Point/Counter

Should an independent regulatory body oversee the FDA drug approval process?

Click here to read the Cover Story, “FDA’s ‘breakneck pace’ accelerates drug access, but concerns persist about lack of complete data.”

POINT

Yes.

Having worked in drug development for 2 decades, including serving as an FDA medical officer/senior medical analyst, I can say with conviction that the FDA mostly does an OK job of reviewing drugs from an efficacy and safety standpoint. However, problems occasionally arise. For instance, a director or deputy director makes the final decision of approval vs. nonapproval — not the FDA medical officer who reviewed the data. The drug is not voted on by a committee, and the FDA is not obligated to hold an advisory committee meeting, or even heed its advice. Instead, it is a single supervisor who ultimately makes the final approval decision. In some cases, that supervisor — an unelected federal employee — makes a contentious choice.

David Gortler, PharmD, FCCP
David Gortler

There should be an option for a review committee for the reviewers who disagree with their supervisors. Ideally, there should be an independent ethics board separate from the agency. It should not necessarily be a congressional committee, because anyone would be hard-pressed to find a member of Congress who has a bachelor’s degree in any science, let alone someone who holds the drug approval process sacrosanct and is able to understand the complexities of clinical pharmacology, drug safety and drug development.

This should be an appointed committee consisting of legitimate medical and pharmacology experts, perhaps separate from the FDA. It should be a paid position for people with drug development and bioethics experience who can address the drug safety and ethics claims to confirm that the best and safest choice is being made for American taxpayers. As it stands now, if a reviewer provides a recommendation, a supervisor can override the reviewer’s decision, even though that reviewer knows a lot more about the drug than his or her supervisor.

When a director or deputy director disagrees with one of his or her reviewers, that supervisor does not have to provide a reason or an explanation for his decision. The disagreeing reviewer has no higher authority on the FDA leadership cascade to turn to, because the director or the associate director has the final say in what to do — even if the supervisor has done little or nothing to review the data.

The Trump administration likely has larger national concerns than evaluating the FDA’s approval of drugs. I am hoping that Scott Gottlieb, MD, the new FDA commissioner, will agree that this is important enough to address.

David Gortler, PharmD, FCCP, is a professor of pharmacology and biotechnology, as well as a pharmacology and drug safety expert with the consulting group FormerFDA.com. He can be reached at gortler@gwu.edu. Disclosure: Gortler reports no relevant financial disclosures.

COUNTER

No.

The last thing we need is another agency overseeing another agency. There is a regulatory body that oversees the FDA — Congress. It has hearings and calls on experts. I don’t think we should replace Congress with an independent regulatory commission, which may add complications.

Bruce A. Chabner, MD
Bruce A. Chabner

The FDA is very open minded, and it approves drugs early when it sees evidence of useful activity. Many pharmaceutical companies have cancer drugs that are active, which they are trying to push through quickly. We would have to wait 10 years to find out if a drug works with OS as the endpoint. A therapy needs to show it is better than the current therapy in instances when there is unclear or marginal evidence of efficacy. But, for many of the drugs being approved, there is no current therapy.

I don’t know what an independent commission would do when it comes to postmarketing commitments. Would it oversee postmarketing trials? In many instances, those trials are out of date and there is no point to doing them once the drug is approved, clearly active and better than the current therapy. Are you going to put those people into a big postmarketing trial? I don’t see the value in that, either.

As long as the FDA continues to hire the best people possible and has a commissioner who knows drugs, there is no need to change. There have been 15 to 20 new drugs approved every year for the last 10 years. Out of a hundred, only a few have been withdrawn or modified, such as gefitinib (Iressa, AstraZeneca) and bevacizumab (Avastin, Genentech). Considering the number of therapies approved and on the market, the agency has not made many mistakes.

The FDA was very skeptical about new drugs and held them back 35 years ago. Now, it is a much better situation and patients are benefitting from the biomedical industry being so active and developing effective drugs. The FDA review process now takes 4 to 6 months; it used to be several years.

The real issue with drugs is high costs, but that is not considered in the approval process. Some people say the FDA should regulate the cost of drugs, but it does not have the tools to do so. When I was at NCI, we made the first attempt to fix a price for a cancer drug — taxol — by pricing it at the same level of other medications for the same indication. But that was a long, involved process and I don’t think the FDA is set up for that.

Bruce A. Chabner, MD, is professor of medicine at Harvard Medical School and director of clinical research at Massachusetts General Hospital Cancer Center. He can be reached at bchabner@partners.org. Disclosure: Chabner reports no relevant financial disclosures.

Click here to read the Cover Story, “FDA’s ‘breakneck pace’ accelerates drug access, but concerns persist about lack of complete data.”

POINT

Yes.

Having worked in drug development for 2 decades, including serving as an FDA medical officer/senior medical analyst, I can say with conviction that the FDA mostly does an OK job of reviewing drugs from an efficacy and safety standpoint. However, problems occasionally arise. For instance, a director or deputy director makes the final decision of approval vs. nonapproval — not the FDA medical officer who reviewed the data. The drug is not voted on by a committee, and the FDA is not obligated to hold an advisory committee meeting, or even heed its advice. Instead, it is a single supervisor who ultimately makes the final approval decision. In some cases, that supervisor — an unelected federal employee — makes a contentious choice.

David Gortler, PharmD, FCCP
David Gortler

There should be an option for a review committee for the reviewers who disagree with their supervisors. Ideally, there should be an independent ethics board separate from the agency. It should not necessarily be a congressional committee, because anyone would be hard-pressed to find a member of Congress who has a bachelor’s degree in any science, let alone someone who holds the drug approval process sacrosanct and is able to understand the complexities of clinical pharmacology, drug safety and drug development.

This should be an appointed committee consisting of legitimate medical and pharmacology experts, perhaps separate from the FDA. It should be a paid position for people with drug development and bioethics experience who can address the drug safety and ethics claims to confirm that the best and safest choice is being made for American taxpayers. As it stands now, if a reviewer provides a recommendation, a supervisor can override the reviewer’s decision, even though that reviewer knows a lot more about the drug than his or her supervisor.

When a director or deputy director disagrees with one of his or her reviewers, that supervisor does not have to provide a reason or an explanation for his decision. The disagreeing reviewer has no higher authority on the FDA leadership cascade to turn to, because the director or the associate director has the final say in what to do — even if the supervisor has done little or nothing to review the data.

The Trump administration likely has larger national concerns than evaluating the FDA’s approval of drugs. I am hoping that Scott Gottlieb, MD, the new FDA commissioner, will agree that this is important enough to address.

David Gortler, PharmD, FCCP, is a professor of pharmacology and biotechnology, as well as a pharmacology and drug safety expert with the consulting group FormerFDA.com. He can be reached at gortler@gwu.edu. Disclosure: Gortler reports no relevant financial disclosures.

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COUNTER

No.

The last thing we need is another agency overseeing another agency. There is a regulatory body that oversees the FDA — Congress. It has hearings and calls on experts. I don’t think we should replace Congress with an independent regulatory commission, which may add complications.

Bruce A. Chabner, MD
Bruce A. Chabner

The FDA is very open minded, and it approves drugs early when it sees evidence of useful activity. Many pharmaceutical companies have cancer drugs that are active, which they are trying to push through quickly. We would have to wait 10 years to find out if a drug works with OS as the endpoint. A therapy needs to show it is better than the current therapy in instances when there is unclear or marginal evidence of efficacy. But, for many of the drugs being approved, there is no current therapy.

I don’t know what an independent commission would do when it comes to postmarketing commitments. Would it oversee postmarketing trials? In many instances, those trials are out of date and there is no point to doing them once the drug is approved, clearly active and better than the current therapy. Are you going to put those people into a big postmarketing trial? I don’t see the value in that, either.

As long as the FDA continues to hire the best people possible and has a commissioner who knows drugs, there is no need to change. There have been 15 to 20 new drugs approved every year for the last 10 years. Out of a hundred, only a few have been withdrawn or modified, such as gefitinib (Iressa, AstraZeneca) and bevacizumab (Avastin, Genentech). Considering the number of therapies approved and on the market, the agency has not made many mistakes.

The FDA was very skeptical about new drugs and held them back 35 years ago. Now, it is a much better situation and patients are benefitting from the biomedical industry being so active and developing effective drugs. The FDA review process now takes 4 to 6 months; it used to be several years.

The real issue with drugs is high costs, but that is not considered in the approval process. Some people say the FDA should regulate the cost of drugs, but it does not have the tools to do so. When I was at NCI, we made the first attempt to fix a price for a cancer drug — taxol — by pricing it at the same level of other medications for the same indication. But that was a long, involved process and I don’t think the FDA is set up for that.

Bruce A. Chabner, MD, is professor of medicine at Harvard Medical School and director of clinical research at Massachusetts General Hospital Cancer Center. He can be reached at bchabner@partners.org. Disclosure: Chabner reports no relevant financial disclosures.