Republicans seize on reports critiquing FDA to push for agency reforms

Sheila Kaplan, STAT News

June 15, 2016

Has Congress’s watchdog agency just given Tennessee Senator Lamar Alexander the ammunition he needs to push a stalled biomedical reform through the Senate?

The Government Accountability Office on Wednesday issued two critical reports suggesting that the Food and Drug Administration isn’t properly planning how to keep pace with medical science.

One report notes that the FDA lacks goals, targets, and time frames for keeping up with scientific advances — and also fails to track the money it spends on these efforts. The other finds fault with the FDA’s strategic plan, which is supposed to speed approvals of drugs and devices, especially combination medical products, such as heart stents that also deliver blood thinners to prevent clots.

The reports land just as time is winding down for the Senate to pass its version of the 21st Century Cures Act, which the House has already overwhelmingly approved. The bill aims to get drugs and devices to market more quickly.

[…]

Some Democrats say it weakens FDA standards on drug and device approval too much. Critics also want more funding for both the FDA and the National Institutes of Health. To try to move things along, Alexander chopped the legislation into a series of smaller bills, but funding remains a sticking point.

A few weeks ago, Alexander took to the floor to urge action. “There is no excuse whatever for us not to get a result this year. And it would be extraordinarily disappointing to millions of Americans if we did not,” he said.

It’s not clear whether the GAO’s concerns about the FDA would be addressed by the 21st Century Cures bill, or by the Senate’s package of biomedical bills.

Neither the HELP committee Democrats nor the FDA were available for immediate comment.

Diana Zuckerman, a critic of the bills and the president of the National Center for Health Research, said the FDA is already focused too much on speeding up drug and device approvals and is not paying enough attention to “doing its job to protect the health and safety of patients and consumers.”

To see original article, click here.

Coalition letter urging Senators to oppose S. 2912, the Trickett Wendler Right to Try Act of 2016

June 14, 2016

Dear Senator,

As members of the Patient, Consumer, and Public Health Coalition, we are writing to express our views as consumers, physicians, scientists, and public health experts regarding S. 2912, the Trickett Wendler Right to Try Act of 2016. We strongly urge you to NOT co-sponsor this bill. This bill is bad for patients, bad for doctors, bad for drug development, and bad for science.

As you may know, the history of the FDA is a history of Congress responding to medical tragedies by requiring FDA to do more to protect patients from harmful medical products. Thalidomide birth defects and deaths and infertility from the Dalkon Shield IUD are just two of many examples. Although the AIDS crisis resulted in a more flexible approach for deadly diseases for which there were no treatments, today’s HIV/AIDS advocates are among the FDA’s strongest supporters, because the scientific evidence that FDA required of pharmaceutical companies resulted in effective treatments and prevention strategies. The FDA’s essential role continues to be to achieve a balance between patients’ rights and societal good. It is critical that, in your role as a Senator, you support the agency in upholding this delicate balance.

The idea behind this bill and other Right to Try laws is to give terminally ill patients access to experimental drugs that have passed phase I testing – testing that is often done on healthy volunteers, not people with serious illnesses. While this type of legislation promises cures, it offers false hope. Since most phase 1 trials include less than 30 patients and are designed only to identify the most immediate side effects, they provide very little information about the drug’s safety and effectiveness. In fact, most drugs with promising phase 1 test results are later found to be too risky or ineffective for patients to use. This is especially true for cancer drugs. Under the proposed bill, desperate patients are likely to be exploited by unethical companies that want to sell their unproven treatments at prices that would bankrupt many families. Health insurance companies will not pay for these unproven treatments – if they did, insurance premiums would skyrocket as patients harmed by unproven treatments required more expensive remedial care.

In the recent past, access to experimental bone marrow transplants for breast cancer was expedited due to an early benefit seen in preliminary data. It was not until many women were harmed and some even died that researchers realized a terrible mistake had been made. If doctors and patients can be fooled into trying such a painful and toxic treatment, imagine how often patients would be fooled by treatments that are less painful but ineffective and expensive snake oil.

This legislation would subject patients to a great risk of harm and they would have no legal recourse if things go wrong. In fact, this bill and similar state Right to Try laws reflect the libertarian philosophy of the Goldwater Institute that created them: the patient is on his or her own without any protections or safeguards. The bill explicitly states that drug and device companies as well as physicians cannot be held liable for any tragic outcomes, even if the company or a physician misled the patient. Even worse, if a patient suffers a terrible medical complication from a Right to Try treatment, the insurance company would not need to pay for the medical care that is needed to treat that complication. The only thing worse than a terminal illness is being terminally ill and suffering a major complication as the guinea pig in an experimental treatment that you had to pay for.

There are many other problems with the bill, but perhaps the most important thing for you to know is that the FDA’s current compassionate use program already provides patients with the opportunity to try experimental drugs, while offering some protections to patients and society. For example, while physicians and drug companies are required to report the outcomes of experimental treatments obtained through the FDA’s compassionate use program, Right to Try laws do not carry such stipulations. Indeed, this bill strips all protections from patients who would use it. Bypassing the oversight of the FDA is not in the best interest of patients or the public health.

The one shortcoming of the FDA’s current compassionate use (“expanded access”) program is also in the Right to Try bill: the company is not required to provide the experimental drug to the desperate patient. In some cases, that’s because the company doesn’t have enough doses to provide to patients not in a clinical trial, and in other cases, the company believes that the patient is more likely to be harmed by the product than helped by it.

In conclusion, Right to Try laws such as S. 2912 will do more harm than good for patients and society. The bill is opposed by experts in the field as well as by legitimate drug companies. We urge you to oppose the Trickett Wendler Right to Try Act of 2016.

Sincerely,

American Medical Women’s Association

Connecticut Center for Patient Safety

DES Action

Jacobs Institute of Women’s Health

MedShadow Foundation

National Center for Health Research

National Physicians Alliance

 

 

The Patient, Consumer, and Public Health Coalition can be reached through Tracy Rupp at 202-223-4000 or tr@center4research.org.

 

Coalition’s comments to the FDA on User Fees for Over-the-Counter Drugs

June 10, 2016

Comments of Members of the Patient, Consumer, and Public Health Coalition at the Public Meeting on OTC Monograph User Fees

Thank you for the opportunity to speak today.  My name is Paul Brown and I am speaking on behalf of 10 members of the Patient, Consumer, and Public Health Coalition.  Our informal coalition of nonprofit organizations represents the interests of patients, consumers, health-care professionals, scientists, and public health experts.

We support providing FDA with additional resources through the implementation of OTC user fees, so the agency can fulfill its mission of protecting the public from dangerous and ineffective OTC products.  Additional funding will help FDA to finalize the remaining OTC monographs, and allow patients and consumers to have confidence in the OTC products they use every day.

Since the OTC drug review process was established in 1972, FDA has struggled to complete the safety and efficacy evaluations of ingredients used in OTC drug products.  More than 40 years later, the monograph process still has not been completed for all ingredients and conditions of use.

As a result, many OTC products without a general recognition of safety and effectiveness (GRASE) determination continue to be marketed, leaving millions of American vulnerable to potentially unsafe products.

A staff of only 18 people cannot effectively regulate 800 active ingredients for over 1,400 different therapeutic uses. We are particularly concerned about how the current process limits FDA’s ability to require new warnings or other labeling changes to address emerging safety or effectiveness issues in a timely manner.

We strongly urge FDA to include funding in user fees to address emerging safety and effectiveness issues.  FDA needs resources to provide ongoing surveillance of marketed products and move quickly when safety signals arise.

This is especially important for products used by children. When the monographs were first developed in the 1970s, FDA lacked specific data on use in infants and children.  So, FDA did what was scientifically customary at the time and extrapolated the data by simply reducing adult doses by a percentage.

Our understanding of pediatric dosing has grown since then and, as a result, data from actual use in the pediatric population is preferred.  Many products continue to be given to infants and children without sufficient safety and effectiveness data. OTC user fees are needed to support the reexamination of the use of these products in children.

OTC product user fees should also support the development of product formulation standards. The monographs set forth the conditions under which a specific active ingredient used in a drug product is generally recognized as safe (GRAS) and generally recognized as effective (GRAE) and not misbranded.

The monographs, however, generally do not dictate what other non-active ingredients can be added, or other aspects of the formulation. For example, we know that many product formulation variables affect how much of a tablet’s medication dose is absorbed. The regulatory science behind generic drugs has shown us that excipients and manufacturing quality control must factor into the determination of a product’s safety and effectiveness. Therefore, we recommend development of standards for drug products, not just their ingredients; and we urge FDA to include funding for this in user fees. (As United States Pharmacopeia (USP) has recommended.)

A user fee system for OTC drugs will have to take into account the way OTC drugs come to market.  Since the monograph system is ingredient-based and not product-based and, since sponsors of monograph drugs are not required to obtain FDA approval prior to marketing, the fee structure will have important differences as compared to that used by the prescription drug programs.

We recommend that user fees be structured as a product listing fee based on a sliding scale proportionate to the complexity and reviewing resources required. We feel this mechanism would provide the agency with a stable and predictable source of funding for the OTC division.

We would avoid structuring the fee as a facility fee since it may have the unintended consequence of pushing sponsors to consolidate operations into as few facilities as possible. This could impact the supply chain and cause OTC drug shortages, if a facility is removed from operation.

In summary:

  • We support the establishment of a user fee program for OTC drugs so that all OTC monographs can be finalized.
  • We urge you to include funding in user fees to address emerging safety and effectiveness issues as well to reexamine use of certain OTC products in infants and children. Additional resources are critical to protect public health.

Thank you for your attention.

American Medical Student Association
MAME
MedShadow
MISSD
National Center for Health Research
Our Bodies Ourselves
Quinolone Vigilance Foundation
The TMJ Association
Washington Advocates for Patient Safety
WoodyMatters

Letter Urging Senators to Protect Patient Safety in Innovation Health Bill & Provide Mandatory $$$ for NIH & FDA

May 24, 2016

The Honorable #{Fname} #{Lname}

United States Senate

Washington, DC 20510

 

Dear Senator #{Lname},

A recent STAT-Harvard poll shows that a majority of Americans oppose changing federal regulations to speed the development and approval of new medical products. They are concerned that speeding approvals would lower safety and effectiveness standards for medical products.1 As members of the Patient, Consumer, and Public Health Coalition, we share those concerns.  We also write to emphasize that any effort to develop safe and effective medical products should include mandatory new funding for the National Institutes of Health (NIH) and the Food and Drug Administration (FDA).

The House’s 21st Century Cures Act and its Senate companion legislation should focus more on ensuring that medical products are safe and effective and less on speeding medical products to the market. We appreciate that the Senate bills were less dangerous for patients than the House legislation. But, we opposed 6 of the 19 bills that the Senate passed out of the HELP Committee because they were not beneficial to patients.

When these bills (which we’ve listed below) reach the Senate floor, we strongly urge you to oppose them because they weaken FDA’s safety and effectiveness standards.

 Current and former FDA Commissioners have expressed their concerns as well. Current FDA Commissioner, Robert Califf recently said, “This legislation, if not carefully crafted, could pose significant risks for FDA and American patients…Innovative therapies are not helpful to patients if they don’t work, or worse, cause harm.” 2 Former FDA Commissioner David Kessler, who in the 1990s led the FDA under presidents from both parties, said, “It’s time to uncouple the promise of research funding from the requirement that FDA standards be lowered.”3

The underlying premise of the House and Senate bills seems to be that the FDA needs to speed up its approval process. This premise is based on the erroneous claim that other countries approve medical products more quickly. Nearly two-thirds of the novel drugs approved in 2015 (29 of 45, 64%) were approved in the United States before being approved in any other country.4 In fact, a Forbes article expressed concern that the pendulum has swung too far and that “the FDA is basically providing a rubber stamp” for drug approvals and that drug approvals are at an all-time high.5 Similarly, research indicates that “it takes the same amount of time or less for patients to gain access to innovative, high-risk medical devices” in the U.S. as compared to Germany, France, Italy, and Britain.6 Approximately 99% of all regulated medical devices are cleared by the FDA through the speedy and less-than-rigorous 510(k) process, and the FDA reviews 90% of those applications within 90 days.7 With the emphasis on speed over safety it is not surprising that millions of Americans have been harmed needlessly from defective medical devices such as surgical mesh, metal-on-metal hips, power morcellators, duodenal scopes, birth control devices (Essure), and inaccurate medical device software. It is clear that we need stronger device safety regulations and better postmarket surveillance. When there are problems with medical devices in the real world, patients need to be notified as soon as possible.

Members of our coalition are particularly concerned about the following bills:

MEDTECH Act (S. 1101)

The MEDTECH Act deregulates Electronic Health Records (EHRs) and other electronic health technology. This prevents the FDA from collecting adverse event data or recalling defective software, even when the devices have life-threatening flaws.  The National Center for Health Research studied FDA-reported recalls of medical software, identifying more than 600 software devices and 1.4 million units recalled for moderate or high risk patient safety issues.  They found that if medical software is removed from FDA regulatory oversight, millions of patients would be at risk from defective software. For example, oncology electronic medical record systems were recalled in the past because they calculated and recorded incorrect drug dosages. Clinical decision support systems used during surgery were recalled because they erroneously switched patient data and failed to warn physicians about dangerous drug reactions.

Advancing Breakthrough Devices for Patients Act (S. 1077)

This bill would set a low bar for medical devices to qualify for breakthrough status, and encourage shorter or smaller clinical trials, which would make it difficult if not impossible to include subpopulations (women, seniors, racial and ethnic minorities) in the analysis of the trials. The bill would also push FDA to rely on post-market studies rather than ensuring safety or effectiveness before hospitals and patients pay for the devices. A 2015 GAO report found that most required post-market device studies are never completed. In other words, if this bill passes, there would be no conclusive evidence pre-market or post-market.

The Advancing Hope Act (S. 1878)

Priority review vouchers allow FDA priority reviews to go to the highest bidder rather than allowing the FDA to prioritize matters of the most public health importance.  The bill’s priority review vouchers would be applied to drugs that are already in development rather than spurring new drugs because of the long drug development cycle.

The Medical Countermeasure Innovation Act (S. 2055)

This bill establishes a new priority review voucher program for medical countermeasures (e.g. antidotes to anthrax and other bioweapons). As with existing priority review voucher programs, it does not necessarily reward innovation and prevents FDA from prioritizing matters of greatest public health importance.

The Combination Product Regulatory Fairness Act (S. 1767)

This bill would broaden the ability of combination products to be classified as devices.  This would inappropriately allow products that should be classified as drugs to go through the FDA’s less rigorous device clearance/approval process.

Patient Focused Impact Assessment Act (S. 1597)

The Patient Focused Impact Assessment Act adds a new layer of bureaucracy to the FDA, does not provide safeguards to ensure that patient perspectives will be free of industry influence, and may provide a mechanism for industry to promote drugs directly to consumers for off-label uses.

REGROW Act (S. 2689)

The REGROW Act is the latest effort to lower research standards and deregulate new treatments. Although this bill was not part of the HELP Committee markups held earlier this year, we understand it might be added to a package bill. The Act allows complex biologic therapies to be conditionally approved without phase III trials and prescribed to patients based on studies in a limited population without proof that the new therapy actually works. If this Act passes, it could lead to the Centers for Medicare and Medicaid (CMS) wasting billions of dollars on medications that were approved by the FDA but do not work.

The Alliance for Regenerative Medicine (ARM) does not support the REGROW Act, because those researchers want  “to ensure access to safe and effective regenerative medicine therapies without putting patients at risk, disadvantaging American therapeutic developers and potentially damaging a very promising field of medicine”(emphasis added).[7]  In her recent article about stem cell research in the New England Journal of Medicine, University of Wisconsin law professor and biomedical ethicist R. Alta Charo said, “The best way to find safe effective cures is through the careful steps of clinical trials and treatment monitoring.”8

 For the remaining Senate FDA-NIH bills, we urge you to only support them if they provide mandatory new funding for the National Institutes of Health (NIH) and the Food and Drug Administration (FDA).   If mandatory funding is not included, we strongly urge you to vote against the bills.

 Funding for NIH & FDA

Prior to last year’s $2 billion one-time funding increase, NIH’s funding remained flat for more than a decade (about $30 billion per year).9 Unfortunately, the Cures Act $9 billion increase in NIH research funding is still below what it would have been had Congress not previously allowed NIH’s annual budget to stagnate.10

The Cures Act also would provide the FDA with $550 million in new mandatory funding over five years, but the amount of money pales to the additional workload that the Act assigns to the FDA. New FDA Commissioner, Robert Califf, has warned that Congress needs to provide the FDA with an “adequate and stable source of funding” for the mandates in the legislation.11

Adequately funding NIH is essential if we are serious about the Vice President’s Cancer Moonshot, precision medicine, and efforts to find treatments for Alzheimer’s disease, autism, depression, schizophrenia, addiction and other maladies.12  Underfunding the FDA is likely to slow the approval of new medical products because the agency’s workload has been significantly increased with unfunded mandates.

Conclusions

Members of our coalition agree with most Americans in opposing regulations that would speed the approval of medical products by lowering FDA’s safety and effectiveness standards. We strongly urge you to oppose any bill that weakens FDA’s safety and effectiveness standards, and to ensure that any legislation aimed at improving medical treatments include new mandatory funding for the NIH and the FDA. 

AMWA

Breast Cancer Action

Center for Medical Consumers

Connecticut Center for Patient Safety

MAME

MedShadow Foundation

MISSD

MRSA Survivors Network

National Center for Health Research

National Consumers League

National Organization for Women

National Physicians Alliance

National Women’s Health Network

Quinolone Vigilance Foundation

The TMJ Association

Washington Advocates for Patient Safety

WoodyMatters

Coalition Comments on FDA Draft Guidance for Generic Abuse-Deterrent Opioids

May 24, 2016

Comments of members of the Patient, Consumer, and Public Health Coalition on “General Principles for Evaluating the Abuse Deterrence of Generic Solid Oral Opioid Drug Products: Guidance for Industry”

Docket No. FDA-2016-D-0785

Thank you for the opportunity to comment on the draft guidance, “General Principles for Evaluating the Abuse Deterrence of Generic Solid Oral Opioid Drug Products: Guidance for Industry.” Development of such products is of great public health importance as a strategy to help make pain medications more affordable to those who truly need them while also reducing opioid abuse.

The Patient, Consumer, and Public Health Coalition is an informal coalition of nonprofit organizations representing the interests of millions of patients, consumers, health-care professionals, scientists, and public health experts. The comments and recommendations of the members of the Coalition follow:

  1. Avoid use of the misleading term “abuse-deterrent”

While the guidance does not specifically address labelling, we urge FDA to avoid use of the term “abuse-deterrent” since it is often misinterpreted to mean “addiction-deterrent.” In fact, a recent study found that nearly half (46%) of physicians think that abuse-deterrent formulations make these medications less addictive than others.[1] Instead, labelling language that more accurately captures the product’s abuse-deterrent properties should be used. For example, if a pill has been made difficult to crush, it should be labeled “crush-resistant.” It is important to use precise language because “abuse” and “addiction” are often used interchangeably.

  1. Provide guidance for all types of abuse-deterrent technologies

The draft guidance includes recommendations to industry on the studies needed to show that a generic opioid is no less abuse-deterrent than the brand-name (or reference) drug, with respect to all potential routes of abuse. It provides testing recommendations for four of the seven categories of abuse-deterrent technologies described in FDA’s “Abuse-Deterrent Opioids: Evaluation and Labeling” guidance: solid oral opioid drug products formulated to incorporate physical or chemical barriers, agonist/antagonists, aversive agents, and combinations of two or more of these technologies. Since this field is continually evolving, we urge the FDA to also develop guidance for products in the remaining three categories so that patients have access to a variety innovative pain management therapies that are also safe and effective.

  1. Require the generic product to show it is not less abuse-deterrent than the reference product with respect to all potential routes of abuse

We agree with FDA that it is necessary for a generic manufacturer to provide studies that show that their product is not less abuse-deterrent than the reference product with respect to all potential routes of abuse. This will help minimize the risk of shifting abuse to another potentially more dangerous route of abuse (e.g. oral abuse to intravenous abuse). It will also prevent the approval of a generic opioid product that is less abuse-deterrent than the reference drug, which could lead opioid abusers to preferentially seek out and abuse generics. It is critical that, in attempting to solve one problem, we avoid creating a new one.

  1. Include a control product in tampering studies

Since the abuse-deterrent properties of currently approved drug products can eventually be compromised, it is important to have a good understanding of the type of manipulation or tampering that renders the opioid non-abuse-deterrent. Therefore, we agree with FDA’s recommendation to include a control product (i.e. a non-abuse-deterrent version of the reference product) in studies of abuse-deterrence. This will help provide a more accurate understanding of the product’s ability to deter abuse.

  1. Apply extra scrutiny to Tier 1 studies

As stated in the draft guidance, FDA recommends that applicants follow a tier-based approach when comparing a generic product to a reference product. This tier-based approach allows for hierarchical testing, starting with simple and gentle manipulations of the product (Tier 1) and progressing to more destructive mechanical and chemical manipulations (Tiers 2, 3, 4, etc.). Using this method, all of the null hypotheses in Tier 1 must be rejected before testing the null hypotheses in higher tiers. For that reason, it is essential that Tier 1 studies are rigorously conducted and documented. We urge FDA to apply extra scrutiny to these Tier 1 studies to ensure the statistical integrity of the tier-based approach.

  1. Enforce post-marketing real-world epidemiological study requirements for reference opioids

While not specifically addressed in this guidance, we strongly urge the FDA to enforce post-market study requirements for reference drugs. Since generic abuse-deterrent opioid formulations use the branded product as the reference for actual ability to deter abuse, it is critical that FDA follow through on its requirement for real-world post-marketing studies. An unsafe and ineffective reference product will result in unsafe and ineffective generic products. Sponsors of brand name products with approved abuse-deterrent labeling must complete the required long-term epidemiological studies to assess their effectiveness in reducing abuse in practice or risk having their approval revoked.

Conclusions

In summary, we applaud the FDA for providing guidance to industry to advance the regulatory science behind abuse-deterrent oral opioid formulations. We agree with many of the recommendations in the guidance and urge the FDA to consider our recommendations regarding the use of the term “abuse-deterrent”, the development of guidance addressing additional abuse-deterrent technologies, the scrutiny applied to Tier 1 studies, and the enforcement of post-marketing study requirements for reference products.

Sincerely,

American Medical Women’s Association

Cancer Prevention and Treatment Fund

Connecticut Center for Patient Safety

MedShadow Foundation

National Consumers League

The TMJ Association

Washington Advocates for Patient Safety

 

References

[1] Hwang CS, Turner LW, Kruszewski SP, et al. Primary Care Physicians’ Knowledge And Attitudes Regarding Prescription Opioid Abuse and Diversion. Clin J Pain. 2016 Apr;32(4):279-84.

 

 

 

 

Why we shouldn’t trade a weakened FDA for more medical research funds

By Ed Silverman, STAT News

May 17, 2016

In a quest to bring new medical products to Americans, Congress is considering a grand bargain.

Legislation passed last year by the House would provide billions more dollars for medical research and encourage faster approval of prescription drugs and devices. The Senate, meanwhile, is currently working on a set of companion bills in hopes of crafting a compromise measure.

“If we succeed, this will be the most important bill signed into law this year,” Senate health committee chairman Lamar Alexander, a Tennessee Republican, said at a committee meeting last month.

This sounds promising. After all, adding $9 billion to the National Institutes of Health’s budget over the next five years to underwrite new cures is a good idea. And in an era when desperately ill patients are clamoring for new medicines, giving the Food and Drug Administration extra tools also makes sense.

But there’s a catch. By linking the extra funds to speedier approvals, Congress appears ready to undermine regulatory standards. And this is a misguided notion that, unfortunately, is more likely to help companies than patients.

For instance, the House bill, which is known as the 21st Century Cures Act, would allow the FDA to approve added uses for a drug without relying on a randomized clinical trial — the gold standard for determining whether a medicine can benefit patients and the extent to which there are serious side effects. Instead, the bill would permit the agency to base such decisions on “clinical experience,” a loosely defined term that means, essentially, anecdotal observations from physicians and patients.

To be fair, the FDA would not have to consider such information. And the Senate has, so far, not adopted this notion. But this is hardly the sort of rigorous scientific data that should be used to establish whether a drug is safe and effective.

“This is a harsh way of putting it, but this is why I call it the 19th Century Fraud Act,” said Harvard University political scientist Daniel Carpenter, who studies the FDA. “This is a part of the bill that threatens to take us back more than a century.”

“I call it the 19th Century Fraud Act.” Daniel Carpenter, Harvard University, said.

The final details of the Senate bill remain uncertain, but some proposals are also prompting objections. As an example, one suggestion for speeding approvals of medical devices is to label certain products as “breakthroughs,” a designation that would offer an expedited review pathway. To qualify as a breakthrough, though, a company need only argue its product is either a significant advance over existing devices or is in the best interest of patients.

The use of the word “or” is problematic, because it creates wiggle room for unproven claims. “It’s very vague and only encourages companies to seek breakthrough status,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. […]

To see the original article, click here.

Coalition’s Comment the Black Box Warning and Patient Decision Checklist for Essure Birth Control System

May 3, 2016

 

Division of Dockets Management
Food and Drug Administration
5630 Fishers Lane, Room 1061 (HFA-305)
Rockville, Maryland 20852

 

Comments of members of the Patient, Consumer, and Public Health Coalition
on the Draft Guidance “Labeling for Permanent Hysteroscopically-Placed Tubal Implants [the Essure System] Intended for Sterilization”

 

As members of the Patient, Consumer, and Public Health Coalition, we appreciate the efforts of the FDA to improve the information available to women and physicians considering the Essure System for Permanent Birth Control, through a “Boxed Warning” (black box warning) and a “Patient Decision Checklist” as part of the product labeling.  However, we believe that the draft guidance is not sufficient.

 

The purpose of the draft guidance is to ensure that women receive and understand information about the risks and benefits of permanent devices (hysteroscopically-placed tubal implants intended for sterilization). The Essure System is the only type of these devices marketed in the U.S.

 

We agree with the FDA that in order to make patients aware of the risks associated with the Essure System, there needs to be “accurate product labeling and effective messaging of that labeling.” We agree with FDA that a boxed warning and patient decision checklist should be included in the labeling sections of the Federal Food, Drug, and Cosmetic Act (FD&C Act).

 

The FDA is seeking comment on this draft guidance regarding the wording and content of the black box warning and the patient decision checklist. Our specific comments are below:
The Boxed Warning

This section of the draft guidance has two bullets. The first covers adverse events associated with the device and its insertion or removal, and the second bullet declares that a statement “noting these risks should be conveyed to the patient during the women’s decision-making process.” This short section is augmented by example of a Boxed Warning included in Appendix A of the draft guidance. The example lists several adverse events that may occur from the use of Essure.

 

We have two concerns about the Black box warning:

 

  • We believe that the phrase “some patients” will be misconstrued as “not many patients.” This is not consistent with the thousands of women who have reported their problems from Essure to the FDA.  We suggest you delete the word “some” or replace it with “Many” or “numerous.”

 

  • Most patients will probably not see the device’s boxed warning. To make it clear to patients that the implants are associated with serious adverse events, the black box warning and a description of why the device has one, should be added to the Patient Checklist.

 

Possible text for the Patient Checklist could read:

 

“I understand that the Essure device has received a black box warning, which is used to call attention to serious or life-threatening risks. I acknowledge that my provider has discussed these risks with me and that I understand those risks.”

 

Patient Decision Checklist

We support the FDA’s requirement that physicians provide women considering Essure or similar implants with a list of risks that the FDA has found to be linked to the device.

 

Women can and do make good decisions about the risks and benefits of drugs and medical devices when they have good information. However, the Patient Checklist needs to be easier to understand and more succinct in order to maximize the likelihood that most patients will read and understand it.

As drafted, the Patient Checklist is too long, the language is at a level that seems designed for college graduates rather than the 6th-8th grade reading level that is recommended by health educators, and the check list emphasizes the permanent nature of the device rather than the risks of the device.  In its current form, it is unlikely that most women will read more than the first few points.  The checklist needs to be completely rewritten.  It should also use the term “sterilization” rather than “permanent birth control.”  Essure patients have advised us that the term “permanent birth control” is confusing.

Some specific language in the example checklist needs to be changed.  On page 12, lines 407-411, it says: “These types of events, although not reported in clinical trials supporting device approval, have been reported to FDA by women implanted with the Essure System.”  We request that you delete: “although not reported in clinical trials supporting device approval.” The inclusion of that line downplays the thousands of serious adverse events reported by women from their real world experience.  For example, the National Center for Health Research study of 1104 women with Essure problems found that 86% reported pain (mostly pelvic or abdominal), 34% reported excessive bleeding (some bled every day of the month instead of a regular period), 22% fatigue, 16% hair loss, 12% hysterectomy, 12% depression, and  7% allergies. While these are not a random sample of Essure patients, they indicate the clear pattern of complications that women report. It is important to note that about 35% had Essure removed and about half of those had a complete recovery.  Only 5% of those who had them removed reported no improvement in symptoms.13

 

In addition, it is essential that the checklist be completed at least one week prior to the Essure procedure, so that the patient is provided the information prior to scheduling the surgery or at the least at a time that she can cancel the surgery if she decides it is not a good idea for her.

 

Additional Research Study

We fully support FDA requiring a “clinical study for Essure to determine heightened risks for particular women.” During the 2015 FDA Advisory Committee meeting, it became clear that many women have been harmed by Essure and it is not known if the risks are higher for some subgroups of women or not.

 

Requiring an additional research study be conducted as soon as possible is essential so that providers and women to make more evidence-based decisions. We urge the FDA to require a study of sufficient size and inclusive of diverse populations (including women of color, women of diverse reproductive ages, body size, for example). The study as a whole and the subgroups must be of sufficient power to detect increased risks for certain women and conducted in a way that short-term and long-term complications are well-documented.

 

Conclusions

Members of the Patient, Consumer, and Public Health Coalition strongly support a Boxed Warning and a Patient Decision Checklist for the Essure Permanent Birth Control device.  However, both need to be improved, and the latter needs to be rewritten to be suitable for the average reading skills in the U.S.

 

One final point, we are very concerned about how the FDA discusses Essure in the press. FDA made a statement on February 29, 2016 that “Essure remains an appropriate option for the majority of women seeking a permanent form of birth control” (emphasis added).14 Until better research is conducted, it is not possible to say what percentage of women or which subgroups of women are most likely to benefit and least likely to be harmed by Essure compared to other alternatives.
Breast Cancer Consortium
MISSD
National Center for Health Research
Washington Advocates for Patient Safety
WoodyMatters

 

 

The Patient, Consumer, and Public Health Coalition is an informal coalition of nonprofit organizations representing the interests of millions of patients, consumers, health-care professionals, scientists, and public health experts. The coalition can be reached through Paul Brown at (202) 223-4000 or at pb@center4research.org.

Coalition’s Comments on FDA’s Efforts to Improve Diversity and Analyses in Clinical Trials

April 29, 2016

 

Division of Dockets Management
Food and Drug Administration
5630 Fishers Lane, Room 1061 (HFA-305)
Rockville, Maryland 20852

 

Comments of members of the Patient, Consumer, and Public Health Coalition on FDA’s Efforts to Improve Diversity and Analyses in Clinical Trials Docket No. FDA-2015-N-4952

 

We are writing as member organizations of the Patient, Consumer, and Public Health Coalition, to express our views on the need for diversity and subgroup analyses in clinical trials.

 

We support efforts to improve the safety and effectiveness of drugs and medical devices for ALL patients that are likely to use them. Women, people of color, and patients over the age of 65 have often been under-represented in clinical trials. Just as that was improving in recent years, efforts to speed drug approval have resulted in smaller clinical trials. So, the numbers of patients in those groups are again shrinking, making it impossible to do meaningful analysis of safety or effectiveness.

 

For example, cancer is most common in the elderly, but many cancer drug trials include very few elderly patients. Cancer drugs are often more toxic and less effective in the elderly. But with so little information on elderly patients when a drug is approved, it can be years later that we learn that elderly patients are more likely to be harmed if approved dosing information is followed.

 

The National Center for Health Research recently examined drugs reviewed by FDA Advisory Committees in 2014. They found that, for the 24 drugs the committees reviewed, 7 sponsors did not even tell FDA how many patients in their studies were 65 and older. That is a crucial subgroup for Medicare to make coverage decisions, and should always be analyzed separately. An additional 2 sponsors included fewer than 30 patients 65 and older. In other words, over onethird of the drugs were not studied on enough patients over 65 to draw even the most preliminary conclusions about safety or efficacy.

 

Similarly, over a quarter of the drugs did not include at least 30 African Americans – some didn’t even include 10! It is impossible to conduct meaningful subgroup analyses with such a small number of patients.

 

And although women were always included when appropriate, 23% of drugs did not include a subgroup analysis to determine if the drug was safe for women.

 

Here’s one egregious example. FDA did not require subgroup analysis of Blacks when the company submitted clinical trials for Singulair for asthma in 2014, despite previous evidence that the drug did not work for Blacks.

 

The FDA should make it clear that the agency will not approve medical products for all populations if meaningful subgroup analysis for safety and effectiveness were not conducted for major demographic groups.

 

We do not believe that the major impediment to diversity in clinical trials is the lack of interest of women, people of color, or patients over 65. Inconvenient locations are a major impediment, however. Companies have not done a better job of recruiting because they know FDA will approve their drugs even when adequate subgroup analyses are not conducted.

 

In the same way that companies recruit the best possible physicians by providing generous incentives to participate in clinical trials, companies should do the same to make participation in clinical trials possible and affordable (and even attractive) for patients of limited financial means or with limited resources.

 

Most important, the FDA needs to make it clear to companies that if they want their drugs approved for women and men, whites and people of color, and adults of all ages (and especially Medicare beneficiaries), they need to study sufficient numbers of those patients in subgroup analyses to make sure the drugs are safe and effective for them in the short-term and long-term.

 

American Medical Women’s Association
Annie Appleseed Project
Breast Cancer Action
Breast Cancer Consortium
Center for Medical Consumers
National Center for Health Research
National Organization for Women Foundation
Our Bodies Ourselves
Washington Advocates for Patient Safety
WoodyMatters

 

 

The Patient, Consumer, and Public Health Coalition is an informal coalition of nonprofit organizations representing the interests of millions of patients, consumers, health-care professionals, scientists, and public health experts. The coalition can be reached through Tracy Rupp at (202) 223-4000 or at tr@center4research.org

Coalition’s Comments on FDA’s Direct-to-Consumer Advertising Survey

April 29, 2016

 

Division of Dockets Management
Food and Drug Administration
5630 Fishers Lane, Room 1061 (HFA-305)
Rockville, Maryland 20852

 

Comments of members of the Patient, Consumer, and Public Health Coalition
on the “National Direct-to-Consumer Advertising Survey
Docket No. FDA-2016-N-0544

As members of the Patient, Consumer, and Public Health Coalition, we appreciate the opportunity to comment on the “National Direct-to-Consumer Advertising Survey.” In general, we support this survey, which will update information from surveys completed in 1999 and 2002.

 

The objective of this research is to gain information from the public about “their experiences with and attitudes toward direct-to-consumer (DTC) advertising of prescription drugs.” 15 Topics covered by the survey include consumer’s knowledge of FDA’s authority over DTC ads, how often they’re exposed to DTC ads, their beliefs and attitudes about DTC ads and the influence of DTC advertising on patient-doctor interactions. The survey will measure other characteristics such as demographics, insurance coverage, and prescription drug use. We want to make sure that the questions about beliefs and attitudes will include gathering information about consumers’ views on how information about the benefits and risks is provided, if it could be provided in a more unbiased way, and how it influences their views of the product being advertised. Ideally, you can provide short DTC videos for respondents to watch and ask them questions about what benefit-risk information they recall from those ads.

 

The new study of DTC ads is designed to reach a wider range of respondents than the previous surveys by weighting the data to make it nationally representative. We agree that the survey should represent the demographic makeup of the United States in sufficient numbers to analyze subgroups based on race, ethnic backgrounds, and sex. The survey should be skewed to include a disproportionate number of Americans over 50, since they are the target of vast majority of DTC ads and are also the most likely to be exposed to those ads on TV and in magazines. Of course, younger adults (18 and over) should also be included. Ideally, the study would include a sufficient number of teenagers who are 14-18 years old, to gather meaningful information about that age group.

 

We agree with the FDA that the DTC advertising landscape has changed since the previous surveys. We are concerned that extremely brief social media ads will not include sufficient information about risks. Since DTC ads focus primarily on selling products, it is unacceptable for the risks of a product to be listed through a link that consumers may not click or the often-used phrase, “for more information, see this month’s issue” of a particular magazine. The survey should directly ask respondents how much information about the risks of the medical products they notice from social media ads.

 

Regarding the survey procedures, we support the mixed-mode methodology where households will be asked to complete a 20-minute online survey with a paper questionnaire sent to those who do not respond on-line. The paper option will ensure that respondents who are not internet savvy will still be included. The survey is designed to have as many as five contacts sent by mail to adults aged 18 and older and this should ensure that the FDA achieves at least its 35 percent response rate for both the pilot study and the main study. These estimated response rate percentages are lower than the telephone surveys conducted in 1999 (65 percent or 960 respondents) and in 2002 (53 percent or 944 respondents) 16 but the total number of respondents (1,765) is estimated to be higher for the new survey.

 

FDA also plans to compare responses between this survey and FDA’s 2002 survey. In 2002, “60 percent [of respondents] felt that ads do not provide enough information about risks.”2 That is a high percentage. We want to know if that percentage has gone up or down, and what steps FDA and industry are taking to provide more information about the risks of medical products. We are particularly interested in the number of people searching the internet for drug and health information, which jumped from 18 percent in 1999 to 38 percent in 2002. Back then, most people were looking for information about risks associated with the medical products.2 The new survey should ask people how often they search the internet regarding medical products and what information they are inquiring about.

 

Conclusions

We are very concerned about the impact of DTC ads on prescriptions, and are especially disappointed at the FDA’s failure to follow through on the agency’s previous proposals to put risk information on a more equal footing with information about benefits. Due to the FDA’s continued acquiescence to companies’ proposed ads, DTC ads use the power of an expensive advertising campaign to persuade patients to use medical products that may not be safe or appropriate for them. Rather than empowering consumers, direct-to-consumer ads expose consumers to the most effective persuasion that money can buy. To truly protect consumers, and reduce unnecessary healthcare costs, Congress and the FDA need to do more than survey the public about DTC ads. They need to propose laws and rules to limit the persuasive power and unbalanced information provided in DTC ads, especially for drugs that have not been tested for long-term safety on a large population.

American Medical Women’s Association
Breast Cancer Action
Connecticut Center for Patient Safety
MAME
MISSD
MRSA Survivors Network
National Center for Health Research
National Organization for Women Foundation
National Physicians Alliance
National Women’s Health Network
Quinolone Vigilance Foundation
The TMJ Association
Washington Advocates for Patient Safety
WoodyMatters

 

The Patient, Consumer, and Public Health Coalition is an informal coalition of nonprofit organizations representing the interests of millions of patients, consumers, health-care professionals, scientists, and public health experts. The coalition can be reached through Paul Brown at (202) 223-4000 or at pb@center4research.org.

Establishing Paid Sick Leave for Federal Contractors

April 12, 2016

Establishing Paid Sick Leave for Federal Contractors

Proposed Rule

RIN 1235–AA13

[Docket No. 2016-03722]

The Patient, Consumer, and Public Health Coalition is an informal coalition of nonprofit organizations representing the interests of patients, consumers, health-care professionals, scientists, and public health experts.   As members of the coalition, we strongly support the proposed rule (Federal Register Docket #2016-03722) based on President Obama’s Executive Order 13706, Establishing Paid Sick Leave for Federal Contractors, which requires that employees that work on federal contracts are entitled to earn at least seven days or 56 hours of paid sick leave per year.

The federal government provides paid sick leave to its employees, but not those who work indirectly for the federal government as contractors. This rule would extend sick leave for an estimated 828,000 employees, including providing paid sick leave for the estimated 437,000 that currently do not receive any.   Currently, these men and women must choose between getting a pay check (and possibly retaining their job) and taking the time off to recover from an illness, get preventative care, or care for a family member.

Paid sick leave improves health and productivity of employees.  It enables those who have physical or mental illnesses to take the time to recover when sick, to get preventative care, to deal with injuries, and to care for sick or injured family members.   At the same time, it reduces the chances that their work colleagues will become sick as a result of their contagious illnesses.  For all those reasons it results in greater productivity in the work place.

Paid sick leave improves community health as well. Employees that cannot afford to take an unpaid sick day unwillingly expose co-workers, clients, and even those they are in contact with on public transportation or other public places to their illness. When employees’ ill children cannot stay home from school, they also expose classmates. Enabling employees to stay home when sick reduces the transmission of illnesses throughout the community.

Providing paid sick leave also helps employers. Besides reducing productivity loss due to the spread of illness through the workforce and “presenteeism” losses1, 2, research shows that paid sick leave increases employee retention and overall productivity3. It also reduces the likelihood of workplace injuries4.

The benefits of paid sick leave for communities are well established and currently 5 states, 22 cities, and one county have passed laws requiring paid sick leave.  A study of six of these jurisdictions found that these laws have not hurt most businesses in the region nor the local economy5.

In conclusion, paid sick day policies like those in the Department of Labor’s proposed rule will save employers, taxpayers and families money, and promote healthier workplaces and communities. We urge you to proceed with implementation without delay.

American Medical Women’s Association
Annie Appleseed Project
Breast Cancer Action
MISSD
MRSA Survivors Network
National Center for Health Research
National Consumers League
National Organization for Women
National Women’s Health Network
Our Bodies Ourselves
Washington Advocates for Patient Safety
WoodyMatters

 

The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202) 223-4000 or pb@center4research.org

1 Kumar, S., Grefenstette, J. J., Galloway, D., Albert, S. M. & Burke, D. S. (2013). Policies to Reduce Influenza in the Workplace: Impact Assessments Using an Agent-Based Model. American Journal of Public Health. 103(8), 1406-1411. www.ncbi.nlm.nih.gov/pmc/articles/PMC3893051

2 Johns, G. (2010) Presenteeism in the workplace: A Review and Research Agenda. Journal of Organizational Behavior. 31, 519-542. onlinelibrary.wiley.com/doi/10.1002/job.630/epdf

3 Hill, H. D. (2013). Paid Sick Leave and Job Stability. Work and Occupations. 40(2), 143-173. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3825168

4 Asfaw, A., Pana-Cryan, R. & Rosa, R. (2012). Paid Sick Leave and Nonfatal Occupational Injuries. American Journal of Public Health, 102(9), e59-e64. www.ncbi.nlm.nih.gov/pmc/articles/PMC3482022

5  National Partnership for Women & Families. (2015, November). Paid Sick Days: Low Cost, High Reward for Workers, Employers and Communities. Retrieved March 2, 2016, from www.nationalpartnership.org/research-library/work-family/psd/paid-sickdays-low-cost-high-reward.pdf