First in a series of videotaped sessions at Boston Children’s Hospital’s recent Global Pediatric Innovation Summit + Awards 2014.
Inspiration for pediatric innovation is everywhere—from hackathons to waiting rooms to research labs—but getting from concept to clinic is a challenge. This panel discussion offers observations, insights and strategies for success in pediatric health, from drug development to caregiver support:
- Naomi Fried, PhD, Innovation Acceleration Program, Boston Children’s Hospital (moderator)
- Pamela Simpkins, MBA, strategic leader of operations and decisions support at Janssen Research & Development
- Henry Wei, MD, senior medical director of clinical innovation, Aetna
- Bill Geary, general partner, Foundation Medical Partners
- Dheeraj Batra, MBA, co-founder and vice-president of business development, Arogya Finance
Stay tuned as we post more sessions from the Pediatric Innovation Summit (also available on YouTube) and read our blog coverage.
Perhaps counter-intuitively, rare diseases can present attractive business opportunities for pharmaceutical companies. As discussed previously on Vector, they generally offer:
1) a population of patients with a high, unmet need, greatly lowering the bar to FDA approval
2) a closely networked disease community, greatly lowering the bar to creating disease registries and mounting clinical trials
3) well-studied disease pathways.
Recoiling from expensive failures of would-be blockbuster drugs, companies like Pfizer, Novartis, GlaxoSmithKline, Sanofi, Shire and Roche are embracing rare diseases, despite their small market sizes, because of their much clearer path to clinic. But in the current risk-averse industry environment, some projects are stalling. Industry may need more incentive to jump in—and Cydan Development is basing its business model on providing it. …
Early-stage researchers face a stark economic reality: decreasing available dollars. To address this barrier, a panel of experts at Boston Children’s Hospital’s National Pediatric Innovation Summit + Awards 2013 discussed the shifting of funding from venture capitalists to larger medical device, informatics and pharmaceutical companies.
Alan Crane, general partner at venture capital (VC) firm Polaris Partners, said it is a very difficult time for early-stage life sciences innovation, especially with respect to resource generation. “There were periods when innovation was stronger,” he said. “In 2000, there were 1,000 VC firms, now there are 350. Also, the size of the funds has decreased substantially, particularly for early-stage life sciences. While some biotech firms are going public, this trend hasn’t trickled to the earlier stages.”
To garner funding, Crane said, researchers need a proof of concept—for a clinical model and a business model. This proof of concept will vary based on the product or the nature of the innovation. …