An enduring movement
Despite the successes brought about by the ODA, the search for new rare disorder treatments is far from over. We know of more than 7,000 rare diseases; scientists discover more every year. Fewer than 5% of rare disorders have an FDA-approved treatment.
Similar to advancements made in other diseases, progress in rare disease care and treatments will continue to require an all-in approach to solve what is a looming and massive public health challenge.
As NORD founder Abbey S. Meyers wrote in her 2016 book, Orphan Drugs: A Global Crusade: “It was all of us working together that built an impregnable movement demanding a solution. In the end, with the help of government and a touch of Hollywood, the forces opposing us could not win.”
References
1. Rare Disease Day 2023 at IndoUSrare – Fireside Chat with Dr. Abbey S. Meyers. 2023 Mar 3. Indo US Organization for Rare Diseases. https://www.youtube.com/watch?v=fGTWUcQJPlU.
2. Mikami K. Soc Hist Med. 2019;32(3):609-30. doi: 10.1093/shm/hkx098.
3. National Organization for Rare Disorders. The Orphan Drug Act Turns 40: NORD Celebrates Its Impact on Rare Diseases. 2023 Jan 4. https://rarediseases.org/the-orphan-drug-act-turns-40-nord-celebrates-its-impact-on-rare-diseases. 4. Swann J. The story behind the Orphan Drug Act. US Food and Drug Administration. 2018 Feb 23. https://www.fda.gov/industry/fdas-rare-disease-day/story-behind-orphan-drug-act.
5. Roberts A-D and Wadhwa R. Orphan drug approval laws, in StatPearls (Internet). StatPearls Publishing. 2023 Jun 5. www.ncbi.nlm.nih.gov/books/NBK572052/#.
6. National Organization for Rare Disorders. Rare disease database. https://rarediseases.org/rare-diseases/.
7. National Organization for Rare Disorders. Rare disease video library. 2023 Jan 19. https://rarediseases.org/video-library/.
8. National Center for Advancing Translational Sciences. Rare disease research and resources. 2023 May 16. Accessed Sep. 17., 2023. https://ncats.nih.gov/rare-diseases.
9. Food and Drug Administration. Rare diseases team. 2023 Aug 29. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/rare-diseases-team.
10. National Organization for Rare Disorders. “NORD Guide to Rare Disorders,” 3rd edition. Philadelphia: Lippincott Williams & Wilkins, 2002.
11. National Organization for Rare Disorders. Cockayne syndrome. 2022 Jun 7. https://rarediseases.org/rare-diseases/cockayne-syndrome/.
12. National Organization for Rare Disorders. NORD Rare Disease Centers of Excellence. 2023 Jun 28. https://rarediseases.org/rare-disease-centers-of-excellence/.
13. National Organization for Rare Disorders. Metachromatic leukodystrophy. 2022 Mar 22. https://rarediseases.org/rare-diseases/metachromatic-leukodystrophy/#complete-report.
14. National Organization for Rare Disorders. The Natural History of Metachromatic Leukodystrophy Study. 2023 Feb 23. https://rarediseases.org/mld-home-study/.
15. The Natural History of Metachromatic Leukodystrophy Study (HOME Study). 2023 Jun 13. https://clinicaltrials.gov/study/NCT04628364?.
16. National Organization for Rare Disorders, Saltonstall PL. Letter to US Congress House Committee on Ways and Means. 2021 Sep 13. https://rarediseases.org/wp-content/uploads/2022/04/NORD_ODTC-Letter_FINAL.pdf.
17. National Organization for Rare Disorders. RareCare®: NORD’s patient assistance programs. 2022 Nov 1. https://rarediseases.org/wp-content/uploads/2022/04/NORD_ODTC-Letter_FINAL.pdf.