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Obtaining patient perspectives during open-label extension trials.
Today’s pharmaceutical industry leaders understand the importance of capturing patient voices. Patient journeys inform the strategies of clinical and commercial teams. Regulatory agencies support the move toward increased patient centricity. To that end, the FDA, last month, released the first of four draft guidance documents addressing how stakeholders can “collect and submit patient experience data” from patients and caregivers for medical product development and regulatory decision-making.
Some companies have fully integrated patient voices into their clinical and commercial decisions; others are just beginning to do so. Companies have long interviewed patients, caregivers, and healthcare professionals (HCPs) about living with and managing a disease. More recently, they have been taking such steps as adding patients to their advisory boards and having patients participate in trial design as members of protocol development teams-piloting studies to identify and address challenges prior to launch. Patient advocacy leadership positions are being created to increase emphasis on patient perspectives in all clinical and commercial endeavors.
These steps help companies understand the impact of a disease on patients and caregivers-but there is more to do. Companies need to understand the impact of their new product on the lives of patients and caregivers over time. For example, most medications prescribed for chronic health outcomes are meant to treat patients across the course of months or even years, which may not always be reflected in the design of a finite, shorter-term clinical trial.
As such, if companies don’t know how the new product affects patients over time, especially as they age or develop comorbidities, they will be unable to clarify what patients should expect over the course of their health outcome and treatment journey beyond just the initial intervention.
In the case of rare diseases or scenarios where there is little known about the outcome, patients are often the sole real-world longitudinal source of information on disease progression, impacts to the body, and sustained efficacy of treatment pathways.
Many biopharmaceutical companies are expanding their patient-centricity initiatives to include patients receiving a new product during clinical trials. This is most often done through interviews of interested participants conducted after database lock, and it can provide the study sponsor with many insights that previously weren’t available. Such research can confirm positioning, determine perceived onset and duration of action, and clarify the new drug’s affect on participants’ and caregivers’ lives. Companies are using these insights to go beyond clinical trials in optimizing patient support services and identifying patient “ambassadors,” compensated individuals who might:
Although the advantages are many, interviewing patients in clinical trials after database lock has some limitations in that it provides only retrospective data and a one-time perspective at that. Data collected prospectively would strengthen the patient voice and paint a comprehensive picture for product development and regulatory decision-making.
Recently, some companies have started forging the commercial-clinical connection via real-time web surveys and telephone interviews with participants during clinical trials, primarily during open-label extension (OLE)
studies. Using this technique, companies can obtain prospective data as well as multiple measurements, with increased feasibility to gather both quantitative and qualitative data. OLE studies offer the advantage of a wide window of time-usually 24 to 48 months-over which to gather insights at regular intervals. What’s more, the patients typically are on the new drug, so interviewing them minimizes the risk of unblinding the study and also generates data with high internal value. At a time when minimizing clinical trial costs and gaining efficiencies in collecting data are central to discussions around R&D, OLE studies that gather passive data or harness preexisting patient populations are a budget-friendly alternative to conducting a new incremental trial.
Companies have several options for collecting patient experience data. A sub-study designed to elicit patient perspectives can either be embedded into the protocol initially or submitted as a protocol amendment. However, this approach places relatively high operational demands on the drug developer and investigators and must be submitted to the institutional review board (IRB).
A better alternative often is a companion study, designed as a separate study run concurrently with the original clinical investigation and outsourced to a third party. For example, eligibility criteria could be “patients who meet inclusion/exclusion criteria in the main clinical protocol,” but participants opt-in separately, and the third-party researcher takes responsibility for efficiently obtaining applicable approvals and participant consent alongside the main study. A companion study avoids the need to submit a protocol amendment for ongoing studies. Like a post-study, a concurrent companion study alleviates workload on the sponsor’s part, needing only low-to-moderate resources.
For many organizations, conducting a companion study is the optimal way to proceed. What then, are some of the considerations? From recruiting materials to the research questions to participants, the study must be carefully designed for compliance as well as generating meaningful data. Research partners with experience in interviewing clinical trial patients, caregivers, and HCPs can determine the right questions to ask (and the right way to ask them), and the target population to interview to obtain the desired data. For example, depending on the study, it may be more fruitful to recruit parents or caregivers to provide the experience perspective, rather than research participants enrolled in the main protocol.
A CRO or external consulting partner can be selected; they should understand the nuances of working with IRBs and ethics committees (ECs) and how the relationship between the main protocol and the companion study might influence submission requirements. For example, the main protocol should reference the companion study if interim findings from the companion study result in real-time modifications to the active main protocol, or if data from the companion protocol is to be submitted along with the main study in support of a marketing application. If this information is not submitted initially, a research partner can support submission of this information through an IRB-approved protocol amendment. Partners can also provide insight into engaging with regulatory agencies and help navigate potential concerns and areas of increased scrutiny.
The research partner’s personnel all should be trained in adhering to good clinical practice (GCP) standards. If the companion study involves gathering personal health information, they must comply with patient privacy laws and regulations, including HIPAA and the General Data Protection Regulation (GDPR). They must understand how to write the documents for the companion study protocol, so that compliance with regulatory criteria is ensured in the event of an audit or inspection.
Partners should also understand the nuances involved in writing for patient communities, including heightened protections for vulnerable populations such as children and pregnant women and exercising sensitivity toward local norms.
Listening to the voices of patients in clinical trials is a powerful tool for enhancing the commercial-clinical connection and improving communication-not just between the company and its product’s end users, but also among the various parties engaged in medical product development and regulatory decision-making.
Aarthi Iyer is a managing director at Kinetiq. Ross Weaver is founder and president of Clinical SCORE