Speeding Innovative Medicines to Market: Any Delay Matters to Patients

FDA Commissioner Makary has embraced the cause of faster review times for drugs and biologics, an area that holds great promise for bringing innovative drugs to patients more quickly.¹

While laudable and likely to succeed, faster drug review times are just one way to address the broader and more important question: “What can the FDA do to help innovative medicines reach patients faster?”  

This column looks at ways to lower regulatory risks, improve the agency’s interactions with industry and patients, and other ways to address this question.

The importance of this inquiry comes from the simple fact that any delay matters to patients. 

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Drug Development Priority: Lowering Regulatory Risk to Encourage Investment

Drug development is expensive, lengthy, and has a relatively low probability of success compared to many other investments. Not only must the therapy work, but the company must also anticipate and overcome extraordinary and unpredictable market and regulatory risks that must be projected five to ten (and possibly more) years into the future. 

Likely reflecting a perception that such investments have grown even riskier, biopharma funding via IPO’s was down by 20% last year (here). There is a temptation to blame this on the capital markets and economic conditions, as if FDA plays no role. 

However,  leadership instability and inconsistent decisionmaking at FDA are very much a part of investor calculations. One need only look at the impact of FDA and CDC policies on vaccine investment in 2025 (here).    

Leadership instability is a familiar story. A by-product is inconsistent decisionmaking or at least the perception of it. As I observed in a column this past December, “no one in authority has the institutional memory to recall how the agency operated on a good day.”(here). 

How can sponsors, investors, and patients (!) be sure of continuity of the decisions and guidance from prior years? That anxiety has been fed by a Center Director who has seemed determined to overturn decisions and policies initiated by his predecessor. 

One good idea for lessening regulatory risk and encouraging investment:  

FDA needs to assure patients and the biopharma community that senior leadership will be stabilized and continuity in decisionmaking will once again be the expected norm. 

An immediate opportunity to demonstrate this commitment is the appointment of new permanent directors to lead CDER and CBER. Those individuals need to be mainstream and public-service-oriented enough that 1/ they want to stay beyond the current administration, and 2/ a new commissioner will not feel the need to automatically replace them (as would have been the case for Dr. Prasad). 

Drug Development Priority: Reinforce and Expand the Partnership Among Industry, FDA, and Patients 

The greatest potential to speed innovative drugs to patients is in strengthening the cooperative relationship that FDA has developed with patients and industry. When FDA and a sponsor agree on a development plan — number and types of trials, endpoints, etc. — things speed up. Months and sometimes years can be gained (or lost) in “time to market.” 

Unfortunately, that productive and healthy cooperative relationship was damaged when the new HHS Secretary denounced FDA as the sock puppet of industry (here) and the new commissioner talked about corrupt relationships and being surrounded by “swamp creatures” (here). Then nearly 20% of agency staff were let go, decreasing the agency’s capacity to work closely with sponsors. 

Commissioner Makary has, by his actions, appeared to have moved on. He now praises FDA staff and seems to understand that shortening the path for innovative technologies inherently requires partnership. Oppositional relationships are sometimes necessary, but only after efforts to work together fail. 

One good idea to reinforce and expand the partnership among industry, FDA, and patients. 

The agency, starting with the Commissioner, needs to flip the narrative by telling the public, patients, and industry that FDA’s open communications policy promotes innovation, benefits patients, and poses no additional safety risks. 

Since this policy largely works, albeit with little visibility, the point of telling the story is to encourage sponsors, patients, and FDA staff to continue to invest in partnerships. That will contribute to more innovative therapies and faster transit through the agency and to the patients who need them. 

Drug Development Priority: More Context-Sensitive Risk-Benefit Calculations

On several occasions (most recently here), I have written about how we are “failing patients with relentlessly progressive chronic neurological, neuromuscular, and genetic diseases. Their plight necessitates a significantly different risk-benefit calculation than that of patients with other types of diseases.” 

My advocacy for this population has always centered on situational analysis and compassion. As part of this article, it is important to add that our failure to use more context-sensitive risk-benefit calculations has slowed the development and review of innovative therapies for these patients. My column also highlights the role of regulatory science in accelerating the delivery of innovative therapies to patients. 

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I plan to write one or more future columns exploring additional ideas to accelerate the delivery of innovative therapies to patients. Please share your ideas with me at sgrossman@fdamatters.com.