Day 1, Tuesday, March 19th, 2024 - EST (Eastern Standard Time, GMT-5)
Day 1, Tuesday, March 19th, 2024 - EST (Eastern Standard Time, GMT-5)
- Gain insight on the shifting landscape, focused on insurance coverage and the regulatory environment
- Address marketplace policy disruptions and potential policy solutions to address accessibility and affordability
- Corey Ford, MHA - Vice President of Reimbursement and Policy Insights, Cencora
Trust and empathy are central to improving healthcare, yet systemic risk aversion and groupthink complacency impede everything. 91% of increasingly empowered consumers believe industry prioritizes profits over people, and just 16% trust healthcare communications. In this talk, Matthew sheds light on how we might innovate our way out of this by learning from CPG use cases, nonprofit communications, digital health solutions, shifting trends in consumer media consumption, shared accountability, and increased federal protections.
- Matthew Zachary - Principal, Matthew Zachary Worldwide
In this session, PhRMA’s Kevin Haninger will provide insights to PhRMA’s most recent Global Access to New Medicines Report, sharing the breakthroughs, strategies, and policies informing the future of health care accessibility.
- Kevin Haninger - Vice President of International Policy, PhRMA
- Kristin Smedley - CEO, Curing Retinal Blindness Foundation
- Gwen Hyland - Head, Global Commercial Excellence, Alexion, AstaZeneca Rare Disease
During this Case Study, hear from the CEO of Perseo Pharma, a start-up biopharma company, as he discusses the importance of partnering early and partnering often to accelerate commercialization.
|
- Yves Dudal, Ph.D - CEO, Perseo Pharma
In this session, engage in a comprehensive discussion and knowledge-sharing on the intricacies of rare disease valuation from an investor's perspective. Delve into the factors influencing risk assessment and return on investment considerations, strategies for making rapid go/no-go decisions, diverse funding models, and innovative approaches to mitigating risk in rare disease drug development. |
- Devin Rosenthal - Vice President, NovaQuest Capital Management
Hear real-world insights, including successes and struggles, and benchmark strategies on launching an ultra-rare product for small populations to ensure successful commercialization for current and future products.
|
- Grant Castor - Senior Vice President, Commercial Strategy & Operations, Sentynl Therapeutics
- Larry Bressler - Head, Global Rare Disease Value (Marketing) and Market Access, Chiesi
- Kitty Colletti - Vice President, Sales, Mirum Pharmaceuticals
At the heart of the rare disease industry lie the patients, who serve as the key motivator for the people working within this space. Mutualistic, respective and consistent engagement between industry and advocacy is essential in not only fostering a productive relationship but also in developing a product efficiently. Understand best practices in this multi-stakeholder engagement, including how industry should coordinate with advocacy as well as how advocacy can quantify their unmet needs in a compelling way. |
- Martine Zimmerman - SVP, Head of Global Regulatory affairs and R&D Quality SVP, Ipsen
- Amanda Moore - CEO, Angelman Syndrome Foundation
- Lilly Stairs - Founder and Board Chair, Patient Authentic and Autoimmune Association
In this compelling Patient Perspective Keynote, join Jonathan Cappiello as he shares his journey with 3-hydroxyacyl coa synthase deficiency, an exceptionally rare genetic disorder affecting just 20 individuals worldwide. Gain insight into Jonathan's inspiring diagnostic odyssey, tracing the challenges of his diagnosis and the transformative role his family played in advancing the Newborn Screening Act. Explore the dynamic evolution of his relationship with advocacy and the powerful concept that an illness requires visibility to be acknowledged as real.
- Jonathan Cappiello - Young Adult Rare Representative, EveryLife Foundation
Join us for an extraordinary Industry Trail Blazer Keynote session featuring two remarkable CEOs who are leading the way in the rare disease pharmaceutical landscape. Katie Landes, joined by Reenie McCarthy, CEO of Stealth BioTherapeutics, and Anish Bhatnagar, CEO of Soleno Therapeutics, will take center stage to share their inspiring stories, unveil their "whys" that drive their relentless pursuit of innovation, and provide invaluable insights into how they built their companies from the ground up |
- Katie Landes - Policy Advisor, Rare Disease Company Coaltion
- Reenie McCarthy - Chief Executive Officer, Stealth Biotherapeutics
- Anish Bhatnagar - CEO, Soleno Therapeutics