With the 5th World Orphan Drug Congress on 12- 14 November in Brussels only just two months away, I conducted an interview to one of our key speakers, Dr Jonathan Morton (Communications Director, Rare and Orphan Diseases Practice at Oxford PharmaGenesis™ Ltd) to give you further insight into his exciting panel discussion as well as his top priority orphan drug challenges he hopes to discuss.
Dr Morton has spent his entire career working in the rare diseases field. Since 2012 he has led the Rare and Orphan Diseases Practice at Oxford PharmaGenesis™ that has provided expert guidance to various stakeholders seeking to improve standards of care for patients with rare diseases. His panel, “A problem shared… how can multi-stakeholder collaboration facilitate patient access initiatives?” will have participants from the MPS Society, PPi Ltd and PTC Therapeutics.
Q: What are some of the biggest challenges you are facing and how does your talk try to address this?
I believe our greatest challenge is to help the rare disease community to collaborate effectively for the benefit of patients.
In the 8 years that I have been working in the rare diseases field, there has been a dramatic increase in the mobilisation of the patient community, ranging from participating in clinical trials to patients and/or carers setting up and driving their own drug development programmes. There is also greater investment in rare diseases by biopharmaceutical companies than ever before, and many European countries now have a specific rare disease plan in place, something that was almost beyond comprehension a decade ago. And yet, as a community we often seem to face the same challenges in ensuring patient access to effective treatments. Isn’t it time we took a step back and took a fresh look at the entire picture?
In our panel discussion, we will consider how multi-stakeholder collaboration might facilitate patient access initiatives. My view is that the potential influence of the rare disease community is greater than the sum of its parts. Thus, it is in the interest of us all – biopharmaceutical companies, clinicians, research scientists, patient organisations, patients and carers – to find ways to collaborate effectively to identify and develop novel life-saving therapies, and to build and communicate a compelling case for their approval and reimbursement. For this to be truly effective, it is essential to engage in an open dialogue with regulators and payers so that we can ensure they have all of the information they need to make objective and appropriate decisions. This is a departure from the traditional model of separation between industry, regulators, payers and ‘customers’, but I believe that it provides the best opportunities for improving patient care.
Q: How do you think changes to licensing and approval processes (i.e. fast track and adaptive licensing) will impact on the sector as a whole?
Any mechanism by which patients may gain early access to potentially life-saving therapies is to be welcomed. The need to continue to gather efficacy and safety data is not a great departure from the situation for many orphan drugs that are granted marketing authorisation under the current system, so the biopharmaceutical industry should have no difficulty adapting to the new processes. It is worth noting that in addition to valuable information on effectiveness and safety, the long-term collection of real-life data can provide invaluable insights into the practicalities of administering a new drug outside the of clinical trial setting; this can have a real bearing on adherence and patient quality of life. My hope is that the advent of adaptive licensing will lead to greater transparency about the benefits and disadvantages of novel therapies, which will in turn lead to more informed decision-making when full marketing authorisation is considered. This is surely to the advantage of patients.
Q: How do you think the role of patients/patient advocates/patient groups will change?
Patients have a unique understanding of their condition and how it impacts on their everyday lives. This perspective is likely to become increasingly important as we look for new ways to measure disease outcomes and understand the real-life benefit of novel treatments. Patient organisations have a key role to play in harnessing the patient voice to help inform research initiatives and drive improvements in standards of care. This is already being achieved in some therapy areas, and it is likely to happen in other areas soon as the rare disease landscape evolves.
Q: What would you like to gain from our congress?
Like many of my colleagues in the rare disease community, my ultimate aim is to find ways to ease the burden faced by patients and their carers. As an output from our panel discussion, “A problem shared… how can multi-stakeholder collaboration facilitate patient access initiatives?”, we would like to be able to agree and document some general principles and mechanisms for working together as a community to develop a comprehensive and compelling case for marketing authorisation and reimbursement, so that regulators and payers can make informed and appropriate decisions. This would be an important step towards improving standards of care for patients with rare diseases.
To hear more please visit our website to book your place so you can meet key speakers like Dr Jonathon Morton as they discuss multi-stakeholder collaborations to benefit patients with rare diseases.