Your Drug Info Digest

References once a diagnosis is complete. Care coordinators or managers can be particularly helpful for patients with rare diseases, or their parents and caregivers, to understand their diagnosis and the next steps, as information may be limited.2

1. Cavazzoni, Patrizia. “CDER Continues to Make Rare Diseases a Priority with Drug Approvals and Programming to Speed Therapeutic Development.” U.S. Food & Drug Administration, 4 Mar. 2022, https://www.fda.gov/news-events/fda-voices/cdercontinues-make-rare-diseases-priority-drug-approvals-andprogramming-speed-therapeutic.

2. “Rare Disease: Access, Reimbursement, and Disease Management A Stakeholder Interchange Report.” American Journal of Managed Care, 3 Feb. 2022, https://www.ajmc.com/view/rare-diseaseaccess-reimbursement-and-disease-management-a-stakeholderinterchange-report.

In a roundtable of payers focusing on rare diseases, participants noted that use of high-cost agents is easier to justify if the agent targets a specific genetic marker associated with the disease.2 Payers also see value in manufacturers providing education and patient support. There may be opportunity for manufacturers to assist payers and providers in identifying these rare diseases, providing support in diagnosis, and accessing care.2

3. “Progress in Fighting Rare Diseases.” PhRMA, https://phrma.org/ Scientific-Innovation/Progress-in-Fighting-Rare-Diseases.

4. “New Drug Therapy Approvals for 2022.” U.S. Food & Drug Administration, 10 Jan. 2023, https://www.fda.gov/drugs/newdrugs-fda-cders-new-molecular-entities-and-new-therapeuticbiological-products/new-drug-therapy-approvals-2022#raredisease.