Congress Shouldn’t Pass The 21st Century Cures Act In A Summer Rush

By Paul Brown, Tracy Rupp, and Steven Findlay, Health Affairs

July 11, 2016

The full Senate may in the next few days consider companion legislation to the 21st Century Cures Act that passed the House last year. The legislation—currently 19 separate bills—makes substantial changes to the way the Food and Drug Administration (FDA) approves drugs and devices. Set to adjourn for an extended election-year summer recess on July 15, the clock is ticking. The congressional calendar in the fall is full and the Senate may simply not have the time to take up the complex legislation, and reconcile it with the House version, before the November elections.

We believe that’s a good thing. The legislation, while containing some useful measures, is flawed. Instead of hastily agreeing to it, Congress should postpone consideration until 2017 and attach the best of the 19 bills (see below) to must-pass legislation on FDA funding through industry user fees.

At that time, we believe the Senate should reject those bills that lower standards for drug and device approvals and safety, and risk adding to the rising cost of prescription drugs.

This is a story two years in the making. Proponents of the proposed legislation—drug and device companies, and members of both parties in the House and Senate—argue that the FDA stifles innovation and advances in treatment by approving drugs and devices too slowly compared to other countries.

That premise is faulty. Nearly two-thirds of the novel drugs approved in 2015, for example—29 of 45, 64 percent — were approved in the United States before being approved in any other country. The proportion was even higher in 2012 and 2013. The majority of these drugs (60 percent) took advantage of existing FDA expedited review programs—fast track, breakthrough, priority review, and accelerated approval—and nearly half (47 percent) were approved to treat rare or orphan diseases.

As for devices, research shows 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.

The House and Senate bills ignore the above facts. They essentially seek to speed-up the approval process by relaxing FDA’s safety and effectiveness standards. And to make that more palatable, sponsors have attached the changes to increases in funding for the National Institutes of Health and the FDA.

But while the public supports increases in biomedical research funding, it is deeply skeptical about lowering the standards for drug and device approval. In the most recent poll on this issue, just under 60 percent of Americans opposed changing federal regulations to speed the development and approval of drugs; 38 percent favored speedier FDA action, according to a STAT-Harvard poll released in May. Respondents’ main concern: faster approval would allow products on the market that don’t work or are unsafe.

The survey echoed a previous poll by Consumers Union that found 82 percent of Americans believe that preventing safety problems is more important than limiting safety testing to speed the clearance or approval of devices or promote innovation.

The 21st Century Cures Act received broad bipartisan support in the House in part because it put a significant amount of money for research on the table. The House bill pledges a $9 billion increase in mandatory funding for the National Institutes of Health over five years, gaining the support of universities and medical schools. It also promises $550 million to the FDA (which, according to the CBO’s analysis, would not fully pay for the additional workload the Act assigns the FDA).

The drug and device industries intensely lobbied House members to pass the legislation. The Pharmaceutical Research and Manufacturing Association (PhRMA) increased its lobbying from $4 million to $5.4 million in the quarter before the 21st Century Cures Act passed. The Advanced Medical Technology Association upped its spending from $550,000 to $740,000 in the same quarter. The Senate’s lobbying database listed more than 1,100 lobbyists working on the legislation. Many patient groups have also voiced their support for the legislation, based on their belief that the bill would result in more treatment options. An analysis of public comments for the House bill completed by Avalere noted that 43 percent were from patient advocacy groups.

The Senate bills are an improvement over the House’s 21st Century Cures Act. But, on balance, they are still weighted heavily in favor of speeding medical products to market by weakening FDA approval standards.

New drugs and devices can, of course, be an improvement over existing products. And when they clearly are, FDA has established pathways to get them to market and patients as fast as possible. The agency, for example, grants more than one-third of requests from industry for “breakthrough” designation for new drugs. But the history of medicine is replete with examples of drugs and devices that caused more harm than good, some of which were approved too hastily — such as Avastin for breast cancer, Vioxx for arthritis, metal-on-metal hip implants, and power morcellators. Innovative drugs and devices simply must be required to actually work and not harm patients. Methods of testing drugs and devices in patients are improving all the time, with new tools that help detect products that warrant further testing or could be dangerous. The default mode of this technical process must always be “safety first.”

Below are our concerns with some of the Senate bills followed by a list of bills we believe would improve public health.

Senate bills that would, in our opinion, increase risks to patients:

  • The MEDTECH Act would prevent the FDA from collecting adverse events due to flawed electronic medical records, and from recalling certain types of defective medical software. Some of this software has had life-threatening flaws in the past, such as oncology electronic medical record systems that calculated and recorded incorrect drug dosages for highly toxic chemotherapy drugs.
  • The PATH Act would allow antibiotics to be approved with minimal evidence of safety and effectiveness through a “limited population” approval pathway. But, antibiotics approved in this way are promoted by companies so that they are more widely prescribed in order to increase sales. As they are, we won’t have information about whether they’re actually safe or effective for those groups of patients. For example, fluoroquinolones are today widely overprescribed for urinary tract infections despite guidelines recommending other antibiotics and data linking their overuse to the development of resistance.
  • The Advancing Breakthrough Devices for Patients Act would encourage shorter and smaller clinical trials for medical devices. Abbreviated clinical trials will make it difficult, if not impossible, to include sufficient participation from subpopulations such as women, seniors, and racial and ethnic minorities in the analysis of the trials. For example, a recent study of high-risk medical devices found that the median number of participants was only 65 patients. With so few patients, it’s difficult to draw reliable conclusions about safety and effectiveness, especially for subgroups of patients. In an increasingly diverse America, this is unacceptable. The bill also pushes FDA to rely on post-market studies for devices rather than ensuring safety effectiveness before hospitals and patients pay for the products.
  • The Advancing Hope Act would continue the existing pediatric priority review voucher program through 2022. The program is currently set to expire at the end of September. The program’s ability to stimulate innovation is questionable: a recent GAO review of the program concluded that the six drugs for which vouchers have been awarded so far were in development before the program existed. By allowing drug makers to buy a priority review, the bill removes FDA’s ability to set its work priorities and resource allocations based on public health needs. In a time of threats such as the Zika virus, our government agencies must be able to prioritize public health, and not be bound by vouchers that were sold to the highest corporate bidder.

Senate bills likely to promote innovation and protect public health:

  • The Preventing Superbugs and Protecting Patients Act will help prevent drug-resistant infections from contaminated duodenoscopes and other reusable medical devices that have caused harm and deaths. If enacted, this bill will require certain reusable medical devices to have validated cleaning, disinfection, and sterilization procedures prior to marketing. This will reduce the likelihood that these devices will transmit dangerous infections from patient to patient.
  • The Advancing Research for Neurological Diseases Act will help people suffering from neurological diseases by providing critical information to researchers as they work on new treatments and cures. The bill requires the Department of Health and Human Services to collect neurological disease surveillance data, such as demographic information, risk factors, outcomes, and treatments. Providing such information is likely to enhance understanding of these diseases and the development of new treatments.
  • The Next Generation Researchers Act recognizes the importance of investing in the brightest young researchers to ensure that the United States remains at the forefront of biomedical research.
  • The FDA and NIH Workforce Authorities Modernization Act will make it easier for FDA to recruit and retain top scientific and technical experts by making salaries more competitive with those offered by industry. It will also lead to the development of standards for regenerative medicine — such as replacing, engineering, or regenerating human cells, tissues, or organs to restore or establish normal function.

Lawmakers are also considering adding the REGROW Act to the Senate’s package of bills. The bill would allow complex regenerative medicine therapies to be conditionally approved based on preliminary evidence. Since at least half of all drugs fail in the last stage of testing, many patients could end up receiving therapies that are later found to be unsafe or ineffective.

Looking Ahead

On Saturday June 25, six former FDA commissioners from Democratic and Republican administrations suggested at the Aspen Ideas Festival that Congress make the agency independent of the Department of Health and Human Services — similar to the Securities Exchange Commission, for example. With regulatory purview over products that represent a quarter of the U.S. economy, the group said the FDA is harmed by an unstable federal budget process and persistent political meddling. The group said they would issue a white paper on their proposal for the next administration. That’s another reason why Congress should postpone consideration of these bills until 2017.

To see original article, click here.

Faster Drug Approval: Winners and Losers

By Kristen Fischer, HealthZette

July 1, 2016

[…] Recently, due to calls for novel treatments that move faster from the (lab) bench to the bedside, a legislative effort is attempting to speed up that process. The 21st Century Cures Act would allow companies to submit evidence of safety based on patient stories instead of actual clinical trials.

The House already passed its own version; the Senate is expected to review it in the coming weeks, STAT reported.

While the measure could speed up the process, some people fear it would also remove a lot of quality control. And when it comes to health, it’s good to know that the medications you take have been tested extensively and are backed by reliable data.

Another concern is any new fast-track process would enable pharmaceutical companies, for the most part, to dictate the efficacy of a drug instead of the FDA as a third party.

Patience for Patients

[…] Dr. David Gortler, a former FDA senior medical officer and current drug safety and FDA policy expert at, told LifeZette he doesn’t agree with speeding up the process because lawmakers typically don’t have an extensive pharmacology background. Circumventing the time-tested FDA process will not help Americans.

“They [lawmakers trying to speed up the process] don’t trust these people who have dedicated their lives to pharmacology and medicine,” Gortler said.

People think they know better, but they are really just putting their lives at risk, he added. “They don’t trust doctors or pharmacists. They trust GNC.”

In a Washington Post editorial, Susan F. Wood, an associate professor at George Washington University’s Milken Institute School of Public Health and a former FDA assistant commissioner, explained that the 21st Century Cures Act is based on an assumption that there will be more cures if drugs and devices are studied more quickly on fewer patients.

She, along with Diana Zuckerman, president of the National Center for Health Research, believes the studies would be too small to allow safety and effectiveness findings to be broken down into subgroups such as male and female.

“This embrace of smaller, more preliminary studies could drastically lower scientific standards. When fewer people are studied, it is more likely that a drug will seem safe and effective even if it has dangerous side effects for many patients — who may not have been included in those small studies,” they wrote.


To see original article, click here.

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.

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.

FDA to shift clinical evidence for medical devices toward postmarket

By AAMI, Medical Design Technology

April 4, 2016

Advances in technology and data collection can help regulators and other parties keep better track of the safety and performance of medical devices once they are on the market, opening the door to potentially faster product development. […]

This focus on faster, less expensive product development coincides with a move by the U.S. Senate to create a “breakthrough pathway” for FDA approval of medical devices. The Advancing Breakthrough Devices for Patients Act of 2015 would allow shorter or smaller clinical studies and quicker measures of success to serve as sufficient premarket evidence for the approval of devices with the potential to “reduce or eliminate the need for hospitalization, improve patient quality of life, facilitate patients’ ability to manage their own care (such as through self-directed personal assistance), or establish long-term clinical efficiencies.”

Following approval by a bipartisan Senate committee, some medical-device safety experts have expressed concern that the bill “sets a low bar to qualify for ‘breakthrough’ status’” and “lowers standards for safety and effectiveness,” as articulated by Diana Zuckerman, president of the National Center for Health Research (NCHR) in Washington, a medical research and advocacy group, in The Wall Street Journal.

“We are concerned that the focus of these bills is on getting medical products to market more quickly, instead of making sure that they are safe and effective,” NCHR and 13 other medical safety groups wrote in a letter to the members of the Senate Committee on Health, Education, Labor, and Pensions Committee. […]

The FDA’s goal is to gain access to 25 million electronic patient records from national and international clinical registries, claims data, and EHRs by the end of 2016. It is also aiming to increase the number of pre- and postmarket decisions that leverage real-world evidence by 40% during that same timeframe.

To see original article, click here

US device industry and FDA “colluded” on legislation to weaken regulatory oversight


DECEMBER 17, 2015


US Food and Drug Administration officials had multiple meetings with leaders of the medical device industry to craft legislation that critics say will severely weaken regulatory oversight of the industry, an investigation by the online news service Inside Health Policy has found.

The revelations, discovered in emails and documents obtained under the Freedom of Information Act, have led to renewed calls by professional and public interest watchdog groups to defeat companion legislation to the proposed 21st Century Cures Act, which has been referred to the Senate. They have also called to oppose the approval of Robert Califf as a nominee for the role of FDA commissioner because he took part in meetings with the Advanced Medical Technology Association (AdvaMed), a trade association for medical technology companies.


Michael Carome, director of the health research group at Public Citizen, a public interest organization, described as “unseemly and inappropriate” the meetings between the FDA and the device industry to craft the language in the act.

Carome said that Califf’s “participation in this collusion with industry” should, at a minimum, put Califf’s nomination as FDA commissioner on hold pending an investigation. Carome said, “The attitudes [Califf] has developed over his decades long history of extensive financial ties to pharmaceutical and medical device companies leave him all too willing to promote the interests of regulated industries over those of public health and patient safety.”

The National Physicians Alliance, together with Public Citizen and six other organizations, wrote a letter to the House of Representatives on 19 May, stating that the 21st Century Cures Act “fails to ensure a . . . scientifically based approach” to drug and device approval and that it will allow “unsafe and ineffective drugs and medical devices to enter the market.”


The FDA defended its meetings with the industry, telling The BMJ that “FDA officials routinely meet with a diverse group of stakeholders.” The agency said that it had met with 12 representatives of public interest and professional organizations who attended a meeting on 28 October, after the bill was referred to the Senate in July.

Diana Zuckerman, president of the National Center for Health Research, whose organization requested the October meeting, told The BMJ that none of the more than two dozen non-profit organizations that are members of the Patient, Consumer, and Public Health Coalition had been invited by the FDA to help develop any provisions of the 21st Century Cures Act or its Senate companion bill.

She said, “There’s a world of difference between talking about approval standards in general and crafting specific legislative language. It is outrageous that FDA officials and regulated industry are sitting down to craft legislative language to give to congressional staff.”


BMJ 2015; 351 Cite this as: BMJ 2015;351:h6820

To read the full article, click here.

The 21st Century Cures Act could be a harmful step backward

Susan F. Wood and Diana Zuckerman, The Washington Post
November 19, 2015

Precision medicine is the next big thing in health care, and it’s also one of the few health goals that Congress and the White House agree on. But while we await treatments targeting the precise genetic makeups of individuals and diseases, medical researchers still are not paying enough attention to the most important kinds of differences among patients: those of sex, age and race.

A clear example of this disconnect is the 21st Century Cures Act, which was passed overwhelmingly by the House of Representatives and is being scrutinized by the Senate. The stated goal of the bill is wonderful: to stimulate the development of new cures for a range of diseases. Many medical schools and patient organizations are supporters, since the proposal would provide almost $9 billion more for the National Institutes of Health — including a boost for precision medicine. The 360-page bill offers other potential benefits as well. But an immediate impact would be to ignore how differences between men and women and younger and older patients influence the safety and effectiveness of many medical products.

Throughout the 20th century, most medical research was conducted on relatively young, healthy men. In recent years, researchers have realized that treatments often affect women and older patients differently than men or younger patients. These differences can affect safety and effectiveness. The sleeping pill Ambien, for example, makes women drowsier for longer periods than it does men, putting them at risk if they drive the next morning. Since most medications are taken by people older than 65 and women of all ages, it makes sense to analyze the effects of age and sex on the drugs’ safety and effectiveness before they can be sold.

But the 21st Century Cures Act is based on the assumption that there will be more cures if drugs and devices are studied more quickly by testing them on fewer patients — in some cases, on just a handful. Unfortunately, such studies would be too small to allow safety and effectiveness findings to be broken down for subgroups such as men, women, young adults and seniors.

This embrace of smaller, more preliminary studies could drastically lower scientific standards. When fewer people are studied, it is more likely that a drug will seem safe and effective even if it has dangerous side effects for many patients — who may not have been included in those small studies.

In addition to allowing smaller studies, the House bill would encourage the Food and Drug Administration to determine a drug or device’s effectiveness based on “clinical experience,” which the bill defines to include the experience of one or more doctors or patients. Scientists call these anecdotes and note that just because one doctor has had success treating a few patients with a particular drug does not prove it is either safe or effective. Worse, the bill specifies that after studying only small groups of patients, drug manufacturers could sell a new treatment to anyone, even if the patient was not among the types studied. In fact, hospitals would be paid extra to make it financially feasible to prescribe more expensive new drugs to Medicare patients, even if the drugs were never studied on patients older than 65 (the age of most Medicare patients).

Similarly, lifesaving medical devices, such as heart valves, could be approved based on case histories, which are written descriptions of the experiences of just one or two patients. They are unlikely to be good predictors of how a treatment helps or harms most patients.

The recalls of drugs such as Vioxx and devices such as metal-on-metal hips in recent years have made clear that inadequate testing can produce ineffective and harmful products. And since new drugs tend to be much more expensive than older ones, the costs of widely used, unproven medical products can be enormous in both human and economic terms.

These sections in the 21st Century Cures Act go in the opposite direction of the push for precision medicine and what we’ve learned about differences between male and female and older and younger patients. The General Accountability Office has concluded that the NIH needs to make a priority of analyzing data related to sex differences. Just three years ago, the House and Senate overwhelmingly passed legislation that directed the FDA to ensure that men and women, old and young, are studied, with results analyzed to see which treatments are safest and most effective for whom. Why is Congress undermining that law?

Congress should surely increase funding for research to find 21st-century cures, but the price should not be returning to early-20th-century standards, when unproven medical products were widely available and often put all Americans at risk.

See the article here.

Speeding Ineffective and Unsafe Treatments to Market in the Name of Patients? Not So Fast

By Karuna Jaggar (Breast Cancer Action), The Huffington Post

July 13, 2015


As the head of Breast Cancer Action, a national watchdog organization for the breast cancer movement, I frequently hear from long-time members who are outraged that breast cancer treatments remain largely the same as they were decades ago. Activists and doctors alike worry that the basic treatments for breast cancer continues to be “slash, burn, poison”–more commonly referred to in medical offices as surgery, radiation, chemotherapy–with too few advances benefiting too few women. Too many women continue to be diagnosed with and die from breast cancer.

Why, then, would Breast Cancer Action and other watchdog groups oppose a bill currently speeding through Congress that some say will help get new medical products and treatments into the hands of patients faster?

Because, quick Food and Drug Administration (FDA) approvals don’t help patients if the treatment or technology doesn’t work in the first place–or worse if it is harmful! […]

Read the full story here.