About Patient-Focused Medical Product Development

Patient-focused medical product development is a mechanism by which stakeholders, including but not limited to medical product developers, academics, and regulators, form a partnership with patients to enhance medical product development, throughout the research, regulatory, and reimbursement processes. PFDD is built on the belief that:

  • understanding patient experiences, goals, and needs leads to: clinical trials that are more efficient and measure outcomes patients really care about;
  • more commercially successful medicines because potential barriers to uptake were identified and addressed earlier;
  • and health technology/value assessment that evaluate drugs based on patient-defined value. 

FDA’s Patient-Focused Drug Development Initiative 

The Food and Drug Administration’s Patient-Focused Drug Development (PFDD) initiative began under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). Originally, FDA’s PFDD initiative referred to a series of disease-specific meetings hosted by the FDA. Following each of these meetings, FDA published a Voice of the Patient summary report. Subsequently, a process was established for patient organizations to host their own PFDD meetings (see Externally-led PFDD). Under PDUFA VI, FDA committed to developing four guidances on PFDD. For the most up-to-date information, please visit the FDA’s PFDD website.

Case Examples

Today, PFDD stakeholders are interested in learning how patient-provided information can be applied to improve medical product development. The National Health Council is collecting and publishing real-world case examples of patient-focused medical product development successes. The list below is intended to be a living document and will be updated with new cases as they are identified. For questions or to submit a case example, please contact Elisabeth Oehrlein.

1. Pediatric Assent

Lilly worked directly with iCAN (International Children’s Advisory Network) and UC-Berkeley to improve its approach to the typical pediatric/adolescent assent form. When a child is too young to sign a consent form, the parent provides the consent. However, the children can also give their assent to participate in the clinical trial. Lilly researchers worked alongside patients in the iCAN program to create assent templates for children/adolescents that tell the story of what it’s like to participate in a clinical trial. Similar to a picture book, the colorful template follows kids as they experience blood draws, X-rays, and other procedures. Here is a more detailed description the program.

  • Key point: Lilly researchers updated assent forms to better engage young patients in clinical trials by working with young patients to create them and incorporating their ideas into the updated version.

2JAKAFI® (ruxolitinib)

Jakafi (ruxolitinib) was approved by the FDA in 2011 for the treatment of intermediate or high-risk myelofibrosis. One of the endpoints used to support FDA approval was demonstrated improvement in myelofibrosis-related symptoms. Symptoms were measured by a novel patient-reported outcome (PRO) measure, the modified Myelofibrosis Symptom Assessment Form (MFSAF) version 2.0 diary. The MFSAF 2.0 was the result of additional qualitative patient interviews, cognitive debriefing, and validation testing conducted by the sponsor in order to meeting the requirements outlined in FDA’s 2009 PRO guidance. See Acquadro and Regnault.

  • The FDA uses this as an example of successful Clinical Outcome Assessment development.

3. Maestro Device

In partnership with FDA, RTI Health Solutions (RTI-HS) conducted a preference study to evaluate the tradeoffs that patients would make among effectiveness, safety, and other attributes of weight-loss devices. Based on these results, the researchers estimated the maximum mortality risk patients were willing to accept for a certain amount of weight loss, and the minimum amount of weight loss sufficient to undergo the risks of a weight-loss device.

  • FDA acknowledges that this was the first time a patient-preference study impacted a new device approval.  

4. NxStage’s home hemodialysis device

In August 2018, for the first time, FDA cleared an expanded indication for a home hemodialysis machine. The device was originally cleared for hemodialysis at home, but only in the presence of a trained care partner. This meant that patients who did not have a trained care partner at home were not eligible for in-home hemodialysis. The sponsor worked with FDA and the Kidney Health Initiative to develop and conduct a patient-preference study to understand whether patients are willing to accept the risks associated with hemodialysis at home without a qualified care partner. The FDA cited this survey in their decision based in part on asking kidney patients about their tolerance for risk.

  • Data stemming from the preference study were used to expand the indication.

5. Rituxan Hycela

Rituxan Hycela (indicated for different lymphomas) was approved by the FDA in June 2017. The FDA approval is based on results from clinical studies that demonstrated subcutaneous administration of RITUXAN HYCELA resulted in non-inferior levels of rituximab in the blood (pharmacokinetics) and comparable clinical efficacy outcomes compared to intravenous Rituxan. Importantly, one of the clinical studies demonstrated that the majority (77%) of patient participants preferred RITUXAN HYCELA over intravenous Rituxan, with the most common reason being that administration required less time in the clinic. These findings are reflected in section 14.4 “Patient Experience” of the product label.

  • This appears to be the first example where a “Patient Experience” section appears in a US product label.

Related resources: