The FDA’s patient-focused drug development (PFDD) guidelines are an attempt to put patient-centricity at the heart of drug development. In the memorable words of Devesh Verma and Rebecca Lorenzo from PharmExec.com, the guidelines signal a shift in culture to “selling health versus pills.”
“To ensure a patient-focused approach to medical product development and regulation, FDA is developing guidance on methods to identify what matters most to patients for measurement in clinical trials; specifically, how to design and implement studies to capture the patient’s voice in a robust manner.”
The FDA PFDD guidelines are broken into four distinct documents that span the entirety of the drug development process – from discovery to post-approval. But how can pharma teams incorporate this new guidance in a way that supports patient centricity?
Let’s unpack each of the four guidance documents, and how they intend to bring a patient-first focus to drug development.
1: Collecting Comprehensive and Representative Input
The first guidance document focuses on how stakeholders can “collect and submit patient experience data” for product development and regulatory decision-making. That’s the comprehensive part. Representative input refers to who stakeholders are gathering data from – the target population for a proposed trial.
Here, the FDA is encouraging pharma teams to ensure they’re gathering accurate patient insights from a diverse and representative population. By doing so, they’re establishing a patient-centric approach to drug development right from the get-go, ensuring that the right individuals are included and listened to.
Within3 clients are already well-equipped to do this, because access to an asynchronous virtual platform removes the roadblocks to patient engagement – such as work or home commitments, access to transportation or travel, and health limitations. By making it easier for patients to engage, life science organizations can ensure more patient representation.
Related content on diverse representation in clinical trials and beyond:
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2: Methods to Identify what is Important to Patients
If the first guidance document was about the who, this second one is about the how and the what.
“Guidance 2 will discuss methods for eliciting information from individuals identified in Guidance 1, gathering information about what aspects of symptoms, impacts of their disease, and other issues are important to patients.”
This guidance document promotes a strong focus on the patient experience – what matters to them, what they like and dislike about the trial process, the challenges they face and how pharma teams can make life easier for them. Identifying what’s important to patients will help teams minimize disruption to their lives – in turn, reducing trial dropout rates and helping pharma companies gather the data they need. It’s designed to be mutually beneficial.
“Feedback from patients brings considerations that were largely overlooked into the forefront of shaping study protocol.”
3: Selecting, Developing or Modifying Fit-for-Purpose Clinical Outcomes Assessments
The third FDA guidance document concerns how pharma teams refine the insights gathered through the first two guidance phases into something actionable and applicable. As the FDA puts it: “Not everything identified as important by patients, caregivers, and clinicians can demonstrate change in a specific treatment trial or is measurable.”
Pharma teams must carefully select which insights to act upon when building a medical product development program to ensure clinical benefit while maintaining a culture of patient centricity. It isn’t always about the quantity or even the quality of data gathered, but how, when, and where teams apply those learnings.
By using technology like natural language processing and sentiment analysis, life science teams can understand insights across engagements with patients and experts alike. When you understand what people are really thinking and feeling during the drug development process, it’s easier to apply those insights to a coherent business strategy with patient centricity at its core.
4: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision Making
The final guidance document in the PFDD series covers the ‘collection, capture, storage, and analysis’ of clinical outcome assessment (COA) data. COA data is a crucial component of patient centricity, often putting the patient experience into patients’ own words. The FDA’s intention is that COAs be incorporated into regulatory decision-making, ensuring patient centricity is encoded throughout the drug development process moving forward.
PFDD has been designed to ensure the needs of patients, pharma companies, and HCPs are all met throughout the drug development process. It reflects the fact that a patient-centric approach to drug development is not just beneficial for patients, but potentially of immense value to pharma companies too.
“87% of patient-centric clinical trials have positive results, vs 68% of traditional trials.”
Adopting a patient-centric culture can help pharma companies gain valuable insights, improve clinical trial retention rates and enhance the overall patient experience. Ultimately, acting on guidance in the FDA PFDD guidelines won’t just be seen as the right thing to do but as a business imperative. Read our blog to learn more about how a patient-centric culture benefits life science organizations, or find out more about regulatory affairs in the pharma industry.