Conference Agenda

18 Orphan Drugs & Rare Diseases 2022 Americas - Day 2

Day 2: Wednesday - 30th November 2022


  • The foundational value of building collaboration through trust, respect, transparency, and empathy
  • Not all advocacy organizations are alike: assessing the landscape
  • Real-world examples of innovative digital solutions driving collaboration among multiple stakeholders
  • Value of partnering with advocacy organizations to create innovative patient-driven solutions


Jodie Sherman Gillon, SVP Corporate Affairs & Patient Advocacy, Peptilogics

Jennifer McNary, Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics

Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis

  • Attendees will come away with a better perspective of when, and how to engage patients, advocacy organizations and caregivers
  • Talk will cover the benefits of early and consistent engagement on recruitment, retention, regulatory and access issues.

Jennifer McNary, Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics

  • Connecting with patients and advocacy organizations early in development to influence directional decisions
  • Ideas on how to ensure the patient voice are heard in development strategy discussions
  • How patient input to clinical trial design will bring benefits to everyone involved.

Wendy Erler, Head of Patient Experience and Insights, Alexion Pharmaceuticals, Inc.

  • Creating patient-clinician relationships that create a sense of ease and trust
  • Addressing issues on practical challenges
  • Improving access and supporting patients
  • Multi-disciplinary approach and patient centered support to improve accessibility and further advances which will impact the next generation aiding patients of the future

Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation

  • Addressing the challenges associated with patient access to these medicines
  • How can patient access larger number of medicines in shorter time
  • Understanding how charities can work with manufacturer
  • Provide meaningful help
  • Impact of patient voice in rare disease drug development
  • Critical challenges to a successful rare disease drug development
    Advantages of incorporating patient voice to accelerate rare disease drug development
    Examples of patient led initiatives that regulators are looking for

Sonal Bhatia, Chief Medical Officer for Rare Disease, Pfizer

  • Education of the process of clinical trials( in easier terms so patients can understand)
  • Listen to the Patient needs and help them to understand what the focus of the trial is. (Break the stigma of Us vs.
    Them, Pointing of fingers)
  • Understanding Pharmaceutical costs of trials and research
  • Dealing with an illness with multiple organ involvement. (how multiple organ disease hinders a patient’s
    involvement in trials)
  • Rare Disease ( Limited pool for Trials, Patient distrust, Diversity in Patients)

Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis

  • 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
  • Legislative incentives to encourage development of orphan rare diseases therapies
  • Impact of orphan diseases treatment on healthcare payment system

Simu Thomas, Vice President & Global Head HEOR, Alexion, AstraZeneca Rare Disease

  • How do you generate RWD and evidence in support of your initiative to ensure meaningful results?
  • What data types and structure is needed in support of your research?
  • The important role of patients in evidence generation and how to keep them engaged?

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech. Evaluation, Independence Blue Cross


  • Creating opportunities for rare disease innovations through state and federal policy
  • The power of rare disease collaborations
  • Keeping patient voice prominent in policy and collaborations

Tara J. Britt, Founder and President, Rare Disease Innovations Institute, Inc.

  • What are the opportunities and challenges of orphan and rare diseases from market access standpoint
  • Innovative launch strategies and guidelines needed for optimal access
  • Evidence needed to secure patient access at the right time and right cost
  • Is there any benefit and derived from different early access programs
  • Real-world cases of what works and what doesn’t
  • Breaking down barriers for access
  • Strengthening health systems around the world and accelerating pathways to diagnosis
  • Stakeholders to address the rare diseases’ ecosystem gaps to collaboratively build a sustainable roadmap
    for better health and a brighter future for the patients suffering from rare diseases.
  • Challenges and opportunities in creating sustainable healthcare for all.



Christine Von Raesfeld, Founder/CEO, People with Empathy

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech. Evaluation, Independence Blue Cross

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