The Evaluation of Drug Innovativeness in Italy: Key Determinants and Internal Consistency.
Journal
PharmacoEconomics - open
ISSN: 2509-4254
Titre abrégé: Pharmacoecon Open
Pays: Switzerland
ID NLM: 101700780
Informations de publication
Date de publication:
May 2023
May 2023
Historique:
accepted:
16
01
2023
medline:
11
2
2023
pubmed:
11
2
2023
entrez:
10
2
2023
Statut:
ppublish
Résumé
Innovative medicines are provided with dedicated funds and immediate market access in Italy. Innovativeness evaluation considers unmet need, added therapeutic value, and quality of the evidence. We aimed to evaluate the internal consistency and drivers of the innovativeness appraisal process. Appraisal reports on innovativeness refer to 1997-2021. We used both a descriptive approach and probabilistic multivariate analysis, using logistic regression models to compute odds ratios and 95% confidence intervals. The dependent variable is innovativeness status (innovative vs. non-innovative; full innovativeness vs. conditional innovativeness). Explanatory variables, besides the three above-mentioned domains, are the year of evaluation, drug type, target disease and population, and the number and type of available studies. Among the 141 medicines scrutinized, 31.9%, 29.8%, and 38.3% were evaluated as fully innovative, conditionally innovative, and non-innovative, respectively. Added therapeutic value and the quality of the evidence were associated with the odds of receiving innovative status, and full compared with conditional innovativeness; unmet need was not a predictive variable. Other factors played a minor role: medicines for both solid tumours and rare diseases are more likely to be judged innovative; conditional innovativeness is more probable for medicines for rare diseases. Innovativeness status is driven by the added therapeutic value and quality of evidence. The appraisal process is internally consistent and predictable. This provides industry with a clear indication of what is needed to ensure that access to their medicines is prioritized.
Sections du résumé
BACKGROUND
BACKGROUND
Innovative medicines are provided with dedicated funds and immediate market access in Italy. Innovativeness evaluation considers unmet need, added therapeutic value, and quality of the evidence.
OBJECTIVE
OBJECTIVE
We aimed to evaluate the internal consistency and drivers of the innovativeness appraisal process.
METHODS
METHODS
Appraisal reports on innovativeness refer to 1997-2021. We used both a descriptive approach and probabilistic multivariate analysis, using logistic regression models to compute odds ratios and 95% confidence intervals. The dependent variable is innovativeness status (innovative vs. non-innovative; full innovativeness vs. conditional innovativeness). Explanatory variables, besides the three above-mentioned domains, are the year of evaluation, drug type, target disease and population, and the number and type of available studies.
RESULTS
RESULTS
Among the 141 medicines scrutinized, 31.9%, 29.8%, and 38.3% were evaluated as fully innovative, conditionally innovative, and non-innovative, respectively. Added therapeutic value and the quality of the evidence were associated with the odds of receiving innovative status, and full compared with conditional innovativeness; unmet need was not a predictive variable. Other factors played a minor role: medicines for both solid tumours and rare diseases are more likely to be judged innovative; conditional innovativeness is more probable for medicines for rare diseases.
CONCLUSIONS
CONCLUSIONS
Innovativeness status is driven by the added therapeutic value and quality of evidence. The appraisal process is internally consistent and predictable. This provides industry with a clear indication of what is needed to ensure that access to their medicines is prioritized.
Identifiants
pubmed: 36763319
doi: 10.1007/s41669-023-00393-3
pii: 10.1007/s41669-023-00393-3
pmc: PMC10169980
doi:
Types de publication
Journal Article
Langues
eng
Pagination
373-381Subventions
Organisme : MSD
ID : MSD Oncology Policy Grant Program
Informations de copyright
© 2023. The Author(s).
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