Patient-Focused Drug Development (PFDD)

Patient Focused Medicines Development (PFMD)

Based on the 21st Century Cures Act in June 2017 the US FDA issued guidance to help us better engage with patients, caregivers, and families to produce improved outcomes. In the EU, the European Medicines Agency (EMA) has issued similar guidance – Patient Focused Medicines Development – PFMD. There are parallel activities of which you should be aware. 

What is the PFDD guidance?

The guidance simply says “patients should be involved with drug development in a meaningful way from pre-IND through commercialization

Additional resources:

FDA PFDD Patient Advocacy Workshop slide deck – March 19, 2018

, click thisScott Gottlieb statement re PFDD, April 2, 2018

FDA PFDD draft guidance, June 2018

FDA chart – impact of PFDD across the drug development life cycle

Gain access to all reports from all of the FDA PFDD meetings – FasterCures PFDD Tracker

How does PFDD change clinical trial execution?

For years, we at IHS have been touting the value of getting patient insights early to inform building protocols with endpoints meaningful  to patients and families, not just researchers. Now, the FDA is asking that you do just that. 

Additional resources:

Inspired Health Strategies case study exemplifying the value of early patient/caregiver input

Search for a clinical trial relevant to you: https://www.clinicaltrials.gov

How does this change commercializing a product?

There is a greater expectation that you will follow what happens with your brand in the real world. Your clinical trials have been performed in sterile, vacuum-sealed environments. What happens when people take your drugs in their real lives? The adverse events you have shied away from collecting can now be seen as early warning signs you can use to inform your business decisions. We encourage you to collect all the data you possibly can to make the best decisions possible for both the patients and your business.

Additional resources:

A lesson from Invokana

What can I do if my drug is already in clinical trials? Going to the IRB to change things takes time, effort and money - we don't have any of that to waste.

You can easily collect parallel data via the myriad of patient-focused communities. You can engage with patient advocacy groups to develop market research instruments to gather behavioral data. Certainly, this data will not be coming from the clinical trial participants but you will get data to help inform business decisions as you prepare for launch. The real deal is, do not plan your launch in a vacuum. Build ongoing opportunities to learn from the patients, caregivers, and families who manage the disease your drug targets on a daily basis. They are the experts!

Additional resources:

Media Corner

Videos and podcasts featuring PFDD

 

How will this change how I do business?

It depends on the segment of the business that you are in, but it certainly means changes for the entire life sciences industry. Below links to examples illustrating how you do business can change. 

 

Additional Resources:

DIA change resulting from improved patient engagement

DIA Patient Engagement Tool Kit

What if my company culture does not consider PFDD a value?

That is expected as PFDD demands a change in thinking, that is the real innovation here. Time will tell, though. As the FDA requires NDAs to include this patient experience data, let’s see whose NDA gets rejected due to not including this data. We bet that will be a motivator. In the meantime, you can help your company along by staying informed, avail yourself of the resources on this site and others. This gradual drip drip of information will eventually impact every organization. Face the fact that the life sciences industry is incredibly slow to change. The real innovative companies will get on the bandwagon early.

Additional Resources:

Harvard Business Review white paper illustrating change in pharma business model

We ARE collecting quality of life data within our clinical trial, isn't that enough?

Well, it is a great start! Kudos to you and your team! Next, ask your self HOW you are collecting this data? Is it via a paper-based questionnaire that is administered at the clinical trial site? Can the questionnaire be updated with information you learn along the way? Can the data be collected in real time? Remember, what we are going for here is to understand better how your drug will impact patients lives in the real-world. 

Additional resources:

Clinical Trials Transformation Initiative (CTTI)

Tufts Center for Drug Development white paper re rates of study completion

Only 11.3% of all clinical trials are completed - can PFDD raise the odds of completion?

We think so, but only if you listen to the patients , caregivers and families in addition to academia and researchers. We have to learn to value this information we can gain from potential study participants. If you need blood collected or other tests during the clinical trial ask yourself how you can make that collection easier for patients – can it be done though a local center rather than a trip to the study site? The more we take down barriers to study participation the more chances we have at people actually completing a trial. Just because you included travel costs in your protocol does not make things easier on patients. Sure, that covers the costs but what about the inconvienence? How long does the participant have to stay out of work or get child care? How does travel impact the overall health of the participant? The more we think about these things and make amendments to protocols to reduce the barriers the better chance we have at greater study completion. Again, this demands a change in OUR thinking.

Additional resources:

Tufts Center for Drug Development white paper re clinical trial completion rates

Free Courses

In this section we provide links to the freee resources we find so you, too, can take advantage of these resources to learn more. Also, check out our Patient Engagement Resources tab on this site for additional free downloadable resources.

Premium Courses

We have experience developing customized training to help your organization to strait to think differently and adapt to PFDD. We can talk about your needs and customize training for you. Every organization is different and demands a different approach. Know that the first thing we will do is work with you to assess where your organization sits on the valuing of PFDD continuum.

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