Abstract
The term „Unmet Need“ is used multiple times in European Union (EU) Health Technology Assessment (HTA) regulations,
the EU pharmaceutical legislation and in HTA guidelines in member states (MS). There is no consensus about the definition of unmet need, yet in most guidance documents it refers
to the unavailability of treatment options for patients with severe conditions or for patients suffering from significant
residual disease. Examples are orphan or neurodegenerative diseases like Alzheimer. Proxies or determinants of unmet
need, referred to as decision modifiers, may be also considered to inform payment decisions in MS. Amongst others, they
include rarity, disease severity, intended treatment aim, innovative treatments or cross-sector benefits. Others have
argued to include other value components in the appraisal, such as the value of knowing or value of hope. However,
these terms are ambiguous and not actionable. For many treatments, a cascade of factors ultimately determines if medical products will be available to patients in MS and, hence,
interpreting unmet need as a binary outcome is not appropriate. Concerns have been raised that prioritising R&D efforts to areas of unmet need should be aligned with the right
incentives to mitigate commercial risks, e.g. by (financial) protection measures.
the EU pharmaceutical legislation and in HTA guidelines in member states (MS). There is no consensus about the definition of unmet need, yet in most guidance documents it refers
to the unavailability of treatment options for patients with severe conditions or for patients suffering from significant
residual disease. Examples are orphan or neurodegenerative diseases like Alzheimer. Proxies or determinants of unmet
need, referred to as decision modifiers, may be also considered to inform payment decisions in MS. Amongst others, they
include rarity, disease severity, intended treatment aim, innovative treatments or cross-sector benefits. Others have
argued to include other value components in the appraisal, such as the value of knowing or value of hope. However,
these terms are ambiguous and not actionable. For many treatments, a cascade of factors ultimately determines if medical products will be available to patients in MS and, hence,
interpreting unmet need as a binary outcome is not appropriate. Concerns have been raised that prioritising R&D efforts to areas of unmet need should be aligned with the right
incentives to mitigate commercial risks, e.g. by (financial) protection measures.
| Original language | English |
|---|---|
| Number of pages | 8 |
| Publication status | Published - Mar 2025 |
| Event | Plattform zur Nutzenbewertung: Frühjahrstagung 2025 - Berlin, Germany Duration: 21 Mar 2025 → 22 Mar 2025 |
Conference
| Conference | Plattform zur Nutzenbewertung: Frühjahrstagung 2025 |
|---|---|
| Country/Territory | Germany |
| City | Berlin |
| Period | 21/03/25 → 22/03/25 |