Updates - Rare Coalition

RDCC Comments on Advancing Rare Disease Therapies Through a FDA Rare Disease Innovation Hub

Rare Disease Biotech Innovators Urge Timely Reauthorization of Rare Pediatric Disease Priority Review Voucher (PRV) Program

RDCC’s Response to 21st Century Cures Act and Cures 2.0 Request for Information

Rare Disease Company Coalition Statement Applauding FDA’s Establishment of Rare Disease Innovation Hub

RDCC Delivers New Findings on Value and Impact of the Rare Pediatric Disease PRV Program

Stacey Frisk Named as New Executive Director of the Rare Disease Company Coalition

RDCC presents 2024 Outlook

RDCC Endorses Bipartisan Accelerating Kids’ Access to Care Act

Thought Leadership

Rare Disease Is Not Rare: Reflections on Rare Disease Week 2024

RDCC Supports Bipartisan Effort to Reauthorize Rare Pediatric Disease Priority Review Voucher Program

RDCC Submits Response to Senator Cassidy’s Request for Information on “Improving Americans’ Access to Gene Therapies”

ORPHAN Cures Act Enables Rare Disease Companies to Pursue Promising Research to Uncover More Treatment Options

Graham Owens Announced as Executive Director of the Rare Disease Company Coalition

Rare Disease Company Coalition Urges Congressional Action in Capitol Hill Briefing on the Risks of Rare Disease Drug Development

New White Paper Examines the Impact of Policy Uncertainty on Investment in Rare Disease Drug Development

Thought Leadership

Project GUARDIAN: Pioneering Genomic Newborn Screening for Rare Genetic Diseases

RDCC Delivers New Set of Policy Recommendations to Promote More Streamlined and Effective Early Diagnosis of Rare Diseases

Revised Inflation Reduction Act Guidance Increases Risk of Rare Disease Drug Development

Thought Leadership

How QALY Discriminates Against People Living with Rare Diseases

RDCC Proposes Policy Solutions to Remove Barriers for Rare Disease Patients Seeking Treatment Options and Increase Equity of Access

RDCC Urges Congress to Reject Proposals that Undermine the Intent of Accelerated Approval for Rare Disease Patients

RDCC Sends Letter of Support for Four Rare Disease Bills to Minnesota State Legislators and Expresses Concern for Fifth Bill that Threatens Drug Development

RDCC Applauds Reintroduction of Cameron’s Law and the Rare Disease Clinical Trial Pandemic Disruption Act

RDCC Participates in FDA Rare Disease Day 2023 Public Meeting

RDCC Responds to New HHS Payment Models Targeting Accelerated Approval Therapies

Hopes, Fears for Orphan Drug Act

Thought Leadership

Congress Can Unite to Help Kids Living with Rare Diseases

Thought Leadership

Celebrating 40 Years of the Orphan Drug Act: Renewing a Commitment to Advancing Innovation for Rare Disease Patients

Learn more about how the Orphan Drug Act enables researchers to discover breakthrough cures for rare diseases

Recognizing the 40th Anniversary of the Orphan Drug Act, The Rare Disease Company Coalition Calls on Policymakers to Renew Commitment to Advancing Innovation for Rare Disease Patients

Vital Health Podcast: The Accelerated Approval Pathway is Vital for Rare Disease Treatments

RDCC Submits Comments on Equal Employment Opportunity Commission’s FY 2022-2026 Draft Strategic Plan

Join RDCC in Support of “Leo’s Law”

RARECast Podcast: Examining the Legislative Landscape for Rare Disease Drug Development

Groups Hope New Congress Will Pass Bipartisan Bills Centered on Rare Diseases

New Study Finds Patient Access to Rare Disease Drugs Would Decline Sharply Under Proposals to Limit Medicaid Reimbursements

Arbitrary Price Controls have a Negative, Disproportionate Impact on Rare Disease Treatment Development

Thought Leadership

The State of the Biotech Industry

RDCC Joins in Call for Repeal of R&D Tax Policy that Hurts Rare Disease Innovation

RDCC Urges Swift Action to Reauthorize PDUFA in Letter to Congressional Leaders

RDCC Joins in Support of “Newborn Screening Saves Lives Reauthorization Act of 2021” in Letter to Senate Leaders

Understanding and Modernizing Newborn Screening

Comments Submitted to Colorado PDAB on Draft Proposed Rule Concerning the Affordability Review of Prescription Drugs

A Patient-Centric Approach to Evaluate Rare Disease Treatments

Amanda Malakoff Announced as Executive Director for the Rare Disease Company Coalition

Thought Leadership

Policy Must Keep Pace with Technology if we are to Reap the Benefits of AI Drug Discovery for Rare Diseases

The Orphan Drug Tax Credit is a Proven Catalyst for Investment and Treatment Development

RDCC Supports Policy Proposals That Recognize the Potential of Rare Disease Therapies and Reinforce the Role of the Accelerated Approval Pathway

The Rare Disease Company Coalition Expresses Concern to CMS on Oregon’s Application for a Section 1115 Demonstration Waiver

RDCC Offers Endorsement of the Bipartisan BENEFIT Act in Support of Patient Engagement in Drug Development