ArticleFrontiers in pharmacology2021
The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy.
Article in Frontiers in pharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
24 citing papers in PubMed, 1 synthesis or guideline pooled it, 43 citations in OpenAlex.
- Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.Orphanet journal of rare diseases · 2025Pooled it
- Comparative Cost Evaluation of Managed Entry Agreement Techniques Using Real-World Data from High-Cost Anticancer Drugs in Thailand.Journal of market access & health policy · 2026Article
- Privacy rights and improving knowledge are not hierarchical needs: data protection and good epidemiologic standard (DP_GOES) checklist for retrospective observational studies using secondary data.BMC medical research methodology · 2026Article
- Stakeholders' Perspectives Toward the Use of Patient Registry Data for Decision-Making on Medicines: A Cross-Sectional Survey.Drug safety · 2025Article
- Planning Post-Launch Evidence Generation: Lessons From France, England and Spain.Clinical pharmacology and therapeutics · 2025Review
- The value-for-money assessment and funding arrangements for high-priced drugs in an era of uncertainty: a comparative analysis of national health technology assessment agencies in South Korea, England, Australia, and Canada.BMC health services research · 2025Article
- The evolving value assessment of cancer therapies: Results from a modified Delphi study.Health policy OPEN · 2024Article
- Advanced Therapy Medicinal Products: Availability, Access and Expenditure in Italy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Article
- Evolving assessment pathways for precision oncology medicines to improve patient access: a tumor-agnostic lens.The oncologist · 2024Article
- Can requests for real-world evidence by the French HTA body be planned? An exhaustive retrospective case-control study of medicinal products appraisals from 2016 to 2021.International journal of technology assessment in health care · 2024Article
- Values, challenges, and responses associated with high-priced potential cures: perspectives of diverse stakeholders in South Korea.Cost effectiveness and resource allocation : C/E · 2024Article
- From Indication-Based Pricing to Blended Approach: Evidence on the Price and Reimbursement Negotiation in Italy.PharmacoEconomics - open · 2024Article
- Analysis of drug pricing drivers under South Korea's pharmaco-economic evaluation exemption policy (2015-2022).Frontiers in pharmacology · 2024Article
- High costs, low quality of life, reduced survival, and room for improving treatment: an analysis of burden and unmet needs in glioma.Frontiers in oncology · 2024Review
- Engaging European society at the forefront of cancer research and care: How discussions at the 5Molecular oncology · 2023Article
- How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy?BMC health services research · 2023Review
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries.Frontiers in pharmacology · 2023Article
- Impact of the COVID-19 pandemic on prescription of sacubitril/valsartan in Italy.European journal of heart failure · 2022Article
- Outcome-based reimbursement in Central-Eastern Europe and Middle-East.Frontiers in medicine · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Italy has a well-established prominent system of national registries to support managed entry agreements (MEAs), monitoring innovative medicinal products (MPs) with clinical as well as economic uncertainties to ensure appropriate use and best value for money. The technological architecture of the registries is funded by pharmaceutical companies, but fully governed by the national medicines agency (AIFA). A desktop analysis was undertaken of data over a 15-year timeframe of all AIFA indication-based registries and associated EMA information. The characteristics of registries were evaluated, comparing orphan MPs vs. all MPs exploring cancer and non-cancer indications. OMP (orphan medicinal product) registries' type vs. AIFA innovation status and EMA approval was reviewed. Of the 283 registries, 182 are appropriateness registries (35.2% relate to OMPs, with an almost equal split of cancer vs. non-cancer for OMPs and MPs), 35 include financial-based agreements [20% OMPs (2 non-cancer, 5 cancer)], and 60 registries are payment by result agreements [23.3% OMPs (4 non-cancer, 10 cancer)]. Most OMPs (53/88) came through the normal regulatory route. With the strengthening of the system for evaluation of innovation, fewer outcomes-based registries have been instigated. AIFA has overcome many of the challenges experienced with MEA through developing an integrated national web-based data collection system: the challenge that remains for AIFA is to move from using the system for individual patient decisions about treatment to reviewing the wealth of data it now holds to optimize healthcare.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.