The lack of published literature in rare disease presents an impediment to progressing disease understanding and advancing therapies. Companies developing medical publishing programs need to consider that this is not your everyday medical publications program for a more common disease. As an example, to have a more tangible impact, rare disease publications must be open access and freely available to all stakeholders in rare, including the patient. Breaking down the process we will:
- Discuss the best methods, timelines, and content requirements for rare disease publications
- Highlight crucial differences between publishing for rare diseases and more common types
- Encourage that rare disease clinical trial results are shared with the patient community as a priority and in a legally compliant manner
The patient voice must move to the forefront of clinical trial planning, or else you are at risk of missed endpoints, low enrollment, and wasted time and money. Dedicated MSL teams can be powerful tools for gathering patient information and, in turn, properly structuring your trials. With their direct access to the patient voice, they are also vital in educating clinical staff about the disease.
- Reorient logistics, consent forms, and follow-ups around the patient voice
- Rely on medical affairs professionals to verify your assumptions about age spread and disease severity
- Strengthen advisory board meetings through medical affairs input
Commercial, medical affairs, and medical communications teams must be closely involved from the earliest stages in order to accurately guide sales. This is especially true for rare disease therapeutics for which doctors might see only one patient per month at the most. The entire clinical and medical affairs management cycles for rare disease will have to significantly change in response to the COVID-19 pandemic.
- Pinpoint the medical affairs and communication partnerships and messaging most essential in new APDS therapeutics
- Track the key steps in expanding rare disease therapeutics for a new pediatric indication
- Adapt your methods and messaging to a post-coronavirus world
- Create trust among stakeholders
- Envision innovative partnerships focused on solutions, not profits
- Prioritize transparency, accountability, and conflict resolution in partnerships
- Grasp how small investments can lead to big returns
- Create trust among stakeholders
- Envision innovative partnerships focused on solutions, not profits
- Prioritize transparency, accountability, and conflict resolution in partnerships
- Grasp how small investments can lead to big returns
- Actionable Implications to Achieve Clinical Development, Product Launch Goals,
Drug Safety and Favorable Outcomes - Building High-performing Multi-stakeholder Teams
- Leading Internal Project Teams Across Functions, Geographies and Therapeutic
Areas - Collaborating Across Organizations -- Science, Business and Non-profits
