Rare Disease Funding Called ‘Modern-Day Civil Rights Issue’ скачать в хорошем качестве

Rare Disease Funding Called ‘Modern-Day Civil Rights Issue’

Crowdfunding, Venture Philanthropy and Foundation-Led Capital Reshape the Path to Cures Because patient populations are small, the traditional health care business model doesn't support investment in rare disease therapies. by Pamela Roberts, National Press Foundation The treatment of rare diseases has driven innovation beyond the lab – creative new funding models are advancing research and cures. Because patient populations are small, the traditional health care business model often does not support investment in rare disease therapies. In response, advocates, families and investors are developing alternative approaches — from crowdfunding to venture philanthropy — to move treatments forward. Rebecca Beattie, CEO of Launch Bio, Casey McPherson, founder and CEO of AlphaRose Therapeutics, and Teri Willey, managing director of Pathway to Cures, discussed the need for an evolution of these new strategies at the NPF Rare Disease Reporting Fellowship. “There’s a critical gap in how rare disease innovation gets funded,” Beattie said. Advances such as gene editing and AI-enabled diagnostics have made once untreatable diseases addressable, she noted, but funding models have failed to keep pace. “It’s more than a market problem, but more of a modern-day civil rights issue. Access to cures shouldn’t depend on where a child is born or whether a disease is commercially viable.” Beattie said they are looking for “new ways to fund hope.” McPherson’s work in regulation crowdfunding (an SEC rule that allows startups to raise funds through small investments from many people) began with his quest to find a cure for his daughter’s rare genetic condition, HNRNPH2-related neurodevelopmental disorder. He created a foundation and with the help of journalists began to share their story. “I was able to raise enough money in the foundation to create a pre-clinical treatment for my daughter due to many $100 donations, $1,000 donations, $20 donations from thousands of people all over the world that bought into Rose’s story.” ‘Benevolent investing’ Willey of Pathway to Cures works at the intersection of for-profit and not-for-profit organizations. Donor capital is invested in early-stage companies with the rigor of a traditional venture fund but is mission-driven; returns are reinvested to support further innovation and patient services. “I see this as a three-legged stool,” Willey explained. “You have the venture, the patient advocacy and the research. It’s a powerful triangulation of resources, particularly for rare disease. Just the finance or the science or the clinical or the law are not sufficient – they’re all critical but they’re not sufficient unless all those smart people play nice together, so one of our roles as a benevolent investing organization is to help facilitate that that cooperation.” Willey also stressed the importance of patient involvement. Rather than the traditional route of developing a drug first and then seeking patients for trials, Willey says engagement should begin at the preclinical stage. Involving patients earlier informs the science and leads to better products that fit the needs of the patient. “Engaging patients early is the right thing to do morally, but it is absolutely critical in bringing the right product to the patient faster, better.” Historically, medicines have been expensive to make and require massive investment to bring a drug to approval, often taking years before returning value to investors. McPherson said rare disease therapies present a different opportunity. “These drugs don’t cost a billion dollars to develop, he said. “They’re actually much cheaper to develop and they’re very high impact drugs. But it has to be outside the current pharma model.” “We have built an ecosystem, with an amoral lack of purpose for how and why we invest,” McPherson said. “Now we have the opportunity with startups that are working with patients, building up these treatments, and then connecting with [funders] who have a purpose with their capital because they want to make money, but they also want to affect patients. There’s this great convergence around these new opportunities. It changes our way of thinking, and it educates the investor.” Speakers: Rebecca Beattie, CEO, LaunchBio, Inc. Casey McPherson, Founder and CEO, AlphaRose Therapeutics Teri Willey, Managing Director and Officer, Pathway to Cures This video was produced within the Evelyn Y. Davis studios. NPF is solely responsible for the content.