OPEN Health reflections on the World Orphan Drug Congress in Barcelona

Written by Gavin Jones on Wednesday 20th November 2019

Having just returned from a very busy but hugely enjoyable few days at congress, I wanted to share our views on the event and key themes that our healthcare communications practices took away from the discussions and presentations. 

It was great to see a higher proportion of attendees from patient groups as early patient engagement was a strong message from a number of presentations.  I was happy and proud of the response to our session which gave the floor to two patient representatives providing their own perspectives on improving communications in rare disease.  We were pleased to see other companies including the patient voice in their presentations, but we must go further and increase the number of patient-led sessions in 2020!  The gauntlet is thrown down and we for one will certainly return with this firmly in focus. 

I was also proud to attend with some brilliant folk from across the OPEN Health who discussed challenges and opportunities in rare disease with many of the delegates attending.  It reinforced to me that our passion and commitment to better outcomes is now deeply embedded in our organisation.  Below is a summary of their reflections: 

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Louise Carrington – representing our Medical Communications practice 

The importance of effective communications to patient and healthcare professionals (HCPs) throughout the development of an orphan drug was a key topic of conversation during this conference. It was truly valuable to hear the perspective of all stakeholders – patients, payers, clinicians, regulators, as well as biotech/pharma R&D, medical and commercial teams.  Recognising the complexity of the therapies and associated referral and treatment pathways; and then building communications plans which are clear, demonstrate the value and reach the right audiences is critical.  

Whilst no doubt there are challenges as we move rapidly into unknown territories, it was great to see the breadth of opportunity to collaborate and build valuable networks with patients and HCPs which is needed more than ever in rare diseases. In particular, the introduction of regulations around the development of patient lay summaries creates an opportunity to involve patients early, co-create with and hear their expert voices, to ensure that these valuable treatments reach the patients that need them.   

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Marie Keetley – representing our Value Insight and Evidence practice 

It was an incredibly stimulating conference that created the opportunity to understand the challenges that both industry and patients are facing in gaining access to drugs. Much of the discussion from the speakers and audience was to encourage early engagement with clinical development, regulatory and health technology appraisal agencies. As randomised controlled trials become less feasible in rare disease, due to small patient populations and advanced treatments requiring longer term follow up, there is an even greater requirement for real-world evidence and patient-reported outcomes.  These will help generate the data required by regulatory authorities and payers to help get drugs to market. 

It was also encouraging to see patients and patient advocates present at the conference sharing their remarkable and personal stories and providing their unique vision on clinical development and access to treatment.  Although there is still some way to go, we are delighted to see that patient involvement in research is being recognised. We are very proud of the fact that it has been our standard practice in our initial study designs and by employing this approach we can ensure the success of our programmes. 

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Ed Senior – representing our Patient and Brand Communications practice 

Pharma is making great strides in involving patients early in their strategies. As there are more and more examples of true patient involvement and decision-making, it will become the norm and the patient voice will be routinely integral to decisions that govern their care. It was great to learn about examples and I hope these form a springboard for more next year. 

The session on improving healthcare communications in rare disease was a truly collaborative and patient-centric presentation, with OPEN Health’s Gavin Jones co-presenting with Benjamin James, patient advocate and David Rose from Rare Revolution Magazine. This session underlined not only the importance of early engagement with patients, but also the crucial role that Patient Advocacy Groups play in the appropriate partnerships with Pharma to deliver better outcomes. Benjamin’s research observations are being developed into formal recommendations for pharma-patient engagement which will be another key step in this process. 

At the conference, smaller biotech companies attended alongside big pharma and it was evident that OPEN Health can play an important role in helping them develop the right communications to patients, public and healthcare professionals. OPEN Health applies multidisciplinary thinking to our client’s challenges, creating bespoke teams to provide these organisations with the structure and expertise to guide new products with early insight and communication solutions before launch. 

I hope OPEN Health will be back at the congress in 2020 and we look forward to meeting friends and colleagues in Barcelona again soon. 

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If in the meantime you would like to discuss how we could help you deliver better outcomes in rare disease then please contact me: 

Gavinjones@openhealthgroup.com