Search results for: rare disease

Orphan Drug Designation: A Head Start, Not a Fre...

Manufacturers looking to optimise access for their orphan drugs need to navigate a complex environme...

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ISPOR Europe 2025: Key Insights, Research Posters ...

ISPOR Europe 2025 brought together global HTA bodies, payers, industry leaders and patient organisat...

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2025 Market Access Check-In: Evaluating the Impact...

2025 was a pivotal year for market access, marked by the growing convergence between scientific inno...

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Comparator Choice: The Question That Defines Marke...

Every payer ultimately asks the same question: “Compared with what?” This single question shapes...

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Revisiting rarity: Would yesterday’s NICE highly...

Objectives:  ​​In March 2025, NICE adopted new HST routing criteria for evaluating technolo...

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Surrogates Under Scrutiny: Proving the Link to Out...

Surrogate Endpoints in HTA Decision-Making The need to stay ahead of competitors and offset losses f...

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Navigating NICE’s HST Pathway: Insights from Bot...

NICE’s Highly Specialised Technologies (HST) Programme offers a vital route for bringing ultra-rar...

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Your 5-step ATMP pricing playbook for launch succe...

Advanced Therapy Medicinal Products (ATMPs) are offering transformative, and often life-saving, trea...

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ATMP Pricing in Practice: Success & Cautionary...

Advanced Therapy Medicinal Products (ATMPs) are leading in healthcare innovation, providing potentia...

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Why Gene Therapies Keep Hitting Price & Access...

The pricing benchmark has shifted faster than systems have adapted. In the UK, prices for one-time t...

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