2026
EveryLife Foundation’s Priorities for the Reauthorization of PDUFA VIII – EveryLife Foundation for Rare Diseases’ priorities for the reauthorization of PDUFA VIII as of May 2026. Priorities reflect the engagement of the Ad-Hoc PDUFA Working Group in 2025 and 2026. – PDUFA VIII, Policy Papers – August 7, 2026
The EveryLife Foundation Submits Comments in Response to Rep. Auchincloss’ Request – EveryLife Foundation submitted comments in response to Representative Jake Auchincloss’ request for feedback on a legislative discussion draft of the Next-Generation U.S. Clinical Development to Accelerate Cures Proposal. – Policy Papers – July 1, 2026
The EveryLife Foundation Submits Comments on the Impacts of PFDD Meetings – The EveryLife Foundation for Rare Diseases submitted comments in response to the FDA’s request for information on the impacts of Patient-Focused Drug Development (PFDD) meetings – PFDD, Policy Papers, Request for Information (RFI) – July 1, 2026
The EveryLife Foundation Submits Comments on Prior Authorization Rules – The EveryLife Foundation submitted comments to the Center for Medicare and Medicaid Services on the proposed rule regarding interoperability standards and prior authorization for prescription drugs. – CMS, Medicaid, Policy Papers, Prior Authorization – June 16, 2026
The EveryLife Foundation Joins 43 Organizations in Letter to Protect Medicaid Funding – 44 organizations urge Congress to exclude additional funding cuts or policies that would further impact Medicaid and the Patient Protection and Affordable Care Act in any future reconciliation package. – Medicaid, Policy Papers, Sign-On – April 22, 2026
The EveryLife Foundation Joins 358 Organizations in Letter to Prevent Medicaid and HCBS Cuts – Members of the Disability and Aging Collaborative (DAC), the Health and Long-Term Services and Supports (LTSS) Taskforces of the Consortium for Constituents with Disabilities (CCD), and allied organizations urge Congress to prevent funding cuts or harmful changes to Medicaid. – Medicaid, Policy Papers, Sign-On – April 14, 2026
2025
EveryLife Foundation and 190 Organizations Sign On in Support of the Give Kids a Chance Act of 2025 – This urgent legislation would reauthorize the highly effective Rare Pediatric Disease Priority Review Voucher (PRV) program for five years – Policy Papers – Everylife Foundation – October 15, 2025
EveryLife Foundation Submits Comments on PDUFA VIII Reauthorization – After compiling community input for the reauthorization of the Prescription Drug User Fee Act (PDUFA VIII), the EveryLife Foundation submitted comments reflecting our coalition’s shared priorities – Policy Papers – Everylife Foundation – August 18, 2025
Rare Disease Patient Community Letter to FDA Commissioner Makary – 101 patient advocacy organizations request the establishment of a complementary town hall-style meeting series to strengthen engagement between the FDA and patient advocacy groups. – Policy Papers – Everylife Foundation – June 24, 2025
Newborn Screening Coalition Letter – 272 organizations committed to the health of our nation’s mothers, infants, children, and families, express their deep concern over the Administration’s recent decision to eliminate the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). – ACHDNC, Advisory Committee for Heritable Disorders in Newborns and Children, Newborn Screening, Policy Papers, RUSP – Everylife Foundation – May 5, 2025
Expedited Program for Serious Conditions — Accelerated Approval of Drugs and Biologics Guidance for Industry – The accelerated approval pathway is working as intended: delivering better health to those in dire need. Unfortunately, non-oncology rare diseases are nowhere near that milestone and account for just 23% of all products approved using the accelerated approval pathway. – Accelerated Approval, Policy Papers – Everylife Foundation – March 10, 2025
Expediting Treatments in the 21st Century: Orphan Drugs and Accelerated Approvals – The typical timings for AA to be confirmed or withdrawn has not changed significantly over the decades, the disease areas utilizing orphan drug designation and AA have changed significantly over time. – Accelerated Approval, Policy Papers – Everylife Foundation – January 22, 2025
2024
EveryLife Submits Comments Supporting Prioritization of FDA Rare Disease Innovation Hub – Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub; Public Meeting; Request for Comments. – Policy Papers – Everylife Foundation, fda, Reuben Domike – November 1, 2024
Statement from EveryLife Foundation for Rare Diseases House Energy & Commerce Committee Markup – On behalf of the EveryLife Foundation for Rare Diseases, we thank you for convening this markup to advance bipartisan legislation that will help to support the needs of people and families impacted by rare diseases and disorders. – Policy Papers – Everylife Foundation – September 18, 2024
NORD-EveryLife IRA Letter to SFC – On behalf of the more than 30 million Americans living with one of the over 10,000 known rare diseases, the National Organization for Rare Disorders (NORD) and the EveryLife Foundation for Rare Diseases thanks the Senate Finance Committee for holding a hearing entitled “Lower Health Care Costs for Americans: Understanding the Benefits of the Inflation Reduction Act.” – Policy Papers – Everylife Foundation – September 17, 2024
EveryLife Foundation Submits Comments to Representatives DeGette and Bucshon on Enhancing the 21st Century Cures Act – EveryLife Foundation submitted comments highlighting relevant provisions of Cures 2.0 that should carry forward and ongoing policy needs that should be incorporated into a Cures 3.0 package. – 21st Century Cures Act, Policy Papers – Everylife Foundation – August 5, 2024
EveryLife Foundation submitted comments to the FDA to inform their efforts to optimize the use of and processes of its advisory committees. – EveryLife Foundation Submits Input on FDA Advisory Committee Reforms – Policy Papers – July 24, 2024
EveryLife Foundation Letter in Support of Report Language Establishing an FDA Rare Disease Center of Excellence – The report language directs the FDA to create an Intercenter Institute for Rare Diseases that will serve as a cross-cutting, capacity-building, collaborative hub for rare disease activity at the FDA. – Policy Papers – Everylife Foundation – June 11, 2024
The Promising Pathways Act aims to support the critical goal of working to bring treatments to communities faster. – EveryLife Issues Statement on Promising Pathways Act – New Therapies, Policy Papers – June 6, 2024
EveryLife Foundation Advocates for Improved Newborn Screening Processes in Recent Comments on ACHDNC – The EveryLife Foundation provided comments on the (ACHDNC) processes for evidence-based review and nomination to the Recommended Uniform Screening Panel (RUSP) – Advisory Committee for Heritable Disorders in Newborns and Children, Foundation Papers, Presentations, Releases & Letters, NBS, Newborn Screening, Policy Papers, RUSP – Everylife Foundation – April 5, 2024
Safe Step Act will ensure employer health plans offer a medically reasonable and expedient step therapy exceptions process. – The EveryLife Foundation offered comments to inform the House Committee on Education and Workforce’s efforts to modernize and strengthen the Employee Retirement Income Security Act (ERISA). – ERISA, Policy Papers, Policy Positions, Safe Step Act – Everylife Foundation – April 5, 2024
Valuing Rare Disease Treatments in Healthcare: Real Experience, Real Impact – The EveryLife Foundation and The Innovation and Value Initiative (IVI)’s project aims to advance the field of patient-centered outcomes research for rare diseases. – Patient‑Centered Core Impact Sets, Policy Papers – Everylife Foundation, IVI – February 7, 2024
2023
EveryLife Foundation submitted comments to FDA on the Draft Guidance document – Comments to inform the Food and Drug Administration on the Draft Guidance document entitled Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products – FDA, Policy Papers – Everylife Foundation, fda – December 22, 2023
EveryLife submitted comments to FDA on Proposed Rule Involving Laboratory Developed Tests (LDTs) – Comments to inform and make recommendations to the FDA on their Proposed Rule Involving Laboratory Developed Tests (LDTs) – FDA, PFDD, Policy Papers – Everylife Foundation – December 7, 2023
EveryLife Foundation Joins Forces with NORD and Leading Patient Orgs to Address Technical Changes to IRA – Advocacy, Blog, Drug Development, Foundation Papers, Presentations, Releases & Letters, Front Page, Legislation, News, Orphan Drug Tax Credit (ODTC), PFDD, Policy Papers, Rare Disease Drug Development – December 6, 2023
EveryLife Foundation Releases Newborn Screening Modernization White Paper – This pivotal document, crafted with insights from over 100 experts, charts a transformative path for the U.S. NBS system. – Policy Papers – Everylife Foundation – September 6, 2023
EveryLife submitted comments to inform the FDA on the Patient-Focused Drug Development Guidance – Comments to inform the U.S. Food and Drug Administration on the “Patient-Focused Drug Development Guidance 4: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making”. – FDA, PFDD, Policy Papers – Everylife Foundation – July 14, 2023
EveryLife submitted comments to CMS on Medicare Drug Price Negotiation Program – Comments on Medicare Drug Price Negotiation Program – CMS, Drug Pricing, Medicare, Policy Papers – Everylife Foundation – April 14, 2023
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery – Brief summary of the primary types of GTTs and an overview of the current state of the science. – Gene therapies, Policy Papers – Melissa A. Parisi, P J Brooks, Tiina K. Urv – March 23, 2023
The EveryLife Foundation Submits Comments to Advisory Committee for Heritable Disorders in Newborns and Children Following February Meeting – Comments related to the evidence review of Krabbe disease for consideration to the Recommended Uniform Screening Panel (RUSP) – Advisory Committee for Heritable Disorders in Newborns and Children, Newborn Screening – Blog, Policy Papers – ACHDNC, Annie Kennedy, Dylan Simon – February 14, 2023
EveryLife submitted comments to CMS about their proposed changes to the health equity and utlization management components of the proposed rule on Medicare Advantage and the Medicare Prescription Drug Benefit – Comments relevant to the health equity and utilization management components of the proposed rule on Medicare Advantage and the Medicare Prescription Drug Benefit. – CMS, Health Equity, Medicare, Policy Papers – Dylan Simon, Priscilla Rodriguez – February 13, 2023
2022
Expert Evaluation of Strategies to Modernize Newborn Screening in the United States – First study to delineate solutions to U.S. newborn screening system challenges and identify the need for modernization to keep pace with innovation. – Newborn Screening – Blog, Policy Papers – Angela Y. Gwaltney, Donald B. Bailey Jr, Holly L. Peay, Katherine Ackerman Porte, Melissa Raspa, Sara M. Andrews – December 29, 2022
The EveryLife Foundation Submits End-of-Year Letter to Congressional Leadership – Request to include provisions contained within the bipartisan Speeding Therapy Access Today (STAT) Act (H.R. 1730, S. 670) – Policy Papers, STATAct – Annie Kennedy, Jamie Sullivan – November 15, 2022
The Rare Disease Diversity Coalition (RDDC) – Appropriations Act and End of Year Priorities – The Continuing Appropriations and Ukraine Supplemental Appropriations Act and End of Year Priorities – Appropriations Act, Policy Papers, Rare Disease Diversity Coalition (RDDC) – Jenifer Ngo Waldrop – November 4, 2022
The EveryLife Foundation Provides Testimony on Newborn Screening Efforts to the Advisory Committee for Heritable Disorders in Newborns and Children – The EveryLife Foundation’s Dylan Simon and the co-chair of the Community Congress Diagnostics Working Group and President of Project Alive Dr. Kim Stephens testified during the ACHDNC. – Newborn Screening – Blog, Policy Papers – Dylan Simon, Kim Stephens – November 3, 2022
Rare Disease Week on Capitol Hill 2021 – Diversity Roundtable Discussions – EveryLife joined by groups specialized in minority health policy and rare disease. – Diversity, Policy Papers – Priscilla Rodriguez – September 21, 2022
EveryLife Foundation Signs Support Letter for FY23 Appropriations – Americans need champions who will assure that Congress and the Administration commit to concluding the FY23 appropriations process on schedule. – FY23 Appropiations, Policy Papers – Everylife Foundation – September 19, 2022
The EveryLife Foundation partners up with Expecting Health for the 4 th annual Newborn Screening Bootcamp – The EveryLife Foundation partners up with Expecting Health for the 4th annual Newborn Screening Bootcamp – Advisory Committee for Heritable Disorders in Newborns and Children, Newborn Screening – Blog, Policy Papers – Dylan Simon – August 30, 2022
EveryLife Foundation comments to CMS annual update of the Inpatient Payment Prospective System (IPPS) that sets payment policies for inpatient hospital-based care – Solicitation on Possible Mechanisms to Address Rare Diseases and Conditions Represented by Low Volumes Within the MS-DRG Structure – CMS, Policy Papers, Uncategorized – Annie Kennedy, Jamie Sullivan – June 17, 2022
EveryLife Foundation comments to Food and Drug Administration (FDA) on setting recommendations for diversity plans that aim to increase clinical trial diversity – Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials: Guidance for Industry – Diversity, FDA, Policy Papers – Jamie Sullivan, Priscilla Rodriguez – June 13, 2022
EveryLife Foundation comments to CMS on a proposal contained within Oregon’s 1115 Waiver Application – Comments to the Centers for Medicare and Medicaid Services (CMS), urging them to reject a proposal contained within Oregon’s 1115 Waiver Application – CMS, Oregon, Policy Papers – Everylife Foundation – April 12, 2022
The National Economic Burden of Rare Disease in the United States in 2019 – Comprehensive assessment of the total economic burden of rare diseases (RD) in the United States (U.S.) in 2019. – Burden Study, Economic Burden of Rare Diseases, Economic Impact, Policy Papers – Anne Pariser, Annie Kennedy, Elisabeth Oehrlein, Grace Yang, Inna Cintina, Jamie Sullivan – April 1, 2022
Patient‑Centered Core Impact Sets: What They are and Why We Need Them – Recommendations on how to move the goal of a patient centered core impact set forward through collaboration, leadership, and establishment of a patient-centered core impact set development blueprint with supporting tools. – Patient‑Centered Core Impact Sets, Policy Papers – Annie Kennedy, Eleanor M. Perfetto, Elisabeth M. Oehrlein, Jennifer Bright, Silke Schoch, T. Rosie Love – April 1, 2022
EveryLife Issues Statement on Rare Disease Community’s Drug Pricing Considerations – Senate Finance Committee Hearing, Prescription Drug Price Inflation: An Urgent Need to Lower Drug Prices in Medicare – Drug Pricing, Medicare, Policy Papers – Everylife Foundation – March 16, 2022
Patient Advocacy Group Comments to CMS in Response to Proposed Decision Memo: Monoclonal Antibodies Directed Against Amyloid for the Treatment of Alzheimer’s Disease – Comment Letter from Patient Advocacy Group – Alzheimer, Monoclonal Antibodies, Policy Papers – EveryLife and patient advocacy groups – February 9, 2022
The Economic Burden Of Rare Diseases: Quantifying The Sizeable Collective Burden And Offering Solutions – Each study employed its own methodology and approach to capture the cost burden of rare diseases in the US, and all four aligned in their core conclusions – Burden Study, Economic Burden of Rare Diseases, Economic Impact, Policy Papers – Anne R. Pariser, Annie Kennedy, Joni L. Rutter, Nick Manetto, Sheldon Garrison – February 1, 2022
Guide to Patient Involvement in Rare Disease Therapy Development – A resource for all stakeholders to utilize as we work to optimize our rare disease product development efforts. – PFDD, Policy Papers – Annie Kennedy, Christine Harhaj, Kate Donigan, Kim McCleary, Maria Apostolaros, Samantha Mayberry – January 25, 2022
Common Challenges and Identified Solutions for State Newborn Screening Programs during COVID-19 Pandemic – During the COVID-19 pandemic, state newborn screening programs faced challenges to ensure this essential public health program continued to function at a high level. In December 2020, the EveryLife Foundation held a workshop to discuss these common challenges and solutions. – Covid-19, Pandemic, Policy Papers – Everylife Foundation – January 18, 2022
The EveryLife Foundation submits comments to the Oregon Health Authority – Application for Renewal and Amendment Oregon Health Plan, Section 1115 Demonstration Waiver – Oregon Health Authority, Policy Papers – Annie Kennedy, Julia Jenkins – January 7, 2022
2021
Expert Evaluation of Strategies to Modernize Newborn Screening in the United States – First study to delineate solutions to U.S. newborn screening system challenges and identify the need for modernization to keep pace with innovation. – Newborn Screening – Blog, Policy Papers – Angela Y. Gwaltney, Donald B. Bailey Jr, Holly L. Peay, Katherine Ackerman Porte, Melissa Raspa, Sara M. Andrews – December 29, 2021
Statement of Support for “The Precision Medicine Answers for Kids Today” Act – Reps. Upton and DeGette introduced their much anticipated Cures 2.0 legislation. – Cures 2.0, Policy Papers – Everylife Foundation, Global Genes, The Assistance Fund – November 16, 2021
Rare Disease Drug Development: Clinical, Scientific, Patient & Caregiver Perspectives (Book) – Broad overview of rare disease drug development – Policy Papers, Rare Disease Drug Development – Alistair Macdonald, Lynn O’Connor Vos, Sir Dennis Gillings, Thomas Pike – November 9, 2021
The EveryLife Foundation Provides Testimony at the Federal Advisory Committee for Heritable Disorders in Newborns and Children – EveryLife’s Newborn Screening initiative is focused on ensuring babies receive lifesaving treatment opportunities through early diagnosis with newborn screening. – Advisory Committee for Heritable Disorders in Newborns and Children, Policy Papers – Dylan Simon – November 9, 2021
Takeda Releases New Report: “Reducing Time to Diagnosis for People Living with Rare Diseases: A Conversation on U.S. Policy Opportunities” – Reducing Time to Diagnosis for People Living with Rare Diseases: A Conversation on U.S. Policy Opportunities – Diagnosis, Policy Papers, Takeda – November 4, 2021
The EveryLife Foundation Joins 13 Health Experts and Organizations in Sending a Letter in Support of U.S. Rep. Anna G. Eshoo’s Legislation to Create an Independent Advanced Research Projects Agency for Health (ARPA-H) – Bill that would authorize an important conduit for bold new health innovations. – ARPA-H, Policy Papers – EveryLife Foundation Joins 13 Health Expert – October 25, 2021
FDA’s Accelerated Approval Pathway: Separating Fact From Fiction – The accelerated approval pathway is working as intended: delivering better health to those in dire need. We must value and protect it. – Accelerated Approval, FDA, Policy Papers – Annie Kennedy, Ken Thorpe, Pat Furlong – October 20, 2021
The EveryLife Foundation Provides Testimony at PDUFA VII Meeting – Invited Stakeholder Reaction Remarks – PDUFA VII, Policy Papers – Annie Kennedy – September 30, 2021
The EveryLife Foundation Releases Statement on the Recent Orphan Drug Tax Credit (ODTC) Changes – EveryLife oposes the recently proposed changes to the Orphan Drug Tax Credit (ODTC) included in the Build Back Better Act – Orphan Drug Tax Credit (ODTC), Policy Papers – Everylife Foundation – September 21, 2021
EveryLife Provides Testimony at Federal Advisory Committee for Heritable Disorders in Newborns and Children – EveryLife’s Newborn Screening initiative is focused on ensuring babies receive lifesaving treatment opportunities through early diagnosis – Early diagnosis, Newborn Screening – Blog, Policy Papers – Liesl Broadbridge – August 12, 2021
The EveryLife Foundation Submits Comments to Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) on Cures 2.0 and ARPA-H – Thoughts on the discussion draft, including responses to questions put forward in the Request for Information (RFI) on the creation of the Advanced Research Projects Agency for Health (ARPA-H). – ARPA-H, Policy Papers, Request for Information (RFI) – Everylife Foundation – July 16, 2021
The EveryLife Foundation Applauds Cures 2.0 Discussion Draft – EveryLife provided Representatives DeGette and Upton a detailed discussion of priority provisions for a future “Cures 2.0” legislation – Cures 2.0, Policy Papers – Everylife Foundation – June 29, 2021
The EveryLife Foundation Introduces an ICD Code Roadmap Resource Guide – ICD Code Roadmap provides all the information to determine whether and how to get involved in the continuous process of refining the diagnostic coding system used in the U.S. – ICD Code, Policy Papers – Everylife Foundation – June 16, 2021
The EveryLife Foundations Submits Sign-on Letter with 110 Rare Disease Partners in Support of the STAT Act – 110 Rare Disease Partners Join STAT Act Sign-on Letter – Policy Papers, STATAct – Everylife Foundation – May 28, 2021
EveryLife Urges Action to CDC on COVID-19 Vaccines and Mask Guidance – EveryLife Foundation sent a letter to CDC leadership outlining these concerns and urging action. – Covid-19, COVID-19 Vaccines, Policy Papers – Everylife Foundation – May 20, 2021
Rare Disease Community Statement on Drug Pricing Policy Priorities – Lowering the cost of health care, including prescription drug therapies, is an important but nuanced goal of multiple legislative proposals under consideration in Congress and state legislatures – Drug Pricing, Policy Papers – Everylife Foundation – May 20, 2021
The EveryLife Foundation Provides Testimony at Federal Advisory Committee for Heritable Disorders in Newborns and Children – Update on recent newborn screening initiatives – Advisory Committee for Heritable Disorders in Newborns and Children, Policy Papers – Everylife Foundation – May 14, 2021
The EveryLife Foundation, in Partnership with the Community Congress COVID-19 Response Working Group, Shares U.S Rare Disease Covid Vaccine Survey Results – Survey designed to better understand the rare disease community’s perceptions and information needs about a vaccine for COVID-19 and to inform policy engagement efforts. – Covid-19, COVID-19 Vaccines, Policy Papers – Everylife Foundation – April 16, 2021
The EveryLife Foundation Releases the National Economic Burden of Rare Disease Study, estimating the cost for 379 rare diseases in the United States totaled $966 billion in 2019 – Economic Burden of Rare Diseases in the U.S. Approached $1 Trillion in 2019, Surpassing Cost Estimates for Many Chronic Diseases – Burden Study, Economic Burden of Rare Diseases, Economic Impact, Policy Papers – Everylife Foundation – February 25, 2021
The EveryLife Foundation Sends Letter to Federal COVID-19 Response Leadership Outlining the Roadblocks the Rare disease Community is Experiencing During the COVID-19 Vaccine Rollout – highlight the ongoing challenges the rare disease community is facing as they seek to obtain COVID-19 vaccines. – Covid-19, COVID-19 Vaccines, Policy Papers – Everylife Foundation – February 18, 2021
The EveryLife Foundation Submits Comments to the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) on the Newborn Screening Saves Lives Reauthorization Act – Continued advocacy for newborn screening – Advisory Committee for Heritable Disorders in Newborns and Children, Policy Papers – Everylife Foundation – February 10, 2021
The EveryLife Foundation’s Community Congress Submits Comments to the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) on the Newborn Screening Saves Lives Reauthorization Act – Comments to inform the Advisory Committee’s ongoing conversations about the review process for new RUSP nomination packages. – Advisory Committee for Heritable Disorders in Newborns and Children, Policy Papers – Everylife Foundation – February 10, 2021
The EveryLife Foundation Sends Letter to President Biden Commending the Appointment of Dr. Janet Woodcock as Acting Commissioner of the Food and Drug Administration (FDA) – The Foundaiton applauds the appointment of Dr. Janet Woodcock for the position of Acting Commissioner of the Food and Drug Administration. – FDA, Policy Papers – Everylife Foundation – January 22, 2021
EveryLife Foundation Writes Letter to President-Elect Biden Laying Out the Rare Disease Community’s Policy Recommendations for 2021 – EveryLife lays out our rare disease community’s policy recommendations – Biden, Policy Papers – Everylife Foundation – January 12, 2021
EveryLife Foundation Joins PIPC Comment Letter on Most Favored Nation Rule – Urge the immediate, full withdrawal of the Most Favored Nation rule – PIPC, Policy Papers – Everylife Foundation – January 7, 2021
2020
Focusing on the Patient: Implementation of Key 21st Century Cures Provisions and Recommendations for the Future – While patient engagement is a broad, growing category, this article will review two provisions of the 21st Century Cures Act: Sections 3001 (Patient Experience Data) and 3002 (Patient-Focused Drug Development Guidance), which sought to achieve two fundamental goals regarding patient engagement policies – 21st Century Cures Initiative, Policy Papers – Annie Kennedy, Lauren Bloch, Nick Manetto, Tim Franson – December 3, 2020
EveryLife Foundation for Rare Diseases Submits Public Comments to the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) – Variety of newborn screening-related issues – Advisory Committee for Heritable Disorders in Newborns and Children, Policy Papers – Everylife Foundation – December 1, 2020
EveryLife Foundation for Rare Diseases Comments on Medical Device User Fee Amendments (MDUFA) – Medical Device User Fee Amendments (MDUFA) for Fiscal Years 2023 Through 2027 – Medical Device User Fee Amendments (MDUFA), Policy Papers – Annie Kennedy, Julia Jenkins – November 27, 2020
The EveryLife Foundation Submits Comments on the Discussion Draft of the Preliminary Framework for Equitable Allocation COVID-19 Vaccine – Discussion Draft of the Preliminary Framework for Equitable Allocation COVID-19 Vaccine, commissioned by the National Institutes of Health and the Centers for Disease Control and Prevention – Covid-19, COVID-19 Vaccines, Policy Papers – Everylife Foundation – September 9, 2020
Value to Whom? Incorporating Patient Perspectives into Value Assessment for Novel Cell and Gene Therapies – Developed by Health Advances in collaboration with the Partnership to Improve Patient Care and the EveryLife Foundation for Rare Diseases – Gene therapies, Policy Papers, Value Assessment – Everylife Foundation, PIPC – August 1, 2020
Stakeholder‑Engaged Derivation of Patient‑Informed Value Elements – Identify patient-informed value elements that can be used to make value assessment more patient centered. – Patient‑Informed Value Elements, Policy Papers – Annie Kennedy, Beverly Butler, Chengchen Zhang, Juan Caicedo, Julia F. Slejko, Susan dosReis, Yoon Duk Hong – July 1, 2020
Taking Stock of PFDD: Envisioning a Vibrant Future for Patient-Focused Drug Development – PFDD works is a collaborative forum for patient advocacy leaders to share learnings and advance understanding of how patient-focused drug development – PFDD, Policy Papers – Annie Kennedy, David Zook, Debra Lappin, Isabelle Lousada, Jamie Sullivan, Jill Yersak, Kim McCleary, Kristen Santiago, Leah Howard, Pat Wildman, Todd Sherer – April 1, 2020
2019
Leveraging Evidence-Based Public Policy and Advocacy to Advance Newborn Screening in California – This case study can serve as a model for advocates looking to expand state newborn-screening programs. – Newborn Screening – Blog, Policy Papers – Bertrand Lubin, Mark Dant, Max G. Bronstein, Richard J. Pan – February 1, 2019
2017
Delay in State Adoption of Newborn Screening Tests – Before the RUSP, there was considerable variation across states with respect to newborn diseases screened. 2 The RUSP was aimed at reducing this variation. – Newborn Screening – Blog, Policy Papers – Ang Xu, Stephanie R. Morain, Vignesh Ganapathy – December 1, 2017
EveryLife Foundation for Rare Diseases Comments on FDA-2017-N-3615: Administering The Hatch-Waxman Amendments – Administering the Hatch-Waxman Amendments: Ensuring a Balance Between Innovation and Access; Public Meeting; Request for Comments; Extension of Comment Period – FDA, Policy Papers – Rachel Klein – November 17, 2017
For Rare Disease Patients, a Pathway to Hundreds of New Therapies – Rare disease patients use drugs off-label based on limited data because they have no better options available – New Therapies, Policy Papers – Chip Chambers, David Fajgenbaum, Emil Kakkis, Max Bronstein – March 21, 2017
2016
Closing the Rare Disease Innovation Gap – The Food and Drug Administration recently approved a novel therapy for Duchenne muscular dystrophy utilizing this special pathway when the science is imperfect but the disease is deadly. We support the approval but also note that the development program left many questions unresolved – FDA, Policy Papers – Emil Kakkis, Max Bronstein – October 7, 2016
Patients as Key Partners in Rare Disease Drug Development – As the technology to produce these complex therapeutics has matured, many more ADCs have been approved or are in late-phase clinical trials. – Policy Papers, Rare Disease Drug Development – Emil Kakkis, Max Bronstein – July 22, 2016
Accessing the Accelerated Approval Pathway for Rare Disease Therapeutics – Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases – Accelerated Approval, Policy Papers – Emil Kakkis, Max Bronstein – April 7, 2016
2015
Comments on Draft Guidance: “Rare Diseases: Common Issues in Drug Development” – Rare disease drug development presents a variety of unique and at times, daunting challenges which may require significant guidance to overcome – Drug Development, Policy Papers – Community Congress Program, Everylife Foundation, Regulatory Science Working Group – October 16, 2015
Recommendations for the Development of Rare Disease Drugs using the Accelerated Approval Pathway and for Qualifying Biomarkers as Primary Endpoints in Pivotal Clinical Studies – Rare Disease challenges have been recognized by all stakeholders who endorse the need for flexibility in the regulatory review process for novel therapeutics to treat rare diseases. I – Accelerated Approval, Clinical Studies, Policy Papers – Emil Kakkis – February 10, 2015
2014
EveryLife Foundation for Rare Diseases Comments in Response to First 21st Century Cures White Paper – Recommendations in response to white papers to spur development of lifesaving therapies. – 21st Century Cures Initiative, Policy Papers – Julia Jenkins – June 13, 2014
2013
Draft Guidance Comments for Docket No. FDA-2013-D-0575 – Expedited Programs for Serious Conditions––Drugs and Biologics – FDA, Policy Papers – Everylife Foundation, Organizations – August 26, 2013
2011
The Potential Investment Impact of Improved Access to Accelerated Approval on the Development of Treatments for Low Prevalence Rare Diseases – Research brings attention to the need for well-defined and practical qualification criteria for the use of surrogate endpoints to allow more access to the AA approval pathway in clinical trials for rare diseases. – Accelerated Approval, Policy Papers – B. Miyamoto, Emil Kakkis – July 6, 2011
