Patient needs

How putting patient needs first improves patient engagement, and makes recruiting a more pleasant experience for everyone involved

Recruiting is a difficult and uncertain process, especially when it comes to rare disease trials. But it can be a lot more productive and efficient if trial designers look beyond what they are trying to accomplish in order to think about what they need to do to make the trial appealing to patients.

Patients with rare diseases often have limited treatment options and less access to information about current research and trial opportunities, which means researchers need to work harder to identify these far flung patients, and to create an environment where they will want to participate in the trial opportunity.

If patients don’t know about a trial, if they don’t trust the researchers/company running the trial, or they think the protocol will too painful, time-consuming, or too far away, the physicians will lose them before they fill out the first form. And if an organization is trying to recruit from a very small, widely-dispersed patient population that loss equates to time and money wasted in the drug development cycle. It seems like a simple approach, but too often researchers get so caught up in what they want to accomplish with a trial that they forget to think about what the patients will need for them to even consider participating.

Adapting the trial design to a more patient-centric approach for rare disease trials doesn’t require a major overhaul. Researchers just need to shift their focus and think about the trial through the eyes of the patient and their caregivers. Taking this view will help them make small changes to the trial design, and avoid the mistakes that might otherwise cause them to miss out on effective recruiting opportunities.

Collaborate with patient support groups. Whether a patient is newly diagnosed with a rare disease, or a long-time sufferer looking for alternative treatment options, the first place they are likely to turn is a patient support group. These groups help them feel like they are not alone, and give them a trusted place to find information about trials, resources, and education about treatment options. These groups can be particularly important for sufferers of rare diseases, who may feel isolated and frustrated by a lack of options. Yet, because there are so few sufferers, these groups are often poorly funded, and may struggle to find the resources or expertise to support their members or to build-out their web presence.

This creates a great opportunity for biopharmaceutical companies and research teams to lend a hand. Leveraging and supporting these groups with research materials, educational support and other patient tools will help the patient community become better informed about their disease and about the research in the space. It will also help them begin to build a trusting relationship with the people most likely to participate in the trials, or to broadcast information to those who might.

Identify barriers to participation. With small and widely dispersed patient populations, researchers focused on rare diseases cannot afford to lose any potential patients. To reduce this risk, researchers should take the time to interview patients, care givers, and thought leaders in the disease space about the biggest obstacles to participation, then work that feedback into the trial plan and protocol. Some of the challenges may involve fears about medical uncertainties or discomfort, while others may be more logistical. Based on these interviews, researchers may find that making small changes – such as scheduling evening appointments or offering on-site childcare– is enough to make the trial more accessible for hesitant recruits.

Build sites around patients. When dealing with a dispersed patient population, which is almost always the case with rare disease research, trial designers need to be more strategic in the way their build their trial infrastructure. Instead of opening 200 trial sites and hoping patients will turn up, start by identifying down potential patients then creating trial sites around them. This eliminates two of the biggest barriers to recruitment in a rare disease trials – geography and trust. By bringing the trial to patients, the research team makes it physically convenient, while enabling them to be treated by their own physicians.

This approach may require some additional time and resources to ensure these sites are trial ready. Trial leaders will likely need to train doctors and support-staff on good clinical practice (GCP) standards, using a program like Quintiles’ Site Training eLearning Program (STeP). They may also need to upgrade basic infrastructure, like adding locked cabinets and temperature controlled storage units, and potentially hire a few support staff to ensure the trial is run correctly. But compared to the cost of opening dozens of trial sites with low numbers of participants, this approach will still cut time and cost from the recruiting and research process. It will also help the biopharma company expand its network of treatment sites and trained investigators for future trials.

Leverage rapid study placement approaches. Rapid study placement techniques are about making sure all the pieces are in place to engage the rare disease patient as soon as they agree to participate. To do that, research teams should:

  • Ensure regulatory pathways are well documented and that the protocol has been reviewed by a Central Institutional Review Board (IRB)/ethics to be sure it is ethical, provides potential benefits, and does not cause unnecessary harm to participants.
  • Have an approach in place for developing the start-up documents and processes needed to make new sites operational, including templates for data collection, toolkits and recommendations for defined SOPs for use with the site.
  • Ensure that a training program is in place for sites that are new to research (e.g., Quintiles’ STeP).
  • Ensure that all human resources are trained and ready to go as soon as the sites are up and running and as soon as patients are recruited. 

These approaches are not necessarily designed to engage patients, rather they are to ensure that once the research team has identified and recruited patients for their rare disease study, they don’t lose them due to unnecessary delays and hiccups in your trial operation.

Remember, when it comes to patient engagement, you need to be ready when they are!