Category Archives: In the News

Free patient booklet on ductal carcinoma in situ (DCIS)

In the News

To view, download, or print a free copy of our patient booklet, here is a PDF of

DCIS: What You Need to Know.

DCIS Booklet

The Cancer Prevention and Treatment Fund has developed a free, easy-to-read 32-page color booklet for women diagnosed with ductal carcinoma in situ (DCIS). DCIS is also known as Stage zero breast cancer. It explains DCIS and commonly used medical terms in plain language and helps women make informed decisions about their treatment.

Experts estimate that at least half of all women diagnosed with DCIS would never develop breast cancer even if they never received any treatment for their DCIS. Since no one knows for sure which women with DCIS will develop breast cancer and which won’t, most women with DCIS choose to get some form of surgery, usually lumpectomy. This booklet focuses on helping women decide what kind of surgery to get and what other kinds of treatment they might want or need.  Patients should keep in mind that if their physician tells them that they have a particularly low-risk type of DCIS, they may want to consider active surveillance or tamoxifen only, rather than surgery in addition to other treatments.

The current booklet was approved and funded by a grant from the D.C. Cancer Consortium through the Department of Health, Government of the District of Columbia, and a grant from the Jacob and Hilda Blaustein Foundation.  To request copies of the free patient booklet, write  info@stopcancerfund.org .

We are currently updating the booklet to include information about active surveillance. 

House overwhelmingly passes bill to speed FDA drug approvals

In the News, Uncategorized
By Carolyn Johnson, The Washington Post
July 10, 2015

 

A bipartisan bill that would make significant changes to the process for developing new drugs and medical devices overwhelmingly passed the House in a 344-77 vote Friday morning.

The bill, called 21st Century Cures, was cheered by rare across-the-aisle support from politicians, with 230 co-sponsors nearly evenly split between Democrats and Republicans. The pharmaceutical industry, patient advocacy groups, and medical organizations also support the bill, which calls for an additional $8.75 billion for the National Institutes of Health.

The bill tries to address the impatience that stems from a major societal problem: despite billions of dollars of research into diseases that range from common cancers to the rarest genetic diseases, we still lack treatments for thousands of conditions. Many of its provisions seek to make the drug approval process less burdensome.

But its laundry list of provisions that tweak the process for approving new drugs or devices have raised significant concern from industry watchdogs and physicians who say the legislation is aimed more at helping drug and device companies than patients. Critics say the bill’s regulatory alterations do not address the real problem with the development of new therapies and could lead to the approval of treatments that don’t work and could even harm vulnerable sick people.

“The bill unfortunately offers a horse trade,” said Vijay Das, a healthcare policy advocate at Public Citizen, a patient advocacy organization. “It increases funding for the world-renowned NIH in exchange for providing perks for the pharmaceutical and medical device industries.” […]

“We share Congress’ desire to increase funding for NIH, but there are dangerous parts of this bill that many members of Congress did not fully understand,” Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank, said in a statement. “As often happens, well-funded pharma lobbying was more effective than experts’ concerns about patient safety.”

Read the full story here.

21st Century Cures drug bill triggers a DC dust-up over relaxed development regs

In the News
By John Carroll, Fierce Biotech
July 10, 2015

 

Lawmakers in the House easily passed the 21st Century Cures Act today, a big step toward once again shaking up the legal framework built to guide drug development in the U.S. while significantly boosting the amount of funding that flows to the NIH.

As for the $9 billion being earmarked for the NIH–which comes along with more funding for the FDA–after years of research budget stagnation, there’s virtually no debate. The bill’s authors have found a way to pay for that by reining in some spending to Medicare prescription drug plans and selling off some of the crude oil in the country’s stockpile, and Democrats are being won over by the notion of increased federal spending for more precise medicines. Research groups in the industry and academia are pushing this hard, and finding open arms in the capitol.

The political trip wire in the bill centers on changes to the way drugs and devices are developed and approved. Among the provisions in the bill, regulators would be given the authority to approve new antibiotics based on preclinical testing combined with small human trials. Biomarkers and other so-called surrogate endpoints could be used more easily to approve new drugs, offering developers a shorter clinical path to developing a drug. And the bill strips away some requirements for reporting physician payments from pharma companies, if they’re associated with education–a very broad categorization. […]

Consumer advocates, though, see the new development regulations as a recipe for unleashing drugs and devices that would later prove dangerous and unhelpful.

“The irony is calling this 21st Century Cures, when they’re talking about standards that were left behind in the 20th century, because they were found to not be good,” Diana Zuckerman, president of the National Center for Health Research, tells the Washington Post.

But there’s also been notable opposition from some well-known experts in the field who aren’t in the least bit happy about a switch in rules that would devalue the current gold standard for clinical development, which relies on randomized, well-controlled drug studies. Harvard’s Jerry Avorn, for example, has a problem with “shorter or smaller clinical trials” for devices as well as new criteria for relying on “evidence from clinical experience,” including “observational studies, registries, and therapeutic use” that could be used to allow for new uses of approved drugs. “Although such data can provide important information about drug utilization and safety once a medication is in use, there is considerable evidence that these approaches are not as rigorous or valid as randomized trials in assessing efficacy,” Avorn writes.

“In our rush to find new effective treatments, we should not harm our patients with ineffective toxic ones,” writes JAMA editor Rita Redberg. That’s the line that marks the boundary between supporters and opponents of the bill.

Read the full story here.

House overwhelmingly passes 21st Century Cures Act

In the News
By Steven Ross, Modern Healthcare
July 10, 2015

 

In a rare act of bipartisanship, House members voted 344-77 on Friday in favor of the 21st Century Cures Act, which supporters say will speed the development and regulatory approval of medical breakthroughs. Critics say that speed would come at the expense of patients’ safety. 

The legislation would provide an additional $9.3 billion in mandatory funding over the next five years to fund the National Institutes of Health and establish a Cures Innovation Fund to support work toward breakthroughs in biomedical research. It also provides $550 million in added funding to the Food and Drug Administration over the same period.

Supporters of the legislation say it will remove regulatory roadblocks in the FDA review process for medications and medical devices. 

It has received strong support from both the Pharmaceutical Research and Manufacturers of America and the Advanced Medical Technology Association. The industry groups argue that the bill would make the review process more efficient and less cumbersome, reducing the costs of bringing a product to market and ultimately lowering the cost of those therapies for patients. […]

The bill also has its critics, who say it would loosen FDA review standards and allow therapies to be sold before enough clinical data is gathered to determine whether they are safe and effective.

“What they’re doing is that they’re replacing the burden of proof for drug companies and device companies with a burden of uninformed decision-making for patients and doctors,” said Diana Zuckerman, president of the National Center for Health Research, a Washington D.C.-based not-for-profit patient-advocacy organization. “You’re replacing the burden of proving that your product works with the burden of having expensive products on the market that may or may not work.”

Read the full story here.

This bill promises to speed up drug approvals so much that it’s making people uncomfortable

News Stories & Editorials
By Carolyn Johnson, The Washington Post
July 8, 2015

The bill slated to land on the House floor on Thursday seems unassailable on its face – the 21st Century Cures legislation promises to modernize medicine and speed the development of lifesaving treatments.

But a vocal chorus of physicians and pharmaceutical industry watchdogs warn that the bill is full of stealth provisions that could actually put sick people in harm’s way, by speeding the development of treatments that are neither safe nor effective.

It’s not exactly easy to oppose a widely-supported bipartisan bill that is often referred to as just “Cures.” But opponents say the proposed law is full of flaws, starting with its name and its key premise: that bottlenecks in the regulatory process are a big reason we haven’t cured cancer, Alzheimer’s, and a panoply of rare diseases.

To drug companies and patient advocates, expedited access to drugs and devices might seem like a huge boon. But critics are worried that the law will relax America’s standards for evaluating new drugs and devices, which are approved based on careful review of evidence — including rigorously designed clinical trials. The bill offers up a slew of new ways to evaluate drugs: for example, allowing antibiotics to be approved based on what would today be considered preliminary evidence — animal and test tube studies and very small trials in people. New medical devices could be approved based on “case histories” — potentially of just a handful of patients.

“The irony is calling this 21st Century Cures, when they’re talking about standards that were left behind in the 20th century, because they were found to not be good,” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit, non-partisan think tank that does not accept money from drug or medical device companies.

Here are a few of the provisions that have sparked debate:

-The bill would allow antibiotics to be approved based on laboratory and animal tests and small, early clinical trials.

-The bill allows companies to seek expedited drug approval based on so-called “surrogate endpoints” — early indicators that a drug is working, such as whether a tumor has stopped growing in cancer.

A study published last month in the journal JAMA Internal Medicine found that efforts to expedite cancer drug approval by using such criteria has resulted in the approval of many cancer drugs that do not extend life, but do have side effects.

-The bill also threatens disclosure requirements that are intended to limit pharmaceutical companies’ influence on physicians. The bill would allow physicians to receive speaking fees and gifts from companies without disclosing them, as long as they were for medical education.

Read full story here.

Startling link between pregnant mother’s exposure to DDT and daughter’s risk of breast cancer

In the News, New Cancer Research, Uncategorized
by Ariana Eunjung Cha, Washington Post
June 17, 2015

Banned by the United States in 1972, the insecticide DDT is best known as the impetus for the modern environmental movement. Since Rachel Carson’s bestseller “Silent Spring” sounded the alarm about the poisonous effects of the chemical on wildlife, the environment and human health, numerous studies have linked it to birth defects, miscarriage and reduced fertility.

Its role in cancer has been less clear. The Environmental Protection Agency classifies DDT as a “probable” carcinogen. Roughly three dozen studies have been published about DDT and breast cancer risk for women who lived during its peak use in the 1950s, but a 2014 meta-analysis of that research found that there was no significant association between exposure and breast cancer risk.

They may have been looking at the wrong generation of women.

A new study published Tuesday in the Journal of Clinical Endocrinology and Metabolism found a startling link between pregnant women exposed to DDT and the breast cancer risk to their daughters.

The study tracked the daughters of women who were part of a study at the Kaiser Foundation Health Plan from 1959 to 1967 near the city of Oakland, Calif. During that time DDT was widely used and accumulated in the fat of animals that we eat and was found in milk, butter, cheese and other products in the food supply. It was also in a number of consumer products, including some wallpaper.

During that period the participants gave birth to 9,300 daughters. Every mother had some measurable level of DDT in her blood. Researchers determined the level of exposure to DDT in utero by analyzing stored blood samples that were taken from the mothers during pregnancy or shortly after they delivered their babies. By using state records and surveying the daughters, who are now in their late 40s and early 50s, they were able to figure out which ones developed breast cancer.

The researchers found that elevated levels of DDT in the mother’s blood were associated with almost a four-fold increase in her daughter’s risk of breast cancer and that this was independent of the mother’s history of breast cancer. They also determined that those with higher levels of exposure were diagnosed with more advanced breast cancer.

About 83 percent of those who got breast cancer had estrogen-receptor positive breast cancer and were more likely to develop HER2-positive breast cancer in which a genetic mutation produces an excess of a protein. In previous studies, DDT has been found to interfere with the function of estrogen and, separately, to activate the HER2 protein, which may explain the link.

Barbara A. Cohn, one of the study’s authors and the director of Child Health and Development Studies at the Public Health Institute in Berkeley, Calif., said the 54-year study is “the first to provide direct evidence that chemical exposures for pregnant women may have lifelong consequences for their daughters’ breast cancer risk.”

Elizabeth Ward, senior vice president of intramural research for the American Cancer Society, said the group of mothers and daughters the researchers are studying is a “unique resource” for studying potential associations between maternal blood levels of chemicals and risk to their children.

“What makes this study interesting is its analysis of in-utero exposure,” she said. However, she said that the number of breast cancer cases was small — 103 — so “the results should be interpreted cautiously.”

In an interview, Cohn said the paper is part of a series of studies on chemicals and their effects on hormones or  development during gestation. Earlier studies she led have looked at the effects of DDT on the time of pregnancy of the daughters of women exposed (they found it could slow their ability to become pregnant or shorten it depending on level of exposure) and on incidence of testicular cancer among the male children (those exposed to the highest levels had an almost three-fold risk  compared with those with lower exposure). She is also studying the effect of other chemicals used for stain control on carpeting and waterproofing for food containers.

“We are looking at a vulnerable period in utero,” she said. “In some ways it is not surprising that early in life is a time when some of these chemicals can have a strong effect.”

Earlier work by Cohn also supports the idea that timing of the exposure matters. In a 2007 paper, she found that DDT affected breast cancer only for women who were exposed before age 14.  The meta-analysis that didn’t find any association between DDT and exposure looked at studies of women who were exposed later in life.

DDT is still widely used in other parts of the world, including regions of Africa and Asia, where it is used to control the spread of malaria.

“Our findings don’t change the perception of benefits, but they do change the perception of risks,” Cohn said. “We are hoping that policymakers will use this information as they continue to debate the use of DDT around the world.”

 

See original article here.

Hope for children’s rare drug approval process

News Stories & Editorials
By Lisa Gillespie, USA Today (Kaiser Health News)
June 7, 2015

Advocates for children with rare diseases are watching closely a congressional effort to streamline the nation’s drug approval process because the bill includes a provision that would extend a federal program that rewards companies making remedies for these young patients.

The reward program, the advocates say, offers hope to families that often have very few options. Approximately 15 million children are diagnosed with rare diseases, and 35% of deaths in the first year of life are caused by them.

“Treatments aren’t getting to kids, and kids deserve more than the leftovers,” said Nancy Goodman, the founder and executive director of the advocacy group Kids v Cancer. Goodman’s 10-year-old son died from brain cancer. She helped push for the original reward program in the hope that children like her son would have access to a wider range of treatments.

The extension of that program is part of the bipartisan 21st Century Cures bill, which seeks to rewrite the rules for drug development to make innovative treatments available faster. The overall bill has generated support on Capitol Hill, but some critics contend that it has the potential to undermine drug safety and to profit drugmakers.

Children’s advocates say there is a shortage of good therapies for rare and often deadly pediatric diseases that can include a wide variety of conditions including cancer, skull deformities or enzyme deficiencies. Pharmaceutical companies have historically been hesitant to test drugs for children because of concerns about potential negative outcomes, children’s ability to consent to treatment and the perception that the market for these drugs was limited. So doctors have often been left to try adult-tested drugs on sick children but without the studies that show pediatric safety or effectiveness. But drugs used on adults don’t always work on children in the same way because of differences in metabolism and maturation of organs.

The advocates say more research needs to be conducted with children. But that testing is a sensitive process. It can be very costly and it requires extra care because there are more stringent ethical protocols to protect these minors, who can’t often give informed consent. Bad results — either injuries or deaths — can set back research efforts and have financial consequences for the company.

With that in mind, Congress in 2011 set up a program to help promote more pediatric drug research. It gives creators of medicine for rare pediatric diseases a voucher that they can use to have another one of their drugs approved quicker than usual — six months vs. a process that can run a year or often more.

Drugmakers can also sell that voucher, which can be a big windfall for a small drug company trying to recoup research and development costs. There have been four vouchers given out since 2014, and one was sold for $67.5 million and a second for $125 million.

The voucher program, which advocates say holds big potential, expires next year. The cures bill seeks to extend it another three years.

“A lot of companies are reluctant to get into pediatric drug development because it’s very difficult if something goes really wrong,” said Alexander Gaffney of the Regulatory Affairs Professionals Society, an association for people involved in overseeing health care or the quality of health care products.

Drugs that are approved for cancer in adults are commonly not approved in kids. “Children are not simply small adults, they metabolize drugs very differently,” Gaffney said.

Critics say that however well-intentioned the voucher program is, it could have some unintended consequences. For example, a company could get a drug approved by theFood and Drug Administration but never bring it to market, if the maker decides it would not generate enough money. Yet the company would still pocket the priority review voucher.

Because of the speed sought by the program, vouchers could be given out without some of the safeguards that come in more traditional testing. For example, the research might not uncover that the drug could be fatal to a child after a few months or years.

Diana Zuckerman, president of the National Center for Health Research, a non-profit group that seeks to represent children and families on health research policy issues, says the rush in moving drugs through the system can obscure problems. Drugmakers “shouldn’t be able to sell it [or use it] unless it works,” she said.

She noted that in some studies as few as 10 kids are included because the disease is so rare. With such a small population size, the company is not likely looking at big profits.

“When you’re doing a study of rare disease, it’s a small sample size and it’s easy to manipulate the data to make it look better than it is,” said. “You don’t want an incentive to represent the company wrongly in the short term,” to get the voucher for another larger drug.

Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, an advocacy group pushing for drug companies to spend more on drug development, wants the pediatric drug voucher program extended. She notes that the program is still too new for officials to evaluate whether it is effective.

One problematic part of the current House version, she said, is that it only extends the program for three years, and drug companies generally need 10 years to scratch up investors and research a new drug. The potential reward of expedited drug review might not be enough to allow a company to make a financial plan for a drug based on the program.

The expanded bill covers more than 60 health issues, including a $10 billion boost in funding for the National Institutes of Health and $550 million in extra money for the FDA over the next five years. Other provisions include creating a database of genomic information from a million U.S. patient volunteers and allowing the FDA to approve drugs without the gold-standard clinical trial, instead using smaller observational studies or clinical experiences.

The bill passed the House Energy and Commerce Committee unanimously in May and is expected to come up for a vote in the full House. Senators are in the early stages of working on a similar bill.

See original article here.

Speeding up drug-approval process could have downside

In the News, News Stories & Editorials
By Ed Silverman

Excerpted from The Wall Street Journal, May 30, 2015.

Would a congressional bill designed to jump-start medical innovation end up lowering standards for approving new uses of existing medicines?

Consumer advocates are raising this concern about the 21st Century Cures legislation, which passed the House Energy and Commerce Committee unanimously last week and, in part, is designed to reform the approval process for drugs. Supporters say the bill is a long overdue move that, among other things, will give the FDA the tools to ensure treatments reach patients faster.

But critics say that a section of the bill devoted to drug development is problematic. Specifically, they point to language that would allow the FDA to approve additional uses for drugs without having to rely on randomized controlled trials. These are considered to be the gold standard for determining whether a medicine offers a benefit, and they help gauge the extent to which there are risky side effects.

The bill, however, pushes aside evidence in favor of something called “clinical experience,” which is defined as a mix of observational studies, patient registries and therapeutic use. None of these, however, are viewed as scientifically rigorous for establishing whether a drug may be effective. Instead, critics say the language in the bill is sufficiently, perhaps deliberately, vague.

“Clinical experience is something that should be considered as additional information, but absolutely never take the place of scientific data,” says Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. “By urging FDA to get away from randomized clinical trials, drug makers may have more power to urge the FDA to consider data that is favorable to their product.”

To read the entire article, including similar concerns expressed by Dr. Steven Goodman from Stanford, see http://www.wsj.com/articles/speeding-up-drug-approval-process-could-have-downside-1432857506

Bill aims to expand drug indications minus randomized trials

In the News
By Robert Lowes,  Medscape
May 08, 2015

Can government regulators speed up the introduction of new treatments in hospitals and physician offices without sacrificing safety and effectiveness? Or does the expression “speed kills” hold true?

That’s the debate surrounding a bill in the Republican-controlled House called 21st Century Cures, which aims to fast-track the development and approval of new drugs and devices. Ironically, the legislative process has been slow-going as lawmakers strike compromises to defuse controversy.

The latest draft of the bill, released last week, won kudos from Democrats and Republicans alike for significantly increasing the budget of the National Institutes of Health (NIH), which funds basic research leading to new treatments. However, some experts still worry that the measure, not yet officially introduced as legislation, lowers the scientific bar for these new treatments to come to market — good for manufacturers, but not so good for patients.

One provision, for example, requires the US Food and Drug Administration (FDA) to evaluate the use of evidence from “clinical experience” — and not just from randomized clinical trials — for assessing a drug’s effectiveness and safety in two regulatory scenarios. One is to “help support the approval” of a new indication for a drug already on the market. The other is helping to support or satisfy postapproval studies that the FDA requires when it has its doubts about a drug.

Evidence from clinical experience, as described by the bill, includes data from observational trials, clinical registries, insurance claims, ongoing safety surveillance, and “patient-centered outcomes research activities.”

Diana Zuckerman, PhD, president of the nonprofit National Center for Health Research (NCHR), a think-tank that focuses on women, children, and families, looks askance at substituting clinical experience for randomized clinical trials. She told Medscape Medical News that the bill “would promote the snake oils and elixirs of the early 20th century.”

“I call it eminence-based medicine — if some hotshot doctor says it works,” Dr Zuckerman said.

The push to consider clinical experience for expanding a drug’s indications, she said, is based on the false assumption that once the FDA deems a drug safe for an initial indication, “it can be used for everything else.” That assumption forgets that the FDA weighs benefits versus risks. The agency, for example, might approve a very toxic drug for a certain lung cancer because nothing else would save a patient’s life. However, the FDA may find the drug’s toxicity unacceptable when it comes to treating early-stage breast cancer, a condition for which there are many alternative treatments, and a far higher survival rate.

“Safety is very, very relative,” said Dr Zuckerman.

21st Century Cures also has come in for harsh criticism from the consumer watchdog group Public Citizen, which said that faster product approval based on weaker evidence will sacrifice safety, and Modern Healthcare magazine, which called the measure the “21st Century Quackery Act.” Yet another critic is Margaret Hamburg, MD, who stepped down as FDA commissioner earlier this year. Addressing the National Press Club in March, Dr Hamburg warned against relaxing the agency’s standards. “Innovation doesn’t matter if the product doesn’t work,” said Dr Hamburg, an appointee of President Barack Obama.

However, Politico Pro quotes another former FDA commissioner as saying that, unlike Dr Hamburg, he views the measure as a “positive step.”

“I don’t share the concern that inherently this means you are going to have to lower standards of safety and efficacy,” said Andrew von Eschenbach, MD, Wednesday, at a media briefing on 21st Century Cures organized by the Alliance for Health Reform. Dr von Eschenbach headed the FDA from 2006 to 2009 under President George W. Bush.

“The FDA’s Not Going to Do Anything That’s Inappropriate”

In an interview with Medscape Medical News, a high-ranking FDA official said that the agency already has begun to approve drugs based on clinical evidence short of randomized trials, and that it is proceeding cautiously.

“The FDA’s not going to do anything that’s inappropriate,” said Janet Woodcock, MD, director of FDA’s Center for Drug Evaluation and Research (CDER).

The FDA has sometimes relied on single-arm trials and case series to establish efficacy for new drug approvals or expanded indications, said Dr Woodcock. This approach holds promise, she said, for approving new uses of targeted cancer drugs already on the market, especially for rare tumor types in patients who don’t have other good treatment options.

At the same time, the FDA recognizes the limits of clinical evidence, including “pure observational data” gleaned from its own nascent Sentinel System, which searches the electronic health records of some 178 million patients for adverse events.

“It isn’t reliable enough [yet],” said Dr Woodcock. “We don’t even make definitive safety findings from that. We use it to bolster our safety assessments.”

So the FDA is still learning how to harness such clinical evidence. The future, she said, will “bring clinical research much closer together with healthcare.”

Less Industry Friendly?

21st Century Cures has been in the making since May 2014, when the initiative was unveiled by Rep. Fred Upton (R-MI), chair of the House Energy and Commerce Committee, and committee member Rep. Diana DeGette (D-CO).

In January 2015, the committee released a first draft of the legislation, which was criticized as catering to industry. Some of the most controversial provisions would have extended market exclusivity for various categories of drugs. The NCHR’s Dr Zuckerman credits lawmakers with removing this language from the latest draft.

Also missing, said Dr Zuckerman, are “micromanaging” provisions on biomarkers, those organic fingerprints such as RNA, proteins, and metabolites that can predict a patient’s response to a drug — one of the keys to personalized medicine. Under the first draft, she said, drug makers could have essentially pressured the FDA to adopt biomarkers of their choosing to expedite clinical trials. The draft legislation released last week merely calls on the agency to develop guidelines for developing biomarkers, a gentler directive for an agency already incorporating these measures into its work.

CDER’s Dr Woodcock told the health subcommittee of the House Energy and Commerce Committee on April 30 that biomarkers must pass the evidence test before the FDA accepts them as proxies for health outcomes.

“You have to know those biomarkers are reliable before you risk them on human life,” said Dr Woodcock.

Bill Exempts Some CME Activities From Sunshine Reporting
The latest version of 21st Century Cures covers a waterfront of issues. Among other things, the measure would:

Require the FDA to establish a priority review program for “breakthrough” medical devices “for which no approved alternatives exist.”

Establish that the FDA can consider registry data, peer-reviewed studies, and data collected outside the United States in evaluating medical devices. Like the proposed acceptance of clinical evidence other than randomized clinical trials, this provision has been criticized as a license for weaker scientific evidence.

  • Require the Centers for Disease Control and Prevention to set up a surveillance system for neurological diseases.
  • Require the FDA to incorporate patient-experience data in its regulatory decisions, a path the FDA is already headed down. “Patients are experts in their disease,” Dr Woodcock told lawmakers last week.
  • Allow NIH to require any researchers it supports to share their data.
  • Create an information system for scientists to use and analyze data from NIH-funded trials.
  • Create a fast-track approval process in the FDA for antibiotics in short supply.
  • Encourage the application of Bayesian statistical modeling in drug makers’ clinical protocols and new drug applications.
  • Exempts some continuing medical education activities of physicians from the controversial Sunshine Act. Drug and device manufacturers wouldn’t have to report giving physicians a medical textbook, tuition to attend an educational event, or a fee to speak at such an event.
  • Allow drug makers to share economic data concerning their products with third-party payers and their formulary committees.

The measure seeks to make electronic health record systems interoperable so that physicians can easily share research and clinical data, but lawmakers have not yet come up with specifics. Details also are pending for a provision seeking to make telemedicine more available to physicians and patients, and to repurpose drugs for serious and life-threatening diseases.

The general thrust of all these measures is to accelerate the development, testing, and market debut of new medical treatments. Bill supporters view the FDA approval process as antiquated and too slow, but Dr Woodcock noted in her House subcommittee testimony last week that her agency has been quicker on its feet lately. Last year, she said, the FDA okayed the highest number of new drugs in almost 2 decades, and the most new drugs for “orphan” diseases since Congress passed the Orphan Drug Act in 1983.

She also suggested that the FDA’s depiction as a bottleneck for innovation is exaggerated. The agency, she said, consistently reviews new drugs faster than its counterparts around the world.

“We don’t take a long time to get things approved,” said Dr Woodcock. “They take a long time to get developed.”

Bigger Budget for NIH, but Not for FDA

Appropriations for NIH shrank for 3 consecutive years beginning in 2011, reflecting across-the-board federal budget cuts that grew out of Congressional gridlock. The latest version of 21st Century Cures would significantly boost NIH funding, making the measure more palatable to Congressional Democrats and delighting the healthcare industry.

“The increased funding for the NIH in the 21st Century Cures draft is an important acknowledgement of the need for robust research,” said Kim Williams, Sr, MD, president of the American College of Cardiology, in a news release.

The draft legislation would lift NIH’s budget line to $31.8 billion in fiscal 2016, up from $30.3 billion in 2015. Increases of $1.5 billion would follow in both 2017 and 2018. The measure would allocate an additional $2 billion a year over 5 years for an NIH Innovation Fund to support, among other things, emerging young scientists and research in precision medicine.

At last week’s hearing of the health subcommittee of the House Energy and Commerce Committees, several lawmakers noted that the measure did not give the FDA more money even though it gave the agency more work to do.

Dr Woodcock acknowledged that the FDA is already “very stretched” and that giving its staff additional responsibilities could slow down its drug review program, which “is going full speed.”

“We have a saying in medicine — first, do no harm,” she said. “In enacting new legislation, you don’t break what’s fixed.”

Jeffrey Shuren, MD, director of the FDA’s Center for Devices and Radiological Health, added that more duties for the same number of FDA employees worsens turnover, as does noncompetitive salaries. “Were essentially a training ground for industry,” said Dr Shuren.

The 21st Century Cures draft needs additional tinkering to address this money problem, Rep. DeGette told Dr Shuren and Dr Woodcock.

“We still need to find the funds for the FDA to do the things you have to do,” DeGette said.

See original article here. 

Bill to speed approvals for drugs is cut back

In the News, News Stories & Editorials
By Sabrina Tavernise,  New York Times
April 30, 2015

WASHINGTON — Legislation that would have accelerated the pace of federal drug approvals in a way that critics said threatened to erode patient safety was formally released this week, in a scaled-back version with many of the most controversial provisions left out.

The  draft bill  presented at a  hearing  in the House on Thursday represents a less aggressive streamlining of the drug approval process, critics of the earlier draft said, and seems to have secured strong bipartisan support.

The legislation, called 21st Century Cures, has been in the works for months. Its lead sponsor, Fred Upton, Republican of Michigan, said it would speed the pace of drug cures by removing unnecessary hurdles from the regulatory process.

Critics, including top officials at the  Food and Drug Administration,    had expressed concern that the changes would risk patient safety — for example, by potentially permitting shorter clinical trials and letting drug companies use alternative measures of health as evidence of a drug’s effectiveness and safety.

The bill’s supporters said it was a work in progress that had been assembled in one of the most collaborative, transparent processes Congress has seen in years. The sponsors held eight hearings and more than a dozen round-table meetings in districts across the country to gather comment.

“We have a chance to do something big, and this is our time,” Mr. Upton said.

His co-sponsor, Diana DeGette, Democrat of Colorado, said the lawmakers had made “tremendous progress,” recalling “that hokey video” that she and Mr. Upton made to promote the effort last year.

The draft — at about 200 pages it is half its former size — seemed to allay fears among some experts that it would fundamentally rewire the way drugs are approved. Earlier proposals to give drug companies broad powers to promote their products for uses other than the approved ones and to market brand-name drugs for a longer time without generic competition were not included.

The F.D.A. has defended its record for drug approval speed, saying that in 2014, it approved the most new drugs in almost 20 years, and that it moves faster than its counterparts in other wealthy countries. Experts note that pathways exist for expedited approval, and that the F.D.A. is held to relatively strict timelines under other rules set by Congress. Instead, they say, the bottleneck for new drugs is often a matter of the years of research it takes to find them.

Diana Zuckerman, the president of the National Center for Health Research, said earlier versions of the legislation had “considerable micromanaging of the F.D.A. and enormous power given to industry.”

She added, “Most of that is gone.”

But she cautioned that the bill still held numerous provisions she believed put people at risk, including ones she described as lowering standards for the approval of medical devices and antibiotics. The bill would increase the work the F.D.A. must perform, but not the money it receives, and Dr. Janet Woodcock, an agency official who testified Thursday, said that was a concern.

“We are very stretched in our resources,” she said.

One point that is likely to concern the F.D.A. is a provision that would reduce its authority to regulate some software associated with medical devices.

An earlier version of the bill would have let drugmakers use quicker measures for a drug’s effectiveness during testing — for example, changes in blood sugar level instead of a more final outcome, like development of  diabetes. The new draft instead suggests ways the F.D.A. could use those alternative measures, called biomarkers.

The F.D.A. says it already uses them in a substantial share of drug approvals, and Dr. Woodcock told the lawmakers Thursday that the agency did not need additional authority on this count. Progress has been slow in adopting more biomarkers, she said, because they often did not produce enough evidence to allow scientists to draw firm conclusions.

“You have to know those biomarkers are reliable before you can take a chance on a human life,” she said.

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