This week, we cover:
- FDA’s new “one pivotal trial” policy
- NIH Director, Dr. Bhattacharya’s appointment to be acting Director of CDC
- An activist HHS Secretary is a new thing.
FDA’s New “One Pivotal Trial” Policy
For many years, FDA’s policy has been “two studies are needed for approval of a new drug, but we will listen to any reasonable argument that one is sufficient.” Commissioner Marty Makary and CBER Director Vinay Prasad are changing this to: “only one study is required unless we tell you a second is needed.”
The good part of this: FDA’s acknowledgement that biological and clinical knowledge has reached the point where confirmatory evidence can come from many sources. It need not be a second trial. By itself, that should have a positive impact on biopharma drug development and investment.
However, this is just saying aloud what is already the case. Over the last five years, about 60% of first-of-a-kind drugs were based on a single trial. This option has existed at least since the late 1990s, and FDA has issued guidance on the topic to sponsors, most recently in 2023.
So, changing the policy may have little practical significance as long as FDA is rigorous in insisting on second trials when needed. But it is also true that FDA could just maintain its current policies and become more open to drug development plans that don’t require a second trial.
The problem — and a serious one — is that the message behind the policy change is all wrong.
Going from “two studies with lots of exceptions” to “one study unless another is required” gives the appearance that patient safety and the efficacy of treatments is no longer FDA’s primary concern in drug reviews.
Shifting to one-trial is also inconsistent with recent FDA rejections that cite a need for additional evidence and with Dr. Prasad’s ongoing rhetoric about how many unsafe drugs are on the market.
Based on multiple press reports, the general reaction to the change has been mixed, leaning negative. Matthew Perrone at Associated Press (story here) reached former CDER director and acting Commissioner Janet Woodcock, who pointed out that the primary beneficiaries of the change would likely be drugs for more common diseases. She added that the impact will be hard to assess until FDA provides more guidance on this new policy.
Two Jobs For One Person: How Can They Do That?
It has been announced that NIH Director, Dr. Jay Bhattacharya will serve as Acting Director of CDC until a new director is appointed and confirmed (here). It is legitimate to wonder how he can manage two massive organizations simultaneously.
My own similar experience of this was when Dr. Andrew von Eschenbach was Director of the National Cancer Institute and for eight months was also acting Commissioner at FDA (2005-2006). He describes his routine–visiting both organizations each day–in this oral history (here).
His dual role was facilitated by the overlapping goals of NCI (in part, trials to support therapies) and FDA (to evaluate therapies). It also raised questions about whether there was a conflict of interest in his holding both jobs. The proximity of the two agencies (5 miles, less than a 20-minute drive) made things easier.
Dr. Bhattacharya will have a more difficult time because of differences in agency mission and the distance (650 miles) between their agency headquarters. Fortunately, we have far better ways to work together remotely than we did 20 years ago.
Why do this at all? In 2005, the FDA did not have a principal deputy whom the White House felt comfortable appointing as acting commissioner. The same could be said today about the CDC. The next best option is someone who has been confirmed by the Senate for a different position, which is what creates the not-infrequent situation of a senior person holding two jobs.
There is also a bigger issue: the appointment of Dr. Bhattacharya to be acting CDC director is clearly part of a larger strategy of postponing the nomination and Senate confirmation hearing of a new CDC director. It would probably be the Administration’s preference to wait until after the mid-term elections in November 2026 if it can find ways to accomplish that under the Federal Vacancies Reform Act (here).
An Activist HHS Secretary Is A New Thing
A reporter asked me how common it has been for an HHS Secretary to be deeply involved in driving policymaking at the agency level. Actually, I addressed that question a year ago, in one of my first columns (here). I wrote:
Because it is not a particularly good pulpit, past Secretaries of HHS have never been chosen for the specific purpose of advocacy leadership. As a rule, they are chosen to run the department. The role of chief spokesperson has been barely more than a secondary consideration. Dr. Louis Sullivan (1989-1993) was probably the last Secretary to legitimately see himself as “hired to be a public health leader.”
The column also examined the considerable management and public accountability experience of every HHS Secretary since 1985. In contrast to Secretary Kennedy’s limited management experience, that group includes four former governors, two leaders of large educational institutions, one head of OMB, one former HHS official who ran a sizable company before becoming Secretary, and one former Member of Congress who was a state Attorney General before becoming Secretary.
