- Legislation incentivizing the pharmaceutical industry to invest in therapeutics for rare diseases
- Advances in sequencing that made orphan drugs development faster and cheaper
- Overcoming the downside trend of abuse on the financial incentives for rare-disease drug development
- Avoiding backlash to the hefty price tags of these medications
Reserved. (TBC shortly)
- What are the challenges and how they can be addressed?
- Overcoming the issues of misdiagnosis
- Challenges facing clinicians who care for affected individuals
- Challenges to investigators regard the difficulty and expense of assembling large cohorts of affected individuals for study, and garnering funding for research
Moderator: Alison Schecter, Global Rare Diseases, External Opportunities, Sanofi Genzyme
Panelist: Radhika Tripuraneni, Vice President, Medical Affairs, Prothena Corporation Plc
- The key attributes of a successful orphan drug launch
- Examples of a successful orphan drug launch
Anthony J Arleth, Managing Director, Pennside Partners
- Understanding the global rare disease policy landscape
- New steps to meet the challenges of rare diseases
- What are the key elements across diverse rare disease programs and how this can affect patients access to care
- Bridging gaps between policy and practice to guarantee access for patients
- Trends in Orphan Diseases partnering deals
- Orphan diseases partnering agreement structure
- At which stage of the drug development does in-licensing occur and what is the reason for this trend?
- What criteria do partners look for when partnering for in and out-licensing?
Moderator: Brian Bronk, Head of External Innovation, Rare Diseases, Sanofi
- Case study: RVT-802 for the treatment of Complete DiGeorge Anomaly (cDGA)
- Overview of expedited regulatory pathways available for innovative rare disease therapeutics
- Managing multi-stakeholder collaboration including academia, patient advocacy, government, and industry
- Novel strategies to promote patient/market access
Alvin Shih, CEO, Enzyvant Therapeutics
• It would be desirable to be able to re-treat patients in life, but the formation of neutralizing antibodies against AAV gene therapy vectors prevents the ability to re-dose AAV
• We have developed synthetic vaccine particles encapsulating rapamycin (SVP-Rapamycin) that have been shown to mitigate immunogenicity against a variety of biologic drugs by inducing immune tolerance to the biologic
• We will present pre-clinical data demonstrating the ability of SVP-Rapamycin to inhibit the formation of anti-AAV antibodies and successfully enable repeat administration of gene therapy in animals and share data from an ongoing Phase 2 clinical trial of SVP- Rapamycin combined with a therapeutic enzyme
Kei Kishimoto, CSO, Selecta Biosciences
Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences
Understanding the epidemiology of the disease to streamline a diagnostic strategy
Justin Bingham, Vice President of Business Development, Centogene
- Efficiencies in developing well-characterized molecules in novel targets in rare diseases
- Speeding up the drug development process to rapidly deliver treatments to patients
- Diversifying a pipeline of potential treatments
David Cory, President and CEO, Eiger Pharmaceuticals
- High – throughput screening for drug repurposing
- “Basket” trials of drugs targeting shared molecularetiologies
- Gene therapy and gene editing
- Platform approaches to clinical trials
Philip J. Brooks, Program Director, Division of Clinical Innovation , NCATS, NIH
• Importance of disease insights to guide development
• Role of increasing access to genetic testing to aid early and accurate diagnosis
• How sponsored genetic testing programs can help patients and clinicians to position for long-term success
Katherine Stueland, Chief Commercial Officer , Invitae Nan Doyle, Senior Manager – Diagnostics , Alnylam Pharmaceuticals
- What innovative technologies in clinical trials on rare diseases are available to speed-up development?
- Limitations and challenges of these technologies
- Considerations for developing orphan drugs required to advance biomarker and endpoint use in clinical development
Senior Representative, Alexion
- Various aspects for clinical trials in rare diseases
- Innovations and flexibility required for appropriate clinical research programs
- Totality of clinical data and patient insights related to therapies for rare diseases
- How clinical research and regulatory review in rare diseases can be improved in the years ahead
Tim Miller, Co-Founder, President and Chief Scientific Officer, Abeona Therapeutics Inc.
- Patient recruitment and retention for a successful clinical trial in rare diseases
- How can you prepare healthcare providers to know about the therapy and identify the right patients for your therapy?
- How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
- What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?
Christian Girard, Co-Founder & CEO, Overlord Pharmaceuticals
Panelist: Leslie Leahy, VP, Medical Sciences, Ovid Therapeutics
Panelist: Luke Rosen, Head of Patient Engagement, Ovid Therapeutics
Panellist: Khrystal K. Davis, CEO and Patient Advocate, Zebra Leaf Publishing
Panellist: Mahesh Kolar, Vice President, Strategy and Business Development , Rarity Health
• Growing up with a fatal hereditary rare disease in my family and knowing I had a 50/50 chance of getting it
• The disease took the lives of 12 family members before we were given a name Hereditary Sensory Autonomic Neuropathy Type 1E (HSAN1E)
• It was through genetic testing from my brother and sister we were able to get a diagnosis.
• Caregiving for my brother and sister
• Deciding to get tested for HSAN1E, living with a negative result
• Starting a non-profit to find other families with HSAN1E
Rachelle Dixon, President / Co-Founder, HSAN1E Society
- Why must we ask why there is no treatment?
- How can we achieve the goal?
- The way forward
Richard A. Basile, Co Founder and CEO, BioPontis Alliance for Rare Diseases
- Review of drivers of pre-approval drug access and the “right to try”
- Overview of relevant regulation and legislation in the US
- Consideration of program guidelines in other countries
- Perspectives of key stakeholders: patients, families, advocates, regulators, media, payers, companies
- Implications for effective program planning
- Special considerations for rare and genetic diseases
- Best practices in program design, implementationm and evaluation
Mark Krueger , Chief Strategic Officer, MK&A Inc.
• Empowering patients (and their families) as the experts
• Understanding the patient journey - participate, listen and learn
• Building partnerships that address unmet needs
Why support must extend beyond our medicines – the role of industry in the community
Vikram Karnani, Executive Vice President, Chief Commercial Officer, Horizon Pharma
• What are more novel areas for collaboration? unmet
needs, diagnosis, access, ancillary support
• What are novel methods and models for collaboration?
• Which other stakeholders should be involved and how? • What’s the ideal we should strive for?
Jodie Gillon, Global Medical Lead, Patient Engagement Rare Diseases, Pfizer Inc.
Isabelle Lousada, CEO & President, Amyloidosis Research Consortium
• Trusted and purposeful relationships between patients and pharmaceutical companies drives patient resources and support programs
• CASE STUDY: Rarity’s 360° Patient Experience
Mahesh Kolar, Vice President, Strategy and Business Development, Rarity Health
• How can leading groups advance understanding and awareness of rare diseases by unifying patients
Moderator: Richard A. Basile, Co Founder and CEO, BioPontis Alliance for Rare Diseases
Panellist: Khrystal K. Davis, CEO and Patient Advocate, Zebra Leaf Publishing
Panellist: Michael A. Swit, Esq., FDA Legal Counsel, Law Offices of Michael A. Swit
Panellist: David Cory, President and CEO, Eiger BioPharmaceuticals
Panellist: Mahesh Kolar, Vice President, Strategy and Business Development, Rarity Health
- Current situation in Europe from a regulatory and a market access perspective
- Pricing and reimbursement: key success factors
- Case studies: stories of success and failure
- Strategic implications for biopharma companies, planning to expand into Europe
Matthias P. Schönermark, Managing Director, SKC
• Key logistics in providing early access
• From early to greater access
Stephan Toutain, SVP, Operations, Anavex Life Sciences
• Drug development is a team sport and it is critical to attract and retain top talent
• Collaboration across the organization is necessary for advancing programs effectively
Bob Ward, Chairman, Chief Executive Officer, Eloxx Pharmaceuticals
- Successful example of value-based pricing in Rare Disease
- Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
- Implementing measures designed to limit their exposure to costs associated with orphan drug reimbursement.
- Legislative incentives to encourage development of orphan rare diseases therapies
- Impact of orphan diseases treatment on healthcare payment system
Moderator: Christian Girard, Co-Founder & CEO, Overlord Pharmaceuticals
Panellist: Matthias P. Schönermark, Managing Director, SKC
Panellist: Stephan Toutain, SVP, Operations, Anavex Life Sciences
Panellist: Bob Ward, Chairman, Chief Executive Officer, Eloxx Pharmaceuticals
Panellist: Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures