When the process of putting together this resource began in 2018, the challenges facing 30 million rare disease patients in the U.S. were already daunting, and they remain so. The Principles outlined here similarly remain every bit as vital as guideposts for access to care and treatment. But are there lessons learned from the pandemic that are relevant to the application of the Guidelines in a post-COVID world? Absolutely. And those lessons present new challenges and inspire new hope in our efforts to ensure access to critical care and therapies for rare diseases.
In this whitepaper report from the Child Neurology Foundation, the Rare Access to Critical Therapies (ACT) collaboration, and Global Genes, discover five core guiding principles to use as a set of fundamental expectations for rare disease patient care. You can read the full press release here or fill out the form below to download! You can also download and share the infographics in the Attachments section of this page (on the top right hand side) to highlight and check out key takeaways of the whitepaper.
Looking for deeper discussions around these topics? Register for the three part webinar series on the guiding principles for rare disease care and patient access, in partnership with Evidera, The Child Neurology Foundation and the National Association of Managed Care Physicians.
If you missed any of the webinars or want to rewatch, simply click on any of the below registration links. You’ll be directed to the same registration page where you can complete the form and watch the recording. If you’ve already registered, you can click “already registered” and simply enter your e-mail address.
- October 6, 2020 from 10am-11am ET: Beyond Scylla and Charybdis: Progress and Promise for Helping Rare Disease Patients Avoid the Diagnostic Odyssey and Get the Treatments They Need
- October 27, 2020 from 10am-11am ET: Rare but Well Done: How do we Ensure Health Technology Assessment and Reimbursement Processes Address the Unique Needs of Rare Disease Patients and Caregivers?
- November 12, 2020 from 10am-11am ET: Mind the Gap: How do we Address Fundamental Needs and Keep Rare Disease Patients from Falling through the Cracks?
Fill out this form to download the Guiding Principles of Rare Disease Care and Patient Access Whitepaper!