FDA Protects Patients While Promoting Innovation

BIOtech Now
Daniel Seaton

Today, 83 percent of children with cancer survive, compared to just 58 percent in the mid-1970s. Hepatitis C, a once-incurable disease, now has a cure rate of more than 90 percent, thanks to new treatments. And HIV/AIDS — once a death sentence – is now a manageable, chronic condition. This is the power of biopharmaceutical innovation, and it’s been truly remarkable to witness.

This progress would be impossible without the help and leadership of the Food and Drug Administration (FDA) – the hard-working public servants who review, approve, and are ultimately responsible for breakthrough medicines coming to the market in a safe and timely manner. Yet, a recent story by ProPublica takes a cheap shot at the Agency’s personnel, alleging that they are “fast-tracking expensive drugs” to support their budget for scientific reviews – a misleading claim that could not be further from the truth.

A more balanced approach would have highlighted the numerous advances in medicine that have been facilitated by FDA’s work and have resulted in lives saved and quality of life improved for millions of patients, rather than presenting negatives as the norm. A few months saved during FDA review can quite literally mean the difference between life and death for many – though the rigorous evaluation process is still required.  Further, the story fails to note that prescription drug user fees also support significant and important post-market safety activities of FDA, including contributing substantially to the use of the Sentinel database.  This database includes real-world data from tens of millions of patients and provides crucial insights into how those drugs are working once they reach the larger population, as well as safety issues that could not be detected in clinical trials that may need further exploration.

Next, the success of the Prescription Drug User Fee Act (PDUFA) program is hard to understate. As Dr. Janet Woodcock testified before Congress last year, “Before PDUFA’s enactment in 1992, Americans’ access to innovative, new medicines lagged behind other countries. FDA’s premarket review process was understaffed, unpredictable, and slow. The Agency lacked sufficient staff to perform timely reviews or develop procedures and standards to assure a more rigorous, consistent, and predictable process.”

PDUFA has provided FDA the ability to hire more highly qualified scientific and medical staff, to be sure.  However, the increase in staff numbers is only one reason that FDA is able to efficiently and effectively evaluate and make available to patients innovative medicines that are safe and beneficial.

For example, developers of new medicines are able, because of PDUFA, to meet earlier with the Agency, to discuss potential roadblocks, such as possible safety concerns, how best to measure whether the proposed new drug is effective, and how to design clinical trials that have the best chance of answering the key question of whether the potential benefits of a proposed drug outweigh its potential risks.  These discussions help lead to more efficient development programs that are most likely to provide meaningful and valid data.  This has led to a significantly greater chance that an FDA decision about an application can be made in the first review cycle – saving FDA time, company time, and patient lives.  However, neither PDUFA funds, nor meetings with FDA, nor the desire of patients, guarantees success or FDA approval. Every drug approved must meet the FDA gold standards for safety and efficacy. Drug development is risky business – an astonishingly small percentage of investigational drugs make it to the finish line.  In fact, the overall likelihood of approval for an investigational new drug starting Phase I trials remains about 10 percent overall, and only 5 percent for oncology drugs, even with PDUFA’s successes.

PDUFA funds also provide FDA with the ability to meet with the public, including academic experts, physicians and other healthcare providers, consumers, and patients, to discuss creative approaches to developing new drugs.  For example, meetings with the public are occurring to discuss the appropriate use of surrogate endpoints, the evaluation and validation of biomarkers, the use of real-world evidence, and the development of computer models and use of artificial intelligence to identify the most promising approaches to study an investigational medicine’s potential safety and effectiveness.  Such public meetings generally have not been within FDA’s budget capability, so it is PDUFA funds that are allowing these robust and important public discussions to occur.  This influx of funding has allowed the Agency to meet with patients and their advocates and caregivers, and to listen to patients’ first-hand views of their disease.  To hear what patients need, to hear what patients expect, and to determine how best to address this need are crucial aspects of the FDA’s mission.

The ProPublica piece also takes an unfair swipe at expedited approval pathways, the goal of which is to help deliver safe and effective therapies to patients suffering from serious diseases. For instance, the Accelerated Approval program allows for review of a drug based on non-traditional endpoints if it is aimed at treating a serious condition and would fill an unmet medical need. It is a designation that is only judiciously granted.  An approval under this designation can be based on a surrogate or intermediary clinical endpoint, but also requires confirmatory studies to prove the product’s clinical benefit.

Other expedited programs, such as Breakthrough Therapy Designation, simply confer additional Agency resources to an application, to help the most efficient development of products for significant unmet needs and where evidence indicates the potential to provide substantial improvement over current care, including for diseases for which there is no therapy and patients for whom there is little or no hope.

Like biotechnology companies, FDA must stand at the cutting edge of science to meet the needs of patients.  It seems as though nearly every month – or even more quickly – we see new technologies and true breakthroughs coming through the pipeline.  And these high-quality and remarkable technologies are changing the face of medicine. Transformative new technologies such as CAR-T cell therapies for blood cancers, gene therapies for genetic diseases, and next generation immuno-oncology drugs represent real breakthroughs for patients that will change the way we treat disease forever. The developers of such transformative technologies provide strong evidence of safety and efficacy.

We all owe patients a careful and efficient FDA review that will ensure these breakthroughs reach them as quickly as possible.


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