Dr. Luca Pani: The Italian OBA Connection
April 22, 2021
Luca Pani on the need for more creative market access agreements – and what Canada can learn from Italy.
A globally recognized expert in pharmacology, regulatory and access strategies, Dr. Pani served as CHMP and SAWP Member of the European Medicines Agency (EMA) and Director General of the Italian Medicines Agency (AIFA) from 2011 to 2016. During his tenure at AIFA, he revolutionized the agency’s approach to drug approval, pricing and reimbursement, elevating Italy to an international leadership position in the use of managed entry and outcomes-based agreements. The co-author of hundreds of scientific papers, Dr. Pani currently holds academic positions at the University of Modena and Reggio Emilia in Italy and the University of Miami, handles regulatory strategy and market access innovation for the influential and technology advanced drug development consultancy VeraSci in Durham, NC, and is arguably the leading global expert on outcomes-based agreements.
Among the disruptive changes you introduced at AIFA, is there one that was especially meaningful to you?
AIFA was the payer who negotiated access for Strimvelis, the world’s first gene therapy. This is for people who can’t produce white blood cells and thus have to spend their lives isolated from infectious agents—in a bubble, as it were. We got the drug listed within 55 days of the dossier request for regulatory evaluation at a price of 594,000 Euros, with reimbursement contingent on results and clear outcomes measurements to guide us.
Did you get support for your approach from the Italian government?
Yes. Our parliament has a law that mandates offering treatments to patients when no reasonable alternatives exist. We also have web-based certified registries which are legally binding in the context of a drug pricing negotiations. Having binding laws, as opposed to mere guidelines, is the key to empowering successful negotiations and timely access, especially for high-cost therapies.
How do registries fit into the market access framework?
Registries define the population (even in sub-strata if needed) with precise inclusion and exclusion criteria and what we call the “value endpoints” and can be used to compare a new treatment to the standard of care. Data from registries are the “fuel” for market access agreements. Most agreements for expensive, life-changing medications requires proof of duration for the clinical response within 2-3 years, which registry data can help establish.
Duration of response is an obviously important decision-making point. Who bears the burden of demonstrating a lasting response?
Nowadays it’s up to the market access professionals to provide the data on duration of response, though access to a medication cannot be held hostage to this evidence when it is limited. You can’t tell a parent you won’t give a medication to their dying child because you don’t know how long it will work. Because so much is at stake for families, regulators may even approve early access for a larger population than that proposed by the manufacturer, as was the case with Zolgensma in Europe. Early access first, formalized reimbursement criteria later—we’ll be seeing a lot more of this model in the future.
“Early access first, formalized reimbursement criteria later—we’ll be seeing a lot more of this model in the future with innovative life-saving therapies.”
What else can manufacturers do to expedite access and set fair pricing?
Non-inferiority data can help a manufacturer get a listing, but payers will understandably expect to see superiority data, with clinically relevant endpoints such as overall survival, to justify a high price. High-quality health-economic data can also strengthen a case. For example, if you can demonstrate that a novel combination treatment prevents the progression of, say, hepatic fibrosis to hepatocarcinoma and reduces the need for costly liver transplants, you can leverage these downstream certifiable savings in pricing negotiations. The problem is that the drug procurement and transplant departments in a healthcare system often operate in silos. We need a One Health model approach, with all departments talking to each other.
We’ve seen incredible innovation in specialty medicine in the past few years. How has this innovation changed the regulator’s role?
Today, regulators are not expected merely to keep ‘bad medicines’ off the market. They must align drug licensing with patients’ needs by granting them timely access to promising new technologies. Of course, quick access comes with a burden to prove effectiveness. This entails more stringent monitoring and data generation throughout the life cycle of the medication.
What would you recommend to help Canada move forward in the OBA space?
I would suggest building an early access scheme, ideally with legal teeth. There should be a centralized body giving binding recommendations on reimbursement criteria, with some additional negotiating room at the provincial level. Regulators and payers need to get together to make it happen.
How important is it for industry to explore innovative market access agreements, such as OBAs?
It’s critically important. Specialty medicine innovation isn’t going away, and affected families won’t put up with protracted negotiations when a loved one’s life hangs in the balance. Payers won’t be pushing for more creative negotiations—they’re used to thinking in terms of caps or discounts—so it’s up to drug manufacturers to take the lead.
Should manufacturers be sharing their OBA experience more transparently within the industry?
We can’t expect manufacturers to reveal pricing details—and we don’t need to know the specific figures. Transparency in OBA implementation processes, however, is much more valuable for shared learnings.
How important is the quality of the data to support OBA-type agreements?
Payers are generally willing to work with real-world data, as opposed to regulatory-grade data, as long as the integrity of the data is assured. Some manufacturers are even venturing into creative new data collection territory. For example, Biogen recently announced a partnership with Apple to investigate how iWatch and iPhone could help in screening and monitoring possible declines in cognitive health.
Has the COVID-19 pandemic taught us anything that can be applied to innovative market access agreements?
The pandemic has shown us that things previously thought impossible are doable. Imagine if we had been told a year ago that over 600 million people could be quickly vaccinated under emergency use authorization—nobody would have believed it. Now that we know how fast and effectively medical science can intervene, it will be harder to justify requesting, say, 2 years of additional safety data before making a life-saving drug available to patients. Science will dictate speed, and early access is the new target. Patients are demanding it and rightly so.