Joint replacements are the #1 expenditure of Medicare. The process of approving these medical devices is flawed according to the Institute of Medicine. It is time for patients' voices to be heard as stakeholders and for public support for increased medical device industry accountability and heightened protections for patients. Post-market registry. Product warranty. Patient/consumer stakeholder equity. Rescind industry pre-emptions/entitlements.
Sunday, June 7, 2015
Saturday, June 6, 2015
Jerry Avorn, M.D., and Aaron S. Kesselheim, M.D., J.D., M.P.H.
June 3, 2015 (FiDA highlight)
In May 2015, the 21st Century Cures Act was introduced in the U.S. House of Representatives, with the goal of promoting the development and speeding the approval of new drugs and devices.1 Championed by the pharmaceutical, biotechnology, and device industries, the bill was approved unanimously (51 to 0) in committee and continues to be debated. If enacted into law, some of its provisions could have a profound effect on what is known about the safety and efficacy of medical products, as well as which ones become available for use.
Some aspects of the bill could indeed enhance the development of and access to new drugs. The legislation calls for annual increases in the stagnating budget for the National Institutes of Health (NIH) amounting to about 3% per year for 3 years when adjusted for inflation. It would also provide an additional $2 billion per year for 5 years to create an “NIH Innovation Fund.” Together, this support would help counteract the effects of sequestration and budget cuts that have reduced the purchasing power of the NIH to its lowest level in years. Given the crucial role that NIH-funded research plays in generating the findings on which so many new drugs are based,2 this boost would be a welcome development. Another useful provision could make deidentified data from NIH-funded clinical trials more available to researchers.
Other proposed changes could lead to less salutary outcomes for patients and the health care system. An underlying premise of the bill is the need to accelerate approval for new products, but this process is already quite efficient. A third of new drugs are currently approved on the basis of a single pivotal trial; the median size for all pivotal trials is just 760 patients. More than two thirds of new drugs are approved on the basis of studies lasting 6 months or less3 — a potential problem for medications designed to be taken for a lifetime. Once the Food and Drug Administration (FDA) starts its review, it approves new medications about as quickly as any regulatory agency in the world, evaluating nearly all new drug applications within 6 to 10 months, an impressive turnaround for such complex assessments.
Nonetheless, as introduced, the 21st Century Cures Act instructs the FDA to consider nontraditional study designs and methods of data analysis to further speed approvals. Adaptive trial designs and the use of Bayesian methods hold promise in some kinds of evaluations, particularly in oncology. However, more problematic proposals include encouraging the use of “shorter or smaller clinical trials” for devices and the request that the FDA develop criteria for relying on “evidence from clinical experience,” including “observational studies, registries, and therapeutic use” instead of randomized, controlled trials for approving new uses for existing 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.
The bill would also encourage the FDA to rely more on biomarkers and other surrogate measures rather than actual clinical end points in assessing the efficacy of both drugs and devices. The FDA already uses surrogate end points in about half of new drug approvals.3 Some biomarkers are accurate predictors of disease risk and can be useful measures of the efficacy of a new drug (such as low-density lipoprotein cholesterol for statins). But though a drug's effect on a biomarker can make approval quicker and less costly, especially if the comparator is placebo, it may not always predict the drug's capacity to improve patient outcomes. Bevacizumab (Avastin) delayed tumor progression in advanced breast cancer but was shown not to benefit patients. Similarly, rosiglitazone (Avandia) lowered glycated hemoglobin levels in patients with diabetes even as it increased their risk of myocardial infarction. In 2013, patients began to receive a new drug for tuberculosis approved on the basis of a randomized trial relying on a surrogate measure of bacterial counts in the sputum — even though patients given the drug in that trial had a death rate four times that in the comparison group, mostly from tuberculosis.4 These provisions in the legislation would not immediately change FDA approval standards, but they would give the agency greater discretion, backed by congressional support, to approve drugs on the basis of less rigorous data.
The proposed legislation would make immediate changes with respect to new antibiotics and antifungals by enabling their approval without conventional clinical trials, if needed to treat a “serious or life-threatening infection” in patients with an “unmet medical need.” In place of proof that the antimicrobial actually decreases morbidity or mortality, the FDA would be empowered to accept nontraditional efficacy measures drawn from small studies as well as “preclinical, pharmacologic, or pathophysiologic evidence; nonclinical susceptibility and pharmacokinetic data, data from phase 2 clinical trials; and such other confirmatory evidence as the secretary [of health and human services] determines appropriate to approve the drug.” Antimicrobials approved in this manner would carry disclaimers on their labeling, but there is no evidence that such a precaution would restrict prescribing to only the most appropriate patients. If passed in its current form, the bill would also provide hospitals with a financial bonus for administering costly new but unproven antibiotics, which could encourage their more widespread use. The bill gives the secretary of health and human services the authority to expand this nontraditional approval pathway to other drug categories as well, if “the public health would benefit from expansion.”
The 21st Century Cures Act goes still further in altering the requirements for approving medical devices — an area long criticized for lack of rigor as compared with drug evaluations,5 though regulatory oversight has improved in recent years. As proposed, the new law would redefine the evidence on which high-risk devices can be approved to include case studies, registries, and articles in the medical literature, rather than more rigorous clinical trials. Another section would allow device makers to pay a third-party organization to determine whether the manufacturer can be relied on to assess the safety and effectiveness of changes it makes to its devices, in place of submitting an application to the FDA. Thus certified by the external company, a device maker would be authorized to continue to assess its own products on an ongoing basis.
Informed consent by patients in drug trials has traditionally been sacrosanct, with exceptions made only when consent is impossible to obtain or contrary to a patient's best interests. But another clause in the proposed law adds a new kind of exception: studies in which “the proposed clinical testing poses no more than minimal risk” — a major departure from current human subject protections. It is not clear who gets to determine whether a given trial of a new drug poses “minimal risk.”
Embedded in the language of the 21st Century Cures Act are some good ideas that could streamline the development and evaluation of new drugs and devices; its call for increased NIH funding may prove to be its most useful component. But political forces have also introduced other provisions that could lead to the approval of drugs and devices that are less safe or effective than existing criteria would permit.
Over the past 80 years, this country's regulatory approach has embraced steadily improving criteria for accurately assessing therapeutic efficacy and risk. Patients and physicians would not benefit from legislation that instead of catapulting us into the future, could actually bring back some of the problems we thought we had left behind in the 20th century.
Tuesday, June 2, 2015
Be there - Grantsville, MD!
Here is the agenda.
Be there - Grantsville, MD!
Here is the agenda.
Friday, May 29, 2015
MAY 28, 2015 Nicholas Kristof I’ve admired the Clintons’ foundation for years for its fine work on AIDS and global poverty, and I’ve moderated many panels at the annual Clinton Global Initiative. Yet with each revelation of failed disclosures or the appearance of a conflict of interest from speaking fees of $500,000 for the former president, I have wondered: What were they thinking?
But the problem is not precisely the Clintons. It’s our entire disgraceful money-based political system. Look around:
• Gov. Chris Christie of New Jersey accepted flights and playoff tickets from the Dallas Cowboys owner, Jerry Jones, who has business interests Christie can affect.
• Senator Marco Rubio of Florida has received financial assistance from a billionaire, Norman Braman, and has channeled public money to Braman’s causes.
When problems are this widespread, the problem is not crooked individuals but perverse incentives from a rotten structure.
“There is a systemic corruption here,” says Sheila Krumholz of the Center for Responsive Politics, which tracks campaign money. “It’s kind of baked in.”
Most politicians are good people. Then they discover that money is the only fuel that makes the system work and sometimes step into the bog themselves.
Money isn’t a new problem, of course. John F. Kennedy was accused of using his father’s wealth to buy elections. In response, he joked that he had received the following telegram from his dad: “Don’t buy another vote. I won’t pay for a landslide!”
Yet Robert Reich, Bill Clinton’s labor secretary and now chairman of the national governing board of Common Cause, a nonpartisan watchdog group, notes that inequality has hugely exacerbated the problem. Billionaires adopt presidential candidates as if they were prize racehorses. Yet for them, it’s only a hobby expense.
For example, Sheldon and Miriam Adelson donated $92 million to super PACs in the 2012 election cycle; as a share of their net worth, that was equivalent to $300 from the median American family. So a multibillionaire can influence a national election for the same sacrifice an average family bears in, say, a weekend driving getaway.
Money doesn’t always succeed, of course, and billionaires often end up wasting money on campaigns. According to The San Jose Mercury News, Meg Whitman spent $43 per vote in her failed campaign for governor of California in 2010, mostly from her own pocket. But Michael Bloomberg won his 2009 re-election campaign for mayor of New York City after, according to the New York Daily News, spending $185 of his own money per vote.
The real bargain is lobbying — and that’s why corporations spend 13 times as much lobbying as they do contributing to campaigns, by the calculations of Lee Drutman, author of a recent book on lobbying.
The health care industry hires about five times as many lobbyists as there are members of Congress. That’s a shrewd investment. Drug company lobbyists have prevented Medicare from getting bulk discounts, amounting to perhaps $50 billion a year in extra profits for the sector.
Likewise, lobbying has carved out the egregious carried interest tax loophole, allowing many financiers to pay vastly reduced tax rates. In that respect, money in politics both reflects inequality and amplifies it.
- Lobbyists exert influence because they bring a potent combination of expertise and money to the game. They gain access, offer a well-informed take on obscure issues — and, for a member of Congress, you think twice before biting the hand that feeds you.
The Supreme Court is partly to blame for the present money game, for its misguided rulings that struck down limits in campaign spending by corporations and unions and the overall political donation cap for individuals.
Still, President Obama could take one step that would help: an executive order requiring federal contractors to disclose all political contributions.
“President Obama could bring the dark money into the sunlight in time for the 2016 election,” notes Michael Waldman of the Brennan Center for Justice at the New York University School of Law. “It’s the single most tangible thing anyone could do to expose the dark money that is now polluting politics.”
I’ve covered corrupt regimes all over the world, and I find it ineffably sad to come home and behold institutionalized sleaze in the United States.
Reich told me that for meaningful change to arrive, “voters need to reach a point of revulsion.” Hey, folks, that time has come.☐
Thursday, May 28, 2015
May 28th, 2015 | By Jane Akre (FiDA highlight)
$100 million in damages.
That is the amount a Delaware jury today awarded to mesh injured woman Deborah Barba. The amount includes $25 million in compensatory and $75 million in punitive damages, established to send a message to the company.
The 51-year-old from Newark, Delaware, sued manufacturer Boston Scientific for her permanent and serious injuries caused by the company’s Advantage Fit and Pinnacle transvaginal meshes. She was implanted in 2009 and has suffered significant complications and endured two surgeries that did not fully remove the devices.
“While we are extremely pleased with this verdict and the relief we hope it will bring to the Barbas for Deborah’s unspeakable suffering, we also hope Boston Scientific and other mesh manufacturers take note of this verdict and resolve all pending cases swiftly. Deborah’s case will hopefully bring more awareness of mesh issues,s however, no woman and her loved ones should have to endure the stress of going to trial and baring their souls publicly to achieve justice,” said Barba’s attorney Fidelma Fitzpatrick of the Motley Rice law firm.
The jury found Boston Scientific was negligent in its design and manufacture of the Pinnacle and Advantage Fit devices and that the warnings were insufficient to unsuspecting doctors and their patients.
“I am thankful for the jury’s verdict and hope my story can help other women who are suffering from mesh complications to receive the resolution they deserve,” said Deborah Barba. “While difficult to share, I hope my case demonstrates to all mesh manufacturers the dangers of their products and the justice they owe victims.”
The case is Deborah Barba v. Boston Scientific Corporation, Superior Court of the State of Delaware in and for New Castle County, C.A. No. N11C-08-050 MMJ.
Jeff Feeley, May 28, 2015 (FiDA highlight)
Boston Scientific Corp. must pay $100 million to a Delaware woman who blamed the company’s vaginal-mesh inserts for leaving her in constant pain and unable to have sex, in the first verdict after the company agreed to begin settling cases over the devices, and the biggest yet.
A state-court jury in Delaware found Thursday that Boston Scientific’s Pinnacle and Advantage Fit inserts, built to buttress sagging organs and treat incontinence in women, were defectively designed and company executives hid the flaws from Deborah Barba.
The 51-year-old former bank teller contends the inserts eroded once they were implanted, leaving her with a scarred vagina and a host of medical problems. The verdict is the largest so far against Boston Scientific over its vaginal-mesh inserts. It eclipsed a $73 million award last year to a Texas woman who blamed the company’s Obtryx sling for her injuries.
The jury also found Boston Scientific engaged in fraud by failing to alert doctors to the devices’ faulty design. It awarded $25 million in compensatory damages and hit the company with a $75 million punitive-damages award.
The vaginal-mesh verdict is also the first since Marlborough, Massachusetts-based Boston Scientific agreed last month to pay $119 million to resolve about 3,000 lawsuits over the devices in the first settlements of claims the inserts damaged women’s organs and made sexual intercourse painful.
Kelly Leadem, a Boston Scientific spokeswoman, said the company disputes the conclusion that the inserts were flawed and caused Barba’s injuries.
“We disagree with the jury’s finding and intend to appeal based on the strength of our evidence,” she said in an e-mail.
The verdict is surprising because it came in Delaware, the most corporate-friendly state in the nation, Erik Gordon, a professor at the University of Michigan’s business and law schools who teaches classes on how drugs and medical devices are developed and regulated, said by e-mail.
“Corporation-friendly Delaware juries rarely award punitive damages,” Gordon said. “A good portion of Delaware’s economy is driven by its business of domiciling most of the country’s largest corporations.”
Delaware, the corporate home to more than half of the U.S.’s publicly traded companies and 63 percent of Fortune 500 firms, had more than 1 million legal entities incorporated in the 900,000-resident state by 2012, officials said.
“The jury spoke loudly and clearly that Boston Scientific’s defective devices injured Mrs. Barba and many other women and they should step and take responsibility for causing that harm,” said Fred Thompson, one of her lawyers.
The U.S. Food and Drug Administration ordered Boston Scientific, Johnson & Johnson and more than 30 other vaginal-implant makers in 2012 to study rates of organ damage and complications linked to the devices after the companies faced a wave of lawsuits over them.
Women such as Barba allege that inserts produced by Boston Scientific and other companies are made of substandard materials and shrink once they are implanted, causing organ damage and persistent pain. J&J moved in June 2012 to pull four lines of inserts off the market.
Many of the more than 70,000 mesh-insert cases have been consolidated before U.S. District Judge Joseph Goodwin in Charleston, West Virginia. Others have been filed in state courts in Delaware, New Jersey, Missouri, Texas and California.
Goodwin has been pushing manufacturers to consider settling the cases before they face billions in jury awards.
Boston Scientific, C.R. Bard Inc. and other makers of vaginal inserts had talks two years ago about creating a global settlement of cases over the devices, according to people familiar with the discussions. J&J, which refused to participate in 2013 settlement talks, has now begun to settle some cases.
While Boston Scientific and Bard couldn’t agree on an overarching settlement program, both companies have begun to settle some individual suits and some lawyers’ inventories of cases.
Boston Scientific agreed to pay the $119 million to resolve nearly 3,000 cases collected by a group of plaintiffs’ lawyers led by Houston litigator David Matthews in April. The settlement provided an average payout of about $40,000 per case.
The Delaware case is Barba v. Boston Scientific Corp., CA No. 11C-08-050-MMJ, Superior Court of Delaware (Wilmington).
Wednesday, May 27, 2015
The Federal Bureau of Investigation has begun looking into whether medical device makers, doctors and hospitals broke the law by failing to report problems linked to a power tool used during gynecologic surgery, according to two people who said they were interviewed by investigators.
The tool, called a morcellator, has rapidly spinning blades that cut tissue into pieces that can be removed from the body through the tiny slits made during minimally invasive surgery. Morcellators have often been used in surgery to remove the uterus, but in some women with undetected cancers they have sprayed malignant cells around inside the abdomen like seeds, speeding the progression of the disease.
The inquiries were first reported on Wednesday by The Wall Street Journal, which said the agents worked out of the F.B.I. office in Newark, N.J.
Celeste Danzi, a spokeswoman for the F.B.I.’s Newark office, declined to confirm the inquiry. “We just don’t comment on the existence or nonexistence of any investigation,” she said.
In an interview with The New York Times, Dr. Hooman Noorchashm, whose wife, Dr. Amy Reed, was harmed by the device, confirmed that they had spoken a number of times to an F.B.I. agent from Newark. A retired pathologist from Pennsylvania, Dr. Robert W. Lamparter, also said he had spoken to investigators. Both men declined to name the agent, saying they had been warned that disclosing too much information could interfere with the investigation.
Dr. Reed, 42, an anesthesiologist, had a hysterectomy because of fibroid tumors in her uterus in October 2013 at Brigham and Women’s Hospital in Boston. Fibroids are benign, but they sometimes hide cancer. A biopsy after Dr. Reed’s surgery found a hidden sarcoma, an aggressive type of cancer. The tumor spread, resulting in advanced Stage 4 cancer. Dr. Reed underwent numerous rounds of chemotherapy and radical surgery, but the cancer recurred in March of this year, near her spine, requiring still more surgery.
The couple, who have six children, have conducted a ceaseless nationwide campaign to ban morcellation. Gynecology groups have resisted, saying that sarcomas are uncommon and that morcellation makes surgery less invasive and safer for the majority of women.
In November, the Food and Drug Administration said that morcellators should no longer be used in “the vast majority” of women. But the agency did not take the devices off the market or ban their use.
Dr. Noorchashm said he contacted an agent from the Newark F.B.I. office last fall, because he suspected that morcellator manufacturers and some doctors and hospitals using the devices had violated a federal law requiring that adverse events be reported to the F.D.A. He said that he and his wife spoke with the agent a number of times over a few months, and that the F.B.I. seemed increasingly interested.
Dr. Lamparter said that he had also recently spoken to the F.B.I., and that the conversation had focused on his 2006 correspondence with Ethicon, the unit of Johnson & Johnson that sold power morcellators. At that time, he warned Ethicon of the potential for the morcellators to spread undetected cancer, according to email correspondence he provided to The Times and other news outlets. Johnson & Johnson withdrew its morcellators from the market last July.
Johnson & Johnson has said that after Dr. Lamparter raised his concerns, it added new language to the instructions for use of the device, and that the company had already recommended that, in patients where a cancer was suspected, doctors should use a special bag to remove the tissue.
In a telephone interview Wednesday, Dr. Lamparter said that he considered the change a “legal fig leaf” and that the gynecologists at his hospital, Evangelical Community Hospital in Lewisburg, Penn., reported that the training they received from the company in using the device did not substantially change after he raised his alarm.
Dr. Lamparter said he initially believed that the morcellator could still be used, but not on women at high risk for cancer. However, he added, “I’ve come to believe that the morcellator, as it is used now, is just a bad idea.”
Ernie Knewitz, a Johnson & Johnson spokesman, said it was unaware of any investigation.
Kate Zernike contributed reporting.
Friday, May 22, 2015
“There are 7,000 known diseases. We have treatments for only 500 of them. We have work to do.”1
For patients and their families, the gap between the number of diseases and the number of treatments is not a statistic; it is their daily struggle. Despite the medical breakthroughs of recent years, for many diseases, treatments or research simply do not exist.
Through legislation such as the Food and Drug Administration Safety and Innovation Act and the PREEMIE Reauthorization Act, which included the National Pediatric Research Network Act, the Energy and Commerce Committee has sought bipartisan solutions to facilitate and accelerate patient access to innovative treatments. Although progress has been made, significant work remains. That is why we launched the 21st Century Cures initiative, and the involvement and guidance of those patients on the frontlines and their advocates is critical. Our goal of accelerating the cycle of discovery, development, and delivery of promising new treatments and cures is shared by many, but perhaps most of all by patients and their families.
The committee appreciates that certain aspects of the discovery, development, and delivery cycle have different meanings for different patients. Depending on the condition or disease at issue, the state of biomedical research, and the translation of such research into treatments and cures, varies. We want to hear about the state of biomedical research and therapeutic innovation for specific diseases and better understand how Congress can help move the ball forward.
To help the committee’s effort, we are seeking input from the patient community on the following questions:
What is the state of discovery of cures and treatments for your disease? Are there cures and treatments now or on the horizon?
1 Statement of Margaret Anderson, Executive Director of FasterCures, at the 21st Century Cures Roundtable held on May 6, 2014, http://www.youtube.com/watch?v=Fr4Re7sfDzE&t=33m21s.
- What programs or policies have you utilized to support and foster research, such as patient registries, public-private partnerships, and venture philanthropy?
- How can Congress incentivize, coordinate, and accelerate basic research for diseases we know relatively little about?
- How can we work together to better translate advances in science into safe and effective new therapies for patients?
- How do you coordinate your research and outreach with other patients?
- How do you learn about new treatments and cures? How do you communicate with other
patients regarding treatments and cures?
- What can we learn from your experiences with clinical trials and the drug development process?
- What is the role of government in your work, including any barriers to achieving your goals and advancing breakthroughs?
- How should regulators evaluate benefit-risk? How do you work with regulators regarding benefit-risk? Can this process be improved?
- What is the role of public and private funding in the research and development of cures and treatments?
- Are there success stories the committee can highlight and best practices we can leverage in other areas?
- How have you worked with other patients to support one another?
- What is the financial burden of your disease? How would better treatments and cures help
save money for your family and the federal government?
- How can Congress help?
Public input is critical to the 21st Century Cures initiative, especially from the patient community. With the staggering gap between the number of diseases and available treatments, there are undoubtedly countless untold stories that will provide guidance and perspective in this effort. We request all submissions and suggestions be sent to firstname.lastname@example.org by June 13, 2014.