Treating patients as research partners has fast become the goal for many sponsor organizations. This shift towards more patient-centric trials is popular for one reason: it works. Given the recruitment and retention difficulties that beset clinical trials, any move towards improving the patient experience is mutually beneficial to patients and researchers.
A vital means to connect and engage with patients is through building dynamic relationships with patient advocates. Sponsors and researchers can learn more about what patients need and want, and how those patients experience their illnesses. This can better guide research design and strategies.
In this post we assess the importance of connecting with patient advocates and how best to make these relationships thrive.
Patient advocates tend to have four key attributes. They’re full of passion; they’re curios; they want change to happen; and they take the initiative to achieve it, writes Bray Patrick-Lake, director of stakeholder engagement at Duke Clinical Research Institute.
She advises sponsors and research teams to look for the patients and caregivers that ask a lot of questions and seem to want more from what scientific research currently offers. These are usually good signs pointing to valuable patient advocate partners.
On a personal level, Patrick-Lake began as patient in need of relief with few remaining options available. She referred herself to a clinical trial via clinicaltrials.gov after her physicians failed to do so. Her first experience of clinical trials was unsatisfactory, with the trial abandoned after low enrollment that she says could have been rectified if patients had been more engaged during the trial’s design stage.
To worsen the bad experience, none of the trial data was or has ever been shared with any of the patients. This motivated Patrick-Lake to demand more from the investigator who, it turned out, was also frustrated by the treatment patients had to endure. Learning more about the whole research process, Patrick-Lake started to speak on panel discussions and build relationships with experts. She also started by connecting with patients on social media, forming a Facebook group for her condition which in turn became a nonprofit disease advocacy foundation.
Increasingly, pharma companies see the value in treating patients and patient advocates as strategic partners rather than passive participants. To make the most of this ambition to achieve cohesive strategizing, pharma companies need to take a holistic patient view, says neuroscientist Joel Beetsch, vice president of patient advocacy at Celgene.
Beetsch’s approach requires deciding whether your organization’s patient advocacy or patient-centric function is research and commercial. The former might be a center for excellence, which patient advocates contribute to the broader research and development goals of the organization. The latter concerns the business function. However, sometimes in organizations with both a research and commercially focused patient advocacy function, there is a failure to communicate between the two.
Guaranteeing effective and regular communication is vital. A better approach is to merge both functions into one department with an overarching governance of all activities.
Perhaps the most obvious area of influence patient advocates can exert in clinical research is through fundraising. And this influence has been particularly felt in cancer research, explains patient advocate Mary Lou Smith.
Patient advocates have persuaded philanthropic organizations to donate money and successfully lobbied Congress to grant funds for research for most cancer centers in the country.
Another key area of focus for patient advocates is education, informing patients about the different types of cancers and how clinical research works to treat them. Without this useful conduit of knowledge, many patients would not be suitably informed about how clinical trials work and why it could be advantageous for patients to enroll.
Yet another influence that patient advocates have on clinical research is through their involvement in advisory boards and committees, such as the National Cancer Institute’s National Clinical Trials Network. Their ability to present patients’ perspectives into the administrative and strategic planning of research delivers significant value.
Duchenne muscular dystrophy (DMD) is a rare disease. So when Alex Johnson’s son Jack was diagnosed with it eight years ago, she sought the best way to help her him. She founded the charity JoiningJack.org, and partnered with the Duchenne Children’s Trust to establish the charity, DuchenneUK.org.
Johnson’s focus on and indeed her reputation for advancing research is significant. She has worked hard to gain support from venture philanthropists and is helping to meet the Duchenne UK’s mission to eradicate the disease.
Patients’ perspectives are influential in helping attract other patients into clinical research and patient advocates are often the medium through which this message travels. The value of having a shared experience can not be overstressed, notes Cindy Padgett, vice president of commercial operations at PharmaCord.
But incorporating the patient’s voice into the message is a two part process, she adds. First, pharma companies need to consider the patient’s experience and potential needs. Second, these organizations and any third-party partners must communicate and share that patient voice.
The two part process is achieved when patients and caregivers are interviewed and listened to, noting that each patient’s journey is unique. The consideration throughout for sponsors and research teams should be to validate the patients and their particular experience of their illness and the research process.
Under review by the FDA Advisory Committee was Exondys-51, a drug to treat DMD. The FDA initially ruled against using it but the patient perspective, presented by patient advocacy groups, shifted their decision for the drug’s approval. Victories like that show just how influential patient advocates be, explain Andrew Butcher, Cécile Matthews, and Bhavesh Patel, Ph.D., life sciences consultants at Charles River Associates.
Patient advocates have also helped achieve reimbursement. Two examples include patient advocates sending letters in support of Tobi Podhaler for people with cystic fibrosis and Exjade for iron overload.
Relationships between pharma companies and patient advocacy groups should be seen as important partnerships. Indeed, this viewpoint is becoming a dominant trend and many pharma organizations have launched special teams to manage the relationship with patient advocates.
It’s best to begin these relationships at the preclinical stage in order to understand disease characteristics, write Butcher, Matthews and Patel. This way the organizations can better plan for patients in later stage clinical programs.
Considering the patient experience — the demands placed on them, and the potential emotional and mental fatigue from undergoing trials — researchers and sponsors now understand the value of patients as partners. This has been a significant shift from the historic research relationship, says Nina Ahluwalia, head of product development and patient insights at Genentech.
She refers to Roche’s research into autism spectrum disorder and the valuable insights gained from partnering with patient advocates. Beginning at the planning stages of the clinical trial, the Roche team invited patients and caregivers to share their experience of the patient journey.
What arises from this collaboration is an increased empathy, which shapes the working partnership. This, in turn, translates into better trial design and ensures adequate communication with patients regarding the design’s purpose, as well as what patients should expect along the way.
Collaboration invariably yields useful information researchers would not have gained otherwise. Ahluwalia explains that people with autism spectrum disorder do not like to be called patients. They prefer “individuals with ASD” or “self-advocates” because they do not consider themselves sick. This knowledge guided how the research team wrote the communication materials.
Pharma companies working with patient advocates need to be fully committed. That means the entire organization must understand the value of this approach and how best to achieve it. “You’re either all in or you’re not,” says board-certified patient advocate Grace Cordovano, Ph.D.
The pharma organization has to work towards an internal culture that values the stories and experiences of patients and their caregivers. Those organizations that do so will soon see the value of such an approach, she writes. All those who listen to the patients’ stories will be inspired, develop empathy and connect with them. The result is a renewed sense of purpose for pharma company employees.
No matter how enthusiastic, supportive and willing the patient advocate organization is, the sponsor company needs to ensure transparency and ethical standards throughout the relationship. For instance, patient advocate organizations should not receive funding from a single source, says consultant Jean Campbell.
But sponsors should also maintain a safe distance between themselves and the patient advocacy groups. When boundaries are blurred — or simply appear to be blurred to others — both parties could suffer from credibility issues. At all costs, patient advocates should not be seen as the sponsor’s mouthpiece, Campbell stresses. Equally, they should not feel indebted to the pharma organization.
Trust and transparency are the foundations upon which to build relationships with patient advocates. Doing so will foster a more collaborative and strategic partnership by which to enhance the patient experience, improve trial design and protocol and even help with the regulatory process.
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