Value Viewpoint: January 31, 2025
Kimberly Westrich
NPC Chief Strategy Officer | Kimpossibility Life Coach & Yoga Teacher | Biopharmaceutical & Health Policy Researcher | Public Speaker
On Tuesday this week, NPC published its first two Policy & Evidence Briefs – a new series of reports that feature original research and analysis to provide decision-makers with timely evidence and data.
Check out the first two briefs here:
Two new journal pre-proof articles were published this week in Value in Health on access barriers to rare disease treatments and social return on investment:
1.???? In Rare Disease Policy in High-Income Countries: An Overview of Achievements, Challenges, and Solutions, the authors examine the historical access barriers to rare disease treatments faced by patients in high-income countries, including the EU, UK, US, Canada, and Japan.
Authors found progress had been made to address rare disease treatment barriers through interventions such as orphan drug development incentives, public-private partnerships, and patient registries to support research and clinical trials. However, many challenges still remain including high therapy costs, limited access to ultra-rare disease innovation, diagnostic delays, and disparities across regions. Authors conclude,
“To move forward, it is crucial to develop sustainable pricing and reimbursement models, enhance global collaboration across public and private sectors, and leverage emerging technologies to improve diagnosis, data collection, and medicine development.”
2.???? In Social Return on Investment analysis: A Mixed Methods Approach to Assessing the Value of Adult Hospice Services (paywalled) study authors used a Social Return on Investment (SROI) framework to assess the social value generated from hospice sites in the UK. They utilized a mixed-methods approach to capture stakeholder experiences and determine the broader social impact of treatment.
Study results showed that the social “output” value generated from both inpatient care and therapy services were significantly higher than “input” values, demonstrating the high return on investment. Study authors conclude,
“Embracing such [value-driven] methodologies fosters a more comprehensive understanding of value, transcending traditional metrics to encompass social, environmental, and long-term sustainability considerations.”
??? Eye on ICER
A calendar of ICER’s upcoming reports & meetings:
Policy White Papers/Special Assessments:
- 3/4/25: Special Assessment to Inform CMS Drug Price Negotiation: Breo Ellipta and Trelegy Ellipta — Final Report
- 10/30/25: Launch Price and Access Report: Drug Approvals from 2023-2024 — Final Report
领英推荐
Value Assessment Reports:
- 2/5/25: Acute Pain — Revised Evidence Report
- 2/6/25: Retinitis Pigmentosa — Draft Evidence Report
- 3/26/25: Retinitis Pigmentosa — Revised Evidence Report
- 3/27/25: Acute Pain — Final Evidence Report
- 4/10/25: Multiple Sclerosis: SPMS — Draft Evidence Report
- 5/12/25: Retinitis Pigmentosa — Final Evidence Report
- 5/27/25: Spinal Muscular Atrophy — Draft Evidence Report
- 5/29/25: Multiple Sclerosis: SPMS — Revised Evidence Report
- 7/15/25: Multiple Sclerosis: SPMS — Final Evidence Report
- 7/17/25: Spinal Muscular Atrophy — Revised Evidence Report
- 9/2/25: Spinal Muscular Atrophy — Final Evidence Report
Meetings:
- 2/28/25: Acute Pain — Public Meeting (Midwest CEPAC)
- 4/11/25: Retinitis Pigmentosa — Public Meeting (New England CEPAC)
- 6/13/25: Multiple Sclerosis: SPMS (California Technology Assessment Forum CTAF)
- 8/1/25: Spinal Muscular Atrophy — Public Meeting (Midwest CEPAC)?
Contributing author: Brian Sils