OPEN Health’s virtual reflections on the World Orphan Drug Congress US

Written by Gavin Jones on Wednesday 2nd September 2020

Another congress attended in this virtual world with learnings gathered and connections made across the rare disease community. It was great to be joined by many colleagues from all our disciplines demonstrating the enduring commitment of our people to make a difference in rare.

The week kicked off with many of the sessions directly focused on patient engagement and involvement in bringing new rare disease treatments to fruition. Hearing the depth of knowledge and experience within patient groups and non-profit organisations was fantastic with clear messages around engaging early and often.

We were privileged that Emily Crossley (Duchenne’s UK) and Betsy Bogard (AVROBIO) joined our session on ‘Succeeding with Star Wars Medicine’. It was great to have both patient and industry views from two people wholly committed to playing their part in bringing gene therapies to patients. Both expressed the need for greater levels of partnership to overcome the unprecedented challenges in bringing gene therapies to market as the trickle becomes a torrent.

Later in the week more focus was placed on topics related to demonstrating value of new innovation in rare disease and advanced therapies. These topics are so important in rare given the unique considerations required to enable sustainable models to future proof access to novel treatments and drive further investment in rarer conditions. We were invited onto a panel looking at experiences of using real world evidence (RWE) to support access. This gave us the perfect opportunity to include our integrated expertise from Pharmerit with the insightful views of our head of RWE in the U.S. - Shelby Corman.

Some of my super colleagues who attending with me will now give their learnings through the lens of their specific disciplines.

Trish Shepherd – representing our Patient and Brand Communications practice

What an amazing week of inspiring stories. There were so many themes covered from innovation to resilience to collaboration and finally the unyielding determination of so many – patients, PAGS, researchers, life-science organisations, regulatory bodies and support agencies alike. It is hard to know where to begin but as a member of the OPEN Health practice committed to delivering thoughtful patient engagement, I feel compelled to concentrate on one word – stories.

A recurrent theme that struck me was the importance of listening. The profound impact listening is having in not only helping to support patients and families through their daily challenges. But, more important, is the recognition that patient journeys and truly understanding the lived patient experience – from a physical, emotional and practical sense – is having in the sphere of drug development and regulatory approvals.

To paraphrase Luke Rosen from OVID and KIF1A – in the context of clinical trial protocol: to ask for feedback on a protocol equates to failure. It is only when we include patients and families in the design and intent of clinical trials programmes where we will we see true influence. Working in the biopharmaceutical industry and as a father of a child with a rare disease he knows the value of listening all too well.

We learned throughout the week that more than an estimated 40% of future drug approvals will concentrate on rare diseases and genetic disorders. However, we have also learned that, even with the significant unmet need that exists with many rare diseases, recent advances still fail to achieve prolonged commercial success.

When we truly stop to listen and ask the right questions before we engage in clinical trials and commercial strategies – we may be able to improve the long-term success of these vital interventions that hold the promise of changing the face of the disease.

If you are interested in learning more about the approach OPEN Health takes to uncover the moments that matter most to patients and families, please get in touch. We would be privileged to take a few steps towards making a positive change in the journeys of so many impacted by rare disease and genetic disorders.

Charlotte Richards – representing our Value Insight and Evidence practice

The standout message around the need for a patient-first approach being integral to the long-term commercialisation strategy in rare disease really resonated. Early engagement with rare disease patients and caregivers allows for development of patient-centred research plans. These plans should be validated with Payers in order to build appropriate clinical trial and real-world evidence generation strategies that demonstrate value across stakeholder groups and reduce value uncertainty at launch.

The need for early and efficient patient identification was another theme that resonated. Using techniques such as machine learning to support healthcare professionals in their diagnostic endeavours is an important tool in rare disease, supplemented by targeted disease awareness and appropriate service provision.

It was great to hear so many passionate speakers throughout the conference, clearly determined to drive access to life-changing treatments for the right patient at the right time - something that we are very much behind.

Louise Carrington – representing our Medical Communications practice

With many experts meeting virtually for this invaluable conference, the theme of ‘collaboration for success’ came across strongly. Orphan drugs and advanced therapies no doubt come with high expectations from patients, carers and clinicians. As such, early (and frequent) collaboration with stakeholders to overcome challenges to commercialise these medicines are imperative.

Improving the diagnosis of rare diseases is a typical challenge and it was interesting to hear the innovative solutions being explored. With collaborative initiatives, such as the Global Commission, it was exciting to see projects using advanced data, AI and ‘intelligent triaging’ (including virtual panel consultations with geneticists). Finding clinician networks, ensuring the cascade of disease awareness and education, and clearly communicating complex disease evaluation and endpoints in clinical trials, is paramount.

Additionally, as fully discussed during the conference, demonstrating the value of orphan therapies is essential for broad and ready access. The continued importance of communicating value of a drug was noted, and in doing so, balancing the needs and definition of value among HCPs, patients, and payers. Equally, it is important to look at value from the “here and now”, but also in the future (particularly with curative therapies).

Finally, the industry’s response to COVID-19 has demonstrated that, together, advances can be made quickly. Employing that sense of urgency, collaborative mindset, and building effective virtual communications that use learnings from the past few months, will be the key to success.

In conclusion, this congress has ‘US’ tagged to its name but it is very clear from the dialogue that we face Global challenges that require Global solutions. Our broad capabilities and footprint mean we are confident we are the right partner to deliver Globally for better outcomes in rare disease.


For information about OPEN Health’s services and how we could support you, please get in touch.


Visit our careers page to view opportunities at OPEN Health.