Qualitative patient-centered research and evaluation of COAs

Adelphi Values PCO are leaders in the conduct of qualitative research to obtain in-depth understanding and information about the experiences, perspectives, priorities and preferences of patients and other stakeholders. The value of this qualitative patient experience data is evident at all stages of medical product development and evaluation.

Role of qualitative research in drug development and evalution

Pre-discovery research

Understand unmet needs
Important/valued theraputic tagets

Drug discovery

Priority setting
Define and refine target population

Pre-clinical development

Develop target product profile
Inform clinical study design and selection of end points

Clinical development

Support development/selection of COA measures
Define meaningful change
Provide insights as to patient experiences of investigational product
Refine clinical study design

Regulatory approval

Facilitate benefit/risk evaluation
Communicate information to patients/precribers (labelling)

HTA approval

Understand the value of new drug to patient
Facilitate economic evaluation

Post-marketing

Patients’ experiences post-approval
Safety monitoring
Education and communication

Qualitative patient-centered research and evaluation of COAs (1)

Insights from qualitative data are notably critical in the development and content validity testing of clinical outcome assessments (COAs). We have in-depth understanding of various qualitative methodologies and possess the flexibility in thinking to apply them appropriately and creatively to COA instrument development and evaluation. Our qualitative research methods are well-accepted by regulatory authorities and have supported many Food and Drug Administration (FDA) and European Medicines Agency (EMA) patient-reported outcome (PRO)/COA submissions, providing the evidence that COAs are fit-for-purpose in the target population. Being well-versed in regulatory expectations for qualitative research we know what aspects are essential and which are ‘nice to have’.

Adelphi Values PCO have experience of conducting qualitative research in a wide range of diverse medical conditions in countries across the world. We are cognizant of cultural, linguistic, and practical considerations associated with the conduct of such research within certain patient populations or samples. Efficient recruitment of participants is critical to the to the success of any qualitative study. We have an extensive network of trusted patient recruitment partners in over 30 countries and regularly partner with patient advocacy groups or other charitable bodies to access specific or hard to reach disease populations. Working alongside our trusted partners for recruitment, moderation and transcription/translation activities, we oversee qualitative research projects from start to finish.

Planet Qualitative patient-centered research

Insights from qualitative data are notably critical in the development and content validity testing of clinical outcome assessments (COAs). We have in-depth understanding of various qualitative methodologies and possess the flexibility in thinking to apply them appropriately and creatively to COA instrument development and evaluation. Our qualitative research methods are well-accepted by regulatory authorities and have supported many FDA and EMA PRO/COA submissions, providing the evidence that COAs are fit-for-purpose in the target population. Being well-versed in regulatory expectations for qualitative research we know what aspects are essential and which are ‘nice to have’.

Concept elicitation interviews and focus groups

We lead the field in the rigorous conduct of concept elicitation interviews and focus groups to high standards to meet clients’ clinical outcome assessment (COA) instrument development goals. Our in-depth understanding of what is important in concept elicitation interviewing and qualitative analysis means that we are able to apply our methods to elicit in-depth, high-quality data from a range of participant groups including patients, caregivers/parents and clinicians. We have particularly strong expertise in conducting interviews with children, in rare diseases and other special populations where greater creativity can be required.

Hallmarks of our approach to concept elicitation research are the following:

Open-ended enquiry and techniques to maximize opportunity for spontaneous elicitation of concepts by participants
Use of creative tasks (e.g., drawing tasks, mood boards, timeline tasks, importance rating) to facilitate discussion and elicitation of concepts where necessary
Use of dynamic probing to ensure full understanding of concepts of interest
Formal analysis of qualitative transcripts/data using software (ATLAS.ti) and based on thematic analysis methods
A focus on organization and visualisation of complex and in-depth qualitative data in concise and compelling ways

We are also experienced in using a wide range of digital methods, alongside or in place of traditional qualitative research methods (e.g., interviews or focus groups), including:

Mobile apps to elicit ‘real time’ disease experience data in a number of modalities (text, audio, photo or video)
Use of bespoke online communities, focus groups or blogs
Instant messaging platforms

These methods can facilitate access to hard-to-reach participants, reduce participant burden, increase flexibility of scheduling, reduce recall bias, improve ecological validity and increase patient engagement in the research.

Cognitive debriefing interviews

It is important that clinical outcome assessments (COAs) employed in research studies are consistently and correctly interpreted by respondents. Adelphi Values have been at the forefront of the development and refinement of cognitive debriefing interviewing as a central activity in the development and testing of COA instruments. Our cognitive debriefing research has been extensively published and is well accepted by both the Food and Drug Administration (FDA) and European Medicines Agency (EMA).

Typically, we employ a mixture of using a ‘think-aloud’ method (asking patients to speak their thoughts aloud as they read and respond to the questionnaire) and following up with specific and detailed debriefing questions to gain insight regarding patient understanding, and the relevance and appropriateness of items, instruction, response options and recall periods. Our skilled interviewers are highly experienced in eliciting feedback from hard-to-reach groups such as young children and patients with low affect.

As part of a mixed methods approach to COA development, Adelphi Values advocate performing pilot testing or feasibility testing prior to the conduct of cognitive debriefing interviews. This involves patients completing the newly-developed patient-reported outcome (PRO) measure (commonly daily diaries completed on an ePRO device) at home in a ‘real world’ setting for a period of several days prior to their interview. This enables respondents to reflect on their actual experience of completing the diary in real-life, rather than having to think hypothetically. This is particularly valuable for children who have difficulty thinking beyond the here and now. Moreover, collection of data during this period can provide early insight into item response distributions and trends which can then be further explored during the cognitive debriefing interviews.

In-trial/exit interviews (Enhance)

In-trial/exit interviews and surveys are qualitative methods used to explore, in-depth, the patient (or other informant) perspective having participated in a clinical trial. Patient experience data collected within the context of a clinical trial can provide unique insights not always available from standalone qualitative studies. This data can provide context to trial outcomes, inform measurement strategies, future trial designs, and support modification or refinement of clinical outcome assessments (COAs). Specifically, in-trial/exit interviews and surveys can be used to:

Increase understanding of the patient experience of symptoms and/or medication benefits and side effects
Understanding treatment satisfaction and provide insights into adherence
Obtain feedback on clinical trial participation, including trial procedures, feasibility of COA completion and device usability
Generate additional qualitative evidence to support the content validity of COA instruments or refining COAs through cognitive debriefing
Obtaining perspectives to aid interpretation of meaningful change for COA measures

Adelphi Values have a wealth of knowledge and experience from conducting in-trial/exit interviews across a range of patient populations and clinical trial designs. We have an appreciation and comprehensive understanding of methods for obtaining patient input, logistical considerations and compliance requirements. As such we are ideally placed to help sponsors conduct interviews to a high standard; generating accurate, meaningful and valuable outputs in an efficient and cost-effective manner. This is reflected in our Enhance™ service for the conduct of in-trial/exit interviews.

Defining meaningful change for COA scores

With a strong understanding of regulatory expectations and our expertise as measurement scientists, Adelphi Values employ a range of qualitative methods to explore which concepts would be most meaningful to change, as well as the level of change on a clinical outcome assessment (COA)/anchor assessment that would be meaningful and why. Investigations of meaningful change are commonly explored independent of clinical trials based on hypothetical changes, but are increasingly being used within in-trial/exit interviews which provide the opportunity to explore actual change experienced due to the investigational treatment.