Impact of Changes in Indications on Clinical Assessments in the EU
Author Information
Author(s): Heikkinen Inka, Goodall Melinda, Steck Natalie, Poulakou Maria, Piso Katherine
Primary Institution: MSD International
Hypothesis
How do changes in proposed indications affect the scoping for Joint Clinical Assessment in the EU?
Conclusion
Changes in proposed indications often narrow the eligible patient population, which can complicate the Joint Clinical Assessment process.
Supporting Evidence
- 67% of products had only editorial changes between proposed and approved indications.
- Most changes reduced the eligible population rather than broadened it.
- The most common change was a shift to a later treatment line.
Takeaway
When doctors decide how to treat patients, they sometimes change their minds about who should get the treatment, which can make it harder for patients to get the help they need quickly.
Methodology
The study analyzed 27 oncology products and 15 Advanced Therapy Medicinal Products for changes in proposed indications and their impact on patient eligibility.
Potential Biases
Potential bias due to reliance on clinical assumptions without expert validation.
Limitations
The study did not include conditionally approved products and relied on general patterns rather than detailed case-by-case analysis.
Participant Demographics
Included oncology and advanced therapy medicinal products approved between 2017 and January 2024.
Digital Object Identifier (DOI)
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