Gottlieb is Stepping Down From the FDA’s Most Forward-Looking Chapter

We’re about to lose the most tech-savvy FDA chief in recent memory. FDA Commissioner Scott Gottlieb, who announced his resignation on March 5, has left quite a mark in his two-year stint at the agency. Under his forward-looking tutelage, the FDA recently proposed several frameworks to streamline healthcare’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, healthcare 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 Manufacturers of these innovations often must take a different tack to reach the market, a path that has laid bare an outdated product approval process that’s in dire need of being revamped. Under Gottlieb’s direction, today’s FDA seemed to be up to the task: The agency has begun to match the innovations crossing its desk with some innovations of its own.  As Gottlieb prepares to serve his final few weeks in office, let’s look at some of the ways that he has transformed the agency’s tech-related approach, and how his legacy will have long-lasting effects on pharma companies and how they do business. 

• Flip the approval model for gene therapies. The evidence needed to approve a gene therapy differs from what’s needed to greenlight 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 easily can 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 that he expects the FDA to approve 10 to 20 gene therapies per year by 2025.) Are manufacturers prepared to over-focus their energy to help the delivery system with logistics versus promotion?

• Make mobile healthcare app approvals scalable. The FDA is radically changing its approach 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 the doctor typically remembers 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 on its process for regulating standalone digital therapeutics that aren’t drug companions. After all, approving digital therapeutics doesn’t require the same level of scrutiny since there’s no molecule entering the patient’s body. As part of the Pre-Cert. pilot program, the agency selected nine companies, including Pear Therapeutics, 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 that are prescribed as product companions should be treated as part of the drug label, not as a separate medical device. And this approach won’t just apply to adherence-helping apps, but also to apps that enable patients to self-identify (and self-diagnose, to some extent), so that 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 the products into the hands of patients who can benefit more quickly. Does this mean that there will be a change in the way that manufacturers package these apps alongside the drug treatments? How can pharma be prepared so that no delays are created on their end?
  
  When services are part of the product, there’s also an opportunity for pharma to differentiate “around the pill.” Moreover, promoting an enhanced label 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 a hand, rather than add to its list of industry challenges.

• Clear the path for AI-based healthcare solutions. 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 healthcare. Moreover, the FDA is working with experts in the field, like its partnership with Harvard University, 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?
  
  But pharma 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 like developing an evidence-gathering strategy and exploring partnerships with data-generating companies to future-proof against AI from the outside in.

  Between Gottlieb, HHS Secretary Alex Azar and CMS Administrator Seema Verma, the U.S. Department of Health and Human Services 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 that they make or the actions they take. It’s interesting to take stock of the recent changes that they’ve each 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 is Managing Associate, ZS Associates.