Professor Luca Pani has spent four years at the helm of the Italian Medicines Agency (Agenzia Italiana del Farmaco – AIFA). In that time he has worked with three Italian governments, witnessed the tensions between austerity and drug reimbursement, and seen off a media furore over a highly-questionable stem-cell based ‘miracle cure’.

He has also become a leading thinker on the development of the adaptive licensing of medicines in Europe. Why has he taken such a proactive approach to this complex issue? Pani says that he and colleagues at AIFA see their role as helping to get the right treatment to the right patient at the right time.

“Regulators are not expected merely to keep ‘bad medicines’ off the market anymore. They must align drug licensing with patients’ needs by granting them timely access to new technologies,” says Pani.

That means giving a restricted patient population early access to medicines, facilitating the generation of additional ‘real-world’ data. The idea, now well understood at the highest levels of EU regulatory bodies, is nonetheless challenging. It upends the status quo and changes the game for everyone with an interest in medicines.

Yet, listening to Pani passionately make the case for fresh thinking, it becomes clear that the status quo is not working for anyone. Industry says getting a drug to market is risky and expensive; patients say they are sick of waiting for access; payors and policymakers want clear evidence that they are getting value for money.

“I went on the record many times about the need for flexible authorisation models,” says Pani. “This problem has sparked a lively public debate and we hope that this will lead to the development of new paradigms for evaluation and reimbursement of innovative molecules.”

Adaptive licensing is coming. But giving early access to new medicines will mean stricter monitoring and data generation throughout the entire life-span of a drug. What kind of data will regulators look for?

“The short answer is that we need as much ‘real world’ data as possible. The bottom line is that an innovative drug should be licensed and reimbursed only if proven effective. That makes identification of responders absolutely paramount,” explains the Italian drug watchdog.

For its part, the AIFA has established a wide range of monitoring registries. “Our registries are dynamic tools placed in the early phases after marketing authorisation of new drugs,” says Pani. “They have a clear purpose and typically a short defined lifespan, which is designed to measure real world safety and effectiveness and apply the Managed Entry Agreements’ procedures.”

He wants to see granular, real-time data on prescription, adherence, effectiveness and safety. “I am pretty confident that tools like these are the solution to the problem of evidence generation in the context of adaptive licensing.”

While this means big changes for regulators, it’s a new world for pharmaceutical companies too. Pani says industry needs to change how it looks at clinical trials. He says huge amounts of money are spent on executing clinical trials but it would be wiser to invest a bit more time and resources strategically designing studies.

“This means moving away from the approach taken in the past. We need innovators inside the industry. Even innovative industries like biotech do not always have innovative approaches to pricing and reimbursement.”

For many, the ultimate question is how all of this impacts on prices. Pani acknowledges that the pricing and reimbursement of drugs approved under adaptive licensing is “one of the hardest puzzles to solve”.

Payors could argue that giving early market access offers an opportunity for companies to recoup their investment more rapidly, thus allowing for lower prices when the product is made fully available. From an industry perspective, if mounting real world data shows that a medicine is adding real value, a higher price would be expected.

“Moving from adaptive licensing to an adaptive reimbursement system – this is the aim of the game,” says Pani.

The full transcript of the interview with Luca Pani can be downloaded here.