The Rapid Pace of Change at FDA in 2018

January 9, 2018

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As 2017 began, there was change in the air for FDA if for no other reason than the passage and implementation of the 21st Century Cures Act (Cures) which succinctly put, stood for the principle of enhancing and speeding innovation. While this legislation put into place a structure for change, the addition of a new FDA Commissioner ushered in a new era and one that stands to change the cultural approach of the agency to the industries it regulates.

Prior to this appointment as Commissioner, Dr. Gottlieb held a number of positions, including some at the agency he would be heading. He is broadly experienced in healthcare – from being a practitioner to being a patient – and he did not step into the position of Commissioner without a good idea of the things he would like to accomplish and how he would like to do it.

The result has been initiatives set into motion on a broad scale and guided by his long-standing principle that regulation should take a risk-based approach – that you apply your resources to those areas that pose the greatest risk. That has translated into policies that are changing the very way FDA has done business in the past.

This past year, FDA announced new initiatives, putting structure to digital health innovations with the pre-certification program announced during the summer; a new policy with multiple guidance documents enunciating a regenerative medicine framework, as well as initiatives aimed at reforming food recalls, and addressing issues in drug compounding. And in keeping with the commitments made during the confirmation process, Dr. Gottlieb has taken steps to make opioid addiction treatments more available and to address drug costs by facilitating approval of generic drugs. In fact, the very first communication from FDA in 2018 was a statement from Dr. Gottlieb showcasing the work done to approve a record number of generics in 2017 and forecast an even greater for 2018.

If 2017 was the year that marked a foothold in changing the approach FDA takes to regulation, 2018 will be the year that the agency travels down the path it has set, specifically with regard to the further development of approaches to bring FDA more in line with a risk based approach and to facilitate innovation in doing so – or as proponents of this approach characterize it – modernizing the agency. To that end, several processes have been put into place that will play out in the coming year with the aim of enhancing innovation while preserving safety and efficacy – that has become a theme for the renovation of FDA by the new commissioner.

This past year FDA saw a number of “firsts” that signify not just the pace of change, but the many different directions in which change is happening with medical treatment:

  • the first pill combined with a sensor to monitor adherence;
  • the first CAR-T gene therapy approvals to treat forms of blood cancers bringing to reality the long sought promise of engaging the patient’s own immune system to battle cancer;
  • the first prescription app to be used in the treatment of opioid addiction;
  • the first treatment for any solid tumor with a specific genetic trait, regardless of its location

These “firsts” stand among others, many of which will happen in the coming years. To that end, many processes have been put into place that will help define the environment not only for new therapies, but for new types of therapies. For 2018, FDA has introduced several mechanisms for addressing the changing environment and to bring some outstanding questions into sharper focus. For example, FDA will hold the first public workshop for digital health and the pre-certification program later this month.

It is not just the types of treatments, but the way we consider their approval. A primary tenet of the Cures Act was to foster greater patient involvement in the development of new treatments – a process already underway with the first meetings of the newly formed Patient Engagement Advisory Committee, which will be looking at a range of issues related to patient input – from clinical trial design to incorporating patient experience into approval considerations.

FDA can do little directly about a major concern raised during Dr. Gottlieb’s confirmation hearings – pricing. But 2017 was a record setting year for the approval of generics and Dr. Gottlieb has stated that he expects 2018 to grow even further.

Finally, there will be a sharpened focus on further steps FDA can take in addressing the opioid epidemic, identified as a concern during his confirmation hearings and repeatedly positioned as a top priority by Dr. Gottlieb. Last year, he announced the formation of an Opioid Policy Steering Committee, which will have a direct impact on new policies with respect to the regulation of opioids.

In short, it remains a question for many whether or not FDA can streamline the regulatory process for approval and oversight of medical treatments without diminishing the agency’s commitment to safety and efficacy. One thing is for sure – the agency is well on its way to finding out.