ISPOR - Addressing Assessment and Access Issues for Rare Diseases

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Addressing Assessment and Access Issues for Rare Diseases

With the promise of more rare disease treatments on the horizon (particularly genetic treatments for hemophilia and sickle cell disease, which promise to be “one and done” therapies), their much higher per patient cost will create market access and other challenges to payers, providers, and patients.

ISSUE HIGHLIGHTS

From the Editor

Being Aware of Rare Disease

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ISPOR Speaks

Amplifying the Impact of HEOR Through Continued Advancement of Our Mission

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HEOR Articles

The Impact of Digital Health Technologies on Health Equity: Designing Research to Capture Patient-Reported Outcomes

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From the Regions

LMICs and ISPOR: A Member-Level View

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HEOR Articles

The Monitor Intervene Predict Value Framework: A Structured Approach to Demonstrating How Digital Health Can Improve Health Outcomes and Reduce Burden of Illness

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VOL. 9, NO. 1

January/February 2023

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COLUMNS

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HEOR Articles

The Role of Education in Shaping an Open-Source Future

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By the Numbers

HEOR & Rare Diseases

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From the Journals

A Data-Driven Approach to Support the Understanding and Improvement of Patients’ Journeys: A Case Study Using Electronic Health Records of an Emergency Department

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Research Roundup

Research Roundup

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HEOR News

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Events & Education

ISPOR Conferences and Events

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ISPOR Launches New HEOR Ambassadors Website

Sep 22, 2026

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