Yes, You’ve Evaluated Every Risk – But Are You Properly Documenting Medical Device Benefits?
Risk. Risk. Risk. Some days it can feel as though our sole mission as medical device regulatory professionals is to raise red flags about what could go wrong. Without a doubt, safety is where the focus should be. Yet, when ISO 14971 instructs us to reduce risks “as far as possible,” it is easy to lose sight that there is a benefit-risk tradeoff. So while the focus of your job may be on reducing risk, the reason we create medical devices in the first place is to offer patient and user benefits. Do you know how to properly evaluate those benefits as part of your risk management and clinical evaluation process?
You’re not the only one to have struggled with this. In late 2016, FDA issued a 32-page guidance document entitled “Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions.” Most of it focuses on risk, but it includes a section discussing the factors that FDA recommends taking into account when performing an analysis of benefits. Here’s what you should consider as part of your analysis.
1 – Type of benefit(s) and medical necessity.
Examine the effect the device will have on patient treatment plans, survival prospects, and quality of life, including the ability to perform tasks associated with daily living. You need to look at how much your device can improve patient function, prevent loss of function, and provide relief from symptoms. Also, if you have a device that has a single intended use now but other intended uses are possible in the future, that’s a benefit you should note while being clear that the device is seeking clearance only for (or has approval for) specific uses. You should also examine whether your device provides benefits that are not currently available with other technologies, and how accessible those technologies may be.
2 – Magnitude of the benefit.
Once you have defined the type of benefit(s) the device offers, you need to assess how substantial that benefit will be. For instance, let’s say that you have an ENT device and associated test that distinguishes bacterial from viral sinusitis infections. What’s the “magnitude” of the benefit? Well, in this case the magnitude is substantial, because 80% of sinusitis patients are prescribed antibiotics and only 10% have a bacterial infection for which antibiotics would be effective. The test, therefore, has a substantial impact on the patients’ condition, because they would be properly diagnosed as having a viral infection early on and thus not treated with antibiotics that will provide no relief.
If you are submitting a new premarket approval (PMA), humanitarian device exemption (HDE) application, or de novo request, you will definitely want to study this FDA guidance issued in August 2019.
3 – Probability of benefit.
If your device has an 80% chance of effectively treating or diagnosing a condition, is that adequate? How about 50%? FDA – like any regulator – wants to know that you have assessed the likelihood that your device will be effective. Using the example above, FDA would want to know the probability of effectiveness in specific patient populations. For example, is your ENT device more effective at diagnosing viral sinusitis infections among children than older adults? Because many medical device manufacturers struggle with how to frame this probability issue in the context of their benefit-risk analysis, FDA encourages companies to contact them for guidance on what information may be relevant.
4 – Duration of the benefit.
Let’s think about another device: dialysis machines. There is little debate that these machines are effective at extending the lives of patients suffering from renal failure, but how should you think about the effectiveness of this treatment when you consider the duration of the benefit? The typical dialysis patient has to endure 4 hours of treatment 3 times per week. That means the duration of benefit from one treatment is roughly 48-72 hours. Is that acceptable? Or should you look at the duration of the benefit in aggregate? In this case, the alternative is not good, so although the duration of the benefit is short, the impact is enormous.
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5 – The patient perspective.
That brings us to the next part of your evaluation, which involves empathy. Set aside your pragmatic regulatory self and put yourself in someone else’s shoes. If you were the one facing renal failure, and this treatment could extend your life by 5-10 years, would your perspective on the benefits change? This immediately brings up a lot of questions, the key one being the quality of your life during those 5-10 extra years. That’s why you also need to think about this in the context of the alternatives for the patient. Imagine if you were 45 years old and given a choice between dialysis and a kidney transplant. (Note that most people never get off the waiting list or don’t get that option.) The “value” of a kidney transplant may far outweigh the risks. The point is that when evaluating the patient perspective, you cannot simply weigh the benefits of your treatment to the patient (living longer). You need to consider their treatment alternatives, the patient’s age, and even factors such as the financial cost of the options available to those who may not have insurance.
6 – Benefits to healthcare professionals or caregivers.
Patients may not be the only ones benefiting from your device, and you need to take this into account. Remember that sinusitis device we mentioned earlier? Imagine you are a physician and a Medicare patient comes to you with a sinus infection. As you have done so many times in the past, you prescribe antibiotics and send the patient home. Four days later the patient returns, unhappy and uncured because the infection was viral and antibiotics don’t work on viral infections. If that physician had access to your device/test in the office, the patient would have been properly diagnosed, received the correct treatment, would likely have been cured, and probably would not have returned. That’s good for the patient, of course, but it also allows the doctor to help more new patients, saves the doctor’s billing staff some time, and saves us (the taxpayers) money.
Benefit-Risk Analysis and ISO 14971
As you are well aware, ISO 14971 is what FDA follows with regard to risk management. Within the new 2019 revision of the standard, section 7.4 asks you to assess whether the medical benefits outweigh the residual risks. That analysis is linked to your clinical evaluation. ISO 14971:2019 does not change the overall risk management process, but it does finally define “benefit” – something ISO 14971:2007 and EN ISO 14971:2012 do not. Benefit is now defined as “Positive impact or desirable outcome of the use of a medical device on the health of an individual, or a positive impact on patient management or public health.” Notice that the definition of benefit extends beyond the impact on the patient.
Also an updated version of ISO/TR 24971:2020 has been released. It is the accompanying guidance document for ISO 14971. The guidance will actually contain many changes you will want to review and will include examples and more guidance on how to determine benefits when conducting a benefit-risk analysis.
Want to learn more about risk management?
The evaluation of benefit-risk is a complex topic and a critical part of your risk management process. If you would like to take a much deeper dive into this topic, consider our intensive medical device risk management training course – which has been updated for ISO 14971:2019. Our consulting team is also available to help you with risk management compliance issues.