Open Health

Delivering change through better rare disease communications

OPEN Health’s Richard Jones, Gemma Allen and Charlotte Richards discuss how pharma can utilise tools, channels and stakeholders to deliver change and enable better outcomes.

Many innovative therapies are being launched in rare disease following years of research that has been driven by the emergence of new technology and regulatory incentives. This brings high levels of hope and expectation to the rare disease community. Consequently, it’s vital that biotechnology and pharmaceutical companies cut through the noise to bring real understanding and informed decision making about the next generation of orphan drugs. Doing this requires communications that encompass the right mix of tools, methodologies, channels and people.

Exploring the challenges in rare disease through better communication strategies

OPEN Health recognises that there are multiple challenges in commercialising new therapies in rare disease and is leading future practice in communications by placing emphasis on a ‘patient first’ approach. This includes supporting Benjamin James’s recent analysis on what the future could look like for outcomes driven communications in this space. Benjamin is a trustee and patient advocate for DMD Pathfinders and has recently completed a Masters in Scientific Communications. This provides us with a valued perspective on how improvements can be made for the benefit of patients. A summary of his findings can be found here.

Benjamin commented: “Rare disease patients present unique healthcare 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.

But, he added, communications are complex with a number of challenges that need to be overcome if patients are to access the life-changing therapies that are coming to market now and in the future.

This complexity can be overcome to deliver better outcomes for patients and the pharmaceutical industry through the application of integrated communications and tools. These can be used to connect HCPs and patients across broad geographic, diagnostic and treatment pathways, while enabling the patient advocacy community to be partners in positive change.

Capitalising on opportunities with digital technologies

Digital technologies have huge potential in rare disease to support connectivity and drive collaboration, improve route to diagnosis, quantify impact of a condition on quality of life, and increase both access to treatment and recruitment into clinical trials.

Connectivity: In terms of adding value within rare disease, the internet and digital technology are the ultimate networking tools, helping people to connect, share information and generate new ideas and possibilities. That is also true for HCPs, who may need to connect to expert peers for advice on managing rare disease patients who are often geographically and clinically diverse. Furthermore, digitally enabled access to information and community support is so important in rare disease, with virtual ad boards, webinar programmes and virtual centre of excellence communities among the ways digital platforms can effectively connect HCPs to share and learn from each other.

Route to diagnosis: Digital health technologies can also empower families to understand their conditions or symptoms more broadly, help to connect the dots and hopefully reach the point of diagnosis much more quickly. It can also be valuable to discount certain conditions as diagnosis by exclusion is often how rare diseases are eventually identified. It is in this space that artificial intelligence can be used effectively to see patterns where perhaps humans, either patients or healthcare professionals, can’t. That might be because they either don’t see the volume of patients with that condition or they’re just unable to see the broader holistic picture. However, for AI to be more effective, the number of patient registries and access to data needs to increase so that there is sufficient scope to develop and train the algorithms.

Impact of condition and value of treatment: Quantifying the impact of a rare disease condition on someone’s quality of life can also be supported by digital technologies. Some rare diseases can manifest themselves uniquely in each patient, so trying to quantify the difficulties and symptom severity can be a challenge for healthcare professionals and researchers. Wearables and other digital tools that provide an objective view of a patient’s life can collectively build a picture of the rare condition across multiple patients. From this baseline, the impact of a treatment can then also be measured, adding critical real-world evidence (RWE) data to the value proposition of a product.

Access to clinical trials and improving RWE data gathering: Developing new treatments requires researchers in rare disease to overcome the challenge of finding and recruiting patients into clinical trials, who are by their nature, rare. It is also common to see long-term follow-up being a frequent hurdle. Patient advocacy groups can be instrumental in connecting researchers to patients, and this is more evident in rare disease. Improving awareness by providing online information to people looking to access clinical trials, many for conditions where no existing therapy is likely to be available, and connecting them to researchers is critical to the development of treatments in this area. Once involved in research, there are also opportunities to improve patient engagement in clinical and RWE studies through gamification theory. This uses the same powerful principles that are central to the gaming industry and brings them into data gathering applications. This can encourage patients to maintain engagement over the longer term, helping to gather more data on these rare conditions.

Ultimately, digital technologies are providing the tools and platforms to empower patients and HCPs to overcome some of the many challenges associated with living with, and treating, rare diseases. The hope is that this continues to drive research, access to treatment and the emergence of new therapies to address the significant unmet need.

Data, evidence and communicating value

Early communication with clinicians and payers is vital to ensure that clinical development programmes meet the needs of these stakeholders. Pharmaceutical companies must engage with patients, clinicians and payers as early as possible to best understand evidence and educational needs. By doing this pharma companies can build a robust aspirational value proposition that resonates across stakeholder groups, identify data gaps and develop plans to generate evidence in order to support this positioning and secure positive HTA outcomes.

Affordability of orphan drugs – and consequently payer acceptance of the value proposition – is a challenge within rare disease. Due consideration should be given to not only what evidence may be needed to strengthen the clinical data package and support a new medicine’s reimbursement dossier, but what long-term data collection requirements exist and how a data collection programme can be optimised in order to meet requirements and further strengthen the data platform.

Patient reported outcomes data are important in demonstrating that investment in a therapy will provide value beyond a clinical trial. One of the key questions to answer early on is which patient reported outcome measure may mean the most to patients and will also resonate with payers and clinicians. Pharmaceutical companies also need to know which of the endpoints payers will consider most important, what the comparator for cost-effectiveness at the time of launch will be, and what impact this has on comparator selection for the clinical trial.

At the same time alternative value and payment models should be considered. Several pharmaceutical companies are now considering innovative funding mechanisms. These include:

• Annuity based payment model:
This model involves payment by reimbursement agencies to manufacturers for a high-cost drug over a set period of time, which can be many months or even years. In a simple annuity model, there is no link to drug performance.

• Subscription models:
A ‘Netflix’ model, or subscription model sees reimbursement agencies and pharmaceutical companies negotiate a contract whereby an upfront fixed fee or a fixed monthly fee is paid for unlimited patient access to a specific treatment for the duration of the contract.

• Value-based pricing:
Under value-based pricing agreements, risk is shared between payers and pharma companies. An agreement is made to link payment for a medicine to value achieved, rather than volume. What constitutes value needs to be carefully defined and agreed by both parties and measured through a registry in order to collect the data needed to execute the agreement.

Whatever payment model is agreed and implemented, it’s important to gain insights from payers around its acceptability and the ability to implement the model within different healthcare systems. The requirement for registry and long-term outcomes data should also be considered, including how to engage patients and clinicians in the process.

Patient advocacy groups are often experts in their disease and can be an active part of the HTA process in many European countries. Therefore, elevating the patient voice is critical to help ensure reimbursement discussions around a particular therapy truly represent patients’ needs.

Tools and communications to engage the rare disease patient

Special consideration needs to be given to communication in rare disease and the tools used to achieve effective engagement. It goes without saying that when working with such a small patient population your communication strategies must be compliant and have transparency of intent at the heart of any engagement. Here are two examples of how patients have been appropriately partnered to inform better communication solutions:

Co-creation in an ultra-rare condition: Developing clinical study protocols in rare and ultra-rare conditions can be extremely challenging. Low patient cohorts that are geographically dispersed mean that you need a study protocol that you know will engage patients and retain them for the duration of the study. We worked with patients, their siblings and carers, and partnered with the patient advocacy group to co-design a clinical trial application to assist in educating patients and caregivers and improve retention. For an ultra-rare condition affecting just one in two million people the engagement was astonishing. Patients travelled the length and breadth of the UK and even flew in from Europe to take part and give their time to try and help improve outcomes for others affected by this condition. We are continually moved by patients’ willingness and desire to give of themselves to help improve outcomes for future generations. Co-creation provides a valuable mechanism to receive their feedback and provide them with a platform to share their views.

Patient registries in gene therapy: One of the core challenges with rare disease gene therapy is that patients are treated by a small number of specialist centres and then often referred back to their local care centre for ongoing treatment and monitoring. However, there’s no mechanism to capture patient outcomes across different care settings. A patient-led registry that encompasses the whole data ecosystem was developed, allowing both centres of excellence and local care centres to input data, and give patients the ability to input quality of life data. The challenge of achieving long-term patient engagement is fundamental to the success of any registry. It’s an interesting challenge; a patient receives a potentially life-changing treatment of gene therapy which allows them to lead a life less restricted by their disease, but then we ask them to participate in a long-term registry to collect important health data and ask them frequently about their disease. We worked with behavioural health psychologists to develop an engagement programme that ensured participation by patients and clinicians in a gene therapy registry over an extended period.

Across its healthcare communications and value and access practices OPEN Health has significant and long-term experience in rare disease but has taken its investment in better patient outcomes much further. The company has invested in a director of rare disease, Gavin Jones, who works across its practices to ensure solutions truly meet the needs of clients and in turn the patients they are striving to serve.

Gavin said: “It’s a real privilege to work with in-house experts like Gemma, Charlotte and Richard, and I also truly value working with committed people like Benjamin across the patient community, industry and healthcare. Harnessing the combined efforts of these individuals will be essential to delivering better outcomes for patients at a time when there are many reasons for hope within the rare disease community. World class communications, and the appropriate tools and channels to facilitate these, will be required to enable change and lead to rapid and sustained patient access to innovation.”

Navigating today’s rare disease landscape requires moving beyond existing ways of working to better translate, and communicate, the potential value of orphan medicines and truly inform decision making across stakeholder groups. In parallel with this, the use of digital tools has an integral role in communications strategies to alleviate the isolation that patients, their caregivers and HCPs may feel when treating or living with a rare disease.

Each rare disease has a unique commercialisation journey and patients’ priorities and needs will differ according to the condition, or conditions, they have. Consequently, elevating the patient voice is critical for ensuring that rare disease patient needs and expectations are met. This in turn will help inform the value story and decision making on treatment and reimbursement decisions which will ultimately lead to better patient access and outcomes.

About the authors

Richard Jones

Richard Jones, Managing Director, Patient Engagement, OPEN Health
Richard is a highly successful, commercially astute leader and problem solver with a proven track record in senior roles in the pharmaceutical industry and healthcare agencies. Richard combines 20 years’ commercial pharmaceuticals knowledge from GSK, Astra Zeneca and Pfizer with 10 years specialist agency experience. This unrivalled pedigree enables Richard to quickly understand his client’s business and offer innovative, differentiated and effective solutions. A senior executive, comfortable at operating strategically, above practice and respected by C-level customers. Richard has a passion for crystallising issues on patient engagement and developing innovative and effective solutions.

Gemma Allen

Gemma Allen, head of digital communications, OPEN Health
Gemma has over 12 years’ experience working in the pharmaceutical industry. She has worked closely with a number of the top 20 pharmaceutical companies to deliver their digital projects. Gemma has specialised in digital healthcare for the last 7 years of her career and has delivered internal data sharing platforms, multichannel campaigns, interactive iPad presentations as well as digital training. She has been responsible for building digital market access tools and educational HCP online communities as well as implementing content and engagement strategies for several web platforms. Gemma has a strong scientific background, previously working in R&D for GlaxoSmithKline and as a consultant in Market Access.

Charlotte Richards

Charlotte Richards, Customer Strategy Director, OPEN Health 
Charlotte has 12 years’ experience in the pharmaceutical industry spanning pharmaceutical marketing and sales, medical communications, strategic consultancy and real-world evidence and market access. She has a keen interest in rare disease and gene therapy and has supported development of a number of integrated programmes across OPEN Health to drive access to these important interventions and improve patient outcomes. Charlotte is a strategy director for OPEN VIE, our value, evidence and access practice.

About OPEN Health

OPEN Health is a global healthcare communications, value and access company with extensive experience and expertise in rare disease. Its best-in-class practices work together seamlessly to provide bespoke solutions that meet the unique challenges in rare disease and deliver world-class communications and tools that deliver better outcomes. OPEN Health is fully invested in this space with a dedicated director of rare disease working across all of our practices to inform its solutions. Gavin supports all of our value, medical and patient and brand communications practices in delivering programmes that truly meet our clients’ needs in rare disease.

Please contact or visit to find out more.

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