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Optimising market research data in rare disease

Helping to provide powerful insights into the burden of disease and the patient-caregiver experience.
Employing market research to map the rare disease patient journey can provide powerful insights into the burden of disease and the patient-caregiver experience.   Understanding more about their emotional journey, pathway to diagnosis and quest for effective symptom management and/or treatment support can help identify more clearly these patients’ unmet needs.

Findings from primary market research among rare disease patients and caregivers can also help inform targeted communications strategies and customer support services for companies developing solutions for these patients, as well as exploring the barriers/drivers to clinical trial participation. Just as importantly, sharing these significant and valuable learnings can add critical information about the disease area into the evidence base.

Healthcare specialist involvement in the design of market research initiatives can enable the research to be conducted in such a way as to verify if the information being researched is worthy of publication and subsequently provide evidence to support external communications and activities. Findings that can be disseminated through these channels can help to broaden reach and increase awareness about the specific issues and challenges facing these rare disease patients and their support network. 

Additionally, patient recorded outcomes are becoming increasingly important and market research can be utilized to define which symptoms are of particular importance to the patient. These may be different than those perceived by the medical profession. However understanding what symptoms are most significant means that they can be built into clinical studies and either be useful for aligning patient needs for therapy or explaining the benefits of treatment in ways that resonate with a patient and healthcare professionals.   

Gaining a robust picture of the entire patient journey and developing a clearer understanding of these rare, often life-limiting, conditions and the burden of disease is not easy due to the smaller patient populations, but is essential.   Having deeper understanding of these diseases can help to identify the most beneficial medical care and appropriate support for these patients and their families. 

It is therefore crucial to employ a multi-pronged approach to recruitment, maximising the opportunity to identify appropriate individuals to participate in market research.   Recruitment with the support of key opinion leaders (KOLs) and collaboration with Patient Advocacy Groups (PAG) or Patient Support Organisations (PSO) and their members is critical.   Building trust, demonstrating ethical working methods, and having mutually beneficial goals allows these relationships to blossom and leads to success.

Careful consideration as to who should be involved in the market research needs to be given. The patient is the main focus point, however if they are not able to participate because they are too young or not physically/mentally able, then the primary caregiver, often the parent, spouse or sibling, acts as the main respondent.  The broader support network within the family unit can also provide unique observations, so it is best practice to include secondary or even tertiary caregivers to make sure a complete picture of the patient journey is gathered.  Caregivers and patients’ families have multiple roles, and are an integral part of the disease and its management. They are not simply a proxy for the patient who is too young or too ill; they are also information seekers, advocates and influencers on the disease course and treatment journey, and they have a huge influence on the choices the patient themselves makes.

As the patient population is typically widely dispersed, a blend of digital and traditional methodologies is the most effective way to ensure sufficient evidence is gathered. Using multiple approaches enriches the length of time or interaction a market researcher has with each participant, providing far more detailed insights, and the opportunity to understand the moments that matter outside of the traditional market research setting. Considering the challenges of recruiting patients/caregivers in rare and ultra-rare diseases, this approach maximises the opportunity to delve deeper and ask as many questions as possible.

The spectrum of unmet needs in rare diseases can be far reaching from both a direct and indirect result of the disease. Unearthing these needs, can help to inform commercial strategy for Pharma companies where they have the ability to influence or provide solutions. Once clear gaps are identified, companies are able to develop valuable targeted activities and customer support services for patients, where none currently exist.  These can be as broad as helping to define a referral pathway, identification and development of support information needs, or providing funding for support services or home equipment for patients.  Given the close-knit nature of rare disease communities, pharmaceutical activities are often carried out in partnership with the PAG/PSOs. This ensures the needs of all involved in the rare disease are met and that the new initiative is adopted/implemented effectively. 

Given the limited clinical or academic research conducted in many rare diseases, in part due to the small number of patients and dispersed nature of the populations, global market research studies can often reflect patient populations either similar or even larger in size to those in the clinical trials. Insights generated and the potential benefit these findings can provide are hugely important, so should, wherever possible, be shared more broadly with clinical/academic circles through publications and abstracts.   Information disseminated through peer reviewed literature can broaden reach and increase awareness about specific issues and challenges facing these patients.

In order for the market research data to be accepted as ‘ethical and appropriate’ within clinical and academic circles, it is essential to involve either KOLs or a steering committee of experts experienced in the therapy area at the outset. They can help to guide the research by refining the objectives, feeding into the design of the materials, and translating insights into meaningful actions for the rare disease community.  Building in planning outside market research maximises the data outputs and can ensure it is of appropriate design to publish.  Therefore, understanding what is good practice from both the market research and publication side is critical.  Opportunity costs are high and often cannot be replicated, so working with an organisation that can provide the expertise to deliver on both these elements is helpful. 

Where it is not possible to publish research findings, sharing findings of the research with the PAG/PSOs within the area, who have supported recruitment of respondents is hugely beneficial to broadening the reach of the findings. Of course, some content of the market research may be deemed commercially sensitive, but by offering any insight into the findings or providing time within the interviews for the PAG/PSO to ask questions pertinent to their needs helps to build as clear a picture as possible about these rare conditions.  

Understanding the full impact of a rare disease on the patient in its widest possible context, mapping the journey to diagnosis and identifying the unmet symptom management and treatment/support needs through market research are instrumental in advancing the understanding of these conditions.  Maximising the exposure/reach of this knowledge by sharing information with key stakeholders and publishing the data in the peer reviewed literature can help overcome delays in diagnosis, improve symptom management and/or treatment support for patients and ultimately can help to improve their quality of life.

Therefore, when considering market research in rare diseases it is important to consider not just how to accomplish the research but its broader value and potential relevant dissemination. Working with a partner organisation who not only has the research skills, but the consulting and communication knowledge across all stakeholders is therefore invaluable.  

The AuthorClaire Ajdukiewicz is a Director at Cello Health Insight,  Tom Atkinson is a Group Business Director at Cello Health Insight and Steve Smith is Chief Commercial Development Officer, Cello Health Communications Europe.  For further information please visit

3rd October 2016



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Cello Health

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