We’re about to lose the most tech-savvy commissioner of the Food and Drug Administration in recent memory. At its core, the FDA is about keeping people safe. But Scott Gottlieb helped transform the agency’s top role from “custodian of safety” to “advocate of innovation” by pushing for progress and refusing to let outdated regulations stand in his way. I hope his replacement doesn’t backpedal to the agency’s more conservative past and undo the progress that he’s made.
Gottlieb, who announced his resignation on Tuesday, has left quite a mark in his nearly two-year stint at the agency. Under his forward-looking direction, the FDA has proposed several frameworks to streamline health care’s adoption of new technologies and has even received industry nods for venturing beyond what’s traditionally thought of as the agency’s purview.
From gene therapies to mobile apps and wearable medical devices, health care has a different look than it did even a decade ago, and Gottlieb seemed to recognize the need to pivot in response to these external pressures.
Manufacturers of health tech innovations and complex therapies must often take a different tack to reach the market, a journey that has laid bare an outdated product approval process that’s in dire need of being revamped. Under Gottlieb’s direction, the FDA now seems to be up to the task, and has even begun to match the innovations crossing its desk with some innovations of its own.
Here are a few of the ways that Gottlieb has transformed the agency’s tech-related approach; how his legacy will have long-lasting effects on pharma and medical device companies — and to a degree the broader health care landscape — and how they do business; as well as some tough questions that stakeholders will need to answer.
Flip the approval model for gene therapies. The evidence needed to approve a gene therapy differs from what’s needed to green light a small-molecule medication. For conventional product approvals, clinical results are a far bigger consideration than the product’s properties. The approval effort for gene therapies, however, is flipped because clinical efficacy can easily be proven but the manufacturing piece is far more complex. It will become even more important to roll out a new approach as the FDA prepares for a spike in gene therapy applications and approvals. (Gottlieb said recently he expects the FDA to approve 10 to 20 gene therapies per year by 2025.) Manufacturers will need to shift their focus from product promotion to customizing the cell and gene therapy manufacturing, distribution, and delivery experience to meet the needs of each patient.
Make approvals for mobile health care apps scalable. The FDA is radically changing its approach to health care apps by scrutinizing the producer rather than the product. For example, the agency’s Software Pre-Certification Pilot Program proposes a modern way to regulate digital health products by giving primacy to the manufacturer’s identity over the product. This is a scenario that pharmaceutical brands have never had to deal with, since prescribers typically remember the product, not the company. How will manufacturers cope with this behavior shift, one that’s common in other industries? Distinguishing between company branding and product branding tactics certainly isn’t a strong suit for pharma, nor is developing software. Will pharma companies need to partner with software companies that excel in these areas?
The FDA is working to quicken the pace for its process to regulate standalone digital therapies that aren’t drug companions. After all, approving digital therapies doesn’t require the same level of scrutiny as drugs since there’s no molecule entering the user’s body. As part of the pre-certification pilot program, the agency selected nine companies, including Apple, Pear Therapeutics, and Roche, to help develop a risk-based framework and to streamline the review process for pre-certified companies.
Fast-track apps that accompany a drug treatment. According to Gottlieb, apps prescribed as product companions to drugs should be treated as part of the drug label, not as separate medical devices. This approach shouldn’t apply just to apps that aid adherence, but also to those that enable patients to self-identify (and self-diagnose, to some extent) so they can select the right over-the-counter products for their ailments. The idea is to avoid lengthy premarket review submissions for qualifying software, thereby getting products more quickly into the hands of patients who can benefit from them. Does this mean the FDA will change the way manufacturers package these apps alongside drug treatments? How can pharma companies be prepared so no delays are created on their end?
When services are bundled with the product, there’s also an opportunity for companies to differentiate “around the pill” by emphasizing increased effectiveness and better clinical outcomes. Moreover, promoting an enhanced label (one that includes the pill and the wraparound services as a package) could be a good defensive strategy against biosimilars and other competitors. In other words, the agency’s aim to fold product companion apps into the drug label could actually lend pharma companies a hand rather than add to their list of industry challenges.
Clear the path for health care solutions based on artificial intelligence. A big advocate of artificial intelligence, Gottlieb has in the last year promised to develop a new regulatory framework to promote innovation and support the use of AI-based technologies in health care. The FDA is now working with experts in the field, like its partnership with Harvard, to prepare for an anticipated onslaught of AI-based submissions in the coming years. What’s stopping pharma from pursuing similar collaborations with research and academic organizations, and making deeper investments in AI? How can pharma change its mindset to speed the adoption of AI?
The pharmaceutical industry needs to think beyond its plans to adopt AI from the inside out. If the FDA’s frameworks help AI gain a foothold in treatment decisions, pharma’s commercial model could begin to fail. To get ahead of that eventuality, pharma must pursue other avenues, such as developing evidence-gathering strategies and exploring partnerships with data-generating companies to future-proof against AI from the outside in.
Thanks to Gottlieb, Health and Human Services Secretary Alex Azar, and Centers for Medicare and Medicaid Services Administrator Seema Verma, HHS has ushered in a progressive and agile way of thinking that matches today’s new conditions — even though I may not agree with all of the claims they make or the actions they take. It’s interesting to take stock of the recent changes each has spearheaded and to look ahead at how the FDA, in particular, will prepare for the next wave of innovation, and whether its new chief will continue what Gottlieb started.
Pratap Khedkar, Ph.D., is managing principal of ZS Associates, an international professional services firm, and leads its global pharmaceuticals practice.