03/23/2026
Rare disease is entering a moment of real possibility โ thanks to alignment in scientific breakthroughs, technology, and patient advocacy โ with the potential for meaningful impactโฆ but only if we meet this moment with urgency and certainty.
CNBC hosted its inaugural Summit earlier in March, and followed it up with last weekโs documentary. The deeply personal stories shared by families living with rare diseases give added urgency to the insights from the Summit:
๐น๐ฅ๐ฎ๐ฟ๐ฒ ๐ฑ๐ถ๐๐ฒ๐ฎ๐๐ฒ ๐ถ๐ ๐ต๐ถ๐ฑ๐ถ๐ป๐ด ๐ถ๐ป ๐ฝ๐น๐ฎ๐ถ๐ป ๐๐ถ๐ด๐ต๐. Nearly 1 in 10 Americans is affected โ but patients, caregivers, family, and community members still spend years searching for diagnoses and access to care.
๐น๐ฅ๐ฒ๐ด๐๐น๐ฎ๐๐ผ๐ฟ๐ ๐๐ป๐ฐ๐ฒ๐ฟ๐๐ฎ๐ถ๐ป๐๐ ๐บ๐ฎ๐ ๐ท๐ฒ๐ผ๐ฝ๐ฎ๐ฟ๐ฑ๐ถ๐๐ฒ ๐ฝ๐ฟ๐ผ๐ด๐ฟ๐ฒ๐๐. Innovation canโt thrive when standards feel inconsistent and pathways unclear, especially for therapies where time matters most.
๐น๐ฆ๐ฐ๐ถ๐ฒ๐ป๐ฐ๐ฒ ๐ถ๐ ๐บ๐ฒ๐ฒ๐๐ถ๐ป๐ด ๐๐ต๐ฒ ๐บ๐ผ๐บ๐ฒ๐ป๐. Medical and technological breakthroughs are turning theoretical ideas into viable treatments and scalable platforms.
๐น๐๐ฐ๐ฐ๐ฒ๐๐ ๐ฎ๐ป๐ฑ ๐ฒ๐ฐ๐ผ๐ป๐ผ๐บ๐ถ๐ฐ๐ ๐ฎ๐ฟ๐ฒ ๐ถ๐ป๐๐ฒ๐ฝ๐ฎ๐ฟ๐ฎ๐ฏ๐น๐ฒ ๐ณ๐ฟ๐ผ๐บ ๐ถ๐ป๐ป๐ผ๐๐ฎ๐๐ถ๐ผ๐ป. Breakthroughs only matter if they reach patients. Patient engagement, research funding, insurance design, reimbursement, and global reach arenโt side issues โ they are the system in which innovation and access take place.
๐น๐๐ ๐ฐ๐ฎ๐ป ๐น๐ฒ๐๐ฒ๐น ๐๐ต๐ฒ ๐ฝ๐น๐ฎ๐๐ถ๐ป๐ด ๐ณ๐ถ๐ฒ๐น๐ฑ. From helping patients navigate complex health information to supporting research, discovery, and development, AI has real potential when paired with the right data and guardrails. As in every business, industry, and sector, AI has the potential to be transformative โ but only if harnessed and applied properly.
๐น๐๐ฑ๐๐ผ๐ฐ๐ฎ๐ฐ๐ ๐ฐ๐ต๐ฎ๐ป๐ด๐ฒ๐ ๐ผ๐๐๐ฐ๐ผ๐บ๐ฒ๐. Again and again, patients, caregivers, parents, and advocates show they arenโt just raising awareness โ theyโre helping discover drugs, funding trials, and moving the system forward when others canโt, wonโt, or arenโt properly incentivized to.
Weโre on the cusp of extraordinary progress in the rare disease space โ but only if we keep on pushing for scientific and technological innovation, regulatory certainty, capital investment, and the involvement of community and advocacy leadership.
Thanks so much to CNBC and Becky Quick for pushing this conversation and movement forward.