FTI Center for Healthcare Economics and Policy’s Jason Shafrin, PhD, Nadine Zawadzki, PhD, Shurui Zhang, MS, and Dhruv Dalal, recently published an article using a 4-step systematic approach for identifying disease analogs to develop and apply to a rare disease, pulmonary arterial hypertension (PAH). The article was published in Journal of Managed Care & Specialty Pharmacy and is titled “A Structured Approach for Identifying Disease Analogs for Pulmonary Arterial Hypertension.” The study abstract is below.
Abstract
Drug manufacturers often use disease analogs to describe diseases—particularly rare diseases—to payers, policymakers, and stakeholders. However, these comparisons are typically anecdotal. We propose a 4-step, systematic approach to identify disease analogs based on prespecified metrics and apply it to a rare disease, pulmonary arterial hypertension (PAH), as a case study. When there is limited knowledge of a rare disease, such as PAH, the application of the systematic disease analog approach presented can help managed care pharmacists leverage their existing knowledge and perspectives applied to other conditions to inform coverage, tier placement, and pricing decisions.
Methodology
A 4-step systematic approach for identifying disease analogs was developed and applied to a rare disease, pulmonary arterial hypertension (PAH), as a case study. After applying several selection criteria, 17 diseases were identified sharing key similarities to PAH. Among them, cystic fibrosis, neuromyelitis optica spectrum disorder, and rheumatoid arthritis were among the closest matches. Comparing PAH with these similar diseases can help health care professionals understand its severity, raise awareness, and inform decisions to improve care.
Conclusion
Through a systematic approach, this process identified disease analogs for PAH, most of which were classified as neurological conditions with notable functional limitations. Specific potential analogs highlighted included cystic fibrosis, NMSOD, rheumatic arthritis, LGS, and myasthenia gravis. This structured framework can aid in the communication of relevant disease aspects of PAH through empirically determined similarities to other diseases to raise awareness and increase advocacy to improve outcomes in PAH. Although this article focused on PAH, the structured approach presented can serve as a framework for health care stakeholders to systematically identify disease analogs across other diseases as well.
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