HEORStrategies consultants have extensive experience with rare, ultra-rare, and neglected disease. We have expertise in identifying individuals with rare diseases in multiple data sets, including electronic medical records, medical charts, registries, and claims data. We work closely with clients and clinical experts to develop algorithms, for identifying patients when disease-specific specific diagnoses are not available. We use rigorous methods and innovative study designs to help clients identify, describe, and understand the prevalence, costs, treatment patterns, and natural history of rare-disease patients.
HEORStrategies’ expertise includes a diverse portfolio of projects in more than 40 different indications. We have conducted natural history studies of rare diseases or rare genetic subsets and have a track record of research in rare-disease incidence and prevalence, natural history, costs and healthcare utilization, treatment patterns and outcomes. Our rare-disease capabilities span the product lifecycle, from early development to launch, marketing, and post-marketing. We have presented workshops, conducted presentations on methods in HEOR studies or rare disease, presented results to regulatory agencies, and published our findings in the scientific literature.
Core Rare-Disease Services:
- Estimate the incidence and prevalence of rare diseases using various strategies, such as querying publicly available databases, collecting and analyzing primary data, conducting systematic literature reviews, and performing complex dynamic modeling.
- Design and conduct burden-of-illness studies, characterizing healthcare resource use and quantifying the costs faced by payers and patients.
- Design and conduct natural history studies of rare-disease cohorts using electronic medical records (EMR), insurance claims data, and primary surveillance data.
- Identify and evaluate real-world external comparators and historical controls for clinical trials.
- Characterize the demographics, clinical characteristics, treatment patterns, outcomes (including potential adverse events), and healthcare utilization of rare-disease populations.
- Prepare epidemiology sections of regulatory submissions such as Orphan Drug Applications for the Food and Drug Administration (FDA), European Medicines Agency (EMA), and United Kingdom Medicine and Healthcare products Regulatory Agency (UK MHRA).
- Present epidemiologic methods and study results at government agency meetings and at scientific meetings and congresses.
- Prepare abstract submissions, scientific poster presentations, and manuscripts for peer-reviewed publications.
- Provide epidemiological and statistical support throughout the orphan product pipeline.