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Patient-Focused Drug Development (PFDD) — the FDA Guidance that Changes How Life Sciences Works


With its draft guidance, Patient Focused Drug Development, the FDA is encouraging the industry to include patients in a meaningful way in drug development — from pre-IND through commercialization. Many manufacturers aren’t fully aware of the implications this guidance will have on their business.  As a result, few have made significant progress incorporating the guidance into their culture. 

PFDD is a byproduct of the 21st Century Cures Act, an act of the US Congress designed to streamline drug development and approval, and support many improvements in healthcare delivery in the US. The Cures Act, as it is known, was issued during the Obama administration and is one of the few Obama-era actions reinforced under the Trump administration. In the act, Congress tasked the FDA with bringing the drug development piece to life, resulting in PFDD.

During the last six years, the FDA has been bringing together patients, patient advocacy groups, researchers, and industry professionals to hear about health management from all perspectives. Though the meetings have been therapeutic-area focused (e.g. cardiovascular, immunology, hematology, gastroenterology, etc.), the emerging general consensus is that the resulting guidance must be therapeutic-area agnostic, especially if PFDD is going to really be about patients. As a result, the FDA is changing its directionality towards medicines from simply randomized clinical trials (RCTs) to include patient input in both the evaluation process during trials, and the process of monitoring products once approved.

A sea change in how the life sciences industry does business

Only 11.3% of all clinical trials are ever completed, often due to the protocol disrupting the study participants’ daily life.* Currently, protocols for clinical trials are developed by researchers and clinical operations teams with limited resources to understand patient perspectives and patient journeys. That is going to change in big way going forward.

Due to the PFDD guidance, we expect to see pharma companies increase their focus on patient centricity in protocol development. With insights from a patient, protocol developers might, for instance, learn that the blood draw they’ve always required be done at the clinical trial site presents a huge inconvenience to a patient. And the protocol team thought all they had to do was pay for travel to keep participants happy! Feedback from patients brings considerations that were largely overlooked into the forefront of shaping study protocol. 

Similarly, in the case of study-required blood draws, patients could weigh in about how they could lose a paid day or days of work if required to come to the clinical site for blood draws, or how it could impact their ability to be at home to take care of others in their lives. Factoring in these considerations, a simpler, and more patient-friendly solution could be developed for the protocol. Perhaps the blood draw could happen locally at a designated lab, or maybe a nurse or technician could come to the patient’s home or workplace for blood draws or monitoring.

Ultimately, adding these types of conveniences would improve the possibility of patients completing trials. The result: the pharma company gets the data it needs, and patients ­— who are generous enough to give their time and bodies for these studies — get to live their lives with little or no disruption while helping the industry advance research.

Applying PFDD in the real world

The chart below from the FDA describes where and how it thinks PFDD can be applied. It also demonstrates that PFDD does indeed span the entire drug development process from pre-IND through commercialization.


With the advent of PFDD, real world data/real world evidence (RWD/RWE) becomes even more important as the FDA puts emphasis on collecting data once a drug or device becomes commercialized and is out in the real world.

During the US government shut down of 2018/2019, then-FDA commissioner Gottlieb announced a restructuring in the FDA’s drug review process to enable better analysis of:

  • Patient-reported outcomes
  • Real-world data
  • Technology use in drug development

Even as Gottlieb departs, the process is in place to acknowledge the impact of these three areas on better assessing drugs and devices at every point of the drug development process.

This also emphasizes the increasing significance of data in healthcare.  We see this in many of our solutions, including our Electronic Visit Verification services, which is also mandated by the 21st Century Cures Act.  This service helps states follow patients throughout their healthcare journey [insights] and uses data to improve efficiencies and health outcomes.

With PFDD, we also know the impact  RWE can make through the work we do handling reimbursement and patient access programs.  

The primary goal for these access programs is to ensure access to a specialized drug for patients managing a rare disease condition. We see a huge opportunity to infuse adherence solutions and drive better results for our clients. Since we’re in two-way conversations with patients as part of these programs, it becomes an opportunity to assess barriers and attitudes around managing the patient’s health. We now gather information that allows us to learn what actions we can take to help improve patient adherence to medications. We’re collecting patient data in real time and leveraging technology and analytics to drive actionable insights. As a result, our clients can see strong improvements in adherence rates for patients, and consequent, improved patient experience and revenue. 

Conduent has the unique ability to support the industry’s efforts to collect, manage data and comply with the requirements of PFDD through how the company currently manages data across payer, provider, and manufacturer.

A new paradigm

PFDD will change the life sciences industry business model. It will help balance the needs of patients, healthcare providers, and brands — and, finally, give companies the leverage they need to make being patient-centered a business imperative, not just an altruistic effort.

FDA leadership considers PFDD such a sensible way to do business that it hasn’t chosen to take steps to ratify the draft guidance into final guidance — the thought being that the industry will recognize the value of being patient-centered without the necessity for PFDD become law.

But, some companies have recognized the value and incorporated PFDD into how they do business instead of waiting for the FDA to ratify the guidance — and already benefitted. More on that in our next installment, along with examples of companies that have incorporated PFDD into their corporate cultures and the impact it has made.

Here’s more on PFDD and its ramifications:

The FDA Draft Guidance – Patient-Focused Drug Development -


*Profiles of New Approaches to Improving the Efficiency and Performance of Pharmaceutical Drug Development, Tufts Center for Drug Development


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