2019 Medical Affairs
for Rare Disease Forum

July 22-24, 2019 | Philadelphia, PA

Sheraton Philadelphia University City

Event Overview

The Accreditation Council for Medical Affairs and Dynamic Global Events are excited to announce a partnership that brings a CE/CME-accredited conference experience to medical affairs professionals specializing in rare diseases. 

As advances in genomic and clinical science have created innovative opportunities to design personalized medicine, the pharmaceutical industry must work to reconstruct their business models to combine market opportunities with clinical health benefits. Join us at the Medical Affairs in Rare Diseases Forum to learn from peers and disruptive newcomers about the role Medical Affairs plays in precision medicine.

Featured Session Topics:

  • Analyzing Medical Affairs’ Role in Orphan Drug Development
  • Working with Patient Advocacy Groups: Best Practices
  • What Regulators are Looking for from Medical Affairs in Rare Diseases
  • Skills & Development Training for Medical Affairs Professionals in Rare Diseases
  • Assessing the Orphan Drug Market: What’s Coming Next?
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Sheraton Philadelphia University City

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3549 Chestnut St
Philadelphia, PA 19104

(215) 387-8000
Hotel Website

Special Event Rate
Book Now!

Presentation by the FDA:

What Regulators Look for from Medical Affairs in Rare Diseases

Plus, join us for the Women’s Leadership in Medical Affairs Breakfast on Day One providing the unique opportunity to build your professional network and engage with peers and colleagues.

Top Reasons to Attend

  • Earn CME/CE credits at the first-ever accredited event brought to you by the Accreditation Council for Medical Affairs (ACMA) and Dynamic Global Events (DGE)
  • Discover how medical affairs teams are creating advanced strategies and achieving success in rare disease markets
  • Optimize your medical affairs’ ability to understand patient needs and create patient-centric medical information
  • Understand the regulatory pathway and nuances for the orphan drug approval process and the value of natural history studies

Who Should Attend

This Conference is Designed for Life Science Professionals responsible for:

  • Medical Affairs
  • Medical Engagement
  • Product Development
  • Medical Science Liaison
  • Medical Information
  • Medical Communication
  • Scientific Communications
  • Rare Disease
  • Patient Engagement
  • Field Medical Teams
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Featured Co-Chairs

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Jaime Arvizu, MD, MPH is a senior executive pharmaceutical physician and Vice President of Medical Affairs with 27+ years of comprehensive global leadership experience. He has been instrumental building, leading and developing Medical Affairs (MA) teams across the world, with growth strategies which lay the foundation for patient-centered, operational excellence at country, region and global levels. Jaime is passionate about transforming the standards of medical care and leaving a legacy of better living for patients with rare and debilitating disease.

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Dr. Anne Arvizu, PharmD, FASCP, CPC, is founder and CEO of RxER Communications, a top consulting firm specializing in Global Medical Affairs (GMA) Shared Services capabilities build. With over 22 years of executive leadership experience in the pharma/biotech industry, she is globally recognized as the go-to expert for GMA operational launch readiness and effective organizational design. Notable career highlights include oversight and subject matter expertise of mission critical, compliance-driven roles such as Global Medical Information, Medical Communications, Publications Strategy, Knowledge Management, Scientific Exchange/MLRC Review, Medical Governance and Field Medical Team Launch Support.

Download the Full Agenda

Monday, July 22nd, 2019 | Workshop Day

 

11:00 AM - 12:00 PM

Registration & Networking Lunch

12:00 PM - 12:30 PM

Opening Interactive Exercises

  • Define your objectives and what you hope to achieve
  • Meet with patients & advocates

Terri Ellsworth - Director, Professional Relations, Patient Advocacy
Accreditation Council for Medical Affairs (ACMA)

Caryl HarrisPatient Ambassador
Illumina
Founder & Executive Director
Avery's Hope

Nadia Bodkin - CEO
Blaze Therapeutics

Nina Daya Patient Ambassador
Illumina
Patient and Family Advisory Council Member (PFAC)
Sutter

Seth Fritts - Corporate Engagement
Global Genes

Stephanie Fischer - Executive Committee
Rare Advocacy Movement

12:30 PM - 2:00 PM

Part 1: How to Leverage the Power of Patients with Medical Affairs and Patient Advocacy

Working in rare diseases, Medical Affairs and Patient Advocacy can work together to leverage the power of the rare disease patient themselves.  Developing ways to listen to the patient will provide a company the opportunity to develop its patient engagement strategy to effectively achieve all participant’s goals.  This session will: 

  • Discuss tactics to engage the patient community
  • Provide a case study in which Medical Affairs and Patient 
  • Advocacy worked together to achieve patient enrollment in a rare disease clinical study
  • Discuss a case study of supporting patients to implement a rare disease patient organization

 

Seth FrittsCorporate Engagement
Global
 
Genes

Amy GroverDirector of Patient Advocacy
Catalyst Pharmaceuticals

David Caponera Head of Patient Advocacy and Patient Services
Dicerna Pharmaceuticals, Inc.

2:00 PM - 2:45 PM

Part 2: Learning Outside of Rare: Discuss How to Design Protocol with Advocates and Create Patient Advisors and Partners

  • Discuss how to compose protocols that involve patients at the beginning of drug development 
  • Learn to build an experience that considers the needs of the patient
  • Examine the changing role of patient advocacy and review how to empower patient advocates 

Helena Chung - Global Patient Reported Outcomes Scientist for Oncology
AstraZeneca

2:45 PM - 3:30 PM

Networking & Refreshment Break

3:30 PM - 3:45 PM

Part 3:  Discuss the Challenges of Patient Access

  • Learn from a case study - Our Rare Journey
  • Discuss cost and coverage
  • Readiness - Patient / Provider
  • Awareness - Patient / Provider

Nina Daya Patient Ambassador
Illumina
Patient and Family Advisory Council Member (PFAC)
Sutter

3:45 PM - 4:30 PM

Part 4:  Public Policy for Industry

  • Discuss why public policy matter and how it can make a difference
  • Learn to engage with patient advocacy organizations on public policy
  • Examine how to Enhance the FDA’s external communication platforms to expand public awareness and help patients, caregivers, and advocates navigate the FDA engagement activities and regulatory processes
  • How to engage with the NIH and stay informed

Stephanie Fischer - Executive Committee
Rare Advocacy Movement

4:30 PM - 5:00 PM

Reflection & Action Plans, Q&A, Closing Remarks

Moderator:
Meredith Cagle Senior Director, Patient Engagement
Global
 
Genes

Terri Ellsworth - Director, Professional Relations, Patient Advocacy
Accreditation Council for Medical Affairs (ACMA)

Caryl Harris - Patient Ambassador
Illumina
Founder & Executive Director
Avery's Hope

Nadia Bodkin - CEO
Blaze Therapeutics

Nina Daya Patient Ambassador
Illumina
Patient and Family Advisory Council Member (PFAC)
Sutter

Seth Fritts - Corporate Engagement
Global Genes

Stephanie FischerExecutive Committee
Rare Advocacy Movement


Tuesday, July 23rd, 2019 | Day 1

 

8:00 AM - 9:00 AM

Registration & Continental Breakfast 

9:00 AM - 9:15 AM

Chairperson’s Opening Remarks 

Jaime Arvizu, MD, MPH - Vice President, Head Medical Affairs, LATAM
Ultragenyx

Anne Arvizu, PharmD, FASCP,  PCC - CEO and Medical Affairs Advisor
RxER Communications Corp

9:15 AM - 10:00 AM

Keynote Address:  Medical Affairs’ Role in Rare Disease Drug Development

As accessibility to medical information has exploded over the last 20 years and moved online, it has changed the way that researchers, clinicians and patients receive information and has had an important influence in the collective decision-making process in what treatment a patient will receive. The most important function across the biopharmaceutical industry in facilitating accurate and balanced information about the treatment choices available is the Medical Affairs function. This is even more evident in the area of Orphan Drugs and Rare Disease where there are often limited FDA-Approved treatment options, an emergence of new investigational drugs and genetic technologies, and often a greater engagement among patients, patient advocacy organizations and disease foundations.

  • How to employ the optimal Medical Affairs strategy to serve effectively in this complex new environment in serving the research, clinical and patient communities with the information they need
  • How to meet the increasing demand for information from medical conferences on a real-time basis for both FDA-approved drugs and investigational drugs in development
  • How to use the Medical Affairs function to guide drug development and increase the probability of success for regulatory approval and to create best-in-class therapies for patients.

Chris GarabedianChairman and CEO
Xontogeny 
Former CEO
Sarepta Therapeutics

10:00 AM - 10:30 AM

Patient Engagement in Drug Development

  • Discuss why it’s imperative for doctors to empathize with patients to improve outcomes
  • Working together to advance treatments and medical care in the rare disease space while keeping the patient first
  • Consider the impact of every touchpoint to fully comprehend the patient journey and learn to understand the complexities of a rare disease

Dr. Anita Gupta -  Senior Vice President, Medical Strategy & Government Affairs
Heron Therapeutics, Inc.

10:30 AM - 11:00 AM

Networking & Refreshment Break

11:00 AM - 12:00 PM

Working with Patient Advocacy Groups: Best Practices 

In this session, we will learn how to optimize medical affairs’ ability to understand patient needs and create patientcentric medical information. We will explore methods for working with patient advocacy groups to engage patients, understanding their needs, and encouraging patient-centered views and developments.

  • Understand what patient advocacy groups want from medical affairs
  • Describe best practices for working with patient advocates
  • Learn about where the patient advocacy space is heading in the next decade

Susan Walther, MS, CGC - Director of Patient Engagement
Friedreich's Ataxia Research Alliance (FARA)

Seth Fritts - Corporate Engagement
Global Genes

Terri Ellsworth - Director of Professional Relations, Patient Advocacy
Accreditation Council for Medical Affairs (ACMA)

12:00 PM - 1:00 PM

Networking Lunch

1:00 PM - 1:45 PM

Creating an Expedited Pathway for Industry to Collaborate with Patients

Nadia Bodkin - CEO
Blaze Therapeutics

1:45 PM - 2:15 PM

What Regulators Look for from Medical Affairs in Rare Diseases

There have been notable regulatory actions taken in the rare disease space. However, specific guidance for orphan drug development is still limited compared to traditional drug development. Discuss what tools the FDA offers to promote and advance orphan drug licensing and how to work with regulators throughout the lifecycle of the drug.

  • Learn about how to effectively engage with regulators focusing on rare diseases
  • Discover the value of natural history studies and their impact on the regulatory pathway
  • Discuss the differences between FDA vs EMA and other regulatory agencies on orphan drug approval

Melanie BlankMedical Officer
Food and Drug Administration  

2:15 PM - 3:00 PM

Collaborating Around Rare Disease

  • Need for innovation
  • W3 background/demo video
  • 3 rare disease case studies of clients who innovated

Jon Kaskey - Senior Director of Business Partnerships
Within3

3:00 PM - 3:15 PM

Networking & Refreshment Break

3:15 PM - 4:15 PM

Set-Up Your Organization for Success: Medical Affairs Departmental Structure in Rare Disease Organizations 

Examine the impact that rare disease has on medical affairs departments and consider the best approach for developing an organizational strategy that aligns with the changing standard in the pharmaceutical industry to personalized medicine.

  • Examine successful organizational models for medical affairs in rare diseases
  • Learn the impact of key roles such as patient diagnosis, patient diagnostics and medical affairs shared services
  • Compare & contrast biotech vs. small to mid-size vs. large pharma organizations in rare diseases

Jaime Arvizu, MD, MPHVice President, Head Medical Affairs, LATAM
Ultragenyx  

Anne Arvizu PharmD, FASCP,  PCC - CEO and Medical Affairs Advisor
RxER Communications Corp

Len Valentino, MD, FAAP VP, Strategy Lead, Hematology
Spark Therapeutics   

4:15 PM - 5:00 PM

Explore Gene Therapy for Rare Disease

  • Discuss the differences of rare disease gene therapy vs. biological or small molecule development
  • Examine the contribution of medical affairs pre and post approval

Paul Korner, M.D., M.B.A. - SVP, Clinical Development & Medical Affairs,
Axovant Sciences

5:00 PM - 5:15 PM

Day One Closing Remarks

Jaime Arvizu, MD, MPH-  Vice President, Head Medical Affairs, LATAM
Ultragenyx

Anne Arvizu, PharmD, FASCP,  PCC - CEO and Medical Affairs Advisor
RxER Communications Corp


Wednesday, July 24th, 2019 | Day 2

 

8:00 AM - 9:00 AM

Networking & Breakfast

9:00 AM - 9:15 AM

Chairperson’s Recap of Day One

Jaime Arvizu, MD, MPH, Vice President, Head Medical Affairs, LATAM
Ultragenyx

Anne Arvizu, PharmD, FASCP,  PCC - CEO and Medical Affairs Advisor
RxER Communications Corp

9:15 AM - 10:00 AM

Medical Affairs Role in Diagnostic Journey of Rare Diseases. Newborn Screening Initiatives with a Structured and Transparent Approach

Develop clear processes and methods to collect and integrate insights from different stakeholders. Leverage these insights by collaborating with R&D and commercial to increase productivity and expedite orphan drug approval and commercialization.

  • Newborn screening (NBS) programs-historical and current overview
  • Rare diseases – NBS screening programs
  • Status of Newborn Screening Programs and Financing  in the United States
  • MPS II (Hunter Syndrome) NBS program at Takeda  - as an example of the cross-functional & multiple stakeholders  collaborative project

Uzma Atif PhD, MPH - Senior Medical Science Liaison, US Rare Disease (LSD) US Medical Affairs, Global R&D 
Takeda  

Ekaterina Wright - Global Medical Lead Rare Diseases, Medical Affairs
Takeda 

10:00 AM - 10:30 AM

Advocacy Partnerships and Making it a Win-Win Collaboration

Review best practices in working with advocacy groups and discuss the role patient advocacy groups can play across the research continuum and how this input can be a value add to the medial affairs team.

  • Review and discuss the use of advocacy groups in understanding factors in the real world setting outside of clinical trials that can help better understand a disease and develop drugs more effectively.
  • Understand factors affecting the patient outside of the clinical trial setting to better understand the disease, patient and caregiver
  • Use RWE to understand the disease outside of the clinical trial. Examine how information from RWE helps to understand the patient needs and ensure that information collected in drug development, throughout the research continuum.
  • Discuss how the patient “voice” can be factored-in and leveraged in improving protocol design; engaging the right patients at the right time and effectively communicating key information during a study to maximize overall study success

Shazia Ahmad - Director, Patient & Physician Services
UBC 

10:30 AM - 11:15 AM

Rare Is Rare: How Many Zebras Are There, and Where Are They Hiding in the Medical Literature?

Rare diseases are an integral part of the medical landscape. By the very nature of their limited prevalence, very little is known about the majority of rare diseases. Consequently, whatever is known needs to be of high quality, easily found, and freely accessible to healthcare providers to enable them to manage the disease in their patients. Just as important, quality information needs to be freely accessible to patients, advocates, and caregivers (PACs) so that they can be well informed and participate fully in their treatment regimens.

  • Understand the prevalence of rare communications in the medical literature database
  • Explore where rare manuscripts are published
  • Take a deeper dive into who has access to the articles, article type, and the acknowledged involvement of PACs, including as authors.

Dan Donovan - Co-Founder & CEO
rareLife solutions 

11:15 AM - 11:30 AM

Networking & Refreshment Break

11:30 AM - 12:15 PM

How Does One Find Key Opinion Leaders for a Rare Disease?

As pipelines continue to shift focus to rare diseases, it is crucial to understand and engage thought leaders to support scientific exchange. Medical affairs teams will need to have a more organized strategy when planning for KOL engagements.

  •  Identify Key Opinion Leaders and Thought Leader Engagement is a core responsibly for Medical Affairs teams
  • Discuss the significant differences in how this should be done for rare diseases compared to other larger more well-known diseases
  • During this session examine what those differences are and how to optimize the process for the rare disease market

Ariel Katz - Partner and Co-Founder
H1 INSIGHTS

Ann Leon Director, Immunology Medical Therapeutic Area
CSL Behring

12:15 PM - 1:00 PM

Skills & Development Training for Medical Affairs Professionals in Rare Diseases

Training medical affairs specialists in rare diseases can be challenging. Finding and developing leaders that can be leveraged for sustained success will ensure your organization will be successfully operating in rare diseases.

  • Learn about the necessary skills needed to prepare your medical affairs organization to be highly functioning in rare diseases
  • Discuss the core areas of training needed for medical affairs professionals working in rare diseases
  • Understand the value of broad skills training for medical affairs professionals in rare diseases

Diana Stefani-Hunyady, MD, MBA, BCMAS  Head of Global Medical Affairs
Viela Bio

1:00 PM - 2:00 PM

Networking Lunch

2:00 PM - 2:15 PM

Chairperson’s Closing Remarks

Jaime Arvizu, MD, MPH - Vice President, Head Medical Affairs, LATAM
Ultragenyx

Anne Arvizu, PharmD, FASCP,  PCC - CEO and Medical Affairs Advisor
RxER Communications Corp


Download the Full Agenda

FEATURING

Speakers & Faculty

Jaime Arvizu

Jaime Arvizu

Vice President, Head Medical Affairs, LATAM 
Ultragenyx

Ann Leon

Anne Arvizu

CEO and Medical Affairs Advisor
RXER Communications

Paul Korner

Paul Korner

SVP, Clinical Development & Medical Affairs
Axovant Sciences

Seth Fritts

Seth Fritts

Corporate Engagement
Global Genes

Shazia Ahmad

Shazia Ahmad

Director, Patient & Physician Services
UBC

Chris Garabedian

Chris Garabedian

Chairman and CEO
Xontogeny 

Helena Chung

Helena Chung

Patient Centricity Excellence Director
AstraZeneca

Nadia Bodkin

Nadia Bodkin

CEO
Blaze Therapeutics

Dan Donovan

Dan Donovan

Co-Founder & CEO
rareLife Solutions

Susan Walther

Susan Walther

Director of Patient Engagement
Friedreich’s Ataxia Research Alliance (FARA)

Terri Ellsworth

Terri Ellsworth

Director Professional Relations, Patient Advocacy
Accreditation Council for Medical Affairs (ACMA)

Jon Kaskey

Jon Kaskey

Senior Director, Business Partnerships
Within3

David Caponera

David Caponera

Head of Patient Advocacy and Patient Services
Dicerna Pharmaceuticals, Inc.

Ariel Katz

Ariel Katz

Partner and Co-Founder
H1 INSIGHTS

Melanie Blank

Melanie Blank

Medical Officer
Food and Drug Administration

Ekaterina Wright

Ekaterina Wright

Global Medical Lead Rare Diseases, Medical Affairs
Takeda

Ann Leon

Ann Leon

Director, Immunology Medical Therapeutic Area
CSL Behring

Caryl Harris

Caryl Harris

Patient Ambassador, Illumina, Founder & Executive Director
Avery’s Hope

Anita Gupta

Anita Gupta

Senior Vice President, Medical Strategy & Government Affairs
Heron Therapeutics, Inc.

Nina Daya

Nina Daya

Patient Ambassador, Illumina
Patient And Family Advisory Council Member (PFAC)
Sutter

Stephanie Fischer

Stephanie Fischer

Executive Committee
RareAdvocacy Movement

Meg Heim

Meg Heim

Vice President, Head of NA Medical Affairs Scientific Communications, CV and Diabetes
Sanofi

Alexis Skoufalos

Alexis Skoufalos

Associate Dean for Strategic Development
Jefferson College of Population Health

Amy Grover

Amy Grover

Director of Patient Advocacy and Engagement
Catalyst Pharma

Len Valentino

Len Valentino

VP, Strategy Lead, Hematology
Spark Therapeutics

Diana Stefani-Hunyady

Diana Stefani-Hunyady

Head of Global Medical Affairs
Viela Bio

 
 

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Continuing Education

All CME/CE is provided by ACMA in conjunction with ScientiaCME.
For more information or questions, please email info@medicalaffairsspecialist.org.

The ACMA is accredited by the International Association of Continuing Education & Training (ANSI/IACET), which is a badge of excellence & distinction indicating that the ACMA maintains the highest standards of quality & excellence in medical affairs and MSL training.

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Please contact Amy Chapman at (561) 571-7687 or achapman@dgevents.com