Orphan Drugs & Rare Diseases

Assuring Success from Regulatory to Global Market Access of Rare Disease Treatments

San Francisco • September 12 • 1:00-5:00

Committed to Improving Health Outcomes

At Mapi, we know that research centered on people is crucial to improving world health. Our unrivaled four decades of operational expertise in generating, synthesizing, and analyzing Real World Evidence and Health Economics Outcomes Research has established Mapi Group as the industry leader and recognized innovator of Outcomes Research methodologies and sciences.

Our research solutions bridge the distance between life science companies and patients, enabling market access and navigating the complex global regulatory environment.

Our commitment runs deep. Mapi Group is the only Health Research Company with a direct-funded non-proit organization dedicated to improving Patient Outcomes and Quality of Life. The Mapi Research Trust supports thousands of independent and academic research programs every year in over 130 countries, offering free and subsidized access to Clinical Outcomes Assessments (COA), their derivatives, and translations.

Selected Presenters

  • Dr. Will Maier
  • Rory Graham
  • Kelly Franchetti

Dr. Will Maier

Chief Scientific Officer, Head of Patient-Centered Sciences, Mapi Group

Mapi’s Chief Scientific Officer, Dr. Will Maier provides scientific leadership to Mapi’s research and consulting services across a range of scientific areas. Dr.Maier has more than 20 years of drug development and commercialization experience. He served as Senior Director of Epidemiology at GlaxoSmithKline and Elan Pharmaceuticals and led research groups conducting observational research to support reimbursement, marketing and drug safety investigations of pharmaceuticals. Dr. Maier is a member of the EMA’s European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.

Rory Graham

Senior Director, Regulatory Affairs, Europe & International Region, Mapi

Mr. Graham has facilitated numerous aspects of drug and medical device development during his 25 year career in the pharmaceutical, biotechnology and device industries. Mr. Graham has held senior positions in the area of regulatory a airs in companies in Europe and Asia-Pacific. He has arranged and directly participated in meetings with many global agencies including FDA, EMA, PMDA, TGA and European National Agencies. He has experience in various therapeutic areas including CNS, Oncology, Hematology, Immunology, Diabetes, Analgesia and Wound-Care. He has achieved orphan designations for pharmaceutical products in the EU, US and Australia. Mr. Graham leads a talented team of regulatory professionals that produce client-specific regulatory and development strategies, as well as solutions for global and local market needs.

Kelly Franchetti

Vice President, Global Patient Insights and Engagement, Mapi Group

Kelly Franchetti has nearly two decades of direct patient experience, gained in both hospital and industry settings including CROs, healthcare technology, patient research, and hands-on patient care across multiple therapeutic areas. Kelly Franchetti has been involved in the development and moderation of both advisory boards and patient, HCP and KOL forums. She also has experience collaborating with Patient and Clinical Advocacy groups. Her experience allows her to provide significant insights into how patients regard their treatment options and how to best approach patient recruitment and retention strategies.

The Agenda

SESSION 1: Partnering with Patients for Clinical Success
Presenters: Kelly Franchetti, Mapi Group

This interactive workshop will introduce participants to a 360° approach to obtaining patient insights and applying them for the successful design and execution of clinical studies. Through engaging activities, we will address how to get to the heart of the patient’s decision-making process, assess their physical/emotional/social needs, and identify their motivators, barriers and influencers as they relate to clinical study participation. Participants will then learn how insights are analysed and applied in order to:
• Design studies that appeal to the targeted patients
• Determine effective and motivating recruitment methods
• Engage and communicate with patients and caregivers
• Identify retention methods that meet the needs of a specific patient population
• Develop materials that resonate with patients and caregivers

SESSION 2: An Introduction to the Science of Endpoint Design in the Current Regulatory and HTA Environment in the US and Europe
Presenters: Rory Graham, Mapi Group

• Regulatory strategies for early scientific advice: FDA & EMA scientific advice, PRIME & adaptive pathways link to scientific advice
• The creation, categorization and hierarchy of endpoints and requirements of different stakeholder groups, including: regulatory agencies, clinicians, HTA bodies, payers, increasingly active patient groups
• Real-life examples of endpoints used in selected therapeutic area & techniques for the development and validation of new endpoints
• Highlighting guidance and publications that will be of use to newcomers to the area
• Solving the non-alignment between different agencies and geographical differences in approach

Networking Break
SESSION 3: Methodologies in Developing Real World Data: Engaging Stakeholders in Developing Evidence Strategies
Presenters: Dr. Will Maier, Mapi Group

• How is expanded access used to provide treatment for patients with rare diseases?
• Compassionate Use Programs in the window between submission and commercialization
• What are the bene ts of conducting clinical studies and registries for rare diseases?
• What are the types of studies conducted during different phases of orphan drug development?
• How are patient registries used to generate real-world data for rare diseases and orphan drugs?
• Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
• What are the unique challenges of conducting rare disease/orphan drug registries?
• How can proactive study management strategies successfully address these challenges?
• Potential risk mitigation strategies, and alternative registration strategies will be discussed

Question and Answer
Presenters: All

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