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W-22: Tell Me More: Exploring Patient Perspective on the Benefits and Disadvantages of Drugs During Clinical Trials

Poster Presenter

Alexis Miller

Acting Global Lead, Global Regulatory Policy and Intelligence
Merck & Co., Inc.
United States

Objectives

To present first use of a new patient-reported outcome (PRO) questionnaire (Patient’s Qualitative Assessment of Treatment (PQAT)) exploring their perception of benefits and disadvantages of drugs during clinical trials (in free text), balance made between them and willingness to continue treatment.

Method

The PQAT was completed on an electronic tablet by 57 English-speaking patients at the end of treatment period (26 weeks) or early discontinuation, in a phase 2 trial of a new injectable treatment in patients with type 2 diabetes. Data were analyzed using qualitative and quantitative methods.

Results

The PQAT was well understood by patients and the mean completion time was approximately 5 minutes. When asking about benefits of treatment received (PQAT open-ended question 1), patients reported objective clinical markers of efficacy (improved or controlled blood glucose levels, weight loss) and more subjective markers not generally considered in clinical decision-making (feeling better, improved energy levels). Disadvantages reported by patients in the 2nd open-ended question were mainly related to drug side-effects (nausea, vomiting, diarrhea, fatigue, stomach pain or headache) or to the injectable mode of administration. A large proportion of patients (70.2%) reported being willing to continue with the treatment at the end of the trial (PQAT question 3), even though many of them reported disadvantages. When asked to balance benefits of the drug received to the disadvantages experienced on a 7-point scale (PQAT question 4), 59.6% of patients reported that the benefits outweighed the disadvantages (+1 to +3), 15.8% reported equal benefits and disadvantages (0) and 24.6% reported that the disadvantages outweighed the benefits (-1 to -3). The majority of patients discontinuing treatment early (n=9/14) reported that disadvantages of treatment outweighed the benefits. Some disadvantages (fatigue, headaches and sleep disturbances due to pain) were only reported by patients who discontinued the new treatment. Among the patients who reported fatigue (n=5), headaches or stomach pain (n=4), 75% to 80% of them were not willing to continue the treatment and all of them reported that the disadvantages of the drug received outweighed the benefits. On the contrary, all patients who reported feeling better (n=10) and improved energy levels (n=7) were willing to continue with the treatment, and the majority of them (70% and 71.4%, respectively) reported that the benefits significantly outweighed the disadvantages by selecting the greatest response on the scale (+3).

Conclusion

The PQAT is a unique PRO tool developed to collect individualized patient-perceived benefits and disadvantages of treatments, and the balance patients make between them. It combines the richness of patient-reported qualitative free-text data with quantitative data that can be used to facilitate statistical comparisons between groups of patients. Compared to patient preference evaluation, which often relies on respondent evaluation of hypothetical scenarios, the PQAT provides data based on patients’ real-life experiences of drugs received during clinical trials. It helps to understand patients’ attitudes toward treatments and the true impact of side-effects. There are a number of learnings from this first administration of the PQAT. Firstly, patients’ responses to open-ended questions were brief and detail was limited. Further instructions and training could be provided to patients for PQAT use in future clinical trials to maximise the depth of information obtained. However, there appeared to be no issues with completion via the tablet device. The PQAT is a cost and time-efficient method for the collection of patient experience data and as a generic measure can be employed widely across multiple studies and conditions. In a context where the patient engagement is increasingly encouraged, personal accounts of patients’ experience of treatments is of value to various stakeholders. Specifically, patient-perceived benefit/risk evaluations can inform regulatory authorities and health technology assessments bodies for product assessment and decision-making; prescribers will have a broader picture of medical products to communicate with their patients and to inform personal treatment decisions; and patients will be pleased to better understand the benefits and disadvantages of treatments shared by their peers before taking a new drug.