Making A Breakthrough Mistake With Medical Devices

REUTERS/Shannon Stapleton/Files

Joseph Gulfo Author, Innovation Breakdown
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The FDA is looking to roll out two policies “intended to expedite access to potentially life-saving devices meant for patients with life-threatening or serious conditions.” The first draft guidance document establishes pathways for the review and expedited approval “for certain medical devices that demonstrate the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions.” The second draft guidance establishes parameters in which post-approval studies can be used to offset some pre-approval study requirements of certain devices.

These are good ideas, but the draft guidance documents are redundant and unnecessary.

For quite some time, the FDA has had mechanisms that address both circumstances for drugs, biologics, and devices. For example, In 1996, I obtained expedited review for a drug called Valstar for bladder cancer. In 2006, I obtained expedited review for a medical device called MelaFind, and in 2011, I came to agreement with the FDA on appropriate post-approval studies for that product.

So, why the wasted effort in drafting and publishing superfluous guidance documents for medical devices?

Well, it is the same reason that the FDA did this with drugs and biologics in the Breakthrough Therapy Designation (BTD) policy, which was completely unnecessary since Fast Track, Accelerated Approval, and Priority Review provisions were already extant. And that reason is public relations.

Fanfare around programs for “important” products does several things. First, it mollifies the critics of the FDA because it advances the appearance of meaningful activity. Second, because of the fanfare and the perceived easier regulatory pathway, these sorts of policies direct the product development efforts to drugs, biologics, and devices meant for patients with life-threatening or serious conditions at the expense of others. Third, there are no criteria for what constitutes life-threatening or serious conditions, so the FDA gets to decide.

These three things create a huge problem – the preferential development of niche products (intended for small groups of patients with advanced diseases) away from products intended for the majority of patients. This is sure to now happen with devices.

Many people call this an intended consequence. I call it Machiavellian genius.

This “unintended consequence” has been welcomed by the FDA because rather than being judged by the public and Congress on all drugs and devices, they get to define which are the “important” products, the ones upon which they should be judged, in their opinion, while the others languish. And these are the “no brainer” approvals, those intended for small patient populations where the benefit to risk calculus is overwhelmingly positive because there are no other therapies. Electrons follow the path of least resistance, and so does the biopharmaceutical industry – if the FDA is going to make it easier to obtain approval for the no brainer drugs and devices, the public is going to get more of these at the expense of products that could offer significant benefit for many, many patients.

This is like a college student being able to tell the university the handful of courses they want counting towards their GPA. And, the truth is, the FDA’s review and approval times on these type of products, with some glaring exceptions (like Ofev and Esbriet for pulmonary fibrosis) is generally within the statute-defined time periods. So, this is tantamount to giving away ice in the winter time, and masterful PR. We have already seen investment dollars flowing away from the major diseases (like diabetes, chronic obstructive pulmonary disease, and cardiovascular disease) to niche (Orphan) diseases precisely due to these policies.


The students do not get to tell the university which courses count toward their GPA. And, Americans deserve more than PR from their government agencies.

I have a better solution – just follow the law. The statutes state that devices requiring substantial data (Pre-Market Approval applications) are to be reviewed in six months and devices akin to others that are already approved (510(k) applications) are to be reviewed within 3 months. If the FDA followed these laws, and were held accountable, acted honestly and consistently in the reviews of products, patients would be well-served, and no one would be complaining. The law already provides for post-approval studies and Priority Review, as well, for products that are intended to treat or diagnose a life-threatening or irreversibly debilitating disease or condition, and meets at least one of the following criteria – (1) constitute breakthroughs, (2) there is no alternative treatment or means of diagnosis, (3) the product offers a significant improvement over others, and the (4) availability of the product is in the best interest of patients.

Why then has the FDA’s Center for Devices and Radiological Health (CDRH) for several years been putting the finishing touches on two conceptually related guidance documents?

Because, it is easier to do PR – to come up with programs that will do nothing, and actually make the situation worse – than attack the root causes of the problems, which are an institutional bias against innovation, an assumption that current medical practice is safer than potential new products, and a complete lack of leadership.

This is a mistake, just as BTD was a mistake with drugs and biologics, and it needs to be stopped.