Patient-Focused Medical Product Development Case Examples

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.

  • Key point: 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.

  • Key point: 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.

  • Key point: 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.

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

6. Addressing Safety Concerns in Device Design: Dexcom G5 Continuous Glucose Monitoring System and Animas Vibe System (Johnson & Johnson)

The FDA discussed with patients, parents/care partners, and patient groups their concerns about the safety of the young pediatric diabetic population using an insulin pump. These conversations included how the device would be used by the young population once approved by FDA and what kind of safety considerations might be relevant.

Based on this feedback, FDA worked with the company to develop additional risk mitigation strategies that included a lockout feature to prevent unintended boluses by young children who might be tempted to play with the insulin pump.

Key point: Meaningful patient input allowed for a safer device to come to the market. The incorporation of the patient perspective in the development of the pump led to a safe device for young people being put on the market and mitigated parents’ concerns over their children managing their own diabetes.

7. Capturing Symptoms Psoriasis Patients Care About:  Cosentyx  (Novartis)In February 2018, the FDA approved an updated label for Cosentyx (secukinumab) based on data gathered using the Psoriasis Symptom Diary, a patient-reported outcome measure. The Psoriasis Diary was created with meaningful patient engagement and addressed issues patients shared that matter to them the most. These included psoriasis symptoms of burning, stinging, and scaling, as well as embarrassment due to plaques and avoiding activities with others due to the look of affected areas. These were gathered through qualitative interviews with psoriasis patients and psoriasis experts.

Key point: The Psoriasis Symptom Diary, a PRO measure, captures what patients report is most important to them and was included in the updated label for a treatment of plaque psoriasis.

8. Patient input into formulation and packaging (Dermira)As Dermira considered the formulation for a new product, staff conducted a focus group of patients for whom the drug was designed. Dermira developed various formulations of the new product and anticipated the most sophisticated formulation would be preferred by patients. However, patients preferred a more traditional formulation – stating they could feel the cream being absorbed into their skin.

Additionally, patients suggested that a plastic tube would be better than a traditional metal tube. Patients want to squeeze all of the cream out of the tube, but when the metal tube is folded over, it can become sharp and cause cuts on hands.

Key point: Patient input was useful in determining a product’s formulation and user-friendly packaging.

9. Chicken or Egg Symptoms? (Johnson & Johnson)For a particular condition, researchers knew that there was a pattern that sleep disturbances often accompanied a disease flare. Based on information from patients, the researchers learned that the sleep disturbance might be predictive that a flare is about to happen. So, patients used a wearable device to monitor sleep disturbances and flares to assess the relationship. Patient input resulted in additional data being collected that informed the natural history of the disease.

Key point: Including the patient voice in medical device development allowed for the collection of data, informed the natural history of the disease, and gave researchers another way to track predictors of a disease flare.

10. Patient Labeling (Merck)Merck works to create patient labels that are easily understood. To create new patient labels, Merck first worked with health literacy experts at Northwestern University and Emory University to conduct a study on health literacy.  The study evaluated participants’ health literacy by asking them to read six medical guidelines. Researchers learned graphics and font need to match a participant’s comprehension level. By the end of the study, participants were more comfortable reading and comprehending medical guidelines when plain English language was used, the layout and typography was clean, and accompanied by clear directive tasks and descriptive graphics.

Key point: Including the patient voice in the development of patient-friendly labels helps ensure sound communications, which can help patients to better understand medical conditions and appropriate treatments available for them.

11. Clinical Trial Design is a Two-Way Street: CoLAB (Eli Lilly & Co.)CoLAB is a clinical-trial project that has been ongoing at Eli Lilly & Company (Lilly) for the past six years. The purpose of the initiative is to integrate clinical researchers, patients, health providers, and study coordinators in drug development through in-person simulations of clinical trials. During simulations, a “dress rehearsal” of a clinical trial occurs, which helps quell the uncertainty and anxiety that can come from the clinical-trial experience. Each simulation gives researchers the chance to reduce study burden on patients. Adjustments to protocols that have results include changes to medication delivery to blister packs from bottles to help people unable to open child-proof packages and reducing the number of invasive procedures needed or eliminating them entirely.

Key point: Engaging with patients early and often throughout the clinical-trial protocol-development process lessens patient burden and allows patients and health care professionals to build stronger relationships and design treatments that patients are more likely to use.

12. Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Guidance for IndustryParent Project Muscular Dystrophy held a policy forum on Duchenne Muscular Dystrophy in December of 2013 that included patients, patient providers, researchers, and members of the FDA to discuss what would be the foundations for their proposed FDA guidance plan. The resulting multi-stakeholder, comprehensive, and patient-initiated draft guidance to the FDA is entitled, “Guidance for Industry Duchenne Muscular Dystrophy Developing Drugs for Treatment over the Spectrum of Disease” and was presented to the FDA in June of 2014.

Key point: This was the first time a patient community drafted its own guidance for the FDA, that the FDA used it as a template for their own guidance.

13. Designing Clinical Trials with Participants in Mind (Janssen)In 2017, Janssen researchers asked patients in a clinical trial for age-related macular degeneration to participate in mock trial initiation at a clinical trial site. Researchers learned that shortening visits and providing transportation to the site would improve people’s experiences participating in the trial. The following year, no patient withdrew from the actual trial, which is a significant improvement over the general drop-out rate of 18%.

Key point: Patient-engagement in trial protocol development can improve trial participation and reduce trial dropout rate.

14. Giving patients access to their health data (Janssen)Patients want access to their personal health data as they participate in clinical trials. Janssen created a way for patients to access some of their data while maintaining the integrity of the research. Patients have access to their lab results and other data collected during trial visits. In one Phase 3 study, patients who opted in for “data return” received the complete set of health data collected during their first exam.

Key point: Personal data are important to participants in a clinical trial so researchers created a platform where participants could access it, no matter what point they reached in the clinical trial process.

15. Patient-reported Outcomes and Endpoints (EMD Serono)EMD Serono partnered with the Accelerated Cure Project (ACP) for Multiple Sclerosis and its people-powered research network, iConquerMS, to integrate patient perspectives into the design, implementation of the clinical trials, and the distribution of patient-facing materials. Patients provided insights and feedback on the choice of patient-reported outcomes (PROs). Specifically, two PROs – the “change from baseline in Patient Reported Outcomes Measurement Information System (PROMIS) MS Physical Function (PF) and the PROMIS MS Fatigue Score at 96 weeks” – were included as secondary endpoints in the trial.

Key point: Based on collaboration with patient organizations from the beginning of drug development to their measurement in clinical trials, ensuring researchers are measuring outcomes MS patients care about.

16. Disease-Expert Panel DEEP Model (Novo Nordisk)In 2015, Novo Nordisk launched their Disease Experience Expert Panel (DEEP). The DEEP model partners with members of the community with the disease of interest and engages members at all stages of research, development, an implementation of clinical trials. In their obesity clinical trial program, researchers worked with DEEP members throughout trial development and learned that going to a doctor’s office was difficult for many because of the worry about being able to comfortably use the furniture in the waiting room and the examination room. From that point forward, clinical trial teams worked to ensure study spaces had furniture that could adequately accommodate participants’ physical needs.

Key point: Patient engagement provided information that was previously unknown by researchers and clinicians that could help to make patient more comfortable with the clinical trials experience.

Related resources: