of insulin at the right time. The first artificial pancreas system, which became available to people with T1D in 2017, is improving lives; and multiple companies have next generation systems in development. The SDP also supports cutting-edge research that could ultimately lead to prevention and a cure for T1D, as well as new treatments and therapies for burdensome and costly diabetes-related complications.
The SDP is supporting research to drive the next generation of innovation. Congress recognizes the importance of this critical research program and continues to provide broad, bipartisan support. Thanks to bipartisan leadership in Congress and the hard work of JDRF volunteers and staff, $300 million for SDP was enacted in late 2017 and early 2018, funding the program through September 30, 2019. JDRF is embarking on another concerted advocacy effort to secure a multi-year renewal of the SDP prior to the end of September. The SDP receives mandatory funding, not appropriations funding that Congress provides on an annual basis. JDRF also supports increasing the overall level of appropriations for the NIH as well as robust support for the Food and Drug Administration (FDA).
Ensure Regulatory Frameworks that Promote Innovation
JDRF is engaging the FDA to encourage regulatory frameworks for T1D therapies that promote continued innovation and improve public health, including:
- Artificial Pancreas: JDRF worked closely with the FDA to establish guidance for artificial pancreas device systems, which was finalized in 2012, and led to approval of the first insulin-dosing system in 2016. To support technological advancements, JDRF has advocated for an additional regulatory pathway for open protocol artificial pancreas components – those from different manufacturers that are interoperable with each other. FDA has created two new device types, the integrated continuous glucose monitor (iCGM) and the alternate controller enabled (ACE) insulin pump which will result in more efficient regulatory processes and, most importantly, increased choices for people with T1D. JDRF continues to work with the agency and the community to shape additional pathways for open protocol artificial pancreas components.
- Prevention Therapies: JDRF is playing a leading role in a consortium seeking regulatory qualification of certain T1D biomarkers, specifically a set of islet autoantibodies that help define the earliest stages of T1D, to facilitate their use in regulatory decision making and accelerate research and development of potential T1D prevention therapies. FDA accepted the biomarkers into the Biomarker Qualification Program and the consortium, part of the Critical Path Institute, is preparing the data and information to submit to the FDA and EMA to support qualification.
- Beta Cell Replacement: JDRF hosts an ongoing educational symposium series with FDA staff focused on beta cell replacement, which involves combining a replenishable source of insulin-producing cells with materials to protect the cells in the body to replace cells destroyed by T1D. The first human clinical trial for such a product was approved to proceed by FDA in 2014 and multiple additional approaches are under development, with some nearing advancement into human clinical trials. JDRF also supports current research policies, put in place by the Bush Administration and updated in 2009, which provide strict ethical oversight for federal research using stem cell-based technologies in encapsulation and cell-based therapies for other diseases.
- T1D Outcomes: Following on the T1D Outcomes Program publication defining outcomes for use in the evaluation of T1D therapies in research, regulatory review, and healthcare reimbursement, JDRF is focused on ensuring that the outcomes are fully integrated into the regulatory paradigm, with a particular focus on hypoglycemia and time in range.
- Pediatric Enrollment in Clinical Trials: Inclusion of pediatric subjects in T1D clinical trials is important for research and development of T1D therapies, but regulatory approval for this has been challenging in some cases. JDRF is partnering with the T1D community to facilitate regulatory approval for the inclusion of pediatric patients in trials through education, development of a white paper and establishment of best practices.
- Immune therapies: To complement the scientific advancements in the area of immune therapies for T1D, JDRF is engaging FDA to translate what has been learned scientifically into the regulatory paradigm. Most notably that has involved a symposium at FDA where JDRF staff, leading academic researchers and people with T1D and their caregivers briefed FDA leadership and staff on the unmet needs and scientific data to date and the group discussed what it means for future development of these therapies.
- Regulatory Pathways for Other Priority T1D Therapies: As additional priority research initiatives are advanced – in areas such as restoring vision, metabolic control, and others – JDRF will work to ensure clear and reasonable regulatory pathways to foster research, development, and approval.
Promote Access to Life-Changing T1D Breakthroughs
JDRF is fighting for access to therapies people with T1D need to stay healthy until there is a cure. Our Coverage2Control campaign advocates for coverage, affordability, and choice for T1D therapies. We are also fighting to preserve insurance protections for those with pre-existing conditions.
Coverage, Affordability and Choice of T1D Therapies
- Affordable Insulin and Diabetes Management Tools: JDRF is advocating strongly for actions by health plans, employers, companies, and the government to make insulin more affordable. This includes:
- Ending the system of prescription drug rebates which is driving up the price of insulin; and, in the meantime, for insurers to pass rebates received to beneficiaries in the form of reduced prices;
- Insurance to cover insulin like the life-saving drug that it is, with low co-payments that stay the same the year around (e.g., treating it like a “preventative medicine” made available without the application of a deductible);
- Regulatory policies that encourage competition, so people with diabetes can choose from more options at the pharmacy;
- More help for the uninsured, such as support for community health centers and other public and private resources. To help our community navigate the complex insurance environment, JDRF’s insurance guide provides advice on picking a plan, getting prior authorizations, appealing a denial, and getting help with costs of insulin and supplies.
- Insulin Pump and CGM Choice: People with T1D should have access to the diabetes management tools that are prescribed by their doctor and work best for them. JDRF strongly opposes health plan policies that limit choice of insulin pumps or CGMs, and advocates for a competitive marketplace that fosters innovation. JDRF strongly opposes additional limitations on insulin pumps put in place in early 2019 by the nation’s largest private insurer, UnitedHealthcare, which limits options for children and adults age 7 and older. JDRF also opposes policies from many health plans excluding coverage of the first implantable CGM on the market.
- Medicare CGM Coverage: After a robust advocacy campaign by JDRF and other stakeholders, in early 2017, the Centers for Medicare & Medicaid Services (CMS) announced that coverage under the Medicare program would be extended to CGM devices approved by the FDA to make treatment decisions. To date, three CGM systems have received such FDA approval and subsequent coverage by Medicare, and in 2018 CMS policy was clarified to allow Medicare beneficiaries to use smart devices in conjunction with a CGM, enabling loved ones to be alerted during diabetes medical emergencies. JDRF calls for Medicare to cover all FDA approved CGM devices so the T1D community can benefit from the full range of technology innovation.
- Artificial Pancreas Coverage: JDRF supports broad healthcare coverage for artificial pancreas (AP) systems. The first insulin dosing system was approved by the FDA in 2016 and came on the market in 2017, with additional versions to follow. JDRF has successfully advocated to private insurers for coverage for these ground breaking devices, and now the 25 largest plans all cover them. JDRF will continue to advocate for coverage of current as well as future systems by private plans as well as Medicare. JDRF will also explore whether there are steps at the federal level JDRF can take to encourage access in Medicaid, given that JDRF is not set up to lobby at the state level.
Protections in Health Reform for Those with Pre-Existing Conditions
JDRF is fighting to preserve insurance protections for people with pre-existing conditions. In 2017, JDRF released a set of healthcare principles and joined with other organizations representing people with chronic, serious, and life-threatening diseases to oppose legislation that violated these principles. A 2018 court decision could threatens these protections, and JDRF has called for Congress to take action if, on appeal, the current protections are ruled unconstitutional. JDRF’s healthcare principles include:
- Preserve protections for those with pre-existing conditions: Individuals with pre-existing health conditions should have access to comprehensive health insurance at rates similar to their counterparts without pre-existing health conditions.
- Allow young adults to stay on their parents’ insurance until the age of 26: Young adults should be allowed to stay on their parents’ health insurance plan until the age of 26.
- Prohibit yearly and lifetime dollar limits for essential health benefits (EHBs): Insurance companies should not be allowed to set annual or lifetime dollar limits on their spending for an individual’s covered benefits.
- Close the donut hole in Medicare Part D: Provisions should remain in place to close the Medicare coverage gap for prescription medicines, known as the “donut hole,” by 2020, which will help Medicare beneficiaries afford insulin and other needed drugs.
Evaluation of New Therapies
- Clinical Evidence: JDRF collaborates with research partners to advance research evaluating the effectiveness of T1D therapies, to provide healthcare decision-makers with adequate information.
- T1D Unmet Need: JDRF has commissioned new analyses of T1D healthcare data, to better understand and educate healthcare decision-makers about unmet needs in the population.
- T1D Outcomes Program: As discussed above, JDRF has organized the T1D Outcomes Program, which has proposed an expanded set of outcomes upon which to evaluate T1D therapies.