We envision a world where people with rare diseases live well and live free.
Rare Medical Affairs provides consulting services for companies and organizations focused on rare diseases.
We focus on small and growing firms to help them solve the most challenging problems facing medical affairs teams.
RMA’s Rare Disease Patient-Centric Framework
Rare Medical Affairs is focused on helping companies in the rare disease space build, structure, change, and innovate their medical affairs function to better meet the needs of people with rare diseases.
RMA has developed this five part framework to inform the unique challenges facing medical teams working in the rare disease space.
Prevalence of Disease
It can be very challenging to determine an accurate prevalence of a rare disease. Frequently, over time, one finds the actual prevalence is higher than first thought.
The Diagnostic and Patient Journey
People with rare diseases experience a long and difficult journey through a maze of healthcare providers and institutions. In some cases, the diagnostic journey can last up to 10 years.
HCP Education
HCP education must focus on diagnosis and understanding of the disease course. Therefore, understanding the patient journey is foundational to informing an effective strategy.
Ongoing Evidence Generation
Generating long-term real world evidence, including patient-reported outcomes, is important in supporting the value proposition for rare disease medicines.
Genetics and Genomics
Variant types, pathogenicity, variants of unknown significance (VUS), new born screening, and genotype-phenotype relationships are important topics for understanding rare genetic diseases.
Let’s talk about how we can work together to improve outcomes for people living with rare diseases.
