Conference

PATIENT DATA

With so few patients, patient data/real-world-data (RWD/RWE) is the key element to advancing the rare disease space. What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access.

NEXT GENERATION THERAPIES

The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. What are the products and approaches that will be making headlines in 5-10 years from now?

PAYERS

US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. Hear from top US and international payers on their strategies for orphan drugs.

CLINICAL DEVELOPMENT & REGULATORY

Understanding the regulatory and clinical landscape for rare disease drug development. How can companies, regulators and patient advocates work together to further patient voice, real world evidence, and decentralized clinical trials.

COMMERCIAL

What are the best commercial strategies for orphan products? How can companies bring products to new markets, build more efficient commercial operations, and develop effective launch strategies?

RARE DISEASE ADVOCACY WORLD

It all starts and ends with patients. So what are the challenges facing rare disease patients and patient organizations today? How can rare disease patient groups rethink funding strategies, partnerships with industry and advance policies around access?

GLOBAL MARKET & PATIENT ACCESS

Access is often the crucial and challenges last step for rare disease patients and developers. How can all stakeholders work together to ensure rare disease patients have access to appropriate treatments globally.

PRICING & REIMBURSEMENT

Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world?

PITCH & PARTNER

The orphan drug space is booming with innovation, M&A activity and partnerships. We play match maker for up and coming biotechs in the rare space and investors & pharma partners looking for their next blockbuster.

ADVANCED THERAPIES - MANUFACTURING

What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production?

ADVANCED THERAPIES - CLINICAL DEVELOPMENT

The regulatory and clinical development landscape is ever evolving for cell and gene therapies. How are companies ensuring continued development of these potentially transformative products and what clinical hurdles remain?

RARE ONCOLOGY

As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients?

ADVANCED THERAPIES - COMMERCIALIZATION

Rare disease patients are uniquely positioned to benefit from the advancements of cell and gene therapies. What are the best strategies for commercializing these innovative products and how do health systems need to evolve to accommodate patient access and reimbursement?

DIGITAL HEALTH & ARTIFICIAL INTELLIGENCE

Approaches in AI and digital health span the full development life cycle of drug development and commercialization. What are the best approaches to implementing these technologies in patient finding, clinical development and commercialization for rare diseases?

DIAGNOSIS

It takes an average of seven years for an accurate diagnosis. How can all stakeholders broaden access to Newborn Screening, Whole Genome Sequencing and Genetic testing to shorten the diagnostic odyssey for rare disease patients?

DISCOVERY

Technologies and processes are ever. How is the growing access to data, and use of AI and machine learning impacting drug discovery in the rare disease space?