8:00 am
Registration & Light Breakfast
8:55 am
Chair’s Opening Remarks
Mastering the Art of Developing a Watertight Go-To-Market Strategy in Rare Disease to Create a Commercially Successful Therapeutic Launch
9:00 am Panel Discussion: Unlocking the Full Potential of The Commercial Team to Work Collaboratively on a Strategic Launch Plan to Create a Clear Streamlined Process with Value for All Stakeholders
Synopsis
- Discover how combinations of Market access, launch success, HEOR, RWE, CCO, commercial excellence, patient access, new product planning, pricing & reimbursement and more roles alike are working together as partners to improve commercial success.
10:00 am Strengthening Relationships & Co-Development Partnerships with Business Development and Research Teams to Create a Clear & Successful Commercial Strategy
Synopsis
- How to successfully work with research and business development teams to inform commercial strategy
- Importance of integrating commercial teams into the early stage of the life cycle of a drug
- Building cross-functional communication and collaboration channels to ensure alignment between research, business development, and commercial teams throughout the drug development process
10:30 am Rare Disease Commercialization: Tailoring Your Launch Excellence Strategies in Uncharted Waters
Synopsis
- Leveraging ideas from blueprint strategies while tailoring them to the unique needs of each rare disease
- Understanding how unique aspects of a treatment, patient experience and health systems may impact your commercialization plans and how we can adapt a basic strategy to each rare disease
- Key considerations in preparing for a launch in an uncharted market, where there are few (or no) precedents
11:00 am
Morning Break & Speed Networking
Synopsis
A prime chance to make the most of in-person networking and forge new connections with an expanding landscape of companies working on commercialization of rare disease therapies. Designed to maximize your introduction to numerous new individuals and serve as a catalyst for ongoing discussions during the summit, connect with peers and build relationships with those facing the same challenges as you.
Accelerating the Forecast of Commercial Viability for Small Patient Populations to Ensure Uptake, Viability & Improve Patient Access
12:00 pm Fireside chat: Creating The Optimum Target Product Profile to Fulfil the Needs of the Patient to Increase Uptake Ensuring Commercial Success
Synopsis
- Defining the ideal product profile based on patient needs, clinical outcomes, and market gaps for rare diseases
- Collaborating with clinicians, patient advocacy groups, and other stakeholders to ensure the product addresses key challenges in rare disease management
- Ensuring flexibility in the TPP to adapt to evolving treatment paradigms and regulatory changes in rare diseases
12:30 pm Understanding Resource Allocation Across the Commercialization Strategy & Across Multiple Assets
Synopsis
- How to develop a portfolio where you can extract as much value as possible without pricing your drug at inaccessible value
- How to identify which area of a commercial strategy to prioritize at which time
- How to prioritize commercial products in launching multiple new products and navigate multiple product launches
1:00 pm
Lunch & Networking
Developing Pricing Analogues & Creating a Strategy for Payer Approval in Rare Disease to Combat Necessary High Price Points
2:00 pm Overcoming the Challenges in the Development of Pricing Analogues for Diseases with No Current Standard of Care
Synopsis
- Understanding how small pricing deviations in rare diseases can significantly impact the overall business case, emphasizing the need for precise pricing assumptions
- Identifying relevant proxy indications and market data to build a pricing framework in the absence of a direct comparator
- Collaborating with key stakeholders, including payers and clinicians, to establish a valuebased rationale for pricing in uncharted disease areas
2:30 pm Enhancing Pricing Analogues with the Epidemiological Data to Justify Price Point Among a Small Population
Synopsis
- Using epidemiological data to refine pricing analogues and ensure the price point aligns with patient population size and unmet needs
- Leveraging historical pricing trends and real-world evidence to support pricing decisions for rare diseases while addressing payer concerns
- Demonstrating the long-term value of treatments through clear data on patient outcomes and cost-effectiveness, even for small patient populations
3:00 pm
Afternoon Break & Networking
Advancing the Use of Data from Diagnosis Rates Through to Data Accessibility in A Rare Disease Population, to Create a Foundation on Which You Build a Successful Commercial Strategy
3:30 pm Overcoming the Challenges Associated with Finding Data from a Newly Discovered Disease Area & In a Widely Under Researched Disease Area
Synopsis
- Identifying obstacles such as lack of established frameworks and data scarcity
- Developing novel data collection strategies, including patient-reported outcomes, natural history studies, and real-world evidence generation
- Building strategic partnerships with academic institutions, patient advocacy groups, and regulatory bodies to accelerate data gathering and validation
4:00 pm Fireside Chat: Ensuring Maximum Data Coverage to Create Confidence in the Identification of a Whole Disease Landscape Allowing for Maximum Uptake of Therapeutics
Synopsis
- Exploring how better patient identification translates to increased treatment adoption and commercialization success
- Integrating data from multiple sources—claims databases, EHRs, genetic testing, and patient registries- to capture a holistic view of the patient journey
- Utilizing comprehensive real-world evidence (RWE) and registry data to map the full disease burden and treatment landscape