While there are many sources of real-world evidence, there’s growing interest and focus on capturing patient generated insights to capture a more specific level of understanding for a given therapy. In today’s complex healthcare landscape, listening to patients can give drug developers valuable information about the lived patient experience, how treatment impacts people’s quality of life and what’s most important to patients.
Today, much more attention is being paid to collecting and amplifying the patient voice so that pharmaceutical companies can develop drugs and build programs that truly work for patients.
Valerie Powell
Chief Patient Officer at CorEvitas
There are a few different methods of collecting patient insights. Holding focus groups or interviewing clinical trial participants, are qualitative in nature, while large-scale global surveys, yield quantitative results. Patient experience studies prioritize understanding patient preferences—being diagnosed with and living with a condition and how patients and their care partners balance risk versus benefit of treatment, including clinical trial participation. Understanding the patient experience is key for clinical research professionals who want to truly understand the disease or condition they’re studying. This perspective can be valuable to pharmaceutical companies in every stage of the product development lifecycle.
Recruitment and retention are perennial challenges for trial sponsors. Pharmaceutical companies need to ensure that their trials generate robust results that satisfy regulators and convince prospective prescribers. But they also need to design trials that patients will participate in—and stay in for the trial’s full duration. Interviewing or surveying trial participants to better understand their experiences can help trial designers create protocols that will be more tolerable to patients in future trials. Surveying patients through advocacy or focus groups before building a protocol can also help sponsors balance patients’ needs with their research objectives. They might, for instance, decide to include fewer invasive procedures – like biopsies – if it’s possible to do so without compromising the integrity of results. Clinical trial simulations [where a patient, principal investigator (PI) and site coordinator all role-play the scenarios that would take place in the trial] can yield powerful insights into what patients will experience in the actual trial. Trials today look quite different from those executed in the pre-COVID world. Many trials are now fully virtual or a hybrid mix of traditional, in-person and virtual visits. The logistics of these new trial designs and the technology required to enable them all impact the patient experience and should therefore be thoroughly tested with patients in the targeted population prior to finalizing plans for clinical trial operations.
Regulators are also increasingly taking patient-reported outcomes (PROs) into account when making approval decisions. The FDA, for example, has undertaken a multi-year effort to identify methods of collecting PROs in clinical trials with appropriate consistency and quality to facilitate scientific rigor. Most recently, guidance addresses incorporating assessment of PROs on product labels. Ultimately the goal is to better enable patients to understand the symptoms and side effects they may experience, as well as how a therapeutic may affect their quality of life.
An important advancement in the patient-engagement space is the development of pharmaceutical company-sponsored long-term patient advisory councils, where companies engage with patients on a broad range of topics and obtain their feedback on strategic initiatives, just as they have with KOLs for years. They are also leveraging rich data sets available from online patient communities to better understand patient experiences. And this information is playing a growing role in informing their strategies and patient programs.
Patient insights research can help identify unmet need and build a manufacturer’s understanding of the patient journey. Patient feedback can be used to improve how patients use and experience pharmaceutical products. This has the potential to improve adherence—and thereby improve patient outcomes over the long term. Taking patients’ needs, preferences and experiences into account as they initiate and remain on therapies, increases the likelihood that patients will adhere to therapy and achieve improved outcomes. When developed in direct partnership with patients, given therapies can improve a patient’s quality of life more holistically.
