A long history low drug prices in Italy have given Italy’s central pricing and reimbursement body AIFA a bad reputation with industry. The recent introduction of risk sharing agreements, claw backs and drug registries have further strengthened this reputation. Therefore, the hypothesis that Italy could become the first European country to fund the high-priced CART-T and gene therapies may sound controversial and surprising but still plausible for many reasons. Here is why.
Firstly, unlike IQWIG in Germany, HAS in France, or the complex Highly Specialized Technology appraisals in the UK, AIFA does not get stuck in a rigid methodological straightjacket of HTA. AIFA works under the radar and only little is published about its work. Our daily experience confirms AIFA as a pragmatic HTA body posing straightforward questions. Who has an urgent need for the new treatment? How many patients are these? Does that fit within our budget? Who can be treated later down the line?
Another reason is the separate, national budget for innovative drugs that is defined by AIFA’s innovation criteria. In December 2017, a plan was approved to set up comprehensive registries for innovative medicines in which the patient is followed in detail, from diagnosis onwards throughout his full treatment pathway. The new registries are designed to overcome the privacy pitfalls of the established oncology drug registries, which have proven effective to limit excessive drug use. Benchmarking on the current registry experience, the new innovation registry and the separate innovation fund are expected to render the use of gene therapy appropriate, predictable and transparent: Which patients are eligible and how do they fare? How are the patients doing that are not (yet) eligible? The new registries are also grafted on Italy’s experience with HCV patient warehousing.
Cost-utility measures are not in the cultural genes of Italy, but cost-benefit is a cornerstone in AIFA assessments. AIFA was sometimes accused of ‘drug bucket thinking’. This is now a thing of the past. The high one-off cost of gene therapy is now compared with the total long-term costs of chronic diagnostic procedures, doctor visits, hospitalizations and interventions. This kind of cost comparison changes the perception of the high cost of gene therapy. Moreover, it overcomes payers’ hostile attitude towards the pharma industry and renders them less anxious to commit paying € 500.000 overnight to treat one patient.
Manufacturers can submit the AIFA dossier 2 months prior to EMA approval (at positive CHMP opinion) and it only takes 4 to 5 months before a funding decision is taken. Straightforward dossiers with clear patient segmentation and convincing budget impact analysis can go fast, and much faster than in France, Germany and UK.
Clearly Italy seems ready to rapidly fund PRIME-approved therapies (orphan and non-orphan) at prices within the expectations of the industry.
Already some years ago Italy was considering staggered payments for exceptionally high cost therapies (e.g. Glybera's 1 million price tag) but it seems that Exondys 51, Kymriah, and Axi-cell will remain below the €500.000 limit. So, this looks as good news for Novartis, Gilead and Sparks when they may get PRIME approval later this year.
From our daily discussions with payers from several European countries we predict that Italy may become the first country to make gene therapies available to patients. A surprising but well-founded hypothesis!
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