Planning Medical Education for Rare Diseases

By Sue McGuinness, PhD, CHCP

Dustin Ensign and Kathleen Moreo presented “A Rare CME Formula – Aligning Strategic Considerations for Orphan, Rare and Ultra-Rare Diseases” at the CBI annual meeting on Sept. 13, 2018.

There are 7,000 rare diseases currently known, impacting approximately 10 percent of the U.S. population. In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people. An additional challenge is that the literature shows it can take nearly a decade for a patient to obtain a confirmatory diagnosis in a rare disease, emphasizing under-recognition and unmet needs in awareness, screening practices and diagnostic barriers among the clinical community.

In independent medical education departments and across industry medical affairs organizations, several stakeholders within a supporter organization may be involved in rare disease education and strategic decision making. In this environment, it’s easy for misalignment to occur. Likewise, providers may not appreciate the importance of communicating and collaborating with multiple external stakeholders in the planning and execution of relevant and impactful education in rare diseases. In this CBI presentation, presenters Ensign and Moreo challenged supporters and educational providers alike to plan differently, partner effectively and design holistically to deliver improved healthcare and clinical outcomes for patients and families impacted by rare diseases.

Ensign discussed the importance of collaboration among the internal team and identified opportunities for IME staff to take a lead role in setting strategic initiatives to reach and educate a disparate and globally located mix of primary, treating and referring providers; clinicians; and the patient/family community. Considerations for the life cycle investment in education of a product were identified in the context of educational objectives and intended participants. Moreo gave examples of actual rare disease activities past and present and challenged attendees to consider whether and to what extent expected objectives and outcomes were met and could have been improved. The significant role of the patient/family in the rare disease community was emphasized, and examples were shared to provide a framework for how patients/families can best be incorporated into the educational program.

Often in rare diseases, resources are limited, and yet the educational needs are great. There may be a need to reach, cultivate and educate centers of excellence around the world and to host programs to encourage scientific exchange at select international meetings where the rare disease community assembles. At the same time, there is an acute need to train front-line, local community clinicians in evidence-based and emerging screening and referral processes. Patients whose lives depend upon timely and accurate diagnosis are often being seen in primary care or specialty settings where screening and referral practices are under- and unrecognized. Ensign and Moreo identified key implications for supporters and medical education providers to consider when planning, executing or evaluating a rare disease activity in the context of this dichotomy.

Through an application-based group exercise, audience members considered how to address an educational plan, selecting one of three rare diseases to tackle in a roundtable format. The exercise allowed supporters and providers to consider and explore ways that strategic goals can be met and to appreciate the viewpoints of different stakeholders in the process. The consensus among the group was that the process of planning medical education for rare diseases is as difficult as it is essential and requires a holistic team effort.

Editor’s Note: Dustin Ensign is Global Lead, Medical Education and Grants in Global Medical Affairs at Ultragenyx Pharmaceutical Inc. Kathleen Moreo is CEO of PRIME Education LLC.

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