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1、2024 Alnylam Rare Disease Trend ReportP E R S P E C T I V E S F R O M H E A LT H C A R E PAY E R S A N D E M P LOY E R S F I F T H E D I T I O N2024 Rare Disease Trend Report23P E R S P E C T I V E S F R O M H E A LT H C A R E PAY E R S A N D E M P LOY E R SP E R S P E C T I V E S F R O M H E A LT H

2、 C A R E PAY E R S A N D E M P LOY E R S2024 Alnylam Rare Disease Trend Report2024 Alnylam Rare Disease Trend ReportTABLE OF CONTENTS Authors .3 Foreword .3 Introduction .4 Key Findings .5 Methodology.6 SECTION 1:Benefit Design And Utilization Management Approach .10 SECTION 2:Infrastructure And Cap

3、abilities .18 SECTION 3:Employer Trends.22 SECTION 4:Technology.24 SECTION 5:Payer Economics .28 SECTION 6:Future Rare Disease Trends .32 Conclusion.34The healthcare community is now well-versed in the life-changing potential of rare disease products,including cell and gene therapies.While the initi

4、al novelty has shifted from groundbreaking anticipation to a rapidly expanding portfolio of treatments,many questions particularly around how to create and sustain access and affordability remain unresolved.As more rare disease products have launched in the United States,stakeholders have explored v

5、arious approaches and policies to manage high costs,while continuing to support innovation.Yet,the debate continues about how best to navigate access challenges as market dynamics evolve.The 2024 Alnylam Rare Disease Trend report continues important discussions with payers and employers,expanding on

6、 previous editions to explore the emerging use of next-generation technologies,such as artificial intelligence(AI),and the impacts of the initial phases of the Inflation Reduction Act(IRA)Medicare Part D Redesign.Published to inspire open dialogue among payers,employers,providers,manufacturers,broke