Every now and then something new comes across my radar from the FDA that comes as a bit of a surprise.
This was the case with their recent report, “Value and Use of Patient Reported Outcomes (PROs) in Assessing Effects of Medical Devices.” As I read through it, it struck me that it has the potential to bring about some significant changes for medical device companies and how we go through the pre-approval process.
As with any new report or guidance that comes out, we tend to be concerned with how radical any changes might be and the significance of those changes for the medical device industry. What’s happening? Here’s my take on the new policy:
Focus on the patient
As I read this report, what became clear to me is that the FDA is very interested in evaluating and ensuring that products meet patient needs. This emphasizes now more than ever the importance of design controls and good documentation practices. Design controls are about safety, efficacy and how a product meets its indications for use. This report seems to be a culmination of that, to show how important that is to the FDA.
Another interesting thing to note is the provisions in the Medical Device Reform Act talking about “evidence-based medicine.” Some guidance came out a few years ago from which this new regulation seems to take the same spirit.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
A few highlights…
CDRH (Centre for Devices and Radiological Health), the umbrella organization at the FDA that governs medical devices talked about a vision that “patients have access to high-quality, safe and effective medical devices.” This is an important point, it has always been the case, but this new PRO program seems to put a heavier emphasis on that.
PRO is a patient’s assessment of their own health status or quality of life. What I really like about this is that it’s starting to change the perception of patients relying on third parties such as clinicians to advise and guide on what they should do. This marks a shift to put some responsibility on the patient. From a medical device company’s point of view, this is powerful because now patients will have the opportunity to have a voice, to speak up and make a case for why one technology or treatment is better than another.
We’ve seen this before in some parts of the industry. For example, I remember driving down a highway and seeing a huge billboard for an orthopedic company describing why their implant was the best as per input from their patients. This PRO push should make the patient’s voice heard more widely, maybe even more so than the industry’s voice.
Patient data to support regulatory reports
This is important because status quo for Class II devices, in particular, has been the entry of that 510(k) submission and demonstrating substantial equivalence with something else that is out there. With this new PRO concept, that may not be good enough anymore. Of note here though, is that it’s not a requirement today, however it may be a leading indicator of things to come from the FDA.
I would anticipate seeing that for some companies, the PRO information and data raises the bar. Not only will the company demonstrate substantial equivalence, but data to show that patients are taking charge of their health and that the product is preferred. The data will be used to help demonstrate that the product is high quality, safe and effective.
The flipside of this is that, given the expected behavior of the FDA by industry, it might increase the time taken to get through the regulatory process and increase costs to produce Class II devices. Why? They’ll have to conduct some sort of PRO study if this becomes a requirement and they’ll need additional funding to support the study activities. It’s a potential game-changer here, and I’m interested to see what happens with PRO on an ongoing basis.
In another section, the FDA states:
PRO measures have been increasingly used for regulatory purposes in CDRH over the last few years.
Specifically, I know the Division of Cardiovascular Devices has been using PROs to support product claims and regulatory submissions, as well as the orthopedic companies I mentioned earlier. The CDRH has a number of divisions other than those for cardiovascular and orthopedic devices, so many product segments have the potential to be impacted by this new PRO focus.
Another point, “CDRH observed greater than 500% increase in pre-market submissions with PROs.” Other parts of the world have been using PRO data for quite some time, so it’s interesting to note that we’re already seeing an increase here.
Impact on reimbursements?
There’s an interesting point here we can surmise about reimbursements. In the U.S., the way the payer networks operate for medical devices is that consumers don’t generally buy devices — it’s generally through CMS or the payer organization for healthcare and medical technologies. The way this works is that once products get cleared through the FDA, they fit under a category at CMS with a reimbursement code for that technology.
The reason this is important is that if an end user of a medical device doesn’t get reimbursed for using a particular product or technology, historically, that product probably won’t be successful in the marketplace. What’s intriguing about PRO is that it’s possible CMS will start to evaluate PRO results to determine which products will be reimbursed. If there is data reporting that PROs are not ideal, I would anticipate that CMS will intervene, which would be a challenge for companies.
Evidence for decision making and pre-market submissions
“The PRO program is about increasing the use and transparency of patient input as evidence in CDRH decision making.” The FDA is making this a strategic priority goal, so it’s quite an elevation from previous practice. CDRH is actively encouraging PRO data in pre-market submissions “where appropriate.” This is potentially revolutionary Although it may have been an expectation within orthopedics and cardiovascular, the encouragement of CDRH indicates that this could be much more widespread in other premarket submissions.
The pre-submission stage is where you might have questions for the FDA and is a vehicle to interact with them. They’re stating that the pre-submission stage is the recommended timeframe to consider including PRO activities. So here’s what we can take from that. If you’re at the pre-submission stage, it may be worth considering PRO data and figuring out how or if it will impact the FDA’s decision-making process.
In the fiscal year 2017, over 75% of approved pivotal, original new study IDEs submitted to the Office of Device evaluation included a PRO measure in the submission.
Referring to Table 2 in the report, it talks about different areas of focus (shown below). It’s very clear from this that there is a lot of interest in this problem overall from the FDA.
Medical Device User Fee Act
Another issue that caught my eye was “Medical Device User Fee Act for MDUFA.” Every few years Congress determines what the fees will be for different types of submissions. MDUFA IV takes place from fiscal years 2018 – 2022. They specifically state a desire to advance the science of patient input as part of this.
The PRO program can potentially have a negative impact on time to market due to the extra studies that may be involved, but the wording in the text indicates that CDRH is at least aware of this, stating a commitment to “least burdensome, predictable and transparent.” At the same time though, we still have priorities that tend to conflict, such as “fast path” vs. ensuring that the product is safe and effective.
Take a minute to look at parts of their conclusion:
PROs play an important role in assessing the patient experience with a medical device, and can provide valuable evidence for regulatory decisions, payers, and health system customers. PROs are commonly used in medical device regulatory submissions. CDRH is working collaboratively with our customers to increase appropriate use and increase the value of PROs as evidence in regulatory decisions and for other, non-FDA purposes.
For me, this really hammers home that as medical device companies, we need to have strong practices around our design controls and ensuring that devices meet their indications for use. This program is new to me, but it indicates that we really need to consider PRO as a potential requirement in future.
Photo: DrAfter123, Getty Images
