How should the pharma industry prepare for FDA plans to even further expedite the regulatory approval process – Interview with Wayne Pines

The U.S. Food and Drug Administration (FDA) is setting a remarkable record for new drug approvals in 2018. As of November 1, 2018, 47 new drugs were approved by the FDA exceeding the previous record in 2017 of 46 new drugs. 

The number of new drugs being approved annually continues to approach 50, with a third or more being for rare diseases and about a quarter for various cancers.

Over the last 10 years, FDA averaged 31 novel drug approvals per year. Thus, the number of new drug approvals the last two years is impressive.

And even while these remarkable achievements are taking place, FDA (specifically CDER, the Center for Drug Evaluation and Research) is seeking to implement new drug development and application review processes that would expedite the regulatory oversight system still further.

CDER’s Annual Novel Drug Approvals: 2008 - 2017

Stig-and-Wayne-Post-Drug-Approval-Chart

Source: Center for Drug Evaluation and Research: “Advancing Health Through Innovation. 2017 New Drug Therapy Approvals.”

I asked my colleague Wayne Pines, President of health care at APCO, and former associate commissioner for public affairs at FDA, what has been the driving force behind FDA’s remarkable achievement:

“There are a number factors. First is the amazing progress that medical science has made in understanding biology and disease better, and thus in being able to develop novel therapeutic approaches. This is true across a wide range of diseases. It goes without saying that for FDA to approve a new drug, the new drug needs to be discovered, developed and tested properly.”

From the FDA standpoint, Wayne believes the agency’s review process is more efficient and focused than ever before, with clear responsibilities and deadlines. The managers of CDER have made meeting deadlines a priority. 

“Furthermore, there have been changes in how we test new drugs. We have more natural histories for certain diseases, which makes it easier to understand what to test and how to do it. There are registries on patients with certain diseases, especially rare diseases, so that helps expedite recruitment into clinical trials. We are learning to use more adaptive trials, where safety and efficacy are measured more frequently so that changes in the protocol can be made. Finally, the FDA staff proactively seeks to help drug sponsors, so that fewer false pathways are being followed.”

Wayne points to the productive collaborative spirit in the drug develop community, which helps bring the process together and facilitate new drug development.       

More changes to new drug approval process coming

According to Wayne, there will be further changes in FDA’s new drug approval process, even as more new drugs are being approved. 

In a recent appearance before the Alliance for a Stronger FDA, Dr. Janet Woodcock, the CDER director, said her intention is to start new review practices in the Office of New Drugs (OND) by the end of 2018. By the middle of 2019 she hopes that an entirely new organizational structure in OND will be in place. In addition, she plans to increase the number of review divisions from five to nine. There will soon be a total of 30 review divisions.

The goal is to facilitate reviews by enabling the appropriate experts within CDER to review data in a more efficient and collaborative manner.

In addition, Dr. Woodcock hopes to select a new OND director to implement all the changes. She has been serving as OND director, as well as CDER director, for more than a year, since the departure of Dr. John Jenkins from the position, which is the epicenter of the drug review and approval process. Whoever is selected as the next OND director would be a candidate to succeed Dr. Woodcock as CDER director, if and when she decides to step down (nothing is believed to be imminent in this regard).

Further, Dr. Woodcock continues to seek to hire more professionals in CDER. (For years, CDER has had hundreds of openings, and thus has had to contract out a number of functions.) And she also told the Alliance that she hopes to put into place a new IT structure in CDER to manage information and decisions better, especially since more information about new drugs is being developed and submitted such as real-world evidence. 

How should pharmaceutical companies adjust to the changes?

How do companies that develop or market new drugs need to adjust to accommodate the changes that are coming at CDER?

Wayne is offering a few thoughts: 

  • With change, especially to large-scale bureaucratic processes, inevitably comes periods of adjustment and in some cases chaos. Companies will need to be patient as CDER institutes changes that in the long term hopefully will expedite the review process, but in the short term might lengthen it.
  • With an increase in the number of review divisions, companies will need to learn to navigate the system under its new structure. Relationships that formerly existed between division staff and company regulatory affairs personnel may no longer be relevant, as new FDA staff moves into their new positions. Understanding the new organizational chart will be a priority for companies with drugs in multiple therapeutic categories.    
  • Companies should prepare the plans for their applications to accommodate a rolling process, where CDER reviewers seek and are willing to accept data as it becomes available, rather than waiting for all the data to be compiled into a single submission. The rolling application, whereby companies submit data periodically rather than all at once, is seen as a better way for FDA to conduct its reviews. 
  • It is well recognized that different FDA medical reviewers bring their own criteria to the evaluation of clinical data and risk tolerance. Companies may be faced with new medical reviewers for their products and will need to learn whether the evaluation standards have changed. 

All these issues must be addressed even as companies face other mega-challenges such as the need to justify the costs of their drugs more credibly and to incorporate the patient voice and patient data into the drug development and review process.

“We all can have confidence in the changes that are taking place at CDER. We can have confidence that they will achieve their objective,” says Wayne. “Since Dr. Woodcock is in the driver’s seat, the reforms have been well thought out and will be successful. That’s been her track record with every initiative she has launched in her three decades at FDA.

But successful reform also will require adjustments by the sponsor companies, as well as patience and understanding on their part. “As patients, all of us, it’s important that the modernization of the drug development and approval move forward successfully,” says Wayne.

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Stig Albinus

Stig Albinus is a senior director in APCO Worldwide’s global health care practice. Read More