Adapting to Change: Analysis of Trend Estimation in AMNOG Dossiers Under Revised GBA Guidelines
Author(s)
Kathrin Gerchow, MPH, Tammo Constantin Viering, PhD, Timotheus Stremel, MSc, Karolin Seidel, MSc, Christian Jacob, PhD.
Cencora, Hannover, Germany.
Cencora, Hannover, Germany.
OBJECTIVES: In 2024, the Federal Joint Committee (G-BA) revised Module 3 requirements of dossiers required under the Act on the Reform of the Market for Medicinal Products (AMNOG). Trend estimates must now focus on expected changes in the number of statutory health insurance (SHI) patients within new drugs’ target populations over the next five years, rather than on prevalences and incidences of underlying diseases. Aim was to assess the implementation of revised requirements, applied methods, and their evaluation by the Institute for Quality and Efficiency in Health Care (IQWiG) assessment.
METHODS: AMNOG dossiers published between November 2024 and February 2025 were analyzed. Module 3 sections 3.2.3 and 3.2.4 were reviewed to determine whether trends were estimated, and which populations were used. Utilized methods were assessed and categorized. IQWiG assessments (section II 1.3.4) were reviewed regarding their trend estimate evaluation. Two reviewers independently verified the categorizations.
RESULTS: In total, N=45 dossiers were submitted, representing N=31 compounds. Most indications were oncology (53.3%), infectious diseases (11.1%), and blood disorders (8.9%). Orphan drugs accounted for 20.0%. Trend estimation was performed in 71.1% of dossiers. Trend assessment was based on incident patients in 66.7%, while prevalent or target populations were each used in 55.6%. In November 2024, all dossiers complied with the old guideline (i.e. prevalence/incidence of underlying disease); by February 2025, only 11.8% did. Now, 52.9% referred exclusively to the target population, and 35.3% used both approaches. Common methods included growth rates (59.4%), population projections (62.5%), and patient numbers from single years (28.1%). While IQWiG comments on methods used to estimate target populations, methodology for trend estimation was described but not evaluated.
CONCLUSIONS: Trend analyses show a shift of trend estimations towards SHI target populations by early 2025 corresponding with G-BA guideline revisions. Growth rates and population projections were common methods. The IQWiG did not criticize used methodologies.
METHODS: AMNOG dossiers published between November 2024 and February 2025 were analyzed. Module 3 sections 3.2.3 and 3.2.4 were reviewed to determine whether trends were estimated, and which populations were used. Utilized methods were assessed and categorized. IQWiG assessments (section II 1.3.4) were reviewed regarding their trend estimate evaluation. Two reviewers independently verified the categorizations.
RESULTS: In total, N=45 dossiers were submitted, representing N=31 compounds. Most indications were oncology (53.3%), infectious diseases (11.1%), and blood disorders (8.9%). Orphan drugs accounted for 20.0%. Trend estimation was performed in 71.1% of dossiers. Trend assessment was based on incident patients in 66.7%, while prevalent or target populations were each used in 55.6%. In November 2024, all dossiers complied with the old guideline (i.e. prevalence/incidence of underlying disease); by February 2025, only 11.8% did. Now, 52.9% referred exclusively to the target population, and 35.3% used both approaches. Common methods included growth rates (59.4%), population projections (62.5%), and patient numbers from single years (28.1%). While IQWiG comments on methods used to estimate target populations, methodology for trend estimation was described but not evaluated.
CONCLUSIONS: Trend analyses show a shift of trend estimations towards SHI target populations by early 2025 corresponding with G-BA guideline revisions. Growth rates and population projections were common methods. The IQWiG did not criticize used methodologies.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA22
Topic
Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas