Medical Affairs & Rare Disease

East Coast

November 4-6, 2020


Event Overview

The Accreditation Council for Medical Affairs and Dynamic Global Events are excited to announce the 3rd Annual Medical Affairs in Rare Diseases Forum. A CE/CME- accredited conference for medical affairs professionals in rare diseases.

Join us for the 3rd Annual Medical Affairs in Rare Diseases Forum to become a strong and collaborative partner who will drive medical strategy of the Launch and Brand Plan of orphan drugs. Learn to better ensure the execution of medical affairs programs, provide medical expertise, training and support to the cross-functional teams and work closely with the patient community.

The Inn at Penn, a Hilton Hotel


3600 Sansom St,
Philadelphia, PA 19104
(215) 222-0200

Hotel Website

Special Event Rate
Book Today!

About the Chairperson


Dr. Matthew T. Roe has been a faculty member at Duke University School of Medine and the Duke Clinical Research Institute (DCRI) since 1999. As a senior investigator at the DCRI, he has been the principal investigator for numerous phase II-IV cardiovascular clinical trials and is currently a co-principal investigator for the ADAPTABLE trial—the first large-scale pragmatic trials being conducted in the PCORnet network. He has also served in leadership roles for several observational registries focusing upon the treatment and outcomes of patients with cardiovascular disease and has served as the Director of the DCRI Clinical Research Fellowship since 2010.

Featured Speakers


Craig Lipset

Head of Clinical Innovation


Jeff Kingsley

Chief Executive Officer
IACT Health 


Dawn Anderson

Managing Director, Life Sciences Strategy and Operations, R&D Practice


Ross Friedberg

General Counsel & Chief Privacy Officer
Doctor on Demand

Top Reasons to Attend

  1. Discover how medical affairs teams are creating advanced strategies and achieving success in rare disease markets
  2. Optimize your medical affairs’ ability to understand patient needs and create patient-centric medical information
  3. Understand the regulatory pathway and nuances for the orphan drug approval process and the value of natural history studies
  4. Earn CME/CE credits
  5. Ensure that key insights/analyses, opportunities and challenges, strategic priorities are developed as part of a cross-functional team approach to maximize alignment

Attendee Profile

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


January 29th, 2020 | Day 1

8:00 AM - 9:00 AM

Registration & Continental Breakfast

9:00 AM - 9:15 AM

Day 1 Opening Remarks

9:15 AM - 10:00 AM

Rare Disease Literature – Rare & Different

The quantity of rare disease literature is disproportionally lower than that of other diseases.

In this keynote session, delegates will:

  • Evaluate the rare disease literature landscape
  • Examine the reasons why rare disease communications and publications are rare and different
  • Look at including patient advocates and caregivers in authorship teams
  • Explore why open access to information is important 

Dan Donovan - Founder
rareLife Solutions

10:00 AM - 10:15 AM

Morning Networking & Refreshment Break

10:15 AM - 11:00 AM

Medical Affairs in Rare Disease

In rare disease, the role and responsibilities of medical affairs are magnified.

This session will cover:

  • Evolution of the medical affairs function in rare disease
  • Magnified outward facing communication functions 
  • Increased internal corporate and clinical responsibilities

Ronald Walls, MD - US Head of Operations, & Business Development, Vice President Clinical Development Medical Affairs 
The CSPC Pharmaceutical Group

11:00 AM - 12:00 PM

PATIENT ADVOCATE PANEL: Open Access to Rare Disease Communications

Patient advocates will discuss current rare disease communications trends and forward-thinking recommendations for future initiatives with a focus on open access.

Panel will discuss:

  • Types of rare disease medical literature patients and caregivers are seeking 
  • Importance of reporting clinical trial results to patients in a timely manner
  • Enhancing knowledge transfer with plain language lay summaries
  • Looking forward: recommendations and suggestions for meeting patients and caregivers’ needs for rare disease medical publications and communications 

Panel Moderator:

Sheila BosePresident
SB LifeSciences Consulting, Inc.

Panelists (invited):

Susan Stein, MPH - Director, Patient Advocacy, Agios Pharmaceuticals
Director/Board Member, Global Genes

Dakota Fisher-VanceFounder, Young Adult Cancer Connection
Patient Advocacy Manager, Horizon Therapeutics

12:00 PM - 1:00 PM

Networking Lunch

1:00 PM - 1:45 PM

Industry Challenges to Accepting & Integrating Patients, Advocates & Caregivers (PACs) as Authors

Why have life sciences companies, heavily involved in rare disease medical publications and with a huge vested interest in PACs been slow to include them as authors?

This presentation will:

  • Explore the benefits and challenges of integrating PACs into authorship teams
  • Discuss PAC’s influences on empowering patients globally with plain language medical publications and communications
  • Evaluate PACs effects on pharma, biotech and medical equipment companies to address patients’ needs in medical publications

Peter Snyder - Sr. Director, Global Publications, Oncology Medical Affairs

1:45 PM - 2:30 PM

Non-profit Initiatives to Provide Plain Language Publications

In an effort to address needs for more rare disease publications, non-profit organizations are providing plain language publications and multi-media communications.

This session will address:

  • Rare disease information needs non-profits work to address
  • Specific diseases within rare that need more attention
  • Public response to non-profit rare disease publications
  • Multi-media rare disease communications (webinars, videos)
  • Pros and cons of patients relying on social media for information

Sika DunyohDirector or Education Programs

2:30 PM - 2:45 PM

Afternoon Networking & Refreshment Break

2:45 PM - 3:30 PM

Including the Patient Voice in Clinical Trial Results

Pharmaceutical companies in Europe submit patient diaries and experiences along with clinical trial results for new drug approvals. The FDA is calling for similar requirements in the USA.

This session will:

  • Explore the FDA’s initiatives to include patient experiences in new drug approval applications
  • Examine methods for enhancing clinical focused medical communications by adding patient pre-diagnosis personal data
  • Highlight the benefits of publishing patient diaries as easily understandable information for the lay community
  • Discuss alternatives for including patients in clinical trial design and informing patients as soon as clinical trial results are known

Susan Stein, MPH - Director, Patient Advocacy, Agios Pharmaceuticals
Director/Board Member, Global Genes

PANEL - Medical Publications & Global Medical Communications

A panel of experienced medical writers, publishers and communications directors will discuss the challenges to publish in rare disease.

3:30 PM - 4:15 PM

Rare Disease Publications & Communications by Life Stages & Age

Where are teenagers and young adults turning for medical information, emotional support, advice and plain language summaries of abstracts and clinical trial results?

Participants in this session will:

  • Discuss the what is missing in rare disease communications by life stages and age
  • Identify the kinds of medical literature patients and caregivers are looking for at different life stages
  • Explore the quantity and quality of rare disease medical publications segmented by life stages
  • Examine the role of of social media as a resource for rare disease information teens, young adults and adults

Dakota Fisher-VanceFounder
Patient Advocacy Manager

4:15 PM - 4:30 PM

Day 1 Closing Remarks


January 30th, 2020 | Day 2

8:00 AM - 9:00 AM

Networking Breakfast & Registration

9:00 AM - 9:15 AM

Day 2 Introduction

9:15 AM - 10:00 AM


Education, inspiration and actionable takeaways – this session will examine challenges in delivering rare disease presentations that are fit for the purposes they need to meet.

Topics include:

  • Challenges of presenting abstracts to mixed audiences -- medical professionals and patients
  • Differences between the delivery and perception of verbal presentations compared to printed publications
  • Current landscape and future trends
  • Best practices for conveying a message that is fit for the purpose the need to meet


Open Forum Q&A

10:00 AM - 10:15 AM

Morning Networking & Refreshment Break

10:15 AM - 11:15 AM

Transitioning from Small Biotech to Mid-size Pharma

For decades, small, scientific-minded companies have focused on rare diseases and orphan drugs. When a small company evolves into a mid-size one, what changes are needed to keep the company competitive?

In this session, participants will learn about:

  • Small biotech’s role in bringing orphan drugs to market
  • Impact of drug commercialization on internal processes and thinking
  • Necessary relationships between publishers and mid-size pharma to market drugs


11:15 AM - 12:00 PM

Knowledge Transfer Challenges in Rare Disease

Rare disease medical publishing faces unique challenges that other disease categories do not encounter. 

In this session, attendees will:

  • Discuss the challenges of small sample size and non-traditional clinical design on rare disease medical publishing 
  • Explore the current landscape and future trends of patient centricity in rare disease medical publishing
  • Identify solutions to expand open access to rare disease communications and publications

12:00 PM - 1:00 PM

Networking Lunch

1:00 PM - 1:45 PM

CASE STUDY – Helping Management Understand the Utility of Medical Affairs & Commercialization

Session topics will include:

  • Real life case study overview
  • Criticality of working with the patients and caregivers to inspire internal decision making
  • Lessons learned


1:45 PM - 2:30 PM

PANEL - Medical Publications & Global Medical Communications

A panel of experienced medical writers, publishers and communications directors will discuss the challenges to publish in rare disease.

2:30 PM - 2:45 PM

Afternoon Networking & Refreshment Break

2:45 PM - 3:30 PM

BRAINSTORMING SESSION for Medical Communications Rare Disease

This forum will provide a platform to:

  • Identify current rare disease information and ease of finding these resources
  • Explore options to increase the visibility, quality and quantity of rare disease communications in rare disease publications
  • Examine the trending landscapes for digital and social media communications

Dan Donovan - Founder

3:30 PM - 4:00 PM

Day 2 closing remarks



Ronald Walls

The CSPC Pharmaceutical Group

US Head of Operations, & Business Development, Vice President Clinical Development Medical Affairs


Dakota Fisher-Vance

Young Adult Cancer Connection

Founder (Also Patient Advocacy Manager, Horizon Therapeutics)


Dan Donovan






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Register 2 Attendees, Get 1 Complimentary Registration*!

Interested in sending groups of 7 or more? Contact us directly for pricing.



Contact Amy Chapman at (561) 571-7687
or achapman@dgeconfs.com




Why Become Board Certified in Medical Affairs?


Certification is becoming an important factor in the healthcare industry in general. A survey of over 1000 key opinion leaders (KOLs) found that over 90% preferred medical affairs professionals who had a Board Certified Medical Affairs Specialist (BCMAS) designation.

Several pharmaceutical companies also look at certification as a way to assess whether an individual possesses the skills and knowledge required for successful performance in a medical affairs role. Why are companies looking for this assurance? They want to hire the best of the best. By becoming board certified in your field of expertise, and by maintaining BCMAS, you are saying that you:

  • Have met standards that have been established and verified by a credible, accrediting body.
  • Are dedicated to maintaining quality of excellence in your profession.

These are powerful statements that speak volumes about you as a professional and the company or organization you represent. Board certification demonstrates that you are committed to your profession and that you want to continue to learn and grow.
Studies have shown that individuals who are certified in their field have higher salaries and better promotion opportunities.

Top 5 Reasons Why You Should Certify with the ACMA:

  1. Accredited: The ACMA is recognized nationally & internationally as a true mark of quality and excellence in the pharmaceutical industry for medical affairs professionals. The ACMA is accredited by the International Association for Continuing Education & Training (ANSI/IACET) which is the gold standard for certification & training around the world. Our program is administered and analyzed using the highest industry standards and state-of-the-art psychometric methodologies.
  2. Distinguish Yourself: Because ACMA offers multiple but related certificates, you can more readily become certified in more than one specialty, making you more valuable and marketable in the field.
  3. Go International: ACMA certification is widely accepted throughout the US as well as internationally with 25% of BCMAS graduates coming from outside the US.
  4. Expand Your Network: Once certified by ACMA you automatically become a member of the 75,000+ strong ACMA organization and gain access to a multitude of benefits, including discounts on continuing education, leadership opportunities, and access to a mentor to help you get ahead.
  5. Be on the cutting edge: ACMA board certification in medical affairs is the future of the life sciences and pharmaceutical industry.

Interested in BCMAS?

By attending any DGE/ACMA conference, receive 10% off of BCMAS registration.

Already BCMAS Certified? Receive 10% off your conference registration!