CFRI is honored to share Voice of the Cystic Fibrosis Patient, a summary report of the experiences, preferences, hopes, and fears shared by those attending CFRI’s Externally-Led Patient-Focused Drug Development Meeting on Cystic Fibrosis held October 29, 2018.

On this momentous day, the diverse and complex patient experience with cystic fibrosis (CF) was shared with representatives of the Food & Drug Administration (FDA), healthcare providers, researchers, biotechnology company representatives, and fellow members of the CF community, so as to expand understanding of disease impacts, the critical need for new therapies, and our community’s willingness to actively participate in clinical trials and to assume risks so as to advance drug development.

To read the report, click here.

The meeting is available for viewing here: http://www.youtube.com/watch?v=9EBhbzyvP1Q

 

Platinum Sponsor: Vertex 

Gold Sponsor: Gilead 

Silver Sponsor: Proteostasis Therapeutics, Genentech, Ionis Pharmaceuticals, Synspira

Bronze Sponsors: Santhera Pharmaceuticals, Translate Bio, Eloxx Pharmaceuticals, Mylan Pharmaceuticals (through an independent medical education grant), and AzurRx BioPharma.

CFRI believes that anyone can be an advocate. Whether you prefer engaging in one-on-one interactions with friends and family to provide CF-related education and resources, or you enjoy speaking with elected officials to advance a specific initiative, your voice matters! This toolkit provides an overview of how to engage in advocacy efforts, while also providing educational materials on specific CF-related topics that can be used for yourself, shared with those in your immediate circle, or offered to key decision makers. Learn about the following and raise your voice:

• Forming Relationships with Legislators
• Sharing your Story
• Oral & Written Testimony
• Leveraging Social Media
• Rare Disease Advisory Councils
• Diversity & Inclusion
• CFTR Modulator Therapies
• Rare Disease Research
• Newborn Screening
• Pharmacy Benefit Managers
• Copay Accumulator Policies
• Prescription Drug Affordability Review Boards
• Step Therapy
• CF & Cancer Awareness

To read the report, click here.

During the COVID-19 pandemic, CFRI engaged in advocacy efforts to promote the health and wellbeing of the CF and broader rare disease community. Protecting individuals with previous serious and chronic health conditions during this unpredictable and vulnerable time became a cornerstone of CFRI’s advocacy priorities during the pandemic. CFRI engaged in the following initiatives to help protect members of our community:

• Letter to ICER urging pause on assessments related to COVID-10 using their cost effectiveness model
• Letter to the Minnesota Department of Health regarding the state’s Crisis Standards of Care
• Letter to Congress urging paid family and medical leave for people with chronic health conditions
• Letter to insurance commissioners asking to maintain supply of vital medications
• Letter to the Health and Human Services Secretary urging for those with CF to receive continued treatment

To read the report outlining these initiatives, click here.

The National Organization for Rare Disorders (NORD), has created a toolkit comprised of simple and easy-to-follow insurance navigation guides that break down essential health coverage topics. The toolkit is intended to help individuals understand how health insurance works, what different terms mean, and how to make informed decisions about medical care. From choosing a plan, figuring out costs, or learning about benefits, NORD’s resources are designed to give you the clarity you need.

To read the toolkit, click here.

To explore NORD’s Health Coverage Basics webpage, visit: https://www.claimyourcare.org/health-coverage-basics/