The Food and Drug Administration may not be the first government agency you think about when wondering how the Trump administration will affect your business and daily life, but it shouldn’t be too far down the list. The organization is responsible for the regulation of everything from your pet’s heartworm medicine to your father’s pacemaker — and all the food, drugs and vaccines in between. The FDA regulates over a trillion dollars’ worth of products — about a quarter of all consumer spending — which has real-life impact on every American multiple times a day.
The U.S. has one of the best safety records in the world when it comes to food and drugs, largely because of the important regulatory oversight the FDA provides.
So how can this vitally important agency be re-engineered and revitalized so that the remarkable engine of American innovation and creativity can be unleashed to provide better, safer, more economical products more quickly for our citizens and for patients around the world?
This will be a huge challenge and, in order to meet it, the next FDA commissioner cannot and should not be a mere figurehead. Of course, the person selected must be competent and able to understand not only the reasons behind policies, but the unintended consequences of those policies. More importantly, they must have demonstrated extraordinary managerial acumen and conviction of purpose that does not bow easily to the pressures of political controversy or media-fueled hysteria.
The agency has such a broad remit that no one person should be expected to have all of the expertise in all of the necessary disciplines to be able to go it alone. In this highly visible agency, the commissioner must be able to select and effectively manage superior individuals who can provide guidance and trusted counsel for all the various disciplines involved in ensuring the safety of our food and drug supply. These include not just medicine and science, but engineering, business, finance and legal aspects of regulatory policymaking and enforcement.
The inarguable highest priority for the FDA is to make decisions based on scientifically rigorous data. With that in mind, too often the agency loses sight of that and important new products or devices are delayed due to political grandstanding, and regulations that may have been well-intentioned morph into onerous, unwieldy and expensive rabbit holes. My hope is that the next FDA commissioner is in the mold of most of President Trump’s cabinet nominees: expertise based on broad and complex real-world experience rather than academic, theoretical or ideological altruism. Given the generous compensation and relative privacy that industry provides compared to government bureaucracy, any appointee who chooses to accept this position must also be recognized for the personal sacrifice required to enter into this important public service.
The FDA is just one of 11 operating divisions and multiple agencies supporting public health interests as part of the Department of Health & Human Services. It has nearly 15,000 employees and close to a $5 billion budget, and its commissioner is one of nearly 4,000 political appointees that President Trump and his cabinet appointees must select. The commissioner has at times been seen as more of a figurehead or public advocate of an administration’s policies rather than the administrator and managing director of one of our country’s most powerful, impactful and important regulatory authorities. That has had important consequences for both the pharmaceutical and biotech industries as well as consumers who are waiting for life-changing therapies. And, given the rapidly changing scientific, technology and healthcare reform environments, it is an agency in desperate need of a certain infamous reset button.
For example, a long-standing benchmark regulation for drug approval has been that any new compound must be proven to be both safe and effective. As scrutiny on drug costs increased and insurers attempted to develop tiered formularies for drugs they would cover and at what cost to the patient, that morphed to mean that a new drug must have a safety and efficacy profile that is at least as good as other drugs of the same class that are currently available. This reduced the attractiveness for pharmaceutical companies to spend time and money developing and promoting “me too” drugs without significant differences from those already available, which was probably a good thing because that research and development money can now be spent on truly new therapies or formulations.
Unfortunately, this regulation has experienced additional scope creep, and the agency has expanded its own authority. Now a new drug must often prove itself to be more effective than other drugs currently on the market.
“Well, that makes a lot of sense,” you might say, and at first blush, it does. However, proving this superiority is much more complex and expensive than the original conscripts of “safe and effective.” Particularly in the realm of new medications that are highly targeted therapies, proving superior effectiveness in a drug that has a marginal difference with a competitor requires huge patient numbers in a clinical trial or multiple trials to give the statistical significance necessary to support the claim that the agency requires.
Another example of the need for restructuring our regulatory environment can be seen as researchers now have increased access to and understanding of powerful analytical tools and real-world, real-time validated patient data that should help to streamline both the cost and time to market for drug development. The ability to strategically target appropriate potential trial subjects based on that analysis — which can dramatically reduce trial length by shortening the time and expense to recruit patients — as well as the number of patients needed to provide statistically significant data, can and will be a game changer in the industry.
Unfortunately, the regulatory requirements are lagging significantly behind available technologies for streamlining research and, instead of increasing the effectiveness of the billions of dollars spent on research each year by the industry, the FDA has increased the length and number of trials per use case as well as the number of procedures per patient necessary to bring a drug to market.
So, in an era of exploding new possibilities for remarkable new therapies and the understanding of how data can be captured and understood more quickly to provide actionable insights earlier in the process, drug approvals have taken much longer and have occurred with greatly reduced frequency over the past 15 years or so. Adding insult to injury is that only about one in five drugs that a pharmaceutical or biotech company chooses to invest in and enter into the multimillion-dollar, multiyear process of regulatory approval actually recoups the costs of development.
The end result? Fewer drug choices for prescribers and patients, and higher costs due to decreased competition. Not to mention, less chance of truly game-changing therapies even getting into the pipeline because of the immense regulatory and financial burden and risk associated with groundbreaking research.
If a new FDA commissioner, with the support of a strong secretary of HHS, can commit to unraveling unnecessarily burdensome regulations and requirements, and look at the future regulatory landscape with an eye toward what is and what is to come more than, “We do it this way because this is the way it’s always been done,” that will be an excellent first step.
About the author: Kelly Moore has sold clinical research and development software solutions to the pharmaceutical and biotech industries for the past several years. She previously spent 20 years in business development for the pharmaceutical research and development field, focusing on multi-study, global clinical programs. She has a bachelor of arts degree in economics from The University of Texas. Any opinions expressed in this article are strictly her own and are not meant to represent those of any employer, client, or organization with whom she is affiliated.Click here for reuse options!
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