With the 5th World Orphan Drug Congress on 12- 14 November in Brussels less than two months away, I conducted an interview to one of our key speakers, Bert Bruce (Vice President, Commercial Development, Rare Disease, Pfizer) to give you further perspective into his session as well as the challenges he faces in making orphan drugs globally affordable.
Bert has a diverse skill set and broad pharmaceutical background, with leadership experiences across multiple therapeutics areas and disciplines and Johnson & Johnson, Wyeth and Pfizer. He has been an integral leader in complex programs including the acquisition team that brought tafamidis (Vyndaqel®) into the Pfizer Rare Disease Portfolio. Well recognised for his strategic vision and collaborative leadership, his session on “New framework for patient and academic collaboration in rare disease medicine development” will illustrate Pfizer’s vision to leverage the expertise of their partners to accelerate accessible drugs for patients.
Why are patient and academic collaborations developing rare disease diagnostics and therapies at the top of your agenda right now?
Pfizer is one of many stakeholders – both large and small – working toward a common goal: to deliver safe and effective medicines that address a part of the vast unmet needs that exist across the rare disease community. We believe that effective rare disease drug development demands an approach that will safely get the most medicines to the most patients, in the shortest amount of time. This is why we engage in collaborations. Through partnership with the patient or scientific community, we know we can not only augment our own expertise, but find ways to add value for others through our resources, expertise, scientific knowledge, or global presence. A recent example of this is our collaborative agreement with the Global Medical Excellence Cluster (GMEC) in the UK. The agreement provides scientists from the GMEC partner universities the opportunity to work with Pfizer scientists on joint drug discovery programs, creating the potential to accelerate the translation of basic science into a new generation of innovative medicines for the treatment of rare diseases.
What are some of the most pressing issues you are facing in doing this and how could this change in the future?
One of the major challenges we are facing is making sure that patients with rare diseases get access to the therapies we develop. Many European health systems are under a lot of financial pressure, and we are finding that each country and sometimes each region within a country are raising a variety of concerns about the affordability of new rare disease therapies, even where their overall budget impact is very low.
Companies have been proactive in efforts to mitigate the short-term costs for health systems in financial distress; at Pfizer we have managed to reach agreements that deliver patient access in many cases, sometimes even in the poorest countries in Europe. However, it can still be slow or sometimes impossible for us to reach agreements in some countries, and hence access for patients is limited or non-existent.
We believe that fundamental arguments about the social priority for tackling rare diseases and the dramatic difference that new orphan medicines can make to patients for a very limited overall budget impact continue to be compelling arguments for health systems and that we can work together to ensure patients have access to the therapies they need and deserve.
What do you think will be the single biggest trend or change to the orphan and rare disease sector between now and 2025?
I think you can look to the agenda for this year’s WODC Europe Congress and see that there are a number of trends influencing this space, so it’s difficult to point to just one trend or change. Generally, I think the continued focus on speeding the availability of therapies to patients is the key driver to change but it is coming in a number of ways. This can include accelerating the drug development and approval process to understanding how to help patients with rare diseases in emerging markets and harnessing the incredible science and technology that’s at the cutting edge of it all. This includes research to determine the incidence, prevalence, and natural history;
the development of improved screening and diagnostic techniques; information to the general public, education programs for healthcare professionals; and infrastructure changes such as biobanks and registries.
What would you like to gain from our congress?
The World Orphan Drug Congress continues to be one of the largest gatherings for the rare disease community in Europe. I’m looking forward to meeting leaders in industry, government, payers, and academia, and, importantly, talking with patients and their advocates.
To hear more please visit http://www.terrapinn.com/template/live/documents/6648/15144 to book your place so you can meet key speakers like Bert as they discuss multi-stakeholder collaborations to benefit patients with rare diseases.