In this exclusive sneak peek feature, we speak with Paolo Morgese (Alliance for Regenerative Medicine; Geneva, Switzerland) ahead of his presentation at the inaugural Real-World Evidence 2020: Rare Diseases and Innovative Therapies meeting. Discover his insight and register to attend RWE 2020 now!
Paolo Morgese is the EU Director of Market Access and Member Relations at the Alliance for Regenerative Medicine (ARM; Geneva, Switzerland).
ARM is a leading, global, non-for-profit alliance, bringing together more than 350 organizations focusing on advanced therapies. One major objective of ARM is to facilitate access to advanced therapies and for this reason ARM is engaged with policymakers and stakeholders around the world to find sustainable solutions to accessing these breakthrough therapies.
What did you want to be when you were growing up?
I wanted to be an archeologist or paleontologist. I like to think I am still on track for it — in this or in another life.
What challenges associated with payer models for rare diseases and orphan drugs could real-world evidence help address?
Advanced therapies, such as gene therapies, offer huge promise to patients and society; some will be able to dramatically change the courses of diseases that previously had little or no treatments available. At the same time, these new technologies are developing very quickly, with hundreds of clinical programs initiated only recently. Due to the rarity of the diseases, the great unmet clinical need and remarkable effects of some of these treatments, the data available when these drugs receive marketing authorization is different from what is normally required by access decision makers.
Once these drugs are approved, health technology assessment bodies and payers need more information (and evidence) on efficacy: real-world evidence is the way to generate this evidence, in the best interest of all stakeholders.”
Traditionally, drug efficacy has been assessed in large randomized clinical trials, with effects measured over a relevant time period. This makes a lot of sense, as traditional drugs often exerted a marginal effect over a limited period of time. By contrast, advanced therapies exert a very large effect on a very small population of patients who have no alternative treatments available. Once these drugs are approved, health technology assessment bodies and payers need more information (and evidence) on efficacy: real-world evidence is the way to generate this evidence, in the best interest of all stakeholders.
Another big systemic challenge is financial sustainability; the number of breakthrough treatments that are going to reach patients is something previously unseen for healthcare. This is fantastic news, but complex to manage from a financial standpoint. Traditional medicines addressing chronic diseases usually have a transient effect; they need to be periodically re-administered to maintain a sustained effect on patients. Many advanced therapies are expected to have an effect lasting many years, if not for the whole patient’s lifetime. If we add to this the small number of prevalent patients with rare or ultra-rare genetic diseases, the price per administration of advanced therapies is comparatively much higher than traditional medicines. This does not mean that advanced therapies will be more expensive over patients’ lifetimes, on the contrary.
The healthcare environment is dramatically changing, and real-world evidence is one of the major areas of innovation that will impact the future of healthcare.”
These peculiarities of advanced therapies challenge existing pricing and payment models and requires new solutions, which are generally called innovative payment models. One example that has received large support from experts and policymakers is the, so-called, annuity-based payment model. With this model, payers do not invest upfront the full cost of an advanced therapy, but split it over some years, eventually linking the payment to the treatment outcome — i.e., no more payments if the effect stops.
What is the importance of real-world evidence for timely access to innovative therapies, such as cell and gene therapies?
Real-world evidence, the infrastructure and methods to generate, collect and use it, represent the present and the future of innovative healthcare. Without a robust and reliable real-world evidence ecosystem, timely access to advanced therapies will be very challenging and will impose a high burden on society. Without real-world evidence, the choice for access bodies will be between taking ill-informed decisions and not taking decisions, which are practically equivalent.
Without giving too much away, what do you hope are the key points for the audience to take away from your talk at Real-World Evidence 2020: Rare Diseases and Innovative Therapies?
The healthcare environment is dramatically changing, and real-world evidence is one of the major areas of innovation that will impact the future of healthcare. Advanced therapies, including cell and gene therapies, have the potential to revolutionize patients’ lives and healthcare. The cross-roads of innovative therapies, real-world evidence and innovative payment models are an exciting space for supporting innovation, helping patients and addressing some crucial challenges of healthcare systems in the years to come.