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Policy Issues

Since its establishment in 1983, NORD has advocated for policy changes to improve the lives of Americans impacted by rare diseases at the federal and state levels. NORD continues this critical work to this day.

Access to Affordable, Adequate Coverage

Rare disease patients need access to affordable, adequate, and accessible health care coverage to maintain their health.

Affordable Care Act

Enacted in 2010 with NORD’s strong support, the Affordable Care Act improved access to quality, affordable health care for millions of Americans, including those with preexisting conditions.

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Copay Assistance

The high cost of prescription drugs has a direct impact on patient access, particularly when more affordable generic versions of medications are not available for the treatment of rare diseases. For some patients, charitable or manufacturer-based assistance is available to help manage the cost of treatment, but not all health care programs and insurers allow copay assistance to be used effectively by patients.

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Emergency Medical Services

Many rare disease patients carry self-administered medications for emergency situations. However, Emergency Medical Services (EMS) protocols often prevent paramedics or other first responders from administering these medications due to liability concerns.

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Ensuring Lasting Smiles

Reconstructive dental or oral procedures to improve, repair, or restore functionality resulting from congenital anomalies or birth defects are often not covered by insurers because they consider this surgery to be cosmetic in nature. Legislation to address this problem has been introduced in Congress.

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Medical Nutrition

Medical nutrition for people diagnosed with digestive or inherited metabolic disorders is often very expensive and is often not covered by insurance.

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Medicare Home Infusion

Although current law provides a limited home infusion benefit for Medicare beneficiaries to access critical treatments, such as chemotherapy; the benefit must be expanded to more fully meet the needs of patients.

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Medicare, Medicaid, and Private Health Insurance Coverage

For rare disease patients, adequate health care coverage means having access to affordable health care, including knowledgeable primary care providers and specialists, when and where it is needed.

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Medicaid Section 1115 Waivers

Section 1115 waivers enable states to waive certain Medicaid requirements and allow a state to direct federal Medicaid funds in ways that would not otherwise be permitted under federal law as part of demonstration projects designed to improve access to health care programs.

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Out of Pocket Prescription Drug Costs

Innovative new medications and treatments are enabling rare disease patients to live longer, healthier lives. Unfortunately, the cost of these medications and treatments that patients must shoulder can often be prohibitive.

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Out-of-State Care for Children on Medicaid

Many rare disease patients have just one or two clinical centers in the entire United States with a specialist who is knowledgeable and can treat a specific rare disease, so it is important that Medicaid cover out-of-state care.

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Section 1332 Waivers

Section 1332 of the Affordable Care Act allows states to pursue innovative strategies for providing their residents with high quality, affordable health insurance. Many states have used Section 1332 waivers to implement reinsurance programs to help stabilize the health insurance marketplace and lower premiums by essentially providing insurance for insurers with respect to high cost beneficiaries.

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Short-Term, Limited-Duration Health Plans and Association Health Plans

Short-term, limited-duration health plans and association health plans are not required to adhere to many key consumer protections for health insurance required by the Affordable Care Act, such as essential benefits and guaranteed issue, and as a result, can exclude or charge higher premiums to employers on the basis of age, gender, occupation.

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SSI/SSDI

Social Security Disability Insurance (SSDI) and Supplemental Security Income (SSI) are two distinct benefit programs for people who are disabled.

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Surprise Medical Billing

Sometimes patients receive unexpected and high bills for Emergency Room and other hospital visits, because a physician is out of network even though the hospital is in network.

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Utilization Management

Practices such as “step therapy” or “prior authorization” are often used by insurers to reduce expenses associated with the high cost of prescription drugs and treatments. When implementing these policies, insurers may require patients to take one or more alternative medications or obtain prior authorization before permitting patients to access the medication prescribed by their provider.

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Access to Affordable Medicines

More than 95% of rare disease patients lack an FDA approved treatment for their condition. Fortunately, scientific innovation continues and more treatments for rare diseases are becoming available. However, the high cost of some of these drugs can sometimes hinder access for rare disease patients. Addressing the affordability of existing and new drugs is critical to ensuring patients can access medically necessary treatments.

Biosimilars

Enacted in 2010, the Biologics Price Competition and Innovation Act, established a pathway for FDA to approve lower-cost versions of critical biologic products that are as safe and effective as their brand counterparts. Since that time, FDA has approved almost 30 biosimilars.

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Federal Drug Pricing Legislation in the 116th Congress

In response to widespread concerns by patients across the country about the impact that high drug prices have had on patient access, the 116th Congress has been considering many legislative proposals to address this issue.

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Generic Drugs

Generic drugs approved by the Food and Drug Administration (FDA) provide a safe and effective alternative to brand name medications. Generics introduce competition to the marketplace and force drug costs down, improving patient access to treatments.

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The Administration's Blueprint

In May 2018, the Trump Administration released their "American Patients First: The President's blueprint to lower drug prices and reduce out-of-pocket costs," which outlines proposals for the Department of Health and Human Services (HHS) to consider when looking to address the issue of high prescription drug prices.

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Value-based Pricing Assessments

In an effort to establish medical product prices based on their value to the healthcare system, health technology assessment entities, such as the Institute for Clinical and Economic Review (ICER), have provided recommendations for such prices. NORD has worked to ensure that such assessments reflect the value from the patient perspective.

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Access to Diagnostics

Many rare disease patients face years of a difficult “diagnostic odyssey” before receiving their correct diagnosis. Obtaining an early and accurate diagnosis is critical for allowing patients to seek effective treatment sooner and preventing morbidity and mortality.

Genetic Testing

Genetic testing holds the promise of ensuring a timely diagnosis and effective, targeted treatment for people with rare diseases, but access and affordability is currently a limiting factor.

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Newborn Screening

Among the most successful public health programs, newborn screening has been saving and improving the lives of children across the United States through early identification of diseases that cause permanent disability or death if not treated early.

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Access to Innovative Medicines and Therapies

The rare disease community faces disparate challenges that require unique considerations throughout the drug and medical device development processes to ensure timely access to innovative treatments.

Access to Pediatric Therapies

Half to two-thirds of the 7,000 rare diseases begin in childhood, so it is critical that innovation in the pediatric orphan drug space continues and that more robust pediatric labeling makes its way onto orphan products.

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Devices for Small Populations

Just as challenges exist for drugs intended for rare diseases, devices intended for use in small patient populations have also needed incentives and regulatory flexibility to bring these products to market.

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Expanded Access

For patients not eligible for clinical trials, FDA's expanded access program can provide an opportunity to access an investigational drug that is not yet FDA approved.

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Gene Therapies

The potential for innovative gene therapies are a particularly exciting development for rare disease patients because many rare diseases are a result of a genetic disorder and currently, most patients do not have any other treatment options.

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MODERN Labeling

The MODERN Labeling Act would give the Food and Drug Administration (FDA) the authority to require that older, generic drugs with no brand counterpart be updatedto reflect new information relevant to the drug and its use.

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Orphan Drug Act

Enacted in 1983, the Orphan Drug Act provides key incentives to spur the development of treatments for rare diseases.

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Orphan Drug Tax Credit

The Orphan Drug Act tax credit allows sponsors to claim a 25 percent tax credit for certain research and development costs for orphan drugs.

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Protecting FDA Standards for Safe and Effective Drugs

FDA-approval is considered across the globe as the “gold standard” because it best ensures that products reaching the market are both safe and effective.

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Patient Focused Drug Development

In recent years, FDA and other stakeholders have recognized the importance of ensuring that patient perspectives are incorporated into drug development at all stages, including FDA's review of product applications.

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Rare Disease Clinical Trials

Clinical trials for rare disease patients present unique challenges due to the small patient populations. Overcoming these challenges when developing therapies requires flexibility in both the design and the analysis of clinical trial results.

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Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)

In coordination with FDA and C-Path, NORD is working on the RDCA-DAP that is intended to serve as a neutral, independent data collaboration and analytics hub to promote the sharing of critically important data across rare diseases in order to accelerate the understanding of disease progression.

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Right to Try

Signed into law by President Trump in May 2018 over NORD objections, the Right to Try Act permits access to treatments that completed Phase I clinical trials, but have not been approved by the Food and Drug Administration (FDA).

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Advancing Rare Disease Research and Regulatory Science

The vast majority of rare diseases have no FDA-approved treatment and hurdles in the development of these therapies are many. It is critical that the research and regulatory environment helps to address these barriers and foster a successful bench to bedside pathway for rare disease treatments.

Ensuring Adequate Funding for NIH

For decades, the National Institutes of Health (NIH) has provided critical funding and support for essential basic and translational research on rare diseases.

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Immigration

Rare disease patients and their caregivers who are willing and able to come to the United States should not face any barriers to doing so.

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PCORI

The Patient-Centered Outcomes Research Institution (PCORI) was created through the Affordable Care Act to fund comparative clinical effectiveness research (CER) studies. The goal of these studies is to understand which treatment approaches are most appropriate for which patients given their needs and preferences.

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RARE Act

Two major challenges faced by the rare disease community are the lack of epidemiological information available on rare disease prevalence and incidence and the lack of provider awareness of rare conditions. The RARE Act would require the Centers for Disease Control and Prevention (CDC) to conduct such epidemiological research and the Agency for Healthcare Research and Quality (AHRQ) to expand activities surrounding provider awareness of rare diseases.

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Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)

In coordination with FDA and C-Path, NORD is working on the RDCA-DAP that is intended to serve as a neutral, independent data collaboration and analytics hub to promote the sharing of critically important data across rare diseases in order to accelerate the understanding of disease progression.

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COVID-19

On March 13, 2020 the federal government declared the COVID-19 pandemic a national public health emergency. The pandemic has had a significant impact on nearly all aspects of our lives, particularly for the rare disease community. NORD continues to work in support of policies to help mitigate the risk of contracting COVID-19 and ensure patients have continued access to vital health care services.

Access to Care

During the COVID-19 pandemic, now more than ever, Americans need access to affordable, quality health care providers, services and treatments.

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Anti-discrimination

With the surging number of cases, the COVID-19 pandemic put our healthcare systems at risk of becoming overwhelmed. As states and hospitals are evaluating and implementing their worst-case scenario planning, it is important that crisis standards of care plans and clinical guidelines do not discriminate against people with underlying medical conditions.

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Funding for Ongoing Science

With laboratory and clinical research largely stalled during the pandemic, it is critical that the US provide bridge funding so that it can resume as soon as possible.

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Paid Leave

During this pandemic, people living with rare diseases and their working family members should be eligible for paid leave if they decide to stay home from work to avoid exposure to COVID-19.

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Telehealth

Telehealth allows patients and physicians to exchange health information without being in the same room. While historically only available to patients living in rural areas, the COVID-19 pandemic has significantly expanded the utilization of telehealth services. Both the federal government and states have the power to regulate aspects of telehealth, including telehealth licensure requirements, reimbursement rates and eligible services.

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Rare Disease Advisory Councils (RDACs)

A Rare Disease Advisory Council (RDAC) acts as an advisory body that gives the rare disease community a stronger voice in state government and makes recommendations as to how their state’s programs and policies should be modified to benefit the rare disease community.