By Jan Nissen and Madeline Geday, Merck
Patient behaviors have changed drastically in recent years. With the widespread adoption of digital technologies, such as wearable devices that transmit patient data, and a surge in health-related information available online, patients have become more knowledgeable and engaged in the management of their own health than ever before.
But while patient behaviors have evolved, the clinical trial process has not changed markedly over the past three decades, and patient participation in clinical trials has been largely stagnant. In the United States, for example, less than 5 percent of patients participate in trials.1 The current drug development process means that there are significant delays in the development of new medicines, resulting in a loss to patients and society. The drug development timeline today is between 12 to 15 years, and it costs an average of over $2 billion to bring a new drug to market.
With intense pressure to reduce drug costs while addressing unmet patient needs, we must find new ways to increase efficiencies in drug development while ensuring patients are at the center of the process.
By involving patients early and ensuring they are a part of the drug development process, we can better understand their points of view, alleviate their existing burdens, and ultimately accelerate research, bringing critical medicines to market faster. Since 2012, the FDA has taken numerous steps to involve the patient in the drug development process including establishing a Patient Affairs team, conducting listening sessions with patients, and issuing guidance documents to help the industry understand how to incorporate patient perspectives.
This increased effort to involve patients will result in benefits in three key areas: recruitment, representation, and retention in clinical trials.
Currently, a staggering 50 percent of clinical trials do not recruit within their planned timeframe.2 Such delays can result in significant financial repercussions. For every day a trial is delayed, the estimated cost to the drug developer is $600,000 — or $8 million in the case of a blockbuster product.3 Meanwhile, patients and caregivers continue to suffer without access to the treatments they so desperately need.
Pharmaceutical companies have an opportunity to drive timelier recruitment by working with patients to develop clinical trial protocols. By engaging with patients early on, we can glean valuable insights into the specific barriers and motivating factors affecting their participation. With this type of insight, we can ensure protocols are better suited to a patient’s preferences and behaviors and may, in turn, recruit in a timelier manner. Close partnerships with patients have demonstrated success — trials that involve patients in the process have a 20 percent higher launch performance.4
At Merck, we have had some success hosting workshops with patients during the planning of some cancer clinical trials, and we’ve seen timely, improved recruitment as a result. We are implementing similar practices across other disease areas.
More Diverse Representation
Minority and ethnic representation is another aspect of clinical trials that would benefit from earlier patient interaction. Diseases occasionally manifest themselves differently in certain demographic subpopulations. A prominent example of this is triple-negative breast cancer, where the incidence of disease is heavily skewed to the African American population. Yet, African Americans represent less than 5 percent of the clinical trial population.5 It is critical that clinical trials are designed to enroll, represent, and reach diverse racial and ethnic groups, particularly those that are genetically predisposed to the disease being studied. By engaging in discussions with a wide range of demographic groups to fully grasp where their challenges lie, we can proactively identify solutions to increase their clinical trial participation.
At Merck, we are implementing a variety of tactics to address representation issues. For example, we’re improving cultural competency through conversations with subject matter experts and development of outreach tools. We’re also working to ensure diversity in our trials by focusing on key partnerships with our sites and monitoring enrollment data by ethnicity to adapt and increase focus among sites.
Higher Patient Retention
The success or failure of clinical trials is also impacted greatly by patient retention. Today, an average of 30 percent of patients will drop out of a clinical trial, costing researchers valuable time and tens of thousands of dollars.6 Trial burden is one of the most commonly cited reasons for increased patient drop-out rates. Major contributing factors include the significant time commitment demanded (given most trials last over 12 months), the difficulty of accessing a trial site, and repeated tests or biopsies considered invasive and unnecessary by patients.
Our team at Merck is examining ways to minimize trial burden. For example, through discussions with potential participants in one of our clinical trials, we learned patients would not come back to a trial site 36 times to provide samples of urine and saliva, nor would they visit the trial website 36 times to submit information. Based on these insights, we developed a for at-home collection of urine and saliva samples. Collection took only 15 minutes, and the sample was then shipped to the research investigator. While the at-home kit wasn’t a more economical solution, it was better aligned with patient preferences and anticipated behaviors — and thus more effective for retention. We’re now considering a similar at-home approach for other trials to minimize disruption to patients’ daily lives.
The healthcare landscape is continuing to shift, and researchers will need to further adapt. Based on the degree to which the FDA has embraced the patient perspective, it is probable that within the next few years the agency will ask pharmaceutical companies how they have incorporated the patient perspective into their new drug applications. Additionally, technological advances will continue to surge. We will undoubtedly see greater use of digital technologies, such as sensors and more sophisticated wearables, for at-home data collection and patient monitoring, which could lead to the realization of virtual clinical trials that decrease the trial burden on patients. While a comprehensive approach to digital clinical trials has not yet been implemented, we can anticipate accelerated adoption. We must prepare for these kinds of shifts now.
By improving clinical trials, we have the potential to realize higher success rates and better meet patient and societal needs. But this can only be accomplished by pharmaceutical companies partnering more closely with patients from the onset. After all, the most important stakeholder in any clinical trial is the patient.
About The Authors:
Jan Nissen is VP of patient innovation and engagement at Merck. She leads Merck’s patient innovation in patient insights, patient and consumer experience, and patient and caregiver alliances and advocacy globally. Nissen has been in the industry for 33 years, in sales, training, marketing, and operational roles. In 2012, she served as a Merck Fellow with Population Services International in Kenya. Nissen has a bachelor's degree from the University of Illinois Medical Center, an MBA from Lake Forest College, and a master’s in population health from Thomas Jefferson University. She currently serves as an executive board member for Clinica Verde, an NGO (nongovernmental organization) in Nicaragua, and on the board of Patient Focused Medicines Development.
Madeline Geday is director of global patient engagement and innovation within the Global Clinical Trial Operations group at Merck, responsible for optimizing clinical trial feasibility and execution through integrated data-driven expert analysis and recommendations. She joined Merck in 2004 and has helped launch the clinical trials microsite on merck.com, led multiple voice of the patient projects, and has served as an industry expert in the field of patient engagement, recruitment, and retention. Geday received a dual bachelor’s degree in psychology and exercise physiology from Ursinus College and is pursuing graduate coursework in marketing from Temple University.