Patient-Reported Outcome Data in Oncology Trials – Part 2: Clinical Benefit & Patient Preference

Sarah Tressel Gary & Rinah Yamamoto | |

In our previous blog, we reviewed current regulatory guidance on the use of patient-reported outcomes (PRO) data in the development and approval of cancer products and presented how PRO-based secondary endpoints are indicative of clinical benefit in terms of patient symptoms and overall quality of life.

Here we present current research on clinical benefits that have been demonstrated through the use of electronic PRO data capture – including improved survival – and review new findings on preference for electronic PRO data capture among patients with cancer

ePRO has shown improved survivability in oncology patients

Demonstrating Clinical Benefit through ePRO

Clinical research by Basch et al. has found that health related quality of life (HRQL) and overall survival are improved in oncology patients completing ePRO.1 In one study, 766 cancer patients initiating chemotherapy were randomized to usual care or web-based symptom reporting (12 common symptoms from the NCI-CTCAE). A computer inexperienced group completed the ePRO at clinic visits; whereas the computer experienced group completed the ePRO at home with weekly email reminders.

An email alert was triggered to a nurse when symptoms worsened ≥ 2 or reached absolute level ≥ 3. A report tracking symptoms was provided to clinician at each visit.  HRQL was measured via the EQ-5D at 6 months and overall survival was assessed in June 2016 (median follow-up period of 7 years). Figure 1 depicts the research findings, which include:

  • HRQL was improved more in the ePRO group than the usual care group (34% vs. 18%) and worsened in fewer (38% vs 53%) (P <0.001).
  • Fewer ePRO subjects visited ER vs. usual care at 1 yr. (34% vs 41%; P =0.02).
  • Overall survival was improved in the ePRO group (31.2 months in the ePRO group vs. 26.0 months in the usual care group (difference, 5 months; P=0.03).

A similar study by Denis and Basch et al. was published in 2019 and found similar findings in lung cancer patients with
overall survival of 22.5 months in the ePRO group vs. 14.9 months in the usual care group.2 The authors conclude that symptom self-reporting engages patients and is associated with clinical benefit.

ePRO improves clinical benefit and survivability in oncology trials

Cancer Patients Prefer ePRO

ERT clinical research finds that cancer patients believe it is important to report on their symptoms and prefer to report their symptoms electronically.  To identify preferences for use of PROs in cancer patients, 185 people with cancer responded to an online survey.  The respondents were asked about their preferences on methods and frequency of reporting daily symptoms. Respondents were also asked about their perspectives regarding the importance of reporting symptoms.

More than 90% of the cancer patient respondents think that it is at least somewhat important to report daily changes in their cancer related symptoms and 74% report that it is quite a bit and very much important to report such daily symptom changes.

Of the respondents, 63% indicated they would prefer to report changes in their daily symptoms in an electronic diary, 18% preferred to report directly to their doctor at study visits, while 14% felt they would prefer a paper diary, and 5% would not want to report their symptoms at all (Figure 2).

Patients prefer electronic PRO data capture in clinical trials

Figure 2: 95% of cancer patients want to report daily changes in symptoms while participating in a clinical trial

Overall 95% of patients want to report daily changes in symptoms while participating in a clinical trial (Figure 2). Most, 88%, would prefer to complete their study questionnaires at home vs. at a clinic.

Taken together, regulatory agencies are placing more focus on the secondary outcome PRO data in addition to survival and overall response when evaluating the clinical benefit of oncology products. Missing data and infrequent collection should be avoided when collecting symptom data, and electronic data capture can help achieve this goal. The use of ePRO has even been shown to improve HRQL and overall survival in oncology patients. In addition, oncology patients prefer electronic data capture using an electronic diary to report their symptoms completed at home, rather than in the clinic.

review the latest regulatory guidance for PROs in oncology trials 

Sarah Tressel Gary, PhD and Rinah Yamamoto, PhD are Senior Scientific Advisors at ERT.

References

1 Basch E, et al. Symptom Monitoring With Patient-Reported Outcomes During Routine Cancer Treatment: A Randomized Controlled Trial. J Clin Oncol. 2016 Feb 20;34(6):557-65.

Basch E, et al. Overall Survival Results of a Trial Assessing Patient-Reported Outcomes for Symptom Monitoring During Routine Cancer Treatment. 2017 Jul 11;318(2):197-198.

2 Denis F, Basch  E et al. Two-Year Survival Comparing Web-Based Symptom Monitoring vs Routine Surveillance Following Treatment for Lung Cancer. JAMA. 2019 Jan 22;321(3):306-307.

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