New Guidelines on AIFA Monitoring Registries
?? The Italian Medicines Agency (AIFA) has recently updated its criteria for the establishment and management of monitoring registries, which are essential tools for ensuring economic sustainability and the appropriate use of therapies reimbursed by the National Health Service (SSN).
??Price and reimbursement requests with negotiation tipology TN 1 are covered by this guideline.
Here are the key highlights:
? Objective: Increase transparency and appropriateness in the use of medicines through platforms like Registries (R), web-based Therapeutic Plans (PTwb), Simplified Registries (RS) and Simplified Multi-Drug Registry (RMS).
??? Key Criteria for Establishment:
1. Economic Impact: Therapeutic indications with an estimated expenditure exceeding €80 million in the first three years of commercialisation(€50 million for orphan drugs for the treatment of rare diseases).
2. Technical-Scientific Evaluation: is crucial in defining the appropriate use of a reimbursed medicine by the National Health Service (SSN) and managing access to reimbursement.
- Identify patient populations with the most robust efficacy evidence and the most predictable safety profile.
- Determine measures to control pharmaceutical expenditure.
- Complex or non-standardized diagnostic pathways.
- High uncertainty regarding efficacy and safety.
- Risk of off-label use, particularly for rare diseases or therapies with high unit costs and regulatory uncertainty.
- Inclusion/exclusion in clinical trials: Restrictions compared to the population authorized by EMA.
- Off-label risk: Use in non-reimbursed indications.
- Complex diagnostics: Non-standardized pathways in clinical practice.
- Limited results: Efficacy demonstrated only in specific patient subgroups.
- Clinical uncertainty: Uncertain treatment outcomes in practice.
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- Therapeutic failures: High frequency of early failures due to toxicity or ineffectiveness.
3. Advanced Therapies: Special attention to high-cost therapies with significant uncertainty in clinical outcomes.
4. Therapeutic Aggregation Criterion: is applies when two or more medicines, with different active ingredients, require monitoring for the same reimbursed therapeutic indication. This includes medicines that are part of the same therapeutic algorithm, even if their indications are not entirely identical.
The criterion is not mandatory for establishing monitoring but becomes a priority if the aggregated therapeutic group has been under monitoring for less than six years. This ensures fair treatment among competing medicines with similar indications. If the monitoring has been in place for over six years, a simplified or no additional monitoring approach may be considered for the aggregated group.
5.?? Managed-Entry Agreements (MEA):serve as tools to enhance the sustainability of high-cost treatments by addressing economic and clinical uncertainties.
Key aspects:
Capping: Sets a maximum treatment cost to manage uncertainty regarding dosage and treatment duration.
Cost-Sharing: Helps mitigate financial risks, particularly for therapies with high early failure rates or significant cost impacts.
Payment-by-Result (PbR) and Risk-Sharing (RS): Focus on clinical outcomes, particularly when there is high uncertainty in real-world efficacy or an elevated rate of early treatment failures.
These agreements define clear criteria for treatment discontinuation (e.g., toxicity or failure) and the time frame for evaluation.
6. Patent Expiry Criterion: This criterion applies when a medicine gains authorization for a new therapeutic indication but is expected to lose patent protection within the next two years, with generics or biosimilars anticipated.
In such cases, establishing a monitoring activity is generally not recommended. This is particularly relevant when the medicine is part of a therapeutic aggregation.
Monitoring may be considered for medicines authorized under L.648/1996 or included in multi-drug registriesestablished for specific regulatory purposes, such as the PPP registry.
This guideline represents a significant step forward in managing innovative therapies in Italy, with a strong focus on sustainability and patient outcomes.
?? Your opinion matters! What impact do you think these measures will have on the pharmaceutical and healthcare sectors? Share your thoughts in the comments! ??