In the mid-1980s the FDA was criticized for being slow in approving new drugs. In fact, a commission was formed as a result of the mounting criticism.
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation, recently gave BioPharma Dive a brief history of the FDA’s approval process while she was in Philadelphia attending Biotechnology Innovation’s annual convention.
Woodcock’s thirty years with the FDA gives her first-hand knowledge of biotech’s growth and the industry’s emergence as a powerhouse in drug development.
The interview covered such topics as fast-tracking approvals and new designs for clinical trials. Woodcock also spoke about her concerns regarding the FDA and the continued debate over accelerated approval.
The Ongoing Debate Over Accelerated Approval
The debate continues over how the FDA balances access to new medicine against requiring sufficient supporting evidence. But it appears that over the past twenty-five years there has been a reversal.
This is evident in a study conducted over several decades showing that during that period, most cancer drugs were approved without submitting data proving survival benefits.
The Price of Cancer Drugs
Woodcock acknowledged the criticism surrounding the price of new cancer drugs. As an example, she mentioned Lilly’s new drug, Lartruvo, with a monthly price tag of $17,000. In its short time on the market, Lartruvo has grossed over $500 million.
About Adaptive Designs
For many years, Woodcock has promoted using “adaptive designs” for clinical trials. The FDA has recently replaced its 2010 industry guide with a new guide that describes methods to design, conduct, and report results from clinical trials.
The new information offers guidance in using adaptive designs in clinical trials to demonstrate the safety and effectiveness of a biologic or drug.
Woodcock believes that they are now beyond pure theory and in the initial stages of “transformation.” In fact, several companies have spoken to the FDA regarding their own adaptive clinical trials.
An adaptive study known as GBM AGILE is slated to test glioblastoma (GMB) treatments. Thus far the industry that deals with this devastating brain cancer has had many trial failures.
Other adaptive studies are planned for Alzheimer’s and breast cancer. Patient groups have already accepted the idea as a means to enhance the results of clinical trials.
One major benefit is that the designs create an ongoing trial structure in an industry where experimental treatments have had high rates of failure.
But drug makers are hesitant about adoptive designs because it requires handing over a certain amount of control as well as creating competitive concerns. Woodcock believes that once companies see a few successful trials, they will be motivated to move forward.
About Continuous Production vs Batch Manufacturing
Pharmaceutical companies tend to move slowly when it comes to adopting new technologies to manufacture products. Industrial sectors are already using continuous production methods but drug makers have held back and are still using batch manufacturing.
Woodcock has been cautiously optimistic about the future of continuous manufacturing among pharmaceutical companies. The reason she is counting on its taking hold is that there are several advantages over batch production.
The advantages involve production volume, energy, quality, and cost. A great deal of time is saved by the elimination of start-up and shutdown steps.
Meanwhile, traditional pharmaceutical batch processing brings together components of a drug by a step-by-step method. The first batch must be completed before the next batch can begin the process. This may involve six or seven steps and multiple pieces of equipment.
About Interchangeable Biosimilars
Small-molecule drugs, such as aspirin or allergy medications, are obviously small and simple in structure. Biosimilars must be “highly similar” to the existing product with little difference in purity, safety or potency.
The FDA recently finalized a guide explaining how biosimilars may acquire interchangeable status. This means that they are allowed to be substituted for prescription medicine without the intervention of a prescriber.
Woodcock acknowledges that doctors in the U.S. have reservations about biosimilars in part due to literature that painted foreign generics as a corrupt, uncontrolled industry. The doctors are concerned that their patients may be switched to another drug without their knowledge. She agrees that there is a need for the FDA to address these concerns.
Looking Forward
Nonalcoholic steatohepatitis is a disease of the liver known as the “silent epidemic.” Woodcock mentioned this disease along with metabolic issues and hypertension. Her point was that there are diseases that cannot be treated by drugs alone.
The diseases she spoke of need to be treated largely through diet and lifestyle choices. This side of the FDA will be responsible for major awareness campaigns to address such challenges.
