Agenda for Day 2 – Wednesday, March 24, 2027Wednesday, 24 March 2027 - ET (Eastern Time, GMT-05:00)

Move past traditional "why we should collaborate" discussions to explore how patient advocacy organizations and manufacturers can build measurable, value-driven partnerships using a structured approach that demonstrates real-world impact and organizational value

Examine practical applications of the Framework to establish meaningful KPIs, assess advocacy readiness and capability and create evidence-based strategies that resonate with both patient communities and internal stakeholders across therapeutic areas

Explore case studies of patient advocacy organizations and manufacturers who have successfully implemented the IMPACT™ Framework to co-create value, generate actionable data and insights and build sustainable collaborations that inform future strategic decision-making

Leave with concrete approaches to assess your current advocacy partnerships, identify gaps in measurement and impact, and develop a roadmap for elevating engagement maturity within your organization

Review a comprehensive analysis of the regulatory and policy environment surrounding China-US biotech collaboration, including recent restrictions, compliance requirements and areas where collaboration remains viable

Break down China's regulatory reforms and accelerated approval pathways for rare diseases

Evaluate the competitive dynamics, influences and opportunities presented by China's growing role in rare disease development, including implications for innovation, intellectual property and strategic competitor partnerships

Examine how the Inflation Reduction Act (IRA) and Most Favored Nation (MFN) policies and international reference pricing are fundamentally reshaping US market access strategies, payer negotiations and US pricing potential for rare disease products

Analyze how global pricing dynamics and policy changes in key international markets are directly impacting US payer coverage decisions, formulary positioning and reimbursement strategies

Explore how current and proposed tariffs are affecting US-based companies' global footprint decisions, including whether to expand internationally or consolidate resources domestically to protect US market positioning

Discover the importance of HEOR in both clinical and commercial development, and learn how to build comprehensive evidence stories that adapt to varying regional payer expectations

Understand strategies for overcoming data scarcity challenges in disease areas to build robust evidence packages that strengthen your evidence base during development and commercialization

Navigate the impact of MFN pricing policies on global launch sequencing and HEOR strategy, including how to structure evidence generation timelines, prioritize market entry decisions and manage pricing implications across geographies

Prepare for the EU Joint Clinical Assessment (JCA) framework by understanding how to align clinical evidence generation with JCA requirements, coordinate multi-stakeholder submissions and integrate JCA outcomes into broader European market access strategies

Explore best practices for developing flexible HEOR strategies that address diverse global requirements while maintaining evidence consistency and managing resource constraints

Gain insights from companies at different lifecycle stages (development, peri-launch and recently launched) and contrast market access strategies to learn best practices in navigating tight payer restrictions and optimizing early payer engagement

Deep dive into strategic approaches for patient support, distribution and specialty networks to counter strict utilization management and accelerate speed-to-treatment

Hear lessons learned from recent launches to best navigate modern reimbursement negotiations, policy changes and evidence generation required for high-cost rare therapies

Understand how to develop internal business education around the strategic shift from early to late-stage commercialization and how individual functions may need to evolve

Learn how to address new regulatory hurdles, post-approval obligations and real-world evidence requirements that emerge as products mature in the marketplace

Explore retrospective launch lessons learned, analyzing the speed of sales growth, effective competitive strategies and how different business models play out in the same market

Discuss lifecycle management strategies and label expansion opportunities

Recognize the evolving role of patient advocacy organizations throughout the product lifecycle and strategies for maintaining meaningful partnerships as commercialization matures

Uncover what hyper-personalization truly means in rare disease and how it differs from traditional patient segmentation, addressing the multidimensional complexities across patients, caregivers and physicians

Discover strategies for designing PSPs that address the full spectrum of ongoing challenges that rare disease patients face, with a focus on adherence, persistence and long-term engagement

Explore the decision framework for determining whether your hub should be mandatory

Understand the operational infrastructure for PSPs and establish best practices in managing vendor partnerships

Learn how to navigate conversations with executive teams to position PSPs as strategic assets, balancing patient-first outcomes with commercial objectives

Examine the paradigm shift from one disease/one treatment to competitive rare disease markets, including the evolving role of patient organizations in this landscape

Discover best practices for market and payer access strategies that facilitate speed to treatment and utilization, including patient support, distribution channels, specialty networks and external partnerships

Learn how to design patient engagement programs, generate differentiating data and build evidence stories that support product positioning in increasingly competitive environments

Evaluate current data from USC on how to value rare disease products, and how rare drugs can be better reimbursed at higher rates

Unpack the factors shaping the evolving pricing landscape, including the impact of FDA regulatory policy, payer pressures and reaction to IRA/MFN drug price negotiations

Learn the characterization of avenues for value-based purchasing concepts and a new approach to HTE assessment that would allow for greater flexibility and better chance at access

Address unique commercialization challenges for one-time curative therapies for very small patient populations

Discuss supply chain, manufacturing compliance and capacity constraints

Cover long-term patient monitoring and durability evidence generation

Highlight the access challenges along the patient journey