Expertise / Rare Disease

A focus on patients with rare diseases

With broad expertise in bringing therapies to the people who need them; our rare disease capabilities cover the full lifecycle. From improving diagnostic and treatment pathways, to impactful scientific and value communications through to innovative patient support and outcomes monitoring.

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We know the ins and outs of the complex rare disease market

OPEN Health is highly invested in the delivery of positive outcomes in rare disease. We have extensive experience across our practices having worked with many clients within the area of orphan and ultra-orphan medicine.

Our broad capabilities help us to recognise that every scenario is unique and we therefore apply bespoke, cohesive solutions to ensure optimal outcomes are achieved for our clients and the patients they serve. Over 300 of our 450 staff have direct rare disease experience, and as a group we have partnered with over 20 rare disease-focused companies in the last three years.

Video screenshot of Gavin Jones.
OPEN Health invest to support outcomes in rare disease

Watch the video »

Gavin Jones Profile.

The face of rare disease expertise at OPEN Health

To optimise our capability and truly meet the needs of our customers we have invested in the deployment of a Director of Rare Disease to advise and work across all of our practices.

Gavin Jones has over 20 years of biopharmaceutical industry experience across numerous roles involved in market access, therapy launch and patient centric services. For 15 years he has developed a specialist interest in rare and ultra-rare disease supporting many orphan therapies as they have been commercialised. He is passionate about supporting optimal patient outcomes through effective therapy awareness; diagnostic and treatment pathway improvement and patient activation.


OPEN Health invested in outcomes with Advanced Therapies

It is a time of high hope and expectation within the rare disease and oncology communities given the increasing numbers of advanced therapies coming to market. OPEN Health has built significant experience in this space but we come with the humility that we are all learning together in these unprecedented times.

We have created a solutions incubator bringing together our capabilities and experience to deliver for our clients and the patients they are striving to serve. This includes the recent publication of a white paper:

White paper

Succeeding with Star Wars medicine: Translating the science and value of gene therapy to your key stakeholders

White paper

Watch Gavin in conversation with two leaders from the patient community and industry discuss the key themes

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A proud partner of Rare Revolution magazine

We always strive for a ‘patient first’ approach in rare disease and are therefore honoured to be partnered with Rare Revolution magazine.  We value the opportunity to share our current thinking on various important topics in rare disease and contribute to their mission of amplifying the patient voice in rare disease.

Rare Revolution magazine logo.

Read the articles

Our thinking in Rare Blood

After many years of creeping evolution we now are in the midst of raid evolution in the management of haemophilia and other rare blood disorders. We look at how Pharma support more personalised approaches to management of these conditions enabling patients to live more freely of their condition whilst feeling protected from adverse events.

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Our thinking in rare oncology

At a time of increased innovation in diagnostics and the growth of more personalised medicine; rare cancers could in fact be seen as becoming more common. We discuss how communications can be effectively delivered to support better outcomes for patients living with rare and less common cancers.

RARE Revolution - RARE Gene Therapy Edition
Our thinking in gene therapy

It is a fascinating time; we are moving from a trickle of gene and advanced cell therapies to a potential torrent of new treatments coming to market. This is going to create unprecedented challenges and opportunities and world class communications will be required to maximise their potential.

Rare revolution winter 2020
Patients as Partners

Patient centricity continues to grow in priority within Bio-Pharma’s clinical and commercial strategies. Given the high stakes and continued unmet need; rare disease communities must be at the forefront of effective patient partnerships that remain compliant and sustainable. We share our ‘patient first’ thinking here.

Professionals and Places

In these challenging times the rare disease community must come even closer together and harness efforts to continue improving outcomes for patients and families living with a rare disease. We look at how to harness this collective power and create connectivity between the various key stakeholders to deliver better outcomes for patients.

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Patient Transition

Given improvements in disease management children and adolescents with rare diseases are living longer. Underneath this positivity is the challenge of effectively supporting these patients into adulthood. We will consider the challenges in transition and how Pharma can support in helping positive patient experience as they transition from paediatric into adult services.

Benjamin James, Trustee of DMD Pathfinders.
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Improving Communications
in Rare Disease

Benjamin James is an active member of the patient community and a Trustee of DMD Pathfinders. He spent the last year completing a Masters in Scientific Communications and based his dissertation on a critical analysis of communications in rare disease using OPEN Health as a case study. 

Rare disease patients present unique challenges including delays in diagnosis, lack of expertise about the disease, limited data and poorly defined patient pathways. 

The unique nature of these rare diseases means communications require innovative approaches to ensure the needs of the rare disease patient are met. This is true in terms of the launch of orphan drugs and their commercialisation.

Communications about these treatments must be delivered in an effective and innovative way, ensuring therapies can be provided to meet the needs of the rare disease patient. These communications are complex and dependent on a number of factors including information, data and evidence, strategy, plan and launch, and patients and stakeholders. This complexity is associated with a number of challenges that need to be overcome. This will ensure patients can access life-changing therapies that are coming to market now and in the future. 

Rare disease launch - key considerations

The challenge of diagnosis
Influence of PAGS & public perception
HCP endorsement across patient pathway
Often limited data platform
Expedited approval timelines
Pricing & reimbursement
Patient pathway ill-defined
Adherence to treatment & follow up

Get in touch

For more information on how we can support your journey in rare disease, get in touch.

Gavin Jones - Director of rare disease, OPEN Health