Lisa Bance co-wrote this blog post with Kristyn Feldman.
This is the final post in a four-part series examining patient centricity within R&D.
To achieve true patient centricity in drug development, pharmaceutical companies need to follow four main principles: gain empathy for patients, uncover and meet patient needs, connect with patients and, finally, engage patients. By asking for and incorporating trial participant input at every level of R&D, the pharmaceutical industry can start to look at patients as true partners.
To ensure that drug development remains focused on the patient, the treatments tested in trials should affect the outcomes that matter most to patients. Often, clinical trial outcomes measures rely on historical, physician-measured metrics to monitor patient progress. But in many diseases, patients are less focused on improving these objective metrics. For example, physicians monitor peak expiratory flow in asthma patients. However, patients are more interested in how their treatment affects shortness of breath, nighttime issues, their use of rescue treatments and daily functioning. This can create a disconnect between what outcomes are being measured and the quality-of-life improvements that really matter to patients. To address this, a growing number of advocacy groups, health foundations and pharmaceutical companies are collaborating with patients to create and measure novel patient-reported outcomes.
In a landmark example, the online patient network PatientsLikeMe used a grant from the Robert Wood Johnson Foundation to create the Open Research Exchange (ORE), an online software platform with tools that simplify the process of developing, testing and sharing new patient-reported outcomes. In one application of this tool, the ORE was used to assess measures for mycosis fungoides and Sézary syndrome, types of cutaneous T-cell lymphoma that affect the skin. Historically, it was difficult for clinicians and researchers to measure the impact of these rare conditions because there were no disease-specific metrics.
Actelion Pharmaceuticals and PatientsLikeMe used the ORE platform to create a free, health-related quality-of-life tool for patients with mycosis fungoides and Sézary syndrome. They hope that the tool will help improve the quality-of-life conversations between patients and care teams, as well as the clinical management and treatment decisions related to these diseases. The PatientsLikeMe forum and its ORE platform have enabled the development of patient-reported outcomes in several other diseases including chronic lymphocytic leukemia, hypertension and multiple sclerosis. Additionally, the platform has produced more general tools like patient-reported outcomes of adverse events and treatment burden questionnaires.
BLOG POST: How to Develop a Patient-Centric R&D Process
Here’s an example of another tack to take: Early in a drug’s development process, Bristol-Myers Squibb creates patient engagement networks consisting of patients, caregivers, study site personnel and patient advocacy groups. The company uses these groups to assess the disease journey, trial design and enrollment barriers, and each patient engagement network meets both virtually and in-person for the duration of the asset or disease area portfolio.
Insights from these networks and other patient conversations have led to changes in trial designs and support services for patients. In a trial for an autoimmune disorder, the patient engagement network provided feedback on the trial plan, leading to the reduction of regular travel to a clinical site. Instead, participants received nurse visits at home or were offered car services, alleviating significant patient burden. Additionally, BMS is now striving to ensure that participant support doesn’t end when the trial does by helping patients transition to an appropriate next therapy, or even additional trials. Lastly, BMS has begun thanking both patients and trial staff in its U.S. advertising campaigns, which has been met with positive feedback from trial participants who often feel forgotten once the last trial visit has concluded.
Similarly, Novartis sponsored the Patient Research Exchange, a platform to facilitate communication about patient engagement in research among patients themselves, patient advocates, patient organizations and other relevant stakeholders. The Patient Research Exchange isn’t intended for marketing or commercial research but is instead aimed at representing patients, fostering collaboration and prompting an idea exchange among all patient engagement stakeholders across diseases and around the globe. This initiative, founded by patient advocates, highlights how the industry can evolve research while engaging patients and sharing best practices and resources.
Our four pillars of patient centricity—empathize, meet needs, connect and engage—establish a clear pathway to putting patients at the center of the pharmaceutical R&D process. Making this shift requires an organization-wide culture change to ensure that the patient-centered mindset is part of every process, transaction and conversation. By embracing patient-centric drug development programs, pharmaceutical organizations can enable patients to transition from study subjects to true research partners
Did you miss the earlier installments? Read them here.