ICYMI: Director of FDA’s Center for Drug Evaluation Discusses Accelerated Approval, Benefits of Master Protocols

According to a recent publication from BioPharma Dive, Dr. Janet Woodcock sat for an interview at the Biotechnology Innovation Organization’s annual conference in Philadelphia earlier this month. Woodcock, the Director of the Food and Drug Administration’s Center for Drug Evaluation and Research, responded to a number of questions that spoke to the changing culture of clinical trials in the United States.

Accelerated Approval – A Persisting Sticking Point

The Food and Drug Administration’s Accelerated Approval Program has been a point of controversy since its introduction in 1992. The Accelerated Approval and related programs (like Parallel Track) are designed to give patients with potentially life-threatening conditions access to drugs still being researched.

In the 1980s, experimental HIV/AIDS and cancer drugs were the focus of significant attention and outcry. The potential harm of these drugs was certainly less serious than the risk posed by the diseases they were being designed to treat.

Accelerated Approval is not meant to create perfect outcomes, argued Dr. Woodcock in defense of the program. “It wouldn’t be accelerated if it has the same results as regular approval.”

The process of getting a drug to market involves a certain trade-off. Lengthier and larger clinical studies can ensure safer drugs once they reach market but the process can take years; years which some rare disease patients don’t have if they can’t get treated.

The alternative, something like the Accelerated Approval program, reduces the amount of empirical data usually required to bring a drug to market. The result is a drug that’s faster to the market, but potentially not as safe or effective as early data suggests it to be.

One such drug, Lartruvo, was the subject of a large plenary conference at the American Society of Clinical Oncology. The soft tissue sarcoma drug received accelerated approval in 2016 — but after several years on the market, it was determined that Lartruvo did not lengthen patients’ lives.

Even more troubling was Lartruvo’s price tag — around $17,000 a month, or some $200,000 annually. By the time Lartruvo was proven to be ineffective and withdrawn from the market, it had already netted manufacturer Eli Lilly and Company $500 million. The skyrocketing cost of Accelerated Approval drugs is one of the main sources of the policy’s continued controversy.

Woodcock argues that ultimately, the Accelerated Approval program is about risk-reward. The guiding wisdom is that failing to approve an effective therapy has a higher cost than approving an ineffective one. Simply put, even if most Accelerated Approvals are ineffective, the few that do ultimately work as intended could save hundreds if not thousands of lives.

Master Protocols and Continuous Manufacturing

In the over three decades of Dr. Woodcock’s career at the Food and Drug Administration, she identified what she saw as inefficiency in the clinical testing and manufacturing processes. In recent years, Dr. Woodcock has advocated for the implementation of “Master Protocols” — an alternative clinical testing model to the one in use now.

Currently, most clinical trials are conducted in sequence and independent of one another, testing different treatments in different study groups. The Master Protocol design would instead propose the implementation of larger trials to simultaneously evaluate the effectiveness of multiple drugs at treating multiple disease populations. Simply put, Master Protocol trials are larger and more complex to organize, but could result in more efficient drug evaluations.

The likelihood of drug companies agreeing to Master Protocol trials is difficult to speculate on — some think it’s unlikely that drug companies would surrender control of the progression of their trials to that of a government standard. However, Woodcock is optimistic. “It is a cultural transformation, but I believe it will occur,” she said at the Philadelphia conference. “I can guarantee you if there is success in a trial like this, [drug makers] will flock to it.”

In a similar vein, Woodcock advocated the implementation of continuous manufacturing by drug producers. Almost all drug manufacturers use batch production — making fixed quantities of a given drug at regular intervals. Limitations in technology have mostly forced pharmaceutical companies to rely on batch production, but Woodcock believes recent technological advances and further research will make “continuous manufacturing” a reality sometime soon.

Such a change in the fundamentals of drug manufacturing could lead to improved access to vitally important drugs.


Years of work and billions of dollars in research go towards the development of new drugs. Why might it be important to occasionally re-evaluate the system by which we develop them? Share your thoughts with Patient Worthy!

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