At the International Pemphigus & Pemphigoid Foundation (IPPF), we advocate on behalf of people affected by pemphigus and pemphigoid (P/P) to affect positive change on many important issues that include:
- Ensuring access to high quality health care for patients coping with a diagnosis of P/P;
- Improving patients’ protections in our current healthcare system;
- Increasing federal funding to advance medical research;
- Providing federal funding for disease awareness and education
The IPPF has led the way in voicing the needs of the pemphigus and pemphigoid community. We work closely with our like-minded advocacy partners on the federal and state level to inspire policies supportive of the needs of P/P and other rare disease patients and their families.
Together, we can drive favorable policies by reaching out to legislators and decision-makers to inform them of our public policy concerns, bring attention to the disease and inform the public about P/P.
American Academy of Dermatology (AAD)
The American Academy of Dermatology was founded in 1938. It is the largest, most influential and representative dermatology group in the United States. With a membership of more than 19,000, it represents virtually all practicing dermatologists in the United States, as well as a growing number of international dermatologists. Additionally, it has an active advocacy branch to its organization and provides physicians and their patients up-to-date information on issues that may affect them. The American Academy of Dermatology Association established principles to guide physician leadership in taking policy positions and advocating for health system reform. At the root of these guiding principles is the need to preserve the patient-physician relationship.
Rare Disease Legislative Advocates is a program of the EveryLife Foundation for Rare Diseases designed to support the advocacy of all rare disease patients and organizations. By growing the patient advocacy community and working collectively, we can amplify our many voices to ensure that rare disease patients are heard in state and federal government. RDLA provides free resources for successful grassroots advocacy.
National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD), a nonprofit organization, is a unique federation of voluntary health organizations dedicated to helping people with rare “orphan” diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.
International Alliance for Dermatological Patient Organizations (IADPO)
The International Alliance of Dermatology Patient Organizations (IADPO) – also known as GlobalSkin – is a unique global alliance, committed to improving the lives of patients worldwide. We nurture relationships with members, partners and all involved in healthcare – building dialogue with decision-makers around the globe to promote patient-centric healthcare. GlobalSkin’s work is founded on three pillars: research, advocacy and support.
The Haystack Project is committed to the Rare and Ultra Rare Disease Community. It brings together Innovators, Patients and Caregivers to educate and advocate for policies that recognize the unique circumstances of extremely rare conditions and treatments.
Global Genes is a global community changing the way the world tackles rare disease. Our partners include nonprofit, government, academic, research, service and industry organizations that believe collaboration is the key to positive change.
Medical Dermatology Society
Members of the Medical Dermatology Society (MDS) represent the heart and soul of our specialty: Medical Dermatology. The MDS is made up of the thought leaders of clinical dermatology and the teachers of the next generation of practicing dermatologists. Our members include academicians, private practitioners, and resident physicians. We specialize in the care of patients with serious dermatologic diseases. Our mission is to improve the care of patients with these conditions.
California Association of Nonprofits
CalNonprofits is an alliance of nonprofits. A crucial role for CalNonprofits is to advance public policies that support a healthy nonprofit ecosystem. We focus on elevating your voice to policy makers, philanthropy, and the public; and strengthening your ability to advocate on behalf of your community.
Are you experiencing healthcare challenges with P/P and do you want to make your voice heard by expressing your thoughts to your elected officials? Do you want to bring awareness to P/P by sharing your story and information about your disease?
The IPPF invites you to become empowered and utilize a variety of advocacy resources provided here that can help you gain attention and support for the issues that are most important to you and the IPPF community.
Elevating Awareness of the Pressing Needs of the Pemphigus Community – This white paper focuses on the urgent needs of pemphigus patients that were defined at our Patient Forum in October, 2020.
Track Legislation – Learn about and track information about legislative issues.
RDLA Advocacy Tools – Advocacy tools designed to help rare disease patients, parents, caregivers and other affected parties learn the basics of legislative advocacy.
Global Genes Resource Hub – Extensive information center for patients and advocates.
NORD Advocacy – Analyses of legislative and regulatory activity.
Haystack Project Resources – Letters, presentations and reports on policy issues.
Sign On Letters
Signed letter recommending reforms to streamline access to out-of-state providers and improve access to effective remote care opportunities for dually-eligible individuals in Medicare and Medicaid.
Signed letter communicating recommendations and
concerns with the Centers for Medicare & Medicaid Services’ (CMS’) Coverage with Evidence Development (CED) study criteria to the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). We urge MEDCAC members to ensure that our patient protection and access concerns are not only considered, but openly discussed within the MEDCAC’s public deliberation on February 13-14, 2023.
Submitted comments on the Notice of Benefits and Payment Parameters for 2024 Proposed Rule. The comments focus on four areas:
1) Continued support for standard plans but urge CMS to set lower copays for specialty tier drugs and ensure prescription drugs are outside the deductible for all metal levels.
2) Express disappointment that CMS continues to not address the growing practice of copay accumulators.
3) Express disappointment that CMS is not addressing the issue of plans designating certain drugs as non-essential health benefits and then collecting the copay assistance from drug manufactures and the growing practice of alternative funding programs.
4) Urge CMS and states to enforce ACA nondiscrimination laws and regulations and conduct adverse tiering reviews.
House Bill 1201 (HB 1201) , legislation that would establish a Rare Disease Advisory Council (RDAC) in Indiana, has been introduced. The bill has been assigned to the House Committee on Public Health. We are leading a sign-on letter to members of the Committee, urging them to add HB 1201 to their agenda.
Joined letter to CMS Administration urging CMS to recognize and cover the significant off-label use rare disease patients encounter; to consider implementing a “rare disease” category of special needs plans so we can benefit from the care coordination/collaboration available in those plans; notes importance of requiring plans to include granularity on provider specialties when it address provider directory shortcomings; strongly opposes a proposal to shift costs of routine care and the “investigation” item from MA plans to FFS within the context of clinical trials initiated to satisfy an NCD with CED requirement because it will disproportionately burden patients with very rare diseases;
Signed letter supporting the BENEFIT ACT addressing: a gap is the lack of a
requirement in law that the FDA include patient experience or patient-focused drug development (PFDD)
data as part of its risk-benefit framework. Examples of patient experience data include:
Patient reported outcomes (how a drug impacts activities of daily living ie: whether they can feed themselves, be independent etc.)
Patient testimonials (qualitative data/patient stories of “living with”)
Patient preference data (how much risk patients are willing to take)
Natural History Data (the natural progression of the disease without intervention)
The agency’s signature tool for evaluating risk-benefit of a drug does not currently explicitly include data from the patient perspective that could be critical to informing the agency’s evaluation and, ultimately, decision on whether or not to approve a product.
We write to request that you include $732 million for NIAMS in the fiscal year 2024 Labor, Health and Human Services, Education, and Related Agencies appropriations bills.
Submitted comments on the Centers for Medicare & Medicaid Services’ (CMS’) proposed rule revising and refining policies in Medicare Advantage (MA) and the Medicare Prescription Drug Benefit (Part D) on:
Utilization Management Requirements
Health Equity in Medicare Advantage (MA)
Medicare Advantage (MA) Provider Directories
Clinical Trial-Related Provisions
Medicare Advantage (MA) and Part D Marketing
Gross Covered Prescription Drug Costs.
Submitted comments on the Patient-Centered Outcomes Research Institute’s (PCORI’s) draft inventory of attributes of patient-centered value.
Signed letter supporting legislation moving in Wisconsin (SB 689 and AB 744) to create a Rare Disease Advisory Council (RDAC)
House Bill 458 (HB 458), legislation that would establish a Rare Disease Advisory Council (RDAC) in Mississippi, was recently introduced. The bill has been assigned to the House Health and Human Services Committee and the House Appropriations Committee. We are leading a sign-on letter to members of both Committees, urging them to add HB 458 to the Committees’ agendas.
Signed letter to establish a Rare Disease Advisory Council (RDAC) in Colorado and help to give a voice to the estimated 1-in-10 individuals living with a rare disease in our state.
IPPF signed a Letter of Support for a Georgia Rare Disease Advisory Council – House Bill 918.
Added IPPF to patient community sign-on letter offering comments on the proposed Notice of Benefits and Payment Parameters Rule for 2023 The proposed rule does not require issuers and PBMs to count copay assistance for prescription drugs towards beneficiary deductible and out-of-pocket maximum obligations, thus allowing the continuation of copay accumulator adjustment programs.
Signed onto comments the CSD will be submitting to the Patient Centered Outcomes Research Institute (PCORI) in regard to their proposed Research Agenda.
Wrote to express our concern that the recent proposed National Coverage Determination (NCD) on anti-amyloid monoclonal antibodies (mAbs) sets a precedent that could have far-ranging and damaging impacts on approval and access to new therapeutics across all disease areas
Signed on to advocate for the Department of Justice to finalize a rule on web and application accessibility before the end of the current administration. As many of society’s interactions and transactions take place in a virtual environment, the impact of inaccessible websites and applications on people with disabilities is clear.
Signed letter to Congressional leadership requesting $3.75 billion for the Centers for Disease Control and Prevention’s National Center for Chronic Disease Prevention and Health Promotion (NCCDPHP) in its mission to help people and communities prevent chronic diseases and promote health and wellness for all.
IPPF was added to a sign-on letter to the Maine legislature urging them to add LD 972 to their agenda that would establish a Rare Disease Advisory Council (RDAC) in Maine, was introduced during the 2021 session and has carried over into this session. It’s currently sitting in the Committee on Appropriations and Financial Affairs awaiting a hearing to discuss whether the Committee wants to move forward with this legislation.
IPPF signed on with organizations representing patients with rare diseases urge Congressional Leadership and the Secretary of Health and Human Services (HHS)to include as part of this year’s Prescription Drug User Fee Act (PDUFA) reauthorization provisions to strengthen the Food and Drug Administration’s (FDA) accelerated approval (AA) pathway and enable patient access to these critical, often life-saving therapies.
Signed onto letter to The Honorable Lina M. Khan, Chair Federal Trade Commission (FTC) for patient groups describing the role PBMs have regarding prescription drug access and affordability in the private insurance market.
Signed letter urging the state of California to avoid policies that would potentially discriminate by relying on inequitable metrics such as the Quality-Adjusted Life Year (QALY) that have detrimental implications for access to needed care and treatment by explicitly barring their use in health care decisions.
(AB 1880) has been introduced in California State Assembly that will improve the review and appeals process for UM by ensuring that a clinical peer, of the same or a similar specialty as the ordering physician, is reviewing prior authorization and step therapy requests. This is especially important for rare disease patients, whose conditions are often not well understood by the broader medical community. The bill also ensures health plans keep key data regarding their UM protocols in the hope of better understanding how and the extent to which these protocols are being used
Signed letter offering comments on how PBMs impact the health and well-being of patients who receive their health coverage through the private insurance market. While most people think insurers make the majority of decisions regarding health coverage and affordability, when it comes to prescription drugs, it is the PBMs that drive much of the decisions as to what medications a beneficiary can access and how much they pay for them. We commend the FTC for its leadership to investigate the impact that PBM practices have on the patient communities we serve and believe this represents a critical step forward to improving patient access and affordability to necessary medications.
Added the IPPF to a sign-on letter to the Senate Health, Education, Labor and Pensions (HELP) Committee, urging they add the RARE Act before voting on FDASLA the week of June 6. The FDA Safety and Landmark Advancements Act (FDASLA), legislation that would reauthorize various user fee acts, including the Prescription Drug User Fee Act (PDUFA), which is of great importance to the rare disease community.
- As you know, Congress is currently working to authorize the FDA user fee reauthorizations (known as PDUFA VII). The House passed H.R. 7667 The Food and Drug Amendments of 2022 today, June 8. The Senate HELP Committee introduced S. 4348, Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act on May 26 and is scheduled to mark it up in committee next Tuesday, June 14.
- Neither the House nor Senate version of PDUFA currently includes provisions to further build upon and improve patient-focused drug development processes at FDA. This is a huge missed opportunity as the bill is only reauthorized every 5 years and is Congress’ main time for addressing FDA policy, especially in an otherwise gridlocked environment.
- The IPPF signed on to a letter to the Committees urging them to include provisions to elevate the patient voice in the drug development process.
- Signed on to letter to the California Health Care Foundation’s (CHCF) urging the Foundation to ensure that ICER does not use discriminatory, biased value assessments or comparative effectiveness metrics as they complete CHCF-funded work.
- Currently, negotiations in Congress to reauthorize critical user fee programs and make other necessary improvements at the FDA have stalled, endangering FDA’s ability to conduct the timely review of critical products that our patients need and leaving many of the additional policy priorities we’ve been advocating for in jeopardy of being excluded from the final UFA package. The IPPF added our organization to a sign-on letter to the leaders of the Health, Education, Labor and Pensions Committee and Energy and Commerce Committee urging them to resume negotiations to develop a comprehensive FDA user fee package.
- Signed a letter calling on Members of Congress to take steps to ensure HHS meaningfully engages affected stakeholders in the negotiation process and to ensure HHS respects the current provisions against discrimination in current law, particularly Section 1182 of the Affordable Care Act barring the use of QALYs in Medicare decisions during its implementation.
- Signed letter to Senators urging them to co-sponsor bill requiring PBMs to pass 100% of any rebates received to patients so the discounts can go where they were intended. It requires PBM transparency, to both the patient and FTC, regarding the cost of prescription drugs, explanations as to why consumer costs increased, and if a PBM is affiliated with the pharmacy to which a patient was referred. It also ensures these new regulations are followed by imposing harsh penalties for noncompliance.
- Submitted comments to the Centers for Medicare & Medicaid Services’ (CMS’) above-referenced proposed rule updating and refining the physician fee schedule and other Part B payment policies (the Proposed Rule).
- Submitted letter to FDA asking them that consideration could be given to appointing a dermatologist to manage the Division of Dermatology and Dentistry.
- Signed letter to Congressional Leadership urging them to pass the bipartisan, bicameral Preserving Patient Access to Home Infusion Act (S. 2652/H.R. 5067).
- Signed letter respectfully requesting that CSL Behring begin offering a patient assistance program for its IVIG product, Privigen.
- Submitted comments to the Centers for Medicare & Medicaid Services’ (CMS’) above-referenced proposed rule streamlining enrollment and renewal in the Medicaid, Children’s Health Insurance Program (CHIP), and Basic Health program (BHP) (the Proposed Rule).
- Signed letter to Congressional leadership urging them to move swiftly to pass a final appropriations bill that includes report language, including the NASEM recommendation that NIH establishes an office to support the coordination of all autoimmune disease research across Institutes and Centers.
- Sent a comment letter to United Healthcare regarding their intentions to move Remicade from preferred to non- preferred coverage in February 2021. This abrupt change by the plan could be disruptive to stable patients and their care, and undermines the treatment regimen set by patients and their prescriber.
- Joined sign on letter to the Biden administration supporting the appointment of Dr. Janet Woodcock as Acting Commissioner of the FDA.
- Signed group letter urging FDA to commit to increased efficiency of generic approvals, and greater attention to the next generation of generic drugs – complex generics – that, by their nature, require a more intensive review.
- Sign on to express strong support for the provision in the COVID relief reconciliation bill (section 3108) adopted by the House Energy and Commerce Committee that will provide life-sustaining support, during this deadly pandemic, for people with disabilities, older adults, and individuals, predominantly low-income women of color, who make up part of the direct care workforce. Modeled on the COVID HCBS Relief Act of 2021 (S. 151/H.R. 525), the bill would provide additional funding for Medicaid Home and Community Based Services (HCBS). This funding must be included in the final COVID relief package.
- Sent letter to California Senate Committee on Health to urge their swift consideration of SB 247 to help enable the creation of a California Rare Disease Advisory Council.
- As Congress works to draft the Labor, Health and Human Services, Education and Related Agencies (Labor-HHS) appropriations legislation for fiscal year (FY) 2022, the [XXX] undersigned organizations request $3.75 billion for the Centers for Disease Control and Prevention’s National Center for Chronic Disease Prevention and Health Promotion (NCCDPHP) in its mission to help people and communities prevent chronic diseases and promote health and wellness for all.
- Request that Congress fund the National Institute of Dental and Craniofacial Research (NIDCR) at $520 million and the National Institutes of Health at $46.111 billion. Funding at these recommended levels will allow for the entities’ base budgets to keep pace with the biomedical research and development price index (BRDPI) and provide meaningful growth of 5%.
- Signed on to a letter to the Biden Administration asking for support for the bipartisan RISE Act, led by Representatives Diana DeGette (D-CO), Fred Upton (R-MI), Eddie Bernice Johnson (D-TX), Anna Eshoo (D-CA) and Anthony Gonzalez (R-OH) in the House and Senators Edward J. Markey (D-MA), Thom Tillis (R-NC), Susan Collins (R-ME), and Gary Peters (D-MI) in the Senate, would provide $25 billion to help restore our nation’s research capacity to its pre-pandemic strength, prevent setbacks against the formidable challenges our nation faces, and further the goal of a robust, diverse, and inclusive STEM workforce.
- Sent letter to Congressional leadership to express our support for the Ensuring Lasting Smiles Act (ELSA). This bipartisan legislation will have a direct impact on patients and will eliminate the existing burdens that prevent access to necessary diagnosis and treatment for those with congenital anomalies or birth defects.
The National Council on Disability (NCD) has brought to light the challenges of policies that rely on quality-adjusted life years (QALYs) to discriminate against people with disabilities and older adults. The result is an exacerbation of health inequities.
Signed letter voicing concerns in light of the upcoming MACPAC meetings on accelerated approval coverage and access. The sign-on letter is regarding the proposed Medicaid rebates for accelerated approvals and how this could impact underserved and minority patients.
Signed letter to Chief Clinical Officer at Cigna about their recent action regarding Cosentyx as an example of coercing a non-medical switch that can potentially harm those suffering from autoimmune diseases and other chronic conditions.
Signed letter in strong support of your legislation, the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act of 2021. The BENEFIT Act would require FDA to include in the benefit-risk assessment framework of a new drug application how patient experience data was considered in the review process. Currently, FDA includes patient experience data in reviews, but does not indicate how such data impacted the drug approval. Providing this information to the public, and patient communities making significant investments in developing PFDD, builds on transparency from PFIA and will accelerate PFDD strategies more broadly.
Signed onto a letter representing individuals with rare diseases in Louisiana, urging the swift action of putting House Bill 460 (HB 460) on the Committee on Health and Welfare agenda for consideration. HB 460 establishes a rare disease advisory council (RDAC), which if passed, would help to give a voice to the estimated 1-in-10 individuals living with a rare disease in the state of Louisiana.
Sent letter to Congressional leadership asking for their support in passing the HEART Act, HR 1184 which contains five provisions critical to the patients we represent with rare diseases:
The FDA must consistently include its own Rare Disease Program staff in reviews for drugs to treat rare diseases.
The FDA must consult directly with patients about any Risk Evaluation and Mitigation Strategies (REMS) for a rare disease drug when those REMS programs call for patient participation.
Experts in rare diseases must be included in FDA Advisory Committee panels when reviewing rare disease drugs.
Each year, the FDA must prepare a report indicating how many rare disease drug applications were reviewed by each division at the Agency, including numbers on the prevalence of those conditions.
The Government Accounting Office must review the EU process for approval of rare disease drugs and provide an assessment of how those processes might apply in the US, including their use of data from open label extension studies.
Sent letter requesting support by Congressional Leadership to increase the appropriation for defense health research programs by five percent plus inflation in the FY 2022 Defense Appropriations Act.
Sign-on letter in support of the Speeding Therapy Access Today (STAT) Act, H.R. 1730 and S. 670. The STAT Act is bipartisan legislation led by Representatives Gus Bilirakis (R-FL) and G.K. Butterfield (D-NC) and Senators Amy Klobuchar (D-MN) and Roger Wicker (R-MS) that aims to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate access to such therapies.
Sent letters to Congressional Members requesting at least $180 million in funding for the Environmental influences on Child Health Outcomes (ECHO) program in the House Fiscal Year (FY) 2022 Labor, Health and Human Services (HHS), Education and Related Agencies appropriations bill and to thank you for your long standing commitment to this program over the last several years.
Wrote thank you for your leadership in introducing the BIOSIM Act (H.R. 2815). Biosimilar medicines hold enormous potential to lower the cost of prescription drugs but face significant barriers to adoption.
Letter of support for SB 247 that would establish a rare disease advisory council (RDAC) in California. Creating an RDAC will give rare disease patients a unified voice in the California state government. Additionally, the RDAC will act as the advisory body on rare diseases to the Legislature and state departments that provide services to, or that are charged with the care of, rare disease patients. It will also be tasked with adopting and implementing regulations, researching and determining the most appropriate method to collect data on rare diseases, and identifying best practices for rare disease care.
Sent letter to Congress stating that they should incentivize states to make the newest and most comprehensive genetic clinical services available where clinically appropriate. Expanding access to next-generation (specifically, whole genome and whole exome) sequencing could move the entire field forward into a more comprehensive testing approach from the moment a clinician suspects a child might be suffering from a rare genetic disease.
Submitted letter acknowledging that we are aware that bill, AB 1130, is under consideration by the California legislature to establish, within OSHPD, the Office of Health Care Affordability to analyze the health care market for cost trends and drivers of spending, develop data-informed policies for lowering health care costs for consumers, set and enforce cost targets, and create a state strategy for controlling the cost of health care and ensuring affordability for consumers and purchasers.
Wrote letter in response to the Request for Information Regarding Reporting on Pharmacy Benefits and Prescription Drug Costs. The patients we represent rely on prescription drugs to treat their health conditions and prevent others. We are pleased that the Biden administration is moving forward with the requirement that insurance plans must report on various data points associated with prescription drug spending. We believe with this greater understanding and transparency of prescription drug costs, you can better implement policies and measures that increase competition, improve prescription drug affordability and access for the American people. As you implement the prescription drug cost reporting requirements for health plans, we recommend that you include the following:
1. Require Plans to Report on the Treatment and Accounting of Copay Assistance.
2. Proper Accounting of Rebates and Pharmacy Benefit Managers.
3. Consider the Benefits of Prescription Drug Costs.
Signed on to letter to the Senate Member, U.S. Senate Committee on Health, Education, Labor and Pensions Health, urging the committee to take action to help people with disabilities and chronic conditions do their part to prepare for emergencies and comply with public health and emergency preparedness advice. The committee should:
- 1. ensure that people who take regular medications, regardless of their insurance coverage status, can have an extra supply on hand, at least to CDC and FEMA recommendations, to plan for emergencies in advance, and/or;
- 2. require that, at the declaration of an emergency, health plans be required to provide early refills, longer refill periods, home delivery, and waive utilization management barriers.
Signed letter outlining some of the proposed policy issues and how they impact patients with chronic diseases, especially those with autoimmune diseases, and we point to policy solutions that could immediately address affordability for patients at the pharmacy counter:
Concerns with Foreign Reference Pricing,
Quality Adjusted Life Years (QALY) Are Inherently Discriminatory and Restrict and Deny Patients with Disabilities and Chronic Illness Access to Medicines
Restricting Access to Vital Medicines Now and Lack of Future Cures
Patient Focused Policy Solutions
Counting Copay Assistance Toward Deductible, and Instituting Out-of-Pocket Caps
Ensuring Discounts Go Directly to the Patient; Addressing Rebates and Pharmacy Benefit Managers
Sent thank you letter to Senators Collins, Kelly and Menendez for introducing S.2022, the “Ending the Diagnostic Odyssey Act of 2021” that will allow states to conduct pilot programs via an increase in the Medicaid Federal Medical Assistance Percentage rate (FMAP) to provide whole genome sequencing clinical services for children on Medicaid with a disease that is suspected to have a genetic cause. We are eager to see this bill signed into law so these services can be offered to families, regardless of income.
Sign-on Letter To Address Out-Of-Pocket Costs in the Medicare Part D Program.Urge you to address the unaffordable out-of-pocket (OOP) costs in the Medicare Part D program as you draft the budget reconciliation package.
CMMI Multi-Stakeholder Administration Support Letter showing support of Biden Administration efforts to pave a new direction for CMMI, one that utilizes a fully collaborative approach in developing, monitoring, evaluating and optimally expanding promising new payment and delivery concepts.
Sent letter to Congressional leadership urging you to work toward the enactment of the fiscal year 2022 Defense Appropriations Act, to ensure that the Defense Health Research Programs, including the Congressionally Directed Medical Research Programs (CDMRP), are fully funded in fiscal year 2022.
Sent letter to Congressional leadership along with 87 organizations representing individuals living with rare diseases in the U.S. urging them to remove the provisions related to the Orphan Drug Tax Credit that would undermine the Orphan Drug Act (ODA) by limiting the availability of the Orphan Drug Tax Credit (ODTC) to only the first approved orphan use of a drug.
Signed letter to Arizona Governor urging him to issue an executive order to establish a Rare Disease Advisory Council (RDAC) in the state. The Arizona Rare Disease Advisory Council will give rare disease patients a unified voice in Arizona’s state government. Additionally, the RDAC will be a valuable advisory body to elected officials and other state leaders on rare disease research, education, diagnosis, and treatment for the care of those with rare diseases. It will also be tasked with providing a report to your office, the Legislature, and other relevant agencies highlighting the Council’s findings, activities, and recommendations for addressing the needs of rare disease patients in the state.
Signed on to letter with The Alliance for Connected Care, National Organization for Rare Disorders (NORD), and ALS Association seeks your endorsement of the below letter urging state governors and legislatures to consider maintaining and expanding medical licensure flexibilities for the duration of the federal public health emergency, reinstating license flexibilities if they have expired, or implementing new flexibilities to better address patient needs during the ongoing pandemic.
Signed onto a letter representing individuals with rare diseases in Wisconsin, we encourage you to co-sponsor LRB 5036/1 to establish a rare disease advisory council (RDAC). If passed, LRB 5036/1 would help to give a voice to the estimated 1-in-10 individuals living with a rare disease in the state of Wisconsin.
Signed letter letting governors across the country know how essential telehealth is to the rare disease community.
Signed comment letter from Haystack Project outlining concerns regarding the new Congressional Social Determinants of Health (SDOH) Caucus including:
Gaps in care, programs, and services that serve as a main barrier in addressing SDOH in the communities
Specific Social Determinants of Health challenges from the COVID-19 pandemic
Federal policies that present challenges to addressing SDOH
The unique role technology can play to alleviate specific challenges (e.g. referrals to community resources, telehealth consultations with community resource partners, etc.)
The role that patient organizations can play in ensuring that their membership reach is representative of the relevant disease population, and that all voices are included in patient registry efforts, natural history studies, and clinical trial recruitment.
Alternative payment models help to measure health care based on its outcomes, rather than its services. What opportunities exist to expand SDOH interventions in outcome-based alternative payment models and bundled payment models?
Sign letter affirming our support for President Biden’s nomination of Dr. Robert Califf as Commissioner of the Food and Drug Administration (FDA). We ask that Senators in the Democratic and Republican caucuses and the Senate HELP Committee vote to confirm Dr. Califf.
Signed letter with leaders in the rare disease community in Michigan, we are writing to urge your support of an amendment to HB-4654, as passed by the House on November 9, 2021, a bill to establish a Rare Disease Advisory Council (RDAC) for the State of Michigan.
Signed letter addressed to key Senate leadership asking them to protect the Orphan Drug Tax Credit in the Build Back Better Act preserving the incentive for the future of care for individuals and families living with autoimmune and immune-mediated diseases.
- Signed on to a letter that would support the requirement that taxpayer-funded research be made available immediately and freely – finally eliminating the current 12-month waiting period for the public to gain access to the outputs of scientific research, including data and articles.
- Signed on to a letter to Congress urging them to enact policies that would help
ensure prompt and equitable access to transformative, potentially curative treatments, such as gene therapy.
- The IPPF signed on to a letter to express our support for HR 1379, the Ensuring Lasting Smiles Act (ELSA). This bill was part of the Health, Employment, Labor, and Pensions Subcommittee’s Jan. 28 hearing; “Expecting More: Addressing America’s Maternal and Infant Health Crisis”.
- Signed on to letter to HHS Secretary Alex Azar to act immediately to restart the activities of the ACHDNC so it can continue its critically important work to improve newborn screening and save babies’ lives.
- Signed onto letter to the Center for Medicare Services (CMS) opposing the proposed rule that would expressly allow insurance plans to exclude drug manufacturer copay assistance from counting towards patients’ annual limitation on cost sharing.
- Letter sent to Congress and the Administration to take action to address the following issues during the Coronavirus (Covid-19) pandemic :
• Require all payers to allow for provision of an emergency supply of medications and supplies by relaxing restrictions on timing of refills and amounts, to at least the CDC-recommended extra 30-day supply and up to a 90-day supply to reduce returns to the pharmacy; Ensure that these protections extend to medications that are controlled substances indicated for and used to treat epilepsy, mental illnesses, cancer and other serious medical conditions, as well as injectables; Require all payers to waive prior authorization and utilization management requirements; Require flexibility regarding use of mail order pharmacies, including out of state mail order pharmacies; and other home delivery methods; Require payers to relax requirements for in-person visits for refills, including allowing visits through tele-health; Require all payers to establish policies to assist patients with cost-sharing related to their emergency supply or allow for delayed payment of out-of-pocket costs for emergency supplies; Increase FDA monitoring and reporting of shortages to ensure consistent supply, especially existing medications approved to treat chronic conditions that may be under study or used to treat COVID-19.
- Help draft a letter to the Trump Administration, requesting that he not take any action that would destabilize the health care supply chain during the Covid-19 crisis.
- Sent letter urging Congress to expand eligibility for the paid family and medical leave program to include individuals determined by the Centers for Disease Control and Prevention (CDC) to be at high risk for adverse complications from COVID-19 and working members of their households
- Signed letter in support of the Part B Access to Seniors and Physicians (ASP) Coalition’s mission to preserve patient access to Medicare Part B covered services. The IPPF is deeply concerned with the current inaccessibility to Part B Medicare-covered drugs for beneficiaries enrolled in Medicare Advantage (MA). MA plans have been using prior authorization restrictions as a barrier to timely delivery of essential Medicare Part B covered drugs to beneficiaries, putting the health of these patients at risk. MA plans are required by law to cover the same services as fee-for-service (FFS) Medicare – plans should not be permitted to use prior authorization to circumvent this requirement.
- Signed on to a letter on behalf of the patients we represent, stating that we strongly oppose recent policy changes in the 2021 Notice of Benefit and Payment Parameters (NBPP) that would allow the exclusion of manufacturer cost-sharing assistance from counting towards patients’ out-of-pocket maximums in the commercial market. This policy will prevent patients from accessing the medications they depend upon and create unnecessary barriers to maintaining effective treatment plans, especially during the global Covid-19 pandemic.
- Sent sign-on letter urging Congressional leadership, as part of the next Coronavirus (COVID-19) legislation, to task the CDC with working with stakeholders to develop a recommendation to ensure the needs of immunocompromised Americans are adequately addressed.
- Signed letter that calls on Congress to address rising out-of-pocket costs in Medicare Part D in the upcoming COVID-19 relief package.
- Signed letter intended to demonstrate a strong show of support to CMS that their latest attempt to undermine copay coupon assistance is not acceptable to patient advocates and others representing vulnerable persons who depend on these programs for their medicines, their health and well-being.
- Sent letter to urge the Congress to prioritize robust federal funding for the critical testing needed to reopen the country. Swift action is needed to ensure that every American, including essential workers, frontline healthcare providers and those at disproportionate risk for COVID-19 have access to vital COVID-19 testing, whether for diagnostic, occupational, return-to-school, public health or surveillance purposes.
- Sent sign-on letter urging Congressional leadership, urge you to work toward the enactment of the fiscal year 2021 Defense Appropriations Act, to ensure that the Defense Health Research Programs, including the Congressionally Directed Medical Research Programs (CDMRP), are fully funded in fiscal year 2021.
- Sent letter to Congressional leadership urging them to rescind the current ban on the use of copay cards in the Medicare Part D program at least through the current Novel Coronavirus (COVID-19) pandemic.
- Open letter to federal healthcare agency leaders asking them to keep their focus on the science and avoid the appearance of political influence on decision-making.
- Helped draft a comment letter to CMS on their annual proposal on doctors’ payments in the office setting of care.
- As a supporter of the Ensuring Lasting Smiles Act (HR 1379/S 560), legislation to require health insurance coverage for treatment of patients with congenital anomalies. We are pleased to report the House Energy and Commerce Committee passed the bill last month. The House Education and Labor Committee and House Ways and Means Committee share jurisdiction on the bill, and we have heard the Education and Labor Committee does not want to waive its jurisdiction. We signed on to a letter asking committee leaders to either mark up the bill or waive jurisdiction so we can get the bill to the House floor as quickly as possible given the limited amount of days left in the 116th Congress.
- The IPPF signed on to two letters that AARDA received from Aimed Alliance addressed to WEA Trust and United Healthcare regarding their recent communication to health care providers detailing provider and medical practice protocols for copay accumulator programs. These programs will negatively impact patients’ ability to access their medications, an issue that we have recognized as vital for patients with autoimmune diseases.
- Signed letter to HHS Secretary, Alex Azar, recommending that HHS provide guidance to state Medicaid programs and Medicaid Managed Organizations (MCOs) clarifying that, during the Public Health Emergency, all FDA-approved treatments for COVID, including those approved under an Emergency Use Authorization (EUA,) must be covered for Medicaid beneficiaries without delay and without prior authorization (PA) requirements. HHS should also clarify that this requirement applies to all therapies – whether inpatient, outpatient, or pharmacy. Finally, if existing contracts with state Medicaid managed care organizations prevent the waiver of PA requirements, states should carve out COVID-19 treatments into the fee-for-service (FFS) benefit.
- Signed letter with ASP along with The Part B Access for Seniors and Physicians (ASP) Coalition calling on Congressional leadership to abandon the MFN Model and focus on patient-centered reforms to protect provider-patient relationships and the high-quality care that our country has consistently been able to offer.
- Signed onto the Friends of NIDCR letter urging congressional appropriators to provide at least the Senate level of $493.234 million for the National Institute of Dental and Craniofacial Research as well as supplemental funding for the National Institutes of Health in fiscal year 2021 spending legislation.
- Signed on to AARDA’s comment letter responding to the Medicare Advantage and Part D proposed rule.
- Signed on to a letter of support for the Ensuring Lasting Smiles Act (ELSA). This link provides an overview for you about the efforts regarding the Ensuring Lasting Smiles Act: https://www.nfed.org/get-involved/advocate/ensuring-lasting-smiles-act/
- Sign-on to draft comments by I Am Essential for the proposed 2020 Notice of Benefit and Payment Parameter. The letter outlines the impact the proposed changes would have on patient access to and affordability of prescription drugs.
- Signed on to a letter with AARDA recommending that Congress allocate $8.0 million in fiscal year 2020 for a stand-alone, dedicated autoimmune disease program in the Department of Defense Congressionally Medical research Program (DoD CDMRP). The CDMRP is located in the US Army Medical Research and Materiel Command to support peer-reviewed medical research programs (PRMRP) to identify and select military health-related research of exceptional scientific merit across 50 or so priority topic areas, including arthritis, diabetes, epilepsy, inflammatory bowel disease, lupus, multiple sclerosis, scleroderma, and other autoimmune diseases.
- Signed a letter in support of Medicare coverage for medically-necessary oral/dental health therapies along with 92 other signatories sponsored by International & American Associations for Dental Research.
- Requested Congressional support for the critical and highly successful defense health research
programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at
the Department of Defense (DoD).
- Sent to Chairman Grassley, Ranking Member Wyden, Leader McConnell, Minority Leader Schumer, and the entire Senate Finance Committee a letter Urging senate leadership to maintain Medicare Part B protections.
- Sent letter to Administrator Seema Verma at the Center for Medicare and Medicaid Services U.S. Department of Health and Human Services, regarding our concern for beneficiaries’ barriers to accessing off-label treatments in Medicare Part D.
- Submitted comment letter regarding the administration’s proposed rule that would dramatically change the regulations that implement Section 1557, the anti-discrimination provision of the Affordable Care Act. The letter focuses on the impact of the proposed changes that impact benefits design and access to care and treatment for people with serious, chronic health care conditions.
- The IPPF joined 17 organizations as part of the American Academy of Dermatology (AAD) Drug Pricing Task Force signed on to the biosimilars principles document that supports common
principles around increasing uptake of biosimilar biological products.
- Sign-on letter to the leadership of the Senate Finance Committee and the leadership of the House Energy and Commerce Committee and the House Ways and Means expressing our continued support for a long-term re-authorization of PCORI, and as they negotiate a health extenders package.
- Submitted comments to the Institute for Clinical and Economic Review’s (ICER’s) proposed changes to the ICER Value Framework for 2020.
- Signed a letter of support for the swift confirmation of Dr. Hahn as FDA Commissioner.
- Signed on to a letter to Senators Jones, Collins, Menendez, and McSally thanking them for introducing the Senate version of the “Ending the Diagnostic Odyssey Act.”
- Signed on to letter to to Representatives Diana DeGette and Fred Upton providing input as Congress considers legislation to build upon the success of the 21st Century Cures Act by facilitating a modernized system of developing new cures combined with a health care system that delivers coverage and access.
- Signed on to letter to Speaker Pelosi, Majority Leader McConnell, Minority Leader McCarthy, and Minority Leader Schumer urging them to complete final negotiations on the fiscal year 2020 Defense Appropriations Act and move the bill expeditiously through the House and Senate toward enactment. Legislation will prevent delay to Defense Health Research Programs, including the Congressionally Directed Medical Research Programs, at the Department of Defense (DoD).
- The IPPF sent a letter to Medicaid Directors across the country emphasizing the importance of preserving patient access to orphan therapies in Medicaid.
- The IPPF joined the Friends of NIDCR requesting research funding of $38.4 billion for NIH, which includes funds provided to the agency through the 21st Century Cures Act, and $477 million for NIDCR, a 12 percent increase over FY2017 levels.
- The IPPF sent a letter to Congress expressing our concern with, and opposition to, the latest version of the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act.
- The IPPF signed on to support the RARE Act (Rare disease Advancement, Research, and Education Act) of 2018 (H.R.5115), introduced by Congressmen Carson and Costello.
- The IPPF signed on to a letter to congress to express our concerns about the impact the proposed rule regarding short-term limited duration plans (STLDs) (CMS-9924-P) will have both on the health insurance marketplaces and the individuals we represent. http://www.heart.org/idc/groups/heart-public/@wcm/@adv/documents/downloadable/ucm_500479.pdf
- Sent letter to the U.S. House of Representatives asking them to amend the Employee Retirement Income Security Act of 1974 to require a group health plan (or health insurance coverage offered in connection with such a plan) to provide an exceptions process for any medication step therapy protocol, and for other purposes (H.R. 2077).
- Signed on to a letter outlines proposals the Administration could take to lower patient cost-sharing since patients with serious and chronic health conditions often shoulder the heaviest cost burden and are faced with high out-of-pocket costs, such as increased premiums, higher deductibles, rising copays and co-insurance. All of which can have an impact on medication abandonment and adherence.
- The IPPF signed a letter that we will be sent to appropriators in advance of their conference on fiscal year 2019 appropriations legislation. The letter requests that appropriators fund of the National Institutes of Health (NIH) and the National Institute of Dental and Craniofacial Research (NIDCR) be at a level equal to or higher than the Senate Appropriations Committee marks of $39.1 billion and $462.024 million, respectively.
- The IPPF signed onto a letter on behalf of the Part B Access for Seniors and Physicians (ASP) Coalition, which represents a broad cross-section of U.S. health care providers and patient advocacy organizations committed to advancing life-saving innovation in the U.S.
- Health Coverage Reform Sign-on Letter to the House Energy and Commerce Committee expressing serious concern about the Preexisting Conditions Protection and Continuous Coverage Incentive Act of 2017.
- Sign-on letter with 200 patient organizations to newly confirmed U.S. Department of Health and Human Services (HHS) Secretary Tom Price urging him to maintain and enforce the ACA’s patient protections as he reviews the law and its implementing regulations.
- Sign-on letter to the Trump Administration about the challenges facing rare disease patients in America and the vital role that federal agencies play in helping to accelerate the research, development, review, and approval of treatments. These agencies must have the ability to hire and retain personnel in order to accomplish their respective missions and to achieve the broader goal of treating and curing diseases.
- Sign-on letter in opposition to the new version of this bill – HR 1313 to the House Education and Workforce Committee . This legislation would fundamentally undermine the privacy provisions in the Genetic Information Nondiscrimination Act (or ‘GINA’), and the Americans with Disabilities Act, allowing workplace wellness programs to require individuals to provide their health and genetic information and that of their families, and to impose severe penalties on those who decline to do so.
- Sign-on letter to Senate Majority Leader and Speaker of the House expressing grave concern about proposals put forth in the American Health Care Act (AHCA) to alter the fundamental structure and purpose of Medicaid, a vital source of health care for patients with ongoing chronic health needs.
- Sign-on letter with The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) are circulating the attached sign-on letter for patient organizations to join us in supporting of timely reauthorization of the Food and Drug Administration’s (FDA) user fee agreements. People with chronic and rare diseases and disabilities rely on FDA to access innovative, safe, and effective treatments.
- Sign- on letter with the Friends of NIDCR to congressional leadership urging them oppose the Administration’s budget proposals, provide the Senate Appropriations Committee approved allocation of $430.5 million for NIDCR in the final FY17 appropriation bill and our recommended level of $452 million for NIDCR in the FY18 bill.
- Sign-on letter with The Patient Access to Community Treatment (PACT) Coalition is concerned that the Medicare Payment Advisory Commission’s (MedPAC) proposed recommendations to reduce Medicare payments for Part B medicines could limit patient access to community-based care.
- Sign-on form urging Congress to engage in trade negotiations supporting policies that foster medical innovation. Patients need new cures and better treatments.
- Sent letter with NORD requesting the establishment of the FDA Office of Patient Affairs to help engage patients more effectively.
- Joined the Cystic Fibrosis Foundation and other rare disease groups in sending a letter to Senate leadership on the importance of Medicaid to our communities and in opposition to cuts to Medicaid.
- We joined 133 organizations in supporting @IAmEssentialCoalition’s response to @HHS’ Request for Information (ROI) on ways to reduce regulatory burdens and improve health care choices to empower patients. http://bit.ly/2uRYbde
- The IPPF joined the National Organization for Rare Disorders (NORD) to express our combined support of the Orphan Drug Tax Credit (ODTC) by sending a letter to Congress.
- Signed FDA letter sponsored by the Sjögren’s Syndrome Foundation and the American Autoimmune Related Diseases Association regarding eye health providing expanded access to generic ophthalmic emulsion drug products.
- The IPPF joined 200 patient organizations in sending this updated letter to Congress in emphatic support of the Orphan Drug Tax Credit.
- Joined Orphan Drug Credit Coalition along with 34 organizations to advocate preserving the Orphan Drug Tax Credit.
- Joined Friends of NIDCR sign-on letter to appropriators with our 2018 funding requests for both NIH and NIDCR.
- Sign on to letters Urge Obama Administration to Enforce ACA Nondiscrimination Provisions; and support of the 21st Century Cures Act (HR 6)
- Sign on to letter for Newborn Screening
- Sign on to letter to Senate HELP Committee Chairman Lamar Alexander and Ranking Member Patty Murray in support of their efforts to advance Innovations legislation bolstered by supplemental funding for NIH.
- Sign on to Rare Pediatric PRV Program as part of 21st Century Cures Act
- Sign on to letter To Bring Treatment to Patients with Rare Diseases
- Sign on to letter for OPEN ACT
- Sign-on letter supporting H.R. 1078, the SOS Act.
- Sign-on letter opposing undoing GINA
- Sign On Letter to Ensure Medicaid patients with rare diseases access to prescribed therapy.
- Sign-on letter supporting H.R. 1078, the SOS Act.
- Sign On to Show Support for Continuing Incentives for Manufacturers Seeking Pediatric Rare Disease Indications.
- Joined National Health Council 21st Century Cures Sign-on Letter
- Signed on to the letter in support of the $10 billion NIH Innovation Fund, which is included in the 2nd draft of the 21st Century Cures legislation.
- Joined United for Medical Research and the National Health Council in opposing the Brat amendment that cuts critical NIH funding in the 21st Century Cures Act through Politico ad campaign.
- IPPF added to Sign-on letter with PPMD to Chairman Alexander and Ranking Member Murray regarding Enhanced Patient Engagement.
- IPPF added to Sign-on letter with NORD supporting the Advancing Targeted Therapies for Rare Diseases Act (S.2030).
- IPPF added to sign-on Letter in support of Dr. Robert Califf for the position of commissioner of the Food and Drug Administration