As we move deeper into the realm of personalized treatments, effective communication and relationships between the pharmaceutical industry, healthcare professionals and patients has never been more important. This is particularly true for rare disease trials. The global rare disease market is set to be worth more than 547 billion US dollars by 2030. However, while clinical trial patient recruitment can be challenging even for non-complex studies, this issue is exacerbated for rare disease trials due to the much smaller patient populations involved. In a recent webinar, Whitney Taynton, our US-based Business Unit Head, was joined by Donna Dale, Director of Patient Recruitment Programmes, Rare Disease, for Pfizer, and Joy Aina, whose mother lives with sickle cell disease, to discuss the importance of patient centricity when recruiting for rare disease clinical trials.



Rare diseases affect up to 446 million people worldwide. In the UK alone, it is estimated that more than 3.5 million people are living with a rare condition. In the US, around 25-30 million people are affected. With more than 7,000 rare diseases, most of which are genetic, it’s vital all patients can access effective treatments. In many cases, there is a large, unmet need, so effective patient recruitment strategies for rare disease clinical trials are essential if we, as an industry, are to develop and deliver new, innovative medicines. However, as the host of our webinar, Pharma Times editor John Pinching, noted, “In the modern healthcare ecosystem, one size does not fit all. Indeed, the challenge of engaging with people from different groups endures most pressingly when it comes to recruiting for rare disease trials.”

Considerations for rare disease clinical trial patient recruitment

Since the emergence of COVID-19, the dynamics of clinical trial patient recruitment have changed. The move to decentralized trials, for instance, has forced the industry to think about the patient perspective within trial design, including patient recruitment strategies. “It’s very important for us to be able to consider our patient population [from the very start],” says Donna Dale. “There are a lot of things we take into consideration and beyond just the clinical trial itself.”

These include

  • a patient’s motivation for taking part in a trial – perhaps it is altruism, a desire to be free of the limitations of their condition or a drive to help develop treatments for future generations
  • what or who might influence a patient
  • long-term benefits for patients who enroll into a trial
  • concerns that patients may have, such as potential side effects, referral hesitancy or a deep-rooted mistrust in clinical research
  • Any geographic or logistical hurdles that may need to be overcome

Living with sickle cell disease

Joy Aina’s mother, Margaret, was diagnosed with sickle cell disease at just three years old. During the webinar, Joy spoke candidly about the impact her mom’s condition had on her family. “I didn’t actually know that my mum was ill until she gave birth to my younger brother,” Joy reveals. “I really didn’t understand why my mum had to be in hospital for months on end. It kind of took a toll on me and made me very upset, especially because I was only nine at the time. One minute she could be fine, literally. We’re out and about doing whatever and then the next minute she’s in hospital and she’s really ill.”

Opening up about the pain her mom often experiences as a result of her condition,  Joy says, “She describes it as a stabbing pain. It’s continuous and sharp at the same time. She has it in her limbs, so her legs, her arms, her chest, and her back. It could be anywhere only part of the body, and I think the tough part out of it is anything can bring it on – so it can be a change of weather, it could be stress, and it could just literally just come on.” It wasn’t until a chance meeting with one of her friends at a Sickle Cell Society event in 2016 that Margaret learned of a new treatment, took the information to her consultant and was eventually prescribed the drug in question. 

Margaret continues to take the medication to this day and has seen a marked reduction in the number of hospital admissions she experiences. Her mental health has improved as a result and her social life has blossomed as she has been well enough to go back to work, socialize with friends and do a lot more things she was not able to before. “It makes us worry a lot less,” says Joy. “We’re also able to go out, experience new things, go on holiday and we don’t have to worry about the weather change, what she’s going to wear or [if] she’s going to fall ill.”

Joy says that, based on her mom’s experiences, more needs to be done to ensure patients are given the most up-to-date information and presented with all treatment options. “We actually found out recently that the drug was approved years ago and it just makes you wonder why she wasn’t put on it and why she has to find out through word of mouth.”

Overcoming clinical trial patient recruitment challenges in rare disease trials

“We need to make sure that we’re targeting patients in the best and most efficient way possible, but also in a way that they feel very heard and cared about,” says Whitney. Information, education, engagement with patient advocacy groups and effective communication are four key factors in accelerating clinical trial patient recruitment in rare disease studies.

Education and awareness

The COVID-19 pandemic threw clinical trials into the mainstream consciousness, but generally speaking, the public’s understanding of trials did not necessarily improve. Unhelpful phrases like ‘guinea pig’ to describe patients taking part in a trial may have stuck in some people’s minds. Others may mistrust medical research for other reasons. Some may simply not know what a clinical trial involves.

It is imperative to work with patients, charities and advocacy groups to ensure everyone understands the process of drug development, including the different phases of clinical trials and the regulatory process. “I would love to see more trials be out there,” says Joy. “I would love to see people using word of mouth, talking about the condition itself and ways to help us.“

Information for patients

Clear information about a trial delivered in an appropriate format and language and tailored to address a patient’s questions and concerns is a key part of successful patient recruitment strategies. However, it is also important to consider a person’s wider circle of support.


“Oftentimes they do have a caregiver or circle around them and they likely won’t make the decision [to enroll in a clinical trial] alone, “ says Donna. “You really have to…provide information to the family, to the extended family, and the caregivers…and whether they move forward or not may likely depend on what the people around them, say to them.”  

Both Whitney and Donna discussed the value of digital technology in enabling sites to share ‘bites’ of information, which can often feel less daunting than receiving a full information brochure, particularly at the very beginning of the recruitment process. 



The way in which information is shared can affect the success of clinical trial patient recruitment. From Joy’s perspective, direct communication between sites or patient recruiters and groups like the Sickle Cell Society can build trust and make patients feel more at ease about participating in a trial. “I think if there was a direct contact between places like the Sickle Cell Society and sites…or people running trials, where they actually target people who have sickle cell and actually bring forward the trial and know so we know that it’s actually just for sickle cell, it will make us feel a lot more comfortable in actually participating.”

The speakers also discussed digital platforms as a way to target and communicate with very specific groups of people, not only by sites but also by charities and patient groups acting as advocates for the communities they serve.

Engage with patient advocacy groups and charities

Patients’ experiences and stories can be powerful tools in developing patient-centered clinical trials and delivering effective clinical trial patient recruitment strategies to enable the timely progress of a study. Building relationships with patient advocacy groups and charities helps to ensure patients’ needs are always kept in mind. Actively engaging with these groups to gather information and advice directly from the patient population in question will also improve our understanding of barriers and challenges, allowing us to develop mitigation strategies.

“I would encourage our sites or sponsors…to not be afraid to go out and to think about how possible it would be to work with patient champions or to hear the patient voice to be able to access patient advocacy groups, even if there are some hurdles or challenges along the way,” says Whitney. “It could bring great success in the future.”

Innovative Trials has extensive experience in accelerating patient recruitment across numerous therapy areas and including rare disease trials. Get in touch to find out how our specialized ‘boots-on-the-ground’ approach can support sites and help you engage more effectively with patient communities in over 70 countries.