How to Integrate the Patient Voice in Rare Disease Clinical Trials

Start early

Companies do not always know how and where to include patients and caregivers in clinical trial activities —some think the only aspect where input can be provided is in the creation of the clinical trial protocol, but patients can be included even earlier. This ensures that the most relevant research questions are being answered and allows for multiple check points throughout the development lifecycle. Creating a treatment or device that is truly patient-centered means including patients and patient advocacy groups in:

• Setting research priorities (for example, during creation of a Clinical Development Plan)
• Designing clinical trial protocols to optimize retention and compliance
• Consulting with solution providers to decrease patient burden through technology such as eConsent, connected devices, and patient portals
• Development of patient materials and information sheets
• Dissemination of clinical trial results, including the creation of plain language summaries

Dive deep to find the right patients

Finding patients and caregivers to include in your research may take time—rare disease patient advocacy groups may not be as visible or easily identifiable, especially for diseases which are not well known or in the “ultra-rare” category. Some may not even have their own patient group and be part of a larger group covering a more general condition.

Making connections with those larger groups or using a social network like We Go Health, where advocates are connecting with other advocates, can be useful. This is especially true as these sites contain public information that can be searched. Using social media more generally can also be effective if you know what you are searching for and what keywords and hashtags to use.

Healthcare professionals can recommend patient advocacy groups, as well as looking at the summaries for relevant webinars, conferences, and patient-authored PubMed articles. Rare disease conditions are often included in these summaries.

When you do find the right patients, making the collaboration process as transparent and simple as possible is essential. If there is more than one patient group, you can work with both, but remember the importance of setting clear inclusion rationale and criteria to avoid any kind of bias.

Understand patients’ day-to-day needs

What the industry or healthcare professionals think living with a condition may entail is often very different from the daily lives of patients and their caregivers. There are three aspects that impact a patient’s ability to participate in a clinical trial: physical, emotional, and financial.

Clinical trial sponsors need to understand the support level required for patients and caregivers to physically be able to participate in the trial—it must fit into their day-to-day living and must work within the unique requirements of their disease. Second, the emotional aspect needs to be managed because for many people clinical trials represent hope. Even more so in rare disease where it may be the only option for treatment. Efficacy, side effects, and overall success (or failure) of the trial must be managed with empathy.

Finally, providing financial support in clinical trials is often considered an “incentive,” but appropriate reimbursement and stipends paid to participants is becoming a basic requirement needed to participate. No patient should be excluded from a trial or suffer a financial burden from participating in a clinical trial. Engaging patients and caregivers early on in the development process will bring these logistical aspects to light so they can be included in the clinical trial design and the trial budgeting process. For the sponsor, this will save money in the long run—any relatively small increased financial burden in terms of planning or logistics is more than made up by the benefits: reducing trial timelines, increasing enrollment rates, and making it easier for patients to complete the trial.

Create a mutually beneficial partnership

While recruitment and retention are obvious sponsor benefits and provide rationale for including patients in the process, studies also show that including patients improves the overall likelihood of successfully launching a drug or device. In Phase II and Phase III trials that are patient-centric, the likelihood of drug launch is higher across disease areas, including oncology, neurology, and rare disease—and in rare disease specifically, there is a significant decrease in recruitment time for patient-centric trials.

Additionally, when it comes to technology, anything that can be done to make the process or trial easier needs to be co-created with patients. For example, in a patient community where dexterity and fine motor skills are a challenge, using a device or digital application with tiny buttons is going to be a challenge at best and potentially make trial participation impossible. One simple thing sponsors can do is ask potential vendors if they have worked with patients on development and ensure that the end-user is driving the design. In some cases, a sponsor could choose to fund a small patient test group prior to the inclusion of a device or application in a clinical trial.

For patients, an effective and successful clinical trial that develops a new treatment is undoubtedly the largest benefit, but what else can sponsors do to support patients? Often patients and patient groups are looking for additional education or exposure in their disease area and including patients in conferences or symposia allow them access that is typically reserved for clinical professionals or scientists.

Provide more choices in clinical trial execution

Over the last few years, decentralized clinical trials have improved patient access to clinical research, but it is important to keep in mind that these trials place more responsibility on the participants and caregivers, so they need to feel confident in taking on that additional responsibility—filling out Patient Reported Outcomes (PROs) Questionnaires in digital or paper format, administering medication, or using a wearable to measure outputs. Additionally, not going to a physical trial site reduces patient opportunity to exchange experiences and feel supported by a physician and study team. Being mindful that decentralized clinical trials may not work for everyone and using it via a hybrid approach, an option within the trial rather than the single solution for the entire trial, will maximize the opportunity to make the trial work for the most patients.

Create many opportunities for feedback

When patients can be involved in the clinical development plan and creation of the trial protocol and are encouraged to provide in-trial feedback, they are more likely to stay in the trial, understand their responsibilities, and adhere to the protocol requirements. Additionally, as a sponsor, you will know if and when a protocol amendment might be required. While this may initially be disruptive, a patient-supported amendment mid-trial could save your research if something is not working or could be improved.

Additionally, once the trial concludes, be clear with patients on how you will capture their feedback and let them know about next steps. Again, offer them choices for providing feedback, whether it is via a survey, one-to-one interview, or an advisory board—allow patients to choose what is easiest for them.

Especially in the Pharmaceutical and Biotechnology space, where drug development can take up to a decade, it is important to have multiple touchpoints with patients as treatment methodologies, available treatments, patient experience and priorities evolve over time. This allows sponsor companies to adapt and refine their strategy in ways that will make their overall program more successful and position them more strongly in the market.