DO REGULATORY AND INNOVATION PROXIES TRANSLATE INTO HTA VALUE? EVIDENCE FROM PORTUGAL
Author(s)
Paula Costa, MSc, BE1, Ana Margarida Advinha, Professor, PhD2, Sofia Oliveira Martins, Professor, PhD3.
1PhD student, Faculdade de Farmácia da Universidade de Lisboa, Lisbon, Portugal, 2School of Health and Human Development, University of Evora, Évora, Portugal, 3Faculty of Pharmacy of University of Lisbon, Lisbon, Portugal.
1PhD student, Faculdade de Farmácia da Universidade de Lisboa, Lisbon, Portugal, 2School of Health and Human Development, University of Evora, Évora, Portugal, 3Faculty of Pharmacy of University of Lisbon, Lisbon, Portugal.
OBJECTIVES: To evaluate the association between regulatory and innovation proxies and the likelihood of obtaining Therapeutic Added Value (TAV) within Portuguese Health Technology Assessment (HTA) framework.
METHODS: This retrospective study analysed a cohort of 226 medicinal products with new active substances, authorised by the European Commission between 2014 and 2024, that subsequently obtained reimbursement in Portugal. Binary logistic regression models evaluated the association between TAV (vs. therapeutic equivalence) and four proxies: EMA Accelerated Assessment (ACC) and Conditional Marketing Authorization (Cond), FDA First-in-class (1st) and Breakthrough Designation (Break). Covariates included ATC classes L01 (antineoplastics) and J05 (antivirals). Univariable and multivariable models were estimated.
RESULTS: TAV was granted to 55.3% of the sample. Baseline frequencies were ACC 12.4%, Cond 13.3%, 1st 31.0%, and Break 30.1%. All proxies were significantly associated with TAV in univariable analyses; however, in multivariable models, only 1st (adjusted OR: 5.87; 95% CI: 2.76-12.48; p<0.001) and Break (adjusted OR: 4.02; 95% CI: 1.82-8.88; p<0.001) remained independently significant. A cumulative gradient was observed, with TAV probability increasing from 28.7% (no proxies) to 100% (four proxies). L01 was strongly associated with TAV (OR: 9.13; p<0.001), while J05 showed a negative association (OR: 0.29; p=0.009).
CONCLUSIONS: Regulatory and innovation proxies are associated with TAV recognition, but only First-in-class status and breakthrough designation retained independent explanatory value in multivariable models, suggesting that not all regulatory or innovation signals translate equally into HTA-recognised value. Therapeutic context substantially influences HTA outcomes. These findings highlight the complexity of converting early innovation signals into assessed clinical value.
METHODS: This retrospective study analysed a cohort of 226 medicinal products with new active substances, authorised by the European Commission between 2014 and 2024, that subsequently obtained reimbursement in Portugal. Binary logistic regression models evaluated the association between TAV (vs. therapeutic equivalence) and four proxies: EMA Accelerated Assessment (ACC) and Conditional Marketing Authorization (Cond), FDA First-in-class (1st) and Breakthrough Designation (Break). Covariates included ATC classes L01 (antineoplastics) and J05 (antivirals). Univariable and multivariable models were estimated.
RESULTS: TAV was granted to 55.3% of the sample. Baseline frequencies were ACC 12.4%, Cond 13.3%, 1st 31.0%, and Break 30.1%. All proxies were significantly associated with TAV in univariable analyses; however, in multivariable models, only 1st (adjusted OR: 5.87; 95% CI: 2.76-12.48; p<0.001) and Break (adjusted OR: 4.02; 95% CI: 1.82-8.88; p<0.001) remained independently significant. A cumulative gradient was observed, with TAV probability increasing from 28.7% (no proxies) to 100% (four proxies). L01 was strongly associated with TAV (OR: 9.13; p<0.001), while J05 showed a negative association (OR: 0.29; p=0.009).
CONCLUSIONS: Regulatory and innovation proxies are associated with TAV recognition, but only First-in-class status and breakthrough designation retained independent explanatory value in multivariable models, suggesting that not all regulatory or innovation signals translate equally into HTA-recognised value. Therapeutic context substantially influences HTA outcomes. These findings highlight the complexity of converting early innovation signals into assessed clinical value.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA332
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes