Recruiting and retaining patients for successful drug development

Patient advocacy group

Craig Stevens, Senior Patient Engagement Specialist, and Amanda Henkel, VP, Strategy from MEDiSTRAVA (an Inizio Company), delve into the challenges and opportunities that surround clinical trial design for rare diseases.

The drug development process requires collaboration at all stages, and is particularly important in the rare disease setting, where in addition to pharmaceutical industry stakeholders, patients and patient advocacy groups play a central role. The ultimate aim of any clinical trial is to find a cure, but many complexities and challenges are faced along the way to success. The rare disease setting is one of the fastest growing areas for biopharmaceutical investment, but rare diseases are inherently heterogenous, with too little known about the disease or its course. Success relies on patient identification and carefully planned and effectively implemented clinical trials – and with low numbers of patients to recruit from, and disease conditions that often add barriers to participation and completion, the clinical trial design is of paramount importance to success.

Opportunities for orphan drug development

Incentives for orphan drug development have changed how the pharmaceutical industry approaches rare diseases. Since 1983 when the orphan drug act was introduced in the US, over 5,000 drugs and biologics have received orphan designation, with the number tripling between 2000 and 20101. Similar incentives and legislation have been put in place across the globe, for example with the EU’s ‘Regulation (EC) No 141/2000’ in 2000. However, the market still has room for growth. Around 350 million people are affected by a rare disease worldwide and the total number of rare diseases is estimated to be between 7000 and 8000 (although the true number could be substantially higher) – and most of these still don’t have a therapeutic option2. 

Although the enhanced focus on rare disease therapeutics has been good for the sector, there are still many challenges to successfully commercialise an orphan drug. For rare disease trials, which are typically complex, utilising data to deliver a targeted patient recruitment and communication strategy can be the difference between trial failure and success.  

Empowering patients 

Clinical studies in the rare disease setting face significant challenges, but also great opportunities for better engagement with patients and their caregivers, as well as with the wider community. One of the biggest difficulties encountered is with patient accrual, ensuring that the resulting data are both impactful and progress knowledge in a timely manner.  

Research has shown that over half of rare disease clinical trials are discontinued or fail to publish their data within four years of completion, mostly due to these challenges with patient recruitment and retention2. This statistic is not only far from ideal, but it also highlights some major issues in clinical trial design.  

To ensure that low patient accrual isn’t a cause for trial failure or inability to publish data, patients should be empowered to help design better, more effective clinical trials. Involving patients in trial design ensures that real value is brought to patients, enabling a better patient and site experience, and ultimately bringing innovative treatments to patients faster. Aligning the right patients with the right study is vital, yet there is more to patient engagement – proactive communication and education are essential, but the first step is to ensure we fully understand the patient’s needs and perceptions.  

Understanding patient needs and maximising engagement

An effective recruitment strategy needs proactive involvement with patient groups and actively listening to understand patient needs – either through advisory boards, one-on-one consultations, or through social listening. These interactions provide crucial insights into the specific needs and challenges experienced by patients with rare diseases that a traditionally obtained patient persona could not achieve.  

Acquiring insights through social listening supports information retrieval from patients in a setting where they are more likely to be candid – something they may find challenging around healthcare professionals who may know less about their disease than they do, or who may use clinical language that unintentionally confuses and alienates them. Social listening and engagement with patient groups early on can open information sources that directly impact trial and recruitment design, maximising the potential for study outcomes to progress knowledge and bring medications closer to approval. 

The patient view is also crucial to determining an effective outcome for a study. Not only does this aid trial design and protocol feasibility for patients, but it allows patients to be more engaged with the process. One issue with collecting data from trials is that some of the parameters being captured can be detached from the realities and context of everyday living – seemingly arbitrary visual acuity scores are less useful to patients than recovering sight to a level where they can sign their name within a pre-marked space or pour a cup of coffee with little to no difficulty. Being involved with patient advocacy groups at an early stage and establishing what is truly important to patients will be critical in keeping patients engaged and active in a trial. 

Decentralisation and data sharing 

The Covid-19 pandemic has accelerated an openness to digital first-treatment options, with attitudes changing regarding telemedicine and how a clinical trial should be carried out. This is evident in the pharmaceutical industry, with several companies moving forward with decentralised trials, for example in late 2021, Roche designed the first fully virtual clinical study in oncology3. 

Patients with rare diseases are typically receptive to telemedicine, which can reduce many of the barriers they face in accessing specialised care. Combined with the inherently smaller patient pool for trial recruitment – a decentralised trial can allow recruitment from across the globe and can also support patient recruitment and retention. 

Many rare diseases have debilitating features, and almost half occur in children. A decentralised trial means patients can avoid travelling long distances to a clinic or spending extended periods away from home – while increasing the pool of patients available. This strategy also lets patients feel valued in the trial process, rather than feeling like “test subjects” that need to be transported to a clinic for study. 

An innovative way to ensure rare disease trials are set up in a way that both aligns with patients’ lives and meets their specific requirements is through a mock clinical trial. Simulating a clinical trial in a role-play scenario allows patients and site staff to naturally discuss the study and identify any potential concerns, challenges, and strengths of the study design – including how information is communicated. The insights gained from this are then used to adapt the study protocol and address any issues that may have been identified – thereby ensuring that the study aligns with the needs of patients. 

A solid communication platform with patients is crucial to acquiring data and keeping them engaged in the trial process. This also needs to be effective and easy for the patient, which is why having an effective patient portal is essential.  

Regular and recorded patient portal contact from an early stage allows issues to be addressed before patients feel the need to exit the study and can improve the study experience for all participants.  

Improving the patient experience 

Obtaining a correct diagnosis in rare diseases is often referred to as a ‘journey’ or ‘odyssey’ and can take six to eight years, typically involving visits to multiple physicians. Perhaps because of this, patients often show a high degree of altruism, with a desire to improve healthcare and research even if they do not benefit personally. On obtaining a diagnosis, they often find no treatment is available and a clinical trial may be their only hope. However, patients need to be able to take ownership and make informed decisions around their healthcare. Education is an essential component to support participation in a clinical trial. Information must be provided at a suitable level, in the patient’s native language, to ensure they can understand the process and give informed consent.  

Strategies to improve the patient experience include simplifying participant forms for informed consent and providing information in multiple formats to inform decision-making. Patients should not be expected to converse or take on information at the level of a medical professional. Information should be tailored to the required audience and communicated in an engaging manner through video summaries, interviews, animations, brochures, and infographics. 

As many rare diseases affect children, the information provided also needs to meet the needs of both them and their parents or guardians. It can be easy to take for granted how important knowledge is to a patient, but it is critical to the success of a clinical trial5. 


To summarise, the key ways to ensure that patient recruitment is effective: 

  • Understanding patient needs: Early acquisition of patient insights through engagement with patient advocacy groups, social listening, and through feedback via mock trials can improve the trial design and enhance recruitment and retention.
  • Relevant outcomes: Aligning outcomes of the clinical trial with outcomes that are relevant to the patient’s needs will ensure better engagement and retention.
  • Patient-centric flexibility: Decentralisation enables wider participation in trials, supporting recruitment and increasing the number of patients available for enrolment.
  • Education: Patients often want to be involved in the process of helping others, but they need to fully understand the trial and exactly what is involved – information needs to be provided in an easily digestible format that they can understand.
  • Proactive communication: Consistent and clear communication with patients regarding everything from the study design to dissemination of findings will increase retention and keep patients engaged in the process.

How we learn from patients and integrate their perceptions and preferences in the development of clinical trials is constantly evolving. A common theme to clinical trial success is early collaboration with patient groups. This collaboration is even more important in the rare disease setting, where patient pools are limited, and the risk of poor trial outcomes is heightened. Patients who participate in clinical studies give a substantial amount of time and energy to the study. Any way in which the process can be improved for them will enhance recruitment and retention, as well as the reputation of the clinical trial sponsor in their patient communities. Given the many challenges on the road to completion of a successful clinical trial, working with outsourcing partners can support this process and bring in the specialist knowledge and experience needed for success. 


  1. Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
  2. Noncompletion and nonpublication of trials studying rare diseases: A cross-sectional analysis 
  4. Patients as Partners in Rare Disease Diagnosis and Research 
  5. The Meaning of Informed Consent: Genome Editing Clinical Trials for Sickle Cell Disease 

About the authors

Amanda HenkelAmanda has over 20 years’ experience in the pharmaceutical and medical communications environments, and is highly skilled in medical affairs, specialising in oncology and rare diseases. Before joining the Inizio Group, Amanda worked for Schering AG in Berlin, where she remained for three years, first as the European Product Manager for MabCampath (CLL) and subsequently as Head of Oncology Product Communications. As European Product Manager for MabCampath, Amanda was involved in strategic global marketing and branding, worked with Medical Affairs to initiate and implement global development studies, and was involved in external expert development programmes and workshops.

Craig StevensCraig has over 23 years’ experience in the pharmaceutical, medical communications, and patient engagement environments. Before joining the Patient Engagement Team within the Inizio Group, Craig was a senior medical writer at CRO IQVIA for over three years working in recruitment and retention for clinical trials. Craig has worked in different areas within research and industry, including in rare diseases at Orphanet for three years, as a freelance medical writer and editor for five years, and in pharma at Sanofi for 10 years.

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