Remarks from FDA Commissioner Scott Gottlieb, M.D., as prepared for testimony before a U.S. Senate Subcommittee on Appropriations on FDA's Fiscal Year 2020 budget

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Mar 28, 2019, 11:38 ET

SILVER SPRING, Md., March 28, 2019 /PRNewswire/ -- Good morning Chairman Hoeven, Ranking Member Merkley, and Members of the Subcommittee. Thank you for the opportunity to appear before you today to discuss the President's Fiscal Year (FY) 2020 Budget request for the FDA.

This is the last time I'll appear before this subcommittee in my current role, and I'm grateful for the opportunity to work with you. I am proud of what we accomplished together, to advance the public health mission of the FDA over the past two years, and I am thankful for your willingness to support us in moments of challenge and opportunity.

We're especially grateful for the funding increases the subcommittee provided for FDA in Fiscal Year 2019. The 2019 Budget includes one of the most substantial increases for the FDA in recent years. The new funding will help the agency modernize our approach to medical product manufacturing and ensure the quality of compounded drugs. You've provided key support for us to advance competition through generic drugs, and to use real world evidence to improve our understanding of the benefits and risks of drugs and devices, across the product lifecycle. We appreciate your support for our efforts to keep pace with rapidly evolving digital health technologies, modernize our information technology systems, and protect the safety of the nation's food supply. I'm personally grateful for the opportunity to engage with all of you directly, and with your staff, in advancing these and many other important goals.

The funding requested in the President's FY 2020 Budget will allow the agency to advance our work to ensure the safety of the food and medical products consumers use every day. We're also seeking new funds to promote competition to bring better and more affordable products to market, support the development of innovative medical products to bring new cures to patients, further modernize our food safety system, strengthen our response capabilities when there's an outbreak of foodborne illness, and continue to implement the 21st Century Cures Act.

Today I want to focus on one of my highest priorities since taking my role two years ago – the FDA's work to combat the opioid crisis. This is the biggest public health crisis facing the agency, and remains one of the biggest crises facing our nation. The contours of this crisis are changing from a crisis that was formed largely in the medical setting, often through prescription drugs that were prescribed lawfully but far too loosely; to a crisis that increasingly involves the use of illicit drugs like super-potent fentanyls. But the fact remains that we still need to take more steps to rationalize prescribing, and too many prescriptions are still being written, and they're being prescribed for durations of use that are too long for the underlying indication.

At the FDA, we're continuing to take new and aggressive steps to combat this crisis. Even as we see some encouraging trends as prescribing declines, we can't lose our focus. We're very far away from fully addressing this crisis. It was a crisis formed over many years, and through many missteps or missed opportunities. To reverse these trends, we need a comprehensive approach. This crisis will remain our highest priority for many years to come.

The 2020 Budget requests $55 million to continue supporting the four main areas of the agency's work. These funds will continue to build on the significant financial investments this subcommittee has provided to the agency for our opioids work over the last two fiscal years. We're focused on decreasing exposure and preventing new addiction, supporting the treatment of those with opioid use disorder, promoting development of novel pain treatment therapies, and increasing enforcement and carefully assessing benefit-risk of opioids. We're also fighting this crisis by implementing the important new authorities that FDA sought and Congress provided in the SUPPORT Act. We've already implemented some of these new authorities, and will implement others very soon, including a requirement that some opioid manufacturers provide unit-of-use packaging for the immediate release (IR) formulations of these drugs. I believe if these IR drugs like Vicodin and Percocet were available in one- or two-day packs, more doctors would default to these more convenient dosing options, and we'd end the bad habit some doctors still have, where they write for 30 tablets at a time, or more.

During the last two years, the FDA worked to change our approach to this crisis. We've committed to act more quickly as we confront new risks and to take a much more aggressive approach to regulatory action. We know that waiting for the accumulation of definitive evidence of harm left us a step behind a crisis that was evolving so quickly, we always remained one step behind its insidious advance. To address this burgeoning epidemic, we need to act forcefully, and sometimes take action before we have perfect information. That's one tragic lesson we have learned.

We need to consider aggressive new steps and embrace options that are tailored to the scope of this crisis and the unique aspects of addiction and opioids. One of the most important steps we could take is to consider the comparative advantage of new opioid approvals relative to other opioids already on the market.

I've previously raised the question of whether there should be an explicit comparative standard for new opioid approvals that requires a demonstration of some advantage over the existing armamentarium, whether it relates to a differentiated way that a new opioid can address pain more effectively than the existing armamentarium or preferably, a characteristic that will also make a new drug safer than an existing alternative. The question is a simple one: In the setting of an opioid addiction crisis of historic proportions, why would the FDA approve a new opioid drug? The supply of drugs doesn't create demand, and it's wrong to assume that just because a new drug is introduced to the market, it's going to lead to greater levels of abuse. But nonetheless, the underlying question is a fair one to ask. In the setting of an addiction crisis, if a novel drug may be eligible for a monopoly, should we demand that a new medicine prove that it has some benefit over existing drugs in terms of how it addresses pain and preferably, how it can help reduce the propensity for diversion and abuse? We wouldn't ask these same questions in other therapeutic settings for a lot of practical reasons. But opioids are different. That is the lesson we've learned from this crisis. And so it's reasonable to ask these questions here.

Today, I come before you to say that, after careful deliberation, I believe that the FDA needs a new, comparative standard for future approvals of new opioid drugs. Given the public health crisis we face, and that American families are still being destroyed by the opioid epidemic, I believe that the FDA should treat opioids, as a class, differently from other drugs.

The FDA has a structured benefit-risk assessment framework that applies to the approval of all new drug products. As part of that benefit-risk assessment, when approving new opioid drug products, the FDA carefully considers the benefits and risks of other available therapies, as well as the broader public health effect of opioids. These include risks related to misuse, abuse, opioid use disorder, accidental exposure and overdose, for both patients and non-patients. The FDA will soon be publishing draft guidance describing this existing benefit-risk assessment as it applies to new opioid analgesic approvals and providing recommendations to sponsors for the types of data that are useful to the agency in carrying out that assessment.

However, based on a careful assessment of the factors that have contributed to the opioid crisis, the FDA has concluded that a new standard is needed to ensure that proposed new opioid drug products offer a comparative advantage over existing opioids already approved to treat the same condition. We need to consider opioids as a class differently than we evaluate other drug classes.

We want to incentivize drug developers to focus on developing new opioid drug products that offer safety advantages, effectiveness advantages, or other material therapeutic advantages over already-approved opioids. For example, safety advantages might be reduced abuse liability, reduced incidence or severity of serious adverse events, or greater tolerability in particular subpopulations. An effectiveness advantage might be improved analgesia for particular subpopulations that could help doctors more narrowly tailor their prescribing of opioids to carefully selected settings that might, in turn, reduce overall prescribing. We hope this new approach would also incentivize developers to increasingly focus their efforts on non-opioid alternatives that could, eventually, begin to replace opioids as the drugs of choice in the armamentarium of drug products used to treat pain and address the risk of addiction.

For this to work, the FDA believes that there should be a premarket demonstration that a new opioid is superior to an already-approved opioid or opioid-containing drug for the same general indication. The standard should be that a new opioid or opioid-containing drug provides a significant advantage relative to an already-approved opioid or opioid-containing drug for the same general indication in terms of greater efficacy or greater safety. We've carefully considered these concepts with the agency's scientific experts. There is a consensus that such a standard could help the agency better address this crisis. Superiority under this standard could result from any physical aspect of the drug product – including the active opioid moiety, other active or inactive ingredients, a device constituent part, the dosage form, packaging, or other product attributes that the sponsor develops. Our goal is for this approach to include abuse-deterrent formulations to promote continued innovation in abuse deterrence properties.

This type of approval framework could operate in conjunction with our existing program for breakthrough drugs, based on the significant advantages these products might offer, or it could be paired with a new designation program for all pain therapies that have preliminary evidence of safer or more effective performance. We want to find new ways to prioritize and make more efficient the development of novel treatments for pain that have the potential to offer significant advantages over our existing options. Drugs that meet the standard could get breakthrough designation or a new designation could be created to further prioritize their review. Eventually, Congress could consider how other incentives, including exclusivity periods, might help drive development toward drugs with safer attributes.

Prescribers and payers also play an important role in combatting the opioids crisis. Our hope is that with a new approach by the FDA – one that's focused on approving safer opioids and new non-opioid alternatives – market actors including payers, health care practitioners, and patients would preferentially select these new options if they became available and as appropriate for the subpopulation requiring pain management; thereby improving the overall safety, and potentially efficacy, of drugs used to treat pain, as well as any other condition of use for which an opioid may be approved.

We understand it would take time to move to a new comparative advantage framework, and to meaningfully change our existing armamentarium to treat pain. But with this change, we can envision an eventual world where enough opioids, opioid-containing drug products, and non-opioid alternatives with comparative advantages are FDA-approved, including generic versions of such products, that the FDA could make the determination – under its current authorities – that either some or all of the older, conventional opioids for a specific indication no longer have a favorable benefit-risk balance; or that the benefit-risk balance of such products is only favorable for certain narrowed indications or under more restrictive conditions of use.

We need to continue to act swiftly and forcefully to get ahead of this crisis. We need to consider novel policy options that are narrowly tailored to opioids. We need to recognize that this crisis is going to be hard to fully reverse. We need to be willing to embrace actions in the setting of opioids that we must accept would be unsuitable to any other drug category. A comparative advantage standard for new opioid drug approvals is one such approach.

We don't want to look back years from now, at an even larger crisis, with regret that there were more aggressive steps that we could have taken sooner to address this tragedy. At the FDA, we've considered all options, and we'll continue to do so. We've concluded that we need to change our approach, and treat opioid approvals differently, in order to have the right tools to fight one of the largest and most complex public health tragedies that our nation has ever faced.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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SOURCE U.S. Food and Drug Administration