How can we make lung cancer trials more patient-centric?

In recognition of Lung Cancer Awareness Month, Olivia Kersey, Patient Engagement Strategist, illustrates current opportunities for the pharmaceutical industry to better meet lung cancer patients’ needs across clinical trials and beyond.




Lung cancer is the biggest cancer killer worldwide (1). Many associate the condition with smoking; although smoking prevalence has declined significantly since 1990 (2), this drop has not been aligned with the rate of population growth and so the number of smokers globally continues to increase. This is partly why lung cancer diagnoses are expected to double over the next three decades (3), however this is far from the full story. Partly due to environmental risk factors such as radon gas exposure (4), people who have smoked little or never throughout their lifetime can still develop lung cancer. Evidently, lung cancer remains a massive burden on healthcare services, and the need for better treatments is strong – especially considering lung cancer patient outcomes are typically poor and progress has been slow compared to that of other cancers (5).

In order to develop treatments which most effectively serve the lung cancer patient population, it is imperative that pharmaceutical companies embed a patient-centric approach to drug development. As expressed by Bonnie J. Addario and Daryl Pritchard of the Bonnie Addario Lung Cancer Foundation, “The current biomedical innovation paradigm, of discoveries moving from the bench to the patient, needs to shift its focus so that clinical researchers first take into consideration the patient and new discoveries move from the patient to the bench and then back to the patient. This will transform research to ensure it is patient driven.”


Getting on the right track


A great first step for making lung cancer trials more patient-centric is conducting patient preference (PP) studies, which can support ascertainment of research priorities and clinical trial endpoints (1) (6). By quantifying how patients make trade-offs in treatment decisions, PP data paint a picture of what an ideal medication might look like for those patients, thus providing a clearer aim for drug developers and more relevant context for assessing how well a treatment has performed in trials. This especially valuable in the early stages of clinical research, as the chance of “getting on the wrong track” is reduced.


Removing unnecessary roadblocks


Clinical trial eligibility criteria have long been excessively restrictive, limiting access to treatment via trials and meaning data available on current treatments are not generalizable to realistic patient populations. Lung cancer trials are no exception. In 2018, a multi-stakeholder working group facilitated by patient advocacy group LUNGevity Foundation identified 14 “restrictive and potentially outdated” exclusion criteria that are seen frequently in lung cancer clinical trials (7). The report of the working group’s activities, published in 2020, notes this may be a result of “cutting and pasting” criteria from previous protocols, a practice which is not only lazy, but specifically neglects to account for how dramatically lung cancer treatment has evolved – and therefore how critical the need for re-evaluating trial eligibility criteria truly is. The frequent inadequacy of eligibility criteria is well-known; even the hugely prominent FDA have weighed in, stating “some eligibility criteria have become commonly accepted over time or used as a template across trials without clear scientific or clinical rationale.” (8)

Considering how commonly lung cancer spreads to the brain (and indeed, the fact that the incidence of this event is increasing (8)), the common exclusion of lung cancer patients with brain metastases is a classic example of how unrepresentative the patient pools of lung cancer trials can be. Unfortunately, this is particularly common in industry-sponsored trials (9) due to numerous factors, including expectations of reduced life expectancy. One can imagine why participants with poor prognosis may not be ideal for trial sponsors; these patients are more likely to pass away prior to trial completion, at the very least increasing the chance of incomplete data collection (which comes with financial inefficiencies) and potentially complicating the ascertainment of the true effectiveness of the drug under investigation.

In reality, evidence shows the relative impact of these metastases on overall survival is in fact modest once other factors (e.g., number of metastatic sites) have been accounted for (10), indicating the presence of brain metastases is unlikely to interfere with assessing the effects of the treatment under investigation anywhere near as much as previously thought. In any case, regardless of whether brain metastases impact duration of survival or not, the key point remains that trials should serve to demonstrate if treatments are appropriate for the patients likely to receive them – even if that comes at the expense of short-term inefficiencies.

Fortunately, with the FDA recently publishing guidance on including patients with brain metastases in cancer trials (8), it’s likely these patients will experience greater equity in novel treatment access and the data generated will be significantly more representative of real-world patient populations. Indeed, allowing lung cancer patients with brain metastases to enrol in clinical trials (albeit in line with certain caveats) may offer valuable opportunities, such as the collection of subpopulation data to support accelerated treatment approval or capturing of important safety (and indeed efficacy) data that would undoubtedly be relevant in clinical practice (8) (10). Thankfully, complete exclusion of patients with brain metastases is decreasing in industry-sponsored cancer trials (11), but continued efforts to critically examine eligibility criteria and adjust in line with more informed, nuanced perspectives remain necessary regardless.


Enrolling informed patients


Patient education is critical in empowering patients to be active players in their own care (12) and is associated with improved outcomes (13) (14). When taking on a big decision like clinical trial participation, it is crucial patients understand their condition and what to expect from the trial well enough to be able to give informed consent for enrolment. All lung cancer patients require such information, but those with rarer subtypes have the greatest need as they can often feel “lost” in the wider lung cancer patient community and find it harder to receive the support they need (15). For example, patients with ALK-positive non-small-cell lung cancer (NSCLC) often lack access to doctors who specialize in this particular cancer (16), and therefore face particular barriers to accessing trusted information on their diagnosis and treatment.

A great step in the right direction came from Jazz Pharmaceuticals earlier this year with the launch of Nothing Small About It, a digital community specifically catered for patients with small-cell lung cancer (SCLC), another relatively rare subtype. Through this digital community, Jazz Pharmaceuticals facilitated convenient access to educational materials, making it easier for this group of patients to access relevant information. The resource was developed in collaboration with various groups, including GO2 Foundation for Lung Cancer and LUNGevity Foundation. This evidence of partnership is particularly encouraging, as according to a report from LUNGevity Foundation, lung cancer patients tend to gravitate towards patient advocacy groups (PAGs) as a source of information on clinical trials (17), rather than prioritizing materials from drug developers. With this preference in mind, pharmaceutical companies would do well to consistently deliver trial education/information in partnership with PAGs if they hope to fulfil the impact potential of such materials.


The patient experience in trials


The late Amy Butler was a medical assistant who was diagnosed with colon cancer at the age of 44 (18). Once the cancer had metastasised to her lungs and progressed there, she started enrolling into clinical trials. Prior to her death, Butler shared powerful insights into how sponsors could have made these trials much more patient-centric; for example, by making the results of individual tests and the overall trial easily accessible to the patients who took part. Butler made particular reference to the multiple, invasive lung biopsies and extensive blood draws performed as part of trials; she was never told if anything interesting was found, despite the noteworthy personal cost of undertaking such procedures. Butler also felt pharmaceutical companies should have provided opportunities to provide anonymous, periodic feedback; indeed, identifying threats to enrolment and retention via such surveys would be an appealingly straightforward way to inform the crafting of more patient-centric trials in future.

Feedback surveys are valuable, but pharmaceutical companies can go to the next level – ensuring insights gathered are as relevant and useful as possible by designing and/or implementing Patient-Reported Experience Measures (PREMs). As the name suggests, PREMs are a useful tool to learn more about the quality of patients’ experiences (19), differing from satisfaction surveys in that they essentially ask “What happened?” as opposed to “How did we do?” (20). Thus, compared to satisfaction surveys, PREMs partially mitigate subjectivity limitations (19) and provide more tangible points for improvement (21).

Although the literature available to date focuses on PREM use in clinical practice, PREMs could also be usefully implemented in trial environments. For example, pharmaceutical companies could use information from trial recruitment and retention evaluations, patient centricity-oriented satisfaction surveys, and patient advisory boards to develop a framework for what a patient-centric trial experience should look like, going on to design and utilise PREMs to establish whether that framework was adhered to. PREM data could then be used to demonstrate patient centricity to regulators (similarly to how increasing emphasis is placed on the importance of patient-reported outcome data in the context of drug approval) and provide guidance for improving trial design/management in future.

PREMs should focus on aspects of experience which matter most to patients (22). For instance, a trial-focused PREM could include questions surrounding travel requirements (a common challenge for patients in trials) or whether the patient felt sufficiently informed or like a partner in the process rather than a passive participant. In oncology, the National Cancer Patient Experience Survey (23) is an example of a commonly used PREM (24), although it is currently unclear whether PREMs specific to lung cancer (either care-based or trial-based) have ever been fully developed and used in practice, not to mention whether any developed are particularly robust.


Conclusion: approval, reimbursement, and beyond


From conducting PP studies at the early stages of trial planning, we come full circle once trials are complete, as PP evidence reduces uncertainty pertaining to the value of treatment effects to patients. Thus, decision-making for drug developers, regulators, payers, and clinicians is eased. Such evidence is especially useful in lung cancer given the large patient population and heterogeneity of treatment characteristics, benefits, and side effects (1).

Evidently, the areas for improvement regarding patient centricity in lung cancer drug development are far from scarce. As outlined above, if pharmaceutical companies were to effect a patient-first mindset and practice shift while setting research priorities, designing study protocols, enrolling trial participants, conducting trials, and demonstrating the value of a new treatment, the landscape of drug development and medicine could look very different – and undeniably, more human.

We do however have a long way to go, particularly for patients with unmet needs such as a lack of targeted treatments. Nonetheless, for those who have the determination and foresight to make the change, a triple win may be achieved and celebrated for patients, pharma, and society. For patients, more relevant research will be conducted, which will inform better-tailored care and improve outcomes. For pharmaceutical companies, trial recruitment and retention challenges will be more easily mitigated (therefore saving costs and speeding time to regulatory approval) as trials become increasingly more appealing and accessible to patients. For society, healthcare systems will be fundamentally fairer and provide faster access to treatment. The time to step up patient-centric drug development has not suddenly arrived; rather, it’s long overdue.


Get in touch with the Prime Patient team via email ( or Twitter (@PrimePEPTalks) to learn more about how we activate patient insights to deliver a triple win for patients, pharma, and society.

Read more about our work in Patient Engagement here 


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