Asthma Clinical Trials Need to Include Patient-Reported Outcomes
Patient-reported outcomes will be required in asthma trials to obtain drug approval.
Innovative PROs are incorporating something adolescents wear everyday — wearable fitness and sleep trackers. One such study saw promise in specifically in FitBit wristband technology for informing clinicians when the 22 teen participants had interrupted sleep and insufficient physical activity.6 FitBit-measured sleep quality moderately correlated with sleep disturbance scores (average r=−.31; P =.01) and was significantly correlated with pediatric asthma impact scores (average r=−.18; P =.02).6
PROs can aid clinicians and engineers in building a better inhaler and understanding how patients use the devices. In a 503-patient study, participants were asked about the performance and convenience of their inhalers.7 According to the participants, the top 3 key features in an inhaler were:
- Assurance that the medicine reaches the lungs
- Ease of inhaling into the device
The inhalers' most important features corresponded to 6 of 7 items in the Patient Satisfaction and Preference Questionnaire (PASAPQ) performance domain rather than the convenience domain. The findings underscore the importance of patients being comfortable with their inhalers because this tends to predict adherence to their regimens.7
Combining objective and subjective measures can predict patients' long-term asthma outcomes. That was the case in a study of 181 patients (average age, 41 years) in which biomarkers interleukin-5 and interleukin-6, and lung function (FEV1) were measured with patient self-reports on asthma behavior (Clinicaltrials.gov identifier: NCT00421018). Lodin and colleagues found that patients whose self-reports had low scores also tended to have worse outcomes than patients who reported more positive answers about their asthma self-management (P <.0001).8
Implementing PROs Into Practice
It is not only clinical trials that use PROs; clinicians have found that the measures help them provide patients with better outcomes. When used in context with objective parameters, both patients and physicians learn from the findings.
“We can learn that they might find it difficult to adhere to their medication regimen. This can help us adjust therapy, for example,” says Dr Fishbein. “Or we might find that they are not nervous about their asthma and are very happy with their management plans. PROs are a nice way to engage the family and understand their perspective. PROs using PROMIS measures are being incorporated into the electronic medical record, and will also allow physicians to monitor symptoms at home. It is another tool that helps in patient assessments and gives a full picture of their disease burden.”
Dr Huang agrees, “PROs are comprised of different domains (eg, pain, fatigue, asthma symptoms) and individual domains capture different and unique aspects of health status. Most importantly, PRO data collection is much cheaper than clinical assessment, and PRO assessment can be done in the clinical and home settings. PROs can inform clinicians of specific health issues that are patient-centered (or really matter to patients but are not captured by regular objective assessment). PRO information can be used to screen potentially abnormal health status and initiate conversation between patients and clinicians during the clinical encounter.”
To take full advantage of PROs in practice, Dr Chipps advises, “Clinicians need to look at risk and impairment domains to assess patients' risk for exacerbations.”
Summary & Clinical Applicability
Clinicians realize that patient-reported outcomes are critical to their understanding of patients' disease burden and the effects of treatment. Although a recent systematic review found that fewer than10% of all asthma clinical trials have included PROs, more trials will include these parameters as a condition for drug approval and more clinicians will adopt them as they strive to deliver more patient-centered care.
- Braido F, Baiardini I, Canonica GW. Patient-reported outcomes in asthma clinical trials [published online October 13, 2017]. Curr Opin Pulm Med. doi:10.1097/MCP.0000000000000440
- U.S. Department of Health and Human Services. Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims. ww.fda.gov/downloads/drugs/guidances/ucm193282.pdf. Accessed November 6, 2017.
- European Medicines Agency. Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products. www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003637.pdf Accessed November 6, 2017.
- Chipps BE, Zeiger RS, Luskin AT, et al. Baseline asthma burden, comorbidities, and biomarkers in omalizumab-treated patients in PROSPERO [published online October 17, 2017]. Ann Allergy Asthma Immunol. doi:10.1016/j.anai.2017.09.056
- Jones CM, DeWalt DA, Huang IC. Impaired patient-reported outcomes predict poor school functioning and daytime sleepiness: the PROMIS pediatric asthma study. Acad Pediatr. 2017;17(8):850-854.
- Bian J, Guo Y, Xie M, et al. Exploring the association between self-reported asthma impact and Fitbit-derived sleep quality and physical activity measures in adolescents. JMIR Mhealth Uhealth. 2017;5(7):e105.
- Davis KH, Su J, González JM, et al. Quantifying the importance of inhaler attributes corresponding to items in the patient satisfaction and preference questionnaire in patients using Combivent Respimat. Health Qual Life Outcomes. 2017;15(1):201.
- Lodin K, Lekander M, Syk J, Alving K, Petrovic P, Andreasson A. Longitudinal co-variations between inflammatory cytokines, lung function and patient reported outcomes in patients with asthma. PLoS One. 2017;12(9):e0185019. doi:10.1371/journal.pone.0185019