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The role of the Italian Medicine Agency (AIFA) Luca Pani, M.D. Director General DG@aifa.gov.it Padova, 12 November 2013. Public Declaration of transparency/interests*.
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The role of the Italian Medicine Agency (AIFA) Luca Pani, M.D. Director General DG@aifa.gov.it Padova, 12 November 2013
Public Declaration of transparency/interests* *Luca Pani, in accordance withtheConflict of Interest Regulations approved by AIFA Board of Directors (26.01.2012) and published in the Italian Government Official Journal on 20.03.2012 according to 0044 EMA/513078/2010 on the handling of the conflicts of interest for scientific committee members and experts Note: For this presentation I am not receiving any compensation
Key discoveries in the basic science of HCV Molecular virology has deciphered the viral replication cycle, identified druggable targets and generated tools for compound screening. Structural biology has provided high-resolution structures of key viral drug targets.
New drug targets in HCV As of May 2013, over 1,600 studies evaluating drugs for the treatment of hepatitis C are listed on the ClinicalTrials.gov website. These trials are investigating a multitude of compounds in different patient populations or subpopulations. Roman numerals in brackets indicate the current clinical phase of development.
Will we have just one anti-HCV pill? It is unlikely that a ‘one size fits all’ approach will be developed given a broad choice of different drugs and a large number of patient characteristics that have an impact on response to treatment. However it seems feasible that many treatment regimens will contain a nucleoside or nucleotide NS5B polymerase inhibitor together with other DAAs, PEG-IFN and/or ribavirin. This presumably will come in the form of a single-pill fixed-dose combination.
AIFA's contribution to existing therapies AIFA, in collaboration with the Universityof Padova, designed an electronic web-based algorithm (255 nodes) that identifies the best pharmaceutical pathways to treat HCV. The algorithm is published on the AIFA's portal. http://www.agenziafarmaco.gov.it/it/content/algoritmi-terapeutici
Simeprevir • Applied indication: Simeprevir (SIM) is indicated for the treatment of chronic hepatitis C (CHC) genotype 1 or genotype 4 infection, in combination with peginterferonalfa and ribavirin, in adults with compensated liver disease (including cirrhosis) with or without human immunodeficiency virus-1 (HIV-1) co-infection who are treatment-naïve or who have failed previous interferon therapy (pegylated or non-pegylated) with or without ribavirin. • Mechanismof action: Simepreviris the third member of direct-acting antiviral agents’ class. It is an inhibitor of the HCV NS3/4A protease and is being developed for the treatment of chronic HCV infection. • Procedure: centralised. • Main efficacy studies: QUEST-1, QUEST-2, PROMISE.
Sofosbuvir • Applied indication: Sofosbuvir (SOF) is indicated in combination with other agents for the treatment of chronic hepatitis C (CHC) in adults. • Mechanismof action: Sofosbuvir is a novel nucleotide prodrug inhibitor. In human hepatocytes, it is converted to an active uridine triphosphate form which acts as an inhibitor of the HCV NS5B polymerase. • Procedure: accelerated. • Main efficacy studies: FISSION, POSITRON, FUSION, NEUTRINO.
What about novel therapies for Hepatitis C? A Regulator’s Challenge • How to cope with uncertainty when deciding on pricing and reimbursement? • What is the cut-off to be considered between therapeutic utility of a new medicine and its major cost? • How can we make difficult decisions in the absence of ideal information?
AIFA’sKeywords: Innovation and Sustainability To supportregulatory and administrativeactivities in the achievement of bothefficiency and efficacy To increasescientificknowledge on real-world setting for obtainingrelevant data to take strategicmeasures • AIFAhasdeveloped some usefultoolsable: Since 2000 the Italian Health System is one of the first NHS where conditional reimbursement agreements have been introduced
MEAs classification Managed entry agreements for pharmaceuticals:The Europeanexperience. April 2013.
The Italiancontext: not only risk to benefit but also benefit to price Managed entry agreements for pharmaceuticals:The Europeanexperience. April 2013.
Managing budget impact Managing uncertainty relating to clinical benefit and cost-effectiveness Managing utilisation to optimize performance Non-Outcome based MEAs Outcome based MEAs Therapeutic plans Budget cap Monitoring registers Volume agreements Cost sharing Orphans Antireumatics Oncologicals Cardiovascular Psoriasis Antidiabetics Risk sharing Payment by results AIFA notes Reimbursement (without conditions) Refusal What are the AIFA's priorities? Supporting the prescription appropriateness and NHS sustainability considering the clinical evidence reported in the national and international guidelines and real life data.
Models of MEAs in Italy Three different ways to share responsibility and risk between pharmaceuticals companies and NHS (third payer). Cost Sharing, discount on price of initial therapy cycle(s) for all eligible patients Risk Sharing, discount on price of initial therapy cycle(s) for non-responder Payment by Results, initial cycle(s) fully reimbursed by marketing authorization holder for non-responder (fully reimbursed by NHS for responders) Risk sharing and Payment by Results are performance based-agreements conditioned on clinical evaluation of specific endpoints, with limitations of cost if the effect is inappropriate. The agreement is for a limited period of time, under specific conditions, and to be re-evaluated.
RegistriesasAdministrative & Research Tools Biomedical research on disease and drug development Marketing authorization bench-to-bedside bedside-to-community Monitoringregistries Drug Access for patient and clinical practice in real life use Efficacy and safety data collection
In conclusion: valorization of true innovation AIFA’snew domains • Innovation must represent a therapeutic advantage • Innovation must be "measurable“ on: • Population selection. Robustness of endpoint(s). Choice of comparators. Duration of therapeutic effect • Innovation should be valued against the National context • Innovation must meet pharmaco-economic studies and HTA standards in order to determine the ratio of incremental cost-effectiveness compared to the standard reference standard AIFA’s new algorithm on innovation
