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PC01 - Reinventing Clinical Trials (ID 83)
- Event: WCLC 2019
- Type: Pro-Con Session
- Track: Advocacy
- Presentations: 5
- Now Available
- Moderators:Ivy Elkins, Jose Luis Gonzalez Larriba
- Coordinates: 9/09/2019, 11:00 - 12:30, Seoul (2007)
PC01.01 - Setting the Stage: Tension Between Patient Safety, Scientific Purity and Patient Inclusion (Now Available) (ID 3555)
11:00 - 12:30 | Presenting Author(s): Jacob Sands
Clinical trials are important to the process of better understanding the therapeutic potential of new drugs while also serving as treatment options for individuals seeking the best possible treatment. When writing protocols, we think about how best to determine the effectiveness and side effect profile of the treatment being studied. This can lead to more limited eligibility in an effort to reduce complications not related to the disease or drug. When seeing patients in clinic, we think about how best to treat each individual, which can lead to preferring less strict eligibility. These two perspectives can sometimes seem to be at odds. How do we best define eligibility in a way that both provides scientific rigor while also providing the trial as an option to as many people as possible? This session sets the stage for an in-depth discussion of these 2 important aspects of clinical trials and how to strike the ideal balance for enrollment eligibility criteria.
As part of this introduction, a highlight of the process of drug development through different phases of clinical trials from “pre-clinical work” to phase 3 studies is helpful. The following is a general structure. Any prospective drug must demonstrate compelling results at one phase to then be studied in the next phase. This starts with lab studies and often treatment of mice with prospective drugs. Any studies before treatment of humans is called “pre-clinical.” When results in this setting are promising, treatment in humans starts with phase 1. In this setting the focus of the study is to find the most appropriate dose, and there are often multiple diagnoses allowed for enrollment within that one study. The strategy for dose finding generally includes starting treatment for a few individuals at a low starting dose. After verifying tolerability, another group starts treatment on the study at a higher dose and so on. Less commonly, studies will allow dose increases for each individual that is tolerating the lower dose with ongoing disease control. Dose escalation often continues until finding the “maximum tolerated dose.” Although the side effects of the drug often weigh heavily in determining the dose for further study, responses to the treatment are certainly considered. After determining a dose for future study, this drug may enter a phase 2 study. In this phase, many more individuals are treated at the determined dose, and the effectiveness of the treatment is studied within a specific clinical setting. Results that suggest efficacy often lead to a phase 3 study, which includes randomization to the new drug, or the standard of care treatment, within a specific clinical setting. Although placebo is sometimes utilized, all individuals should get at least the best known therapy. For example, KEYNOTE-189 enrolled individuals with metastatic nonsquamous, non-small cell lung cancer (without sensitizing EGFR or ALK mutations) to the standard of care first line chemotherapy, pemetrexed and a platinum, with randomization to also include either pembrolizumab or placebo. Although people received placebo, it was given along with the chemotherapy, as was pembrolizumab.
This introduction is followed by two related session. One is focused on crafting trials to allow more broad enrollment with the acknowledgment of trials as a treatment option. The other is focused on trial development with full attention toward scientific rigor and study of the drug. A discussion of the balance of these goals follows.
PC01.02 - Real World Research Groups - ROS1ders (Now Available) (ID 3556)
11:00 - 12:30 | Presenting Author(s): Merel Hennink
Presentation Title: Real World Research Groups - ROS1ders
Session: Reinventing Clinical Trials
Lung cancer research has made great progress in the last decade. Discovery of more treatable mutations have paved the way to more treatment options. We are moving away from a “one-size-fits-all” chemotherapy towards personalized medicine. (1)
However, these developments require accurate diagnostics to identify the right treatment options. Furthermore, as a common cancer like lung cancer is split into rare subgroups, it also poses additional challenges. Rare mutations in NSCLC (for instance ROS1) result in small patient populations for trials and less bio-materials for research. The main question here is how to navigate this changing landscape.
The ROS1ders – a community
The ROS1 rearrangement was first described in 2007 and occurs in about 1% of patients with NSCLC. Of the estimated 1.5 million new cases of NSCLC worldwide each year, approximately 15,000 may be driven by oncogenic ROS1 fusions.(2) In 2015, little was known about the ROS1 rearrangement and only a single drug was available to treat the disease. Also in 2015, a group of ROS1 positive lung cancer patients formed an international community on social media, The ROS1ders, allowing them to connect with each other. Through social media, patients are becoming increasingly engaged, and discussing the diagnostic and treatment options available in their countries or hospitals. In addition, these national and global online communities, where information is shared, can highlight (inter)national disparities in treatment access.
Crizotinib is currently the only FDA- and EMA–approved agent for the treatment of ROS1-rearranged NSCLC. Unfortunately several healthcare systems across the world have difficulty with reimbursing the drug due to lack of data from randomized clinical trials (UK, South Africa, Canada, New Zealand). In Canada, Real World Data from the ROS1 Facebook group helped in the approval process. “The Committee expressed that they were impressed with the patient input of 259 ROS1 positive patients and caregivers from 32 countries who supported the use of crizotinib. Overall, from the perspectives of patients with ROS1-positive NSCLC, they value a chance to extend their life and spend more time with their families by having a treatment that is effective, and improves their symptoms and outcomes.”(3)
Global ROS1 initiative – from advocacy to active research
The ROS1ders transitioned from advocacy to active research in the past few years. Reaching out to academic cancer centers, they strove to increase the amount of cancer models available to stimulate ROS1 research. The Global ROS1 Initiative is a partnership connecting researchers, patients, caregivers, and physicians worldwide. Patients who have an upcoming biopsy contact the national study nurse, and can choose to donate extra material to generate a cell line, a patient-derived xenograft (PDX) mouse model, or both. The goal is to create models and data accessible to all researchers. All models and characterization data will be made available as an open access database to researchers and collaborators. The Global ROS1 initiative has already generated four cell lines at the University of Colorado, and three PDX mouse models are in development. Patients are in the driver seat!(4)
Testing is essential to select the right treatment. Nevertheless, systematic diagnostic testing for ROS1 in metastatic NSCLC is not yet included in the European Society of Medical Oncology recommendation, but only suggested. (5) In Europe, access to molecular testing and new medicines differs between individual countries, even within the same country. In the Netherlands, a retrospective study in all patients that tested negative for EGFR and KRAS, showed an increase in ROS1 testing between 2013 (10%) and 2017 (61%) (6). Given the demonstrated added value of patient-driven organizations, both for patients and research, we need a more systematic testing for ROS1, in order to offer effective treatment to more ROS1 patients and strengthen our international community.
ROS1 patients as research partners in Europe
The Global ROS1 Initiative requires streamlined protocols to be successful. Currently, European ROS1ders cannot donate tissue for a variety of reasons. Therefore, a pan-European collaboration should be implemented, that centralizes expertise on the small number of potential tissue donations. In this network, tissue processing should be centralized in a single lab, and the resulting data and models could be distributed to collaborating partners. However, the setup of such network is very difficult, in part due to national differences in legislation.
Furthermore, although we are a European Union, patients are not really united, and bringing together the European ROS1ders is not an easy task. We deal with language, boundary and policy issues. Due to European public health systems, all responsibility is given to doctors. Most patients see less benefit in getting informed and organized. Data protection laws and less use of social media make it more difficult to connect patients. Next, the number of non-governmental organizations supporting lung cancer patients is small - in particular in Europe. And finally, fundraising activities are rather uncommon in Europe. Overall these aspects make it harder for patient advocates to make a difference. As such, international networking and solidarity become even more important.
In the Netherlands, www.stichtingmerelswereld.nl was founded to raise awareness and accelerate research. As a result, research on drug resistance has been initiated in cell lines and PDX mouse models. In parallel, drug resistance is also studied in France and Germany. Combining these individual studies might result in a 1+1 equals 3 scenario.
We strive to unite our national voices to influence European policies and use our European voices to connect national studies.
Understanding the role of personalized medicine https://www.lungcancer.org
Jessica J. Lin MD & Alice T. Shaw (2017) “Recent Advances in Targeting ROS1 in Lung Cancer” in Journal of Thoratic Oncology . https://doi.org/10.1016/j.jtho.2017.08.002
Xalkori for ROS1 positive advanced lungcancer. 2019 (4)
D. Planchard et al. (2018) “Metastatic non-small cell lung cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up.” Annals of Oncology 29 (Supplement 4): iv192–iv237, 2018 doi:10.1093/annonc/mdy275
Kuijpers Chantal C.H.J. et al (2018). “National variation in molecular diagnostics in metastatic lung cancer. “ Nederlands Tijdschrift voor Geneeskunde 162:D1607
PC01.03 - Real World Research Groups - ALK+ Group (Now Available) (ID 3557)
11:00 - 12:30 | Presenting Author(s): Linnea Olson
There is a natural tension inherent to clinical trials—between scientific rigor and a participant’s hope that an experimental therapy shall prove effective. However, it is important to never lose track of the reason for clinical trials. To quote Dr. Richard Pazdur, Director of the FDA’s Oncology Center of Excellence, ‘We have to understand that the clinical trials are here to serve the patients. The patients are not here to serve the clinical trials.’ (1)
The decision to enroll in a clinical trial—particularly phase I where both maximum tolerable dose and the safety profile are being ascertained—is not a casual one. Generally the prospective participant has exhausted traditional therapies. With the emergence of actionable mutations and targeted therapies, the paradigm for participation is evolving. It is foreseeable that a participant can have a response to a therapy measured not only in months but in years. And when progression does occur, that same participant may enroll in yet another clinical trial.
For many patients with advanced lung cancer, clinical trials now offer the best option for therapeutic treatment.
It is critically important to find the balance between advancing medical research and extending individual lives.
To this end, exclusion criteria need to be reevaluated. A patient who is desperately ill is often willing to tolerate a far higher degree of risk. Arguments for exclusion criteria fail to take into account the fact that this is a representative population; should the drug receive FDA approval, this will be the consumer profile. (2,3)
It is also vital to understand the burden that clinical trial participants take on. First, financial. There is an overriding misperception that participation in a clinical trial is cost free. In reality, it is often only the experimental therapeutic that is provided free of charge.
Clinical trials are procedure rich—from pharmacokinetics to additional scans/MRIs/EKGs/biopsies. In most cases a participant’s insurance is billed for these procedures, resulting in quickly maxed out deductibles and added copays. More frequent visits to the site of the trial require additional time and travel on the part of the participant. Transportation, lodging and meals are often the responsibility of the patient. There are lost wages, for either the patient or their caregiver or both. Often there is also the burden of childcare.
The financial toll adds to the high degree of stress that a patient and their families are already living under. It also means that clinical trials are an option only for those with both the financial means and a solid support system in place. As economically distressed families are unevenly distributed among minorities, the clinical trial population is not representative. Not only does this mean there is an inherent disparity in access to clinical trials, it also impacts the science, as clinical trial participants in an ethically diverse country such as the US are overwhelmingly caucasian. In addition, it is important to point out that healthy volunteers to clinical trials are almost always compensated for both their time and even small things that are typically not covered in a clinical trial for oncology patients—such as parking. (4,5,)
And then there is the physical toll upon a patient. Excessive scanning, multiple biopsies, exhaustion associated with additional visits to the site.
“When you begin to look at a trial from the patient’s perspective and consider the complexity of the trial and what we are asking them to do, is it any wonder that so many patients are refusing to participate? And is it any wonder why so many patients choose to withdraw from a clinical trial?” (6)
As accrual is an ongoing issue in clinical trials, it is imperative for both the sake of patients as well as medical science, that the voice of the patient be heard. Patient reported outcomes (7) often fail to capture the actual experience of a trial participant. Rather than being the one size fits all that is commonly handed to participants at each visit now, why not write the PRO with the assistance of actual participants? Humans, unlike their rodent counterparts in the lab, are sentient beings. Potentially a wealth of anecdotal information is being lost simply because no one thinks to ask.
Clinical trials cannot happen without the cooperation of human participants. That cooperation is referred to as compliance. A patient who is noncompliant risks ejection from a trial. This creates a relationship that is inherently unbalanced.
It is possible to address both accrual and the needs of the participant if clinical trials become truly patient centric. To do so one must consider the burden of participation. A clinical trial should be viewed as an opportunity, albeit one that is not risk free. Lessening the burden and removing some of the barriers to participation will better address the needs of both patients and the field of medical research.
1. NCCS Celebrates “Focus on the Care” Reception in Honor of Dr. Richard Pazdur and Ellen Goodman Oct 22 2015 https://www.canceradvocacy.org/news/nccs-celebrates-focus-on-the-care-reception-in-honor-of-dr-richard-pazdur-and-ellen-goodman/
2. Clinical Trial Patient Inclusion and Exclusion Criteria Need an Overhaul, Say Experts
April 23, 2018 Redfearn, S https://www.centerwatch.com/cwweekly/2018/04/23/clinical-trial-patient-inclusion-and-exclusion-criteria-need-an-overhaul-say-experts/
3. You Can’t Sit With Us for This Study: Exclusion Factors in Clinical Trials Feb 4, 2019 Krebill, C, NU SCI https://nuscimag.com/you-cant-sit-with-us-for-this-study-exclusion-factors-in-clinical-trials-44a8f6efbd8
4. Clinical Trials and Their Financial Barriers: Increasing Participation, Lowering Financial Toxicity Jan 07, 2019 Rolleri,C, ASCO Communications https://connection.asco.org/magazine/features/clinical-trials-and-their-financial-barriers-increasing-participation-lowering
5. Payment for participation in clinical research: Review of proposals submitted to the ethics committees Perspect Clin Res. Apr-Jun, 2018 9(2): 64–69. Marathe, PA; Tripathi, RK; Shetty, YC; Kuyare, SS; Kamat, SK; That, UM; atte2https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5950612/
6. Merck Changes The Paradigm On Clinical Trials May 11, 2016 Miseta, E Clinical Leader https://www.clinicalleader.com/doc/merck-changes-the-paradigm-on-clinical-trials-0001
7. Patient involvement in clinical research: why, when and how Apr 27, 2016 Sacristán, JA; Aguarón, A; Avendaño-Solá, C; Garrido, P; Carrión, J; Carrión, A; Kroes, R; Flores, A; NCBI PMC https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4854260/
PC01.04 - Lung Cancer Patients Should Have Better Access to Clinical Research: Relax the Eligibility Criteria (Now Available) (ID 3558)
11:00 - 12:30 | Presenting Author(s): Janet Freeman-Daily
Clinical trials are essential for improving treatment of cancer patients. However, only 2%-3% of adult cancer patients participate in clinical trials, and many trials close because they do not enroll enough patients. Others are slow to accrue patients, which prolongs the time required to obtain results. Those trials that meet accrual goals often have cohorts that don’t reflect the demographics or performance status of the real-world population of patients who have the disease.
Why do trials have trouble enrolling enough patients? Some of the significant barriers to clinical trial participation stem from over-restrictive trial eligibility criteria. Unger et al (2019) found 21.5% of patients did not enroll in a clinical trial because they were not eligible. A Kaiser Permanente study of non-small cell lung cancer patients found 80% of the patients were not eligible for two NSCLC treatment studies.
Trial eligibility must balance opposing factors. It must be narrow enough to ensure the effect of the treatment can be determined, yet broad enough that the population of patients is meaningful. Researchers often use “common” eligibility criteria without giving due consideration to how those criteria may impact trial recruitment and the real-world applicability of their study. A population of younger patients with no health issues other than lung cancer might make it easier to identify the effect of the experimental treatment, but this population gives no real insight into how the treatment affects the typical population of lung cancer patients (which has an average age of 71).
Recommendations to modernize eligibility criteria have been recently published by a joint effort of the American Society for Clinical Oncology and Friends of Cancer Research, as well as the US Food and Drug Administration and the National Cancer Institute. The recommendations include relaxing restrictions on brain metastases, minimum age, HIV/AIDS, organ dysfunction, and prior and concurrent malignancies while ensuring patient safety.
Other aspects of a trial may cause patients to decide not to enroll even if they meet the eligibility requirements. Locations of trial sites might require the patient to travel, yet the patient might not be able to afford time or cost of travel, or their insurance might not cover treatment at any trial site. The trial protocol may prohibit certain previous treatments, or require weeks of washout from previous tyrosine kinase inhibitor (TKI) treatments (which raises the possibility of TKI flare).
Preliminary evidence shows that relaxing trial eligibility requirements could result in a greater number of patients becoming eligible for clinical trials. Harvey et al (2019) conducted a retrospective study of 10,500 CancerLinq records of patients with lung adenocarcinoma. They found 47.7% of patients were excluded from clinical trials by traditional exclusion criteria (no brain metastases, no other malignancies, and creatinine clearance greater than 60 mL/minute), while only 1.5% of patients were excluded by the ASCO-Friends recommended expanded criteria that removed these restrictions.
Relaxing clinical trial eligibility while maintaining safety is in the best interest of the patient. Patients are not for clinical trials. Clinical trials are for the patient.
Gore, L, et al. (2017). "Modernizing Clinical Trial Eligibility: Recommendations of the American Society of Clinical Oncology–Friends of Cancer Research Minimum Age Working Group." Journal of Clinical Oncology 35(33): 3781-3787
Harvey RD, et al. (June 2019). “Impact of broadening clinical trial eligibility criteria for advanced non-small cell lung cancer patients: Real-world analysis.” Presentation at ASCO Annual Meeting 2019, Chicago, IL. https://meetinglibrary.asco.org/record/178360/abstract
Jin, S, et al. (2017). "Re-Evaluating Eligibility Criteria for Oncology Clinical Trials: Analysis of Investigational New Drug Applications in 2015." Journal of Clinical Oncology 35(33): 3745-3752.
Lichtman, SM, et al. (2017). "Modernizing Clinical Trial Eligibility Criteria: Recommendations of the American Society of Clinical Oncology–Friends of Cancer Research Organ Dysfunction, Prior or Concurrent Malignancy, and Comorbidities Working Group." Journal of Clinical Oncology 35(33): 3753-3759.
Lin, NU, et al. (2017). "Modernizing Clinical Trial Eligibility Criteria: Recommendations of the American Society of Clinical Oncology–Friends of Cancer Research Brain Metastases Working Group." Journal of Clinical Oncology 35(33): 3760-3773.
Sharpless NE, Doroshow JH. “Modernizing Clinical Trials for Patients With Cancer.” JAMA. Published online January 23, 2019. 321(5):447–448. doi:10.1001/jama.2018.18938
Uldrick, TS, et al. (2017). "Modernizing Clinical Trial Eligibility Criteria: Recommendations of the American Society of Clinical Oncology–Friends of Cancer Research HIV Working Group." Journal of Clinical Oncology 35(33): 3774-3780.
Unger JM, Hershman DL, Fleury ME, Vaidya R. “Association of Patient Comorbid Conditions With Cancer Clinical Trial Participation.” JAMA Oncol. Published online January 10, 2019 5(3):326–333. Doi:10.1001/jamaoncol.2018.5953
PC01.05 - Representation of Minorities and Women in Oncology Clinical Trials (Now Available) (ID 4059)
11:00 - 12:30 | Presenting Author(s): Narjust Duma
Abstract not provided
MA16 - Prioritizing Use of Technology to Improve Survival of Lung Cancer Subgroups and Outcomes with Chemotherapy and Surgery (ID 142)
- Event: WCLC 2019
- Type: Mini Oral Session
- Track: Treatment in the Real World - Support, Survivorship, Systems Research
- Presentations: 1
- Now Available
MA16.01 - Project PRIORITY: A Patient-Founded and Patient-Driven Research Partnership on Real-World Data on EGFR-Positive Lung Cancer (Now Available) (ID 2918)
15:45 - 17:15 | Presenting Author(s): Ivy Elkins
Despite increases in PFS in EGFR-positive lung cancer patients due to EGFR TKIs, patients eventually develop resistance to these drugs. Project PRIORITY (Patient Reported Initiative On Resistance, Incidence, Treatment studY) is a patient-founded and patient-driven longitudinal study aimed at understanding unmet needs of the global EGFR-positive lung cancer community.Method
A comprehensive 103-question, IRB-approved patient-facing survey about the diagnostic and treatment journey of patients (including risk factor exposure, treatments, symptom and side-effect management, access to biomarker testing and clinical trials) was developed with input from US FDA statisticians and expert clinicians, and then pilot-tested among English-speaking patients both locally and internationally. Differences between US and international participants were analyzed by Chi-square (for categorical variables) and ANOVA.Result
Of the 253 respondents, 27.7% were international participants. In line with previous studies with EGFR patients, participants reported low rates of active tobacco exposure (16.4%) and high rates of second-hand tobacco exposure (34.7%). Also, first-line use of afatinib (OR = 2.5, p <0.05) and erlotinib (OR = 3.3, p< 0.05) were associated with the development of a T790M mutation reflecting similarity in clinical characteristics.
US participants were more likely to report childhood exposure to secondhand smoke, family history of cancer (other than lung cancer), use of more than one line of therapy, and combination first-line therapy (P<0.05 for all variables).
International participants were more likely to report first-line treatment with 1st/2nd generation TKI, less use of tissue and plasma NGS, lower clinical trial participation, and more use of whole-brain radiation for brain metastasis (P<0.05 for all variables).
This first-of-its-kind international study provides a comprehensive picture of the treatment of EGFR-positive lung cancer patients in the real-world setting and highlights the existence of diagnostic (low NGS rates) and treatment gaps (low clinical trial participation and different treatment sequencing) both within the US and internationally.
P1.14 - Targeted Therapy (ID 182)
- Event: WCLC 2019
- Type: Poster Viewing in the Exhibit Hall
- Track: Targeted Therapy
- Presentations: 1
- Coordinates: 9/08/2019, 09:45 - 18:00, Exhibit Hall
P1.14-29 - Disrupting the Paradigm: Partnering with Oncogene-Focused Patient Groups to Propel Research (ID 1498)
09:45 - 18:00 | Author(s): Ivy Elkins
Genomic alterations drive more than 60% of adenocarcinoma cases of non-small cell lung cancer (NSCLC). About 20% of cases will have an oncogenic driver (EGFR, ALK, ROS1, BRAF, NTRK, etc.) that can be treated with approved targeted therapy drugs, and more (RET, Exon 20 insertions, etc.) have clinical trial options. Patients and caregivers dealing with these cancers have organized globally into oncogene-focused groups (“Groups”—see Table 1) and are building partnerships that seek to provide support, increase awareness and education, accelerate and fund research, and improve access to effective diagnosis and treatment.
We partnered in a variety of ways to accelerate research. While each Group sets its own research priorities, we’ve found successful collaborative research has the following seven characteristics. It includes patients from the start, in all aspects of the project. It addresses questions meaningful to patients. It develops patient-centered measurements. It accommodates patients’ clinical realities. It leverages social media and patient groups. It shares progress with participants frequently. It makes results rapidly and freely available.Result
These methods have enabled the Groups to collaborate successfully with clinicians, researchers, advocacy organizations, and industry to generate ideas for next steps in research for their disease, forge new studies and clinical trials for a specific oncogenic driver, create new patient-derived models of oncogene-driven cancers to study acquired resistance, develop registry-based studies to collect real-world data, and guide patients to clinical trials.Conclusion
Oncogene-focused patient-caregiver groups are creating new paradigms across the research continuum. They have demonstrated that their partnerships with advocacy organizations, clinicians, researchers, and industry, can increase available patient-derived models, patient data, and specimens among geographically distributed, oncogene-driven cancer populations.
P1.16 - Treatment in the Real World - Support, Survivorship, Systems Research (ID 186)
- Event: WCLC 2019
- Type: Poster Viewing in the Exhibit Hall
- Track: Treatment in the Real World - Support, Survivorship, Systems Research
- Presentations: 1
- Coordinates: 9/08/2019, 09:45 - 18:00, Exhibit Hall
P1.16-48 - Experiences of Pts on 1st Line Care (EP1C): Symptoms and Impacts of EGFR TKI Therapy on Real-World Daily Lives of NSCLC Pts (ID 1223)
09:45 - 18:00 | Presenting Author(s): Ivy Elkins
Approximately one third of patients with NSCLC have tumours harbouring actionable EGFR mutations (Zhang et al. Oncotarget 2016). These patients are routinely treated with EGFR-tyrosine kinase inhibitors (TKIs) that are approved based on their efficacy and safety in clinical trials. Clinical trials do not fully reflect patient experiences as felt and perceived by patients, and in the real world, symptoms related to EGFR-TKIs and impact on patients’ daily lives are not well documented or understood. EP1C assessed patients’ experience with EGFR-TKIs and their impact on patients’ daily lives. Patient insights gained from EP1C can help guide symptom management strategies and improve communication with patients, helping to improve outcomes and quality of life.Method
This pilot, non-interventional, US-based, real-world study involved individual interviews with adult patients. Eligible patients were those diagnosed with EGFR-mutated metastatic NSCLC, taking one of three US-approved EGFR-TKIs (erlotinib/afatinib/osimertinib) as first-line treatment. Exclusion criteria included: major surgery or radiation therapy three months before treatment, chemotherapy or other therapy as first-line treatment, a second active cancer, or a cognition or sensory issue. Trained qualitative interviewers used a semi-structured interview guide, and conducted all interviews by telephone. Rating questions were included for the severity and degree of bother caused by their symptoms (0–10 point response scale: 0=not at all severe, 10=extremely severe). All interviews were audio recorded, transcribed and coded (ATLAS.ti software) for analysis of similar themes.Result
A total of 19 patients participated in the interviews. The average age was 54.0 years (range 37–76). The sample was 73.7% female (n=14), and 89.5% (n=17) had an education level of college or above. The most frequently reported symptoms were respiratory and gastrointestinal symptoms (n=18; 94.7% each), skin-related sympoms (n=18; 94.7%), discomfort and pain (n=17; 89.5%), hair and nail-related symptoms (n=17; 89.5% each), fatigue and other energy-related symptoms (n=15; 78.9%).
Ratings of severity and bothersomeness trended toward the mid-range of the scale (4.0 to 6.0) for most of the more commonly reported symptoms. The higher ratings were seen for very specific symptoms reported by only one or two patients, including constipation (8.0 severity/8.0 bother), armpit rash (7.0/8.0), tightness in throat (8.0/8.0), mouth soreness (8.0/9.0) and stinging/burning in the genital area (10.0/10.0).
All 19 patients reported impacts on their daily performance and emotional health. Sleep difficulties were reported by 12 patients (63.2%), and 9 patients (47.4%) reported limitations with inter-personal relationships and social functioning. The impacts on patients’ daily activities (work, chores, daily routine; 7.8) and emotions (anxiety, worry, fear and depression; 7.2) were found to be the most difficult to cope with. Several specific impacts, reported by fewer patients, included decreased independence (n=2; 7.0), economic burden (n=6; 7.7), and childcare difficulties (n=2; 8.5).Conclusion
In EP1C, real-world interviews allowed patients to express a broader range of symptoms and impacts compared with clinical trials, which mostly focus on symptoms. Some of the traditionally less commonly reported symptoms had a greater impact on patients’ daily lives. Clinicians should also consider these when assisting patients in managing their symptoms.