Updates - Rare Coalition

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

Thought Leadership

The Case for Rare Diseases as a Public Health Priority

Our Policy Focus Areas

Thought Leadership

“One-Size-Fits-All” HTA would be a Major Setback for Rare Disease Patients

RDCC Advocates for “Newborn Screening Saves Lives Reauthorization Act of 2021” in Letter to Congressional Leaders

The Rare Disease Company Coalition Announces 2022 Executive Committee and Leadership Appointments

The Rare Disease Company Coalition Submits Comments on Oregon’s Application for a Section 1115 Demonstration Waiver

Thought Leadership

The Unique Clinical and Economic Challenges of Rare Disease Diagnosis

Cutting the Orphan Drug Tax Credit is Not Drug Pricing Reform

The Rare Disease Company Announces New Members and Continues Growth in its Inaugural Year

Introduction of Cures 2.0 Applauded by Coalition Dedicated to Rare Disease Treatment Development with Call to Congress to Recognize Both Challenges and Opportunities in Rare Disease R&D

Congressional Leaders Continue to Place Politics Over Rare Disease Patients with Proposed Changes to Orphan Drug Tax Credit in Build Back Better Act