The Dry Eye Zone

Rebecca's Blog


Study: Involving patient preferences in the early drug development process

Ooh, ooh, ooh, don’t look now!

It’s that magic phrase: “Patient preference”.

Do you have any idea how rarely that comes up in my PubMed feeds about dry eye or anything related to it?

Hello. I’m a patient. If you’re trying to fix my problem, it might help if you had a quick word with me. You know, so you don’t end up hacking all the way through the wrong proverbial jungle?

So I’m excited and headed to look up the authors. Whoops - I see it’s a Novartis study. I’m swallowing my tongue and striving mightily to stifle my bias and keep an open mind about my expectations.

Then I read the paragraph.

Naw. Same old same old. I feel profoundly skeptical when I read phrases like:

…working to embrace fully the patient perspective in product development and healthcare decision-making”

You want to embrace the patient perspectives? No lip service, no marketing companies. First involve patient groups and nonprofits (like the Dry Eye Foundation) and don’t attempt to superadd an interpretation of patient views to a longstanding foundation of assumptions.

Going about it as described here might do what you want it to: add a patina that appears patient-friendly and please everyone for whom “patient-centric” and “patients as stakeholders” are THE current buzzwords. But it won’t actually be what it claims to be. Period.

Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs. Cook et al, Front Med (Lausanne). 2019 Apr 24.

Although patient preferences have been studied broadly for marketed products or around the time of submission to authorities and launch, patient preference studies have rarely been used during the early drug development phases. In this paper, we formulate three hypotheses supporting the use of patient preference studies in early product development: (1) integration of the patient perspective into the development process from phase 1 onwards will result in healthcare solutions with outcomes that best address patients' needs; (2) a structured process to build patient-based evidence involving partnerships between patients and other key stakeholders will improve alignment of development activities with the needs of patients; (3) quantitative patient preference research built on robust qualitative insights is necessary to strengthen development decisions in the interests of patients. To illustrate such a structured process, we describe qualitative insights research (social media analysis and online bulletin boards) and quantitative patient preference studies in dry eye disease and non-alcoholic steatohepatitis conducted during early product development by a pharmaceutical company to generate patient-based evidence. The outputs from such early patient preference studies are being used to inform patient reported outcome strategies, clinical development strategies, product design and delivery features, and form the basis for early dialog with regulators, health technology assessment (HTA) bodies and payers to ensure focus and alignment on patient-relevant endpoints. Furthermore, to discuss and theoretically substantiate our hypotheses, we review how different groups and organizations are working to embrace fully the patient perspective in product development and healthcare decision-making. The hypotheses are commensurate with the general trend toward patient-centered healthcare and the activities initiated by regulators, HTA agencies, and patient organizations. We advocate that all healthcare players should actively contribute to aligning on best practices concerning choice of methodologies and engage in multi-stakeholder dialog along the entire product development chain, to realize health technologies that best meet the needs of patients.