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“I live inside the systems I’m working to change.”

Our team recently heard from Lilly Grossman, founder and CEO of Beyond The Box Advocacy, who was kind enough to share her insights and experience working in disability policy and advocacy.

"When barriers are invisible, they are interpreted as personal shortcomings. When barriers are identified and documented, they become design flaws,” Lilly offered to explain her work challenging systems that limit people with disabilities from fully succeeding, connecting the dots between policy research and direct advocacy.

These challenges are all too common for people with disabilities, rare disease, and other devastating conditions – which is why we’re working every day to challenge conventional approaches when it comes to connecting patient communities, advocacy groups, industry, policy- and decision-makers, and other stakeholders.

Rare disease is entering a moment of real possibility – thanks to alignment in scientific breakthroughs, technology, and patient advocacy – with the potential for meaningful impact… 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 week’s documentary. The deeply personal stories shared by families living with rare diseases give added urgency to the insights from the Summit:

🔹𝗥𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝗶𝘀 𝗵𝗶𝗱𝗶𝗻𝗴 𝗶𝗻 𝗽𝗹𝗮𝗶𝗻 𝘀𝗶𝗴𝗵𝘁. Nearly 1 in 10 Americans is affected – but patients, caregivers, family, and community members still spend years searching for diagnoses and access to care.
🔹𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝘂𝗻𝗰𝗲𝗿𝘁𝗮𝗶𝗻𝘁𝘆 𝗺𝗮𝘆 𝗷𝗲𝗼𝗽𝗮𝗿𝗱𝗶𝘇𝗲 𝗽𝗿𝗼𝗴𝗿𝗲𝘀𝘀. Innovation can’t thrive when standards feel inconsistent and pathways unclear, especially for therapies where time matters most.
🔹𝗦𝗰𝗶𝗲𝗻𝗰𝗲 𝗶𝘀 𝗺𝗲𝗲𝘁𝗶𝗻𝗴 𝘁𝗵𝗲 𝗺𝗼𝗺𝗲𝗻𝘁. Medical and technological breakthroughs are turning theoretical ideas into viable treatments and scalable platforms.
🔹𝗔𝗰𝗰𝗲𝘀𝘀 𝗮𝗻𝗱 𝗲𝗰𝗼𝗻𝗼𝗺𝗶𝗰𝘀 𝗮𝗿𝗲 𝗶𝗻𝘀𝗲𝗽𝗮𝗿𝗮𝗯𝗹𝗲 𝗳𝗿𝗼𝗺 𝗶𝗻𝗻𝗼𝘃𝗮𝘁𝗶𝗼𝗻. Breakthroughs only matter if they reach patients. Patient engagement, research funding, insurance design, reimbursement, and global reach aren’t side issues – they are the system in which innovation and access take place.
🔹𝗔𝗜 𝗰𝗮𝗻 𝗹𝗲𝘃𝗲𝗹 𝘁𝗵𝗲 𝗽𝗹𝗮𝘆𝗶𝗻𝗴 𝗳𝗶𝗲𝗹𝗱. 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 – but only if harnessed and applied properly.
🔹𝗔𝗱𝘃𝗼𝗰𝗮𝗰𝘆 𝗰𝗵𝗮𝗻𝗴𝗲𝘀 𝗼𝘂𝘁𝗰𝗼𝗺𝗲𝘀. Again and again, patients, caregivers, parents, and advocates show they aren’t just raising awareness – they’re helping discover drugs, funding trials, and moving the system forward when others can’t, won’t, or aren’t properly incentivized to.

We’re on the cusp of extraordinary progress in the rare disease space – 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.

How CNBC Cures is bringing rare disease stories to a national audience