Latest Drugwonks' Blog
Solid directional guidance fom Bob Hugin, CEO of Celgene and the new Chairman of PhRMA.
Supporting the ecosystem of medical innovation is the most important thing we can do to improve health care for patients with unmet medical needs in the United States.
Medical innovation is a crown jewel of America and its economy. It has been the greatest source of longer life and economic prosperity in our country and around the world. In the course of just one decade, from 1999 to 2008, medical innovation brought about a 45 percent reduction in deaths from cardiovascular disease. Biopharmaceutical therapy for a devastating disease like Alzheimer’s can reduce nursing home admissions by 50 percent. HIV has gone from an untreatable, fatal disease to a serious but manageable condition. And in cancer, nearly 50 million life-years have been saved for patients since 1990 because of innovative approaches to cancer prevention and treatment.
How did we accomplish this? By working together – large and small companies, academic researchers, government agencies, and patient organizations alike. By taking risks and investing in innovation. And those investments have paid off in scientific advancement and economic growth. For example, the US government spent $3.8 billion over thirteen years investing in the Human Genome Project, generating enormous private sector activity. Every $1 invested in the Human Genome Project created $140 in economic value, repaying the government for its investment via tax revenue many times over, and producing transformative scientific and medical advances for patients.
We cannot take this ecosystem for granted. In fact, it is very much at risk. Short-sighted policies that limit scientific and medical innovation today could negatively affect patients for decades in the future. Consider Alzheimer’s Disease. The Alzheimer’s Association reports that without new disease-modifying treatments, by 2050, at least 13.5 million Americans will have developed Alzheimer’s disease, costing this country $1 trillion per year – a crushing expense. A new therapy that delays the onset of Alzheimer’s by five years would reduce by nearly 45% the number of people with the disease by 2050, and save $447 billion per year. We cannot afford NOT to invest in the discovery and development of such a potential treatment today.
So how do we protect the ecosystem of medical innovation? Fortunately, while the challenges are greater than ever, so too are the opportunities. We are only at the early stages of being able to capitalize on the revolution in molecular biology and information technology. With more than 5,000 new treatments in development, the promise and potential of recent scientific and technological advances is enormous. We must be bold and courageous and stay focused on developing innovations that meet patients’ most pressing unmet needs. And to do this, we must have a policy environment that supports innovation, values the entire ecosystem of medical progress – academic medical centers, voluntary health associations, government agencies like the FDA, CMS and NIH, and, crucially, the biopharmaceutical company scientists, entrepreneurs and investors responsible for discovering, developing and bringing to patients the vast majority of new treatments.
Part of a supportive environment for innovation is ensuring that patients have access to the most innovative treatments. New therapies that reduce hospitalizations, promote productivity and improve quality of life have the potential to make real reductions in the overall cost of health care, but access to these treatments is critical. Medicare Part D is an example of how the public and private sectors can work together to maximize benefit to patients and value to society. This government supported, privately delivered program based on competition and choice has expanded access for good prescription coverage to 30 million seniors, with 90 percent satisfaction among participants, at a cost more than 40 percent lower than projections, and substantial demonstrated savings in other parts of the medical system. We must work to protect this model and also ensure patient access to therapeutics in Medicare Part B, other government-funded health programs, and in plans offered through health exchanges.
We can improve health care in the United States by fostering a policy environment that strengthens the ecosystem of innovation, so that working together, we can pursue bold innovations that create longer life and better health for patients.
"Dr. Doshi’s renown comes not from solving the puzzles of cancer or discovering the next blockbuster drug, but from pushing the world’s biggest pharmaceutical companies to open their records to outsiders in an effort to better understand the benefits and potential harms of the drugs that billions of people take every day. Together with a band of far-flung researchers and activists, he is trying to unearth data from clinical trials — complex studies that last for years and often involve thousands of patients across many countries — and make it public."
You see, his renown comes froms digging up old data and proving that Steve Nissen and Andrew Wakefield were right all along!!
"He met Dr. Jefferson, a prominent skeptic of the flu vaccine, after researching whether the Centers for Disease Control was exaggerating the deadliness of the disease.
“We were both lone wolves in the field of influenza,” Dr. Doshi recalled.
Just great. Wakefield redux and the NYT believes that undermining the support for immunization is a important as curing cancer!
Transparency is a tool for people who hate the drug industry to make challenges based on statistical manipulation of data sets... Avandia and Paxil are great drugs for the right people and have saved lives. The second guessing has lead to both medicines being underused while treatments that have even clearer adverse events have been overprescribed. I have seen what researchers have done with the data.. Tamiflu works brilliantly for a subset of patients. The problems it now has is that the viral strains it was designed to block are mutating.
Moreover, what is submitted to the FDA and peer -reviewed publications has nothing to do with financial ties and everything to do with the exclusion criteria and the way studies are conducted. What do these guys contribute to improving or moving the ball forward towards personalized medicine...nothing. Anyone who claims that these drugs DONT work are liars and building their reputations on the bodies of people who are dead because they bought the bullshit. That goes for Ben Goldacre in particular who seems to get off on making TED speeches.
Datasharing that re-examines hypothesis or established science should be encouraged. But it has to be conducted as if people's lives are at stake. I hate to say it, but this is the kind of ass kissing 'stick it to the man' bias that allowed Wakefield to spread fear about vaccines.. Do you think the NYT would have ran the story if it was about giving a medicine a second life for dying patients? And would these 'whistleblowers' have gone after a company that pulled a drug working in a sub-group.
It seems that lately, when it comes to healthcare policy, we are living in the land of unintended consequences.
Per the Supreme Court’s recent 5-4 decision that makers of generic drugs cannot be sued under state law for adverse reactions to their products, the one-eyed man is measuring the throne room.
Writing for the majority, Mr. Justice Alito opines that state law cannot trump federal laws regarding prescription medicines whose design has been approved by the Food and Drug Administration. So, the obvious first question is, why doesn’t Federal Preemption hold for innovator products. Will the Court revisit Wyeth v. Levine? Not likely.
And then there’s the more important question – that of patient safety. While there is no language in the Supreme Court decision on either bioequivalence or therapeutic interchangeability, the unintended consequence of their decision is that many payers and physicians, now that the burden of potential legal entanglements has been lifted, will now consider generics as “the same” as their innovator forefathers and prescribe accordingly.
And since the Supremes’ made no special mention of Narrow Therapeutic Index, well, Katie un-bar the door for anti-seizure meds, anti-depressants, etc.
Not good.
The Court’s decision also undermines the urgency of educating payers, physicians, and patients on the subject of generic drug quality – and this goes further than just excipient changes.
It’s one of our nation’s most important and yet invisible problems. How to educate the various relevant publics on “bioequivalence?” Not that we were trying very hard before – but as we approach the reality of both small molecule patent expiry and biosimilars, the issue becomes less and less academic and more and more practical.
For example, how will the Court’s decision impact the INN debate?
The Court’s decision has thrown yet another cloak of invisibility over the urgent need for quality maximization.
William Gladstone famously said that, “Justice delayed is justice denied.” Well, getting the right medicine to the right patient at the right time (as good a definition as any of “personalized medicine”) is healthcare justice – and the Supreme Court’s decision is going to be a barrier to achieving justice for all.
The battle over ObamaCare will reignite soon, and the next front will be the war for public opinion. The American public remains deeply skeptical of the new law. Many Americans say they will not sign up for insurance in the new “exchanges” scheduled to open October 1, 2013. As a result, the Obama administration is preparing a high-profile public relations blitz to again sell the law to the public.
Here are 4 talking points ObamaCare advocates will attempt to promote — and 4 questions Americans should ask in response.
1) “Free” benefits
One of the supposed selling points of the new law will be “free” benefits, such as “free” birth control, well-woman visits, STD (sexually transmitted disease) prevention counseling, and a variety of preventive services.
Of course, nothing is “free.” Others will have to pay for these services in the form of increased insurance premiums or higher taxes. If anyone touts “free” benefits, we should ask, “Who is really paying for them? And what else could they be doing with their own money if they weren’t compelled to do so?”
2) “Coverage”
One of the goals of ObamaCare is near-universal “coverage.” But “coverage” is not the same as actual medical care. The American Medical Association predicts a “silent exodus” of physicians as ObamaCare is phased in, worsening the already existing physician shortage.
The New York Times notes the already growing disconnect between theoretical “coverage” and actual medical care in parts of California: “Patients still get care, but the process is often slow and difficult. In Riverside, it has left residents driving long distances to doctors, languishing on waiting lists, overusing emergency rooms and even forgoing care.” These problems will worsen under ObamaCare.
Read the full piece here.
It’s rare that I find myself in agreement with Public Citizen. This is one of those times.
In a 5 to 4 vote the Supreme Court has ruled that makers of generic drugs cannot be sued under state law for adverse reactions to their products, a decision that consumer advocates called a blow to patient safety.
The majority opinion, written by Justice Samuel A. Alito Jr., said the state’s law could not run against federal laws regarding prescription medicines whose design has been approved by the Food and Drug Administration.
The consumer watchdog group Public Citizen said the Supreme Court decision Monday undermines patient safety at a time when about 80 percent of U.S. prescriptions are filled with generic medicines.
“Today’s court decision provides a disincentive for generic makers of drugs to monitor safety of their products and to make sure that they have a surveillance system in place to detect adverse events that pose a threat to patients,” Michael Carome, director of Public Citizen’s Health Research Group, said in an interview.
Wither innovator preemption?
Medicine was once considered a place where patients could confide in their doctors about their most intimate concerns and doctors had time to listen. Notes were one- or two-line jots in a chart. We’d spend the extra time because we were valued for our skills and for our knowledge and there was more to it than just pay. We had skin in the game. We got paid in chickens. We knew our patients. Back then, seven-minute appointments didn’t exist.
Now, doctors are cultivated as shift workers. Patients have Google. Everyone has information at their fingertips. Our new story line has become there are no limits to what patients can have in health care. Perfect data. Perfect health care access. Error-free health care with perfect delivery. Perfect communicating doctors. Always. We’re building our medical Utopia.
But this effort to school doctors on our path to Nirvana has a serious downside for health care workers on the front lines.
As I’ve said before, there’s a growing culture of hostile dependency that continues to grow toward doctors these days. The theme is like an adolescent who realizes his parents have feet of clay. He comes out of his childhood bubble and realizes his parents have failures and limitations because they are human beings. This results in the adolescent feeling unsafe, unprotected and vulnerable. Since this is not a pleasant feeling, narcissistic rage is triggered toward the people he needs and depends on the most. None of this occurs at a conscious level. Most of us understand this behavior simply as “adolescent rebellion,” not understanding the powerful issues at play. So when we spotlight one side of what doctors should do for patients, be it improve communication or empathy (or whatever) without acknowledging the realities health care workers face like looming staffing shortages and pay cuts, we risk fanning the flames of narcissistic rage against the very caregivers whom we depend on the most - the very caregivers who are striving to communicate, do more with less, check boxes while still looking in the patient’s eyes, meet productivity ratios, all while working in a highly litigious environment.
Read the full piece here.
Actually Padzur personifies the problems inherent in what Eric Topol has called the "contrived clinical trial environment." Here's Padzur's punchline:
“I am extremely disappointed in the sponsor’s proposed labeling for this drug,” Pazdur said during a long aside quoted in The Cancer Letter. “There is no survival curve in the proposed labeling. There is no hazard ratio— there’s difference in the means and the explanation in one or two sentences confounding by crossover.
“But if this drug is approved, one would have to have a very careful conversation with the patient about this potential negative impact on overall survival. And how would you do this? I’ve been playing this in my mind in several scenarios, and any logical patient that I could think of would say, ‘Doc, if you are so uncertain about this most important endpoint, don’t we have any other drug to use here?’”
If my doctor used terms like survival curves, endpoints and hazard ratios in discussing my cancer treatment, I wouldn't stick around for an explanation. Why waste months debating such crap when my life is at stake?
The fact that Richard Padzur or any regulator is doing the thinking on behalf of patients underscores just how dysfunctional FDA review has become. It’s how drugs work in the real world that matters. Yet the most recent data shows that it actually takes cancer drugs more time to get to patients than any other area of medicine. And orphan designation is simply a way to get more patent life, not speed treatments to patients. An orphan designation is associated with more time in development, not less. Why are we still using phase 2 and 3 randomized trials to test medicines where a driver mutation is known? Why are we allowing insurance companies to force advanced stage patients to ‘fail first’ on cookbook treatments when we have can match mutation to treatment? The call for ‘more evidence’ is a deceitful dodge because the ‘evidence’ people like Padzur and others call for is randomized trials where survival curves mean more than real survival.
Padzur is not the problem. He is obviously a well-intentioned -- and caring -- public servant. Rather he is part of broken system where most patients die in less time than it takes to organize and conduct clinical trials.
Washington, D.C. – In advance of the upcoming U.S.-India Strategic Dialogue, U.S. Senators Rob Portman (R-Ohio), former U.S. Trade Representative, and Bob Menendez (D-NJ), Chairman of the Senate Foreign Relations Committee, today led a bipartisan letter signed by 40 total Senators to U.S. Secretary of State John Kerry urging him to work with the Government of India to improve the business operating environment in India and end its trade and economic practices that discriminate against American companies.
While calling India a “crucial ally” and “an important export market for American-made goods and services,” the Senators wrote: “…we are very concerned that India's recent actions to force the local production of certain information technology and clean energy equipment and to deny, break or revoke patents for nearly a dozen lifesaving medications risk undermining our broader partnership. This is particularly troubling against the backdrop of a generally deteriorating environment for intellectual property protection in India.”
“If India is to truly embrace its ‘Decade of Innovation,’ India’s policymakers must begin to recognize the value of intellectual property. The stakes are too high for India to ignore. India’s deteriorating IP environment is bad for investment, bad for innovation, and bad for international trade,” said Mark Elliot, executive vice president of the U.S. Chamber of Commerce’s Global Intellectual Property Center, co-chair of the Alliance for Fair Trade with India.
“India’s unfair and discriminatory trade practices are hurting manufacturers in the United States. It’s important that this issue is raised at the highest levels to ensure India abides by global trade rules to protect America's competitiveness. Manufacturers look forward to working with Congress and the Administration to ensure a level playing field,” said National Association of Manufacturers (NAM) President and CEO Jay Timmons. The NAM is co-chair of the Alliance for Fair Trade with India.
The Senators urged the State Department “to press for swift action,” adding: “A level playing field for American businesses, farmers and workers in India and other overseas markets is an essential U.S. diplomatic objective.”
Portman and Menendez were joined by U.S. Senators Kelly Ayotte (R-NH), Tammy Baldwin (D-WI), John Barrasso (R-WY), Roy Blunt (R-MO), Richard Burr (R-NC), Maria Cantwell (D-WA), Tom Carper (D-DE), Saxby Chambliss (R-GA), Jeff Chiesa (R-NJ), Dan Coats (R-IN), Susan Collins (R-ME), Chris Coons (D-DE), Bob Corker (R-TN), William “Mo” Cowan (D-MA), Mike Crapo (R-ID), Joe Donnelly (D-IN), Mike Enzi (R-WY), Dianne Feinstein (D-CA), Kay Hagan (D-NC), Martin Heinrich (D-NM), Johnny Isakson (R-GA), Mike Johanns (R-NE), Amy Klobuchar (D-MN), Mary Landrieu (D-LA), Carl Levin (D-MI), Claire McCaskill (D-MO), Patty Murray (D-WA), Bill Nelson (D-FL), Mark Pryor (D-AR), James Risch (R-ID), Pat Roberts (R-KS), Marco Rubio (R-FL), Brian Schatz (D-HI), Debbie Stabenow (D-MI), John Thune (R-SD), Pat Toomey (R-PA), Mark Udall (D-CO), and David Vitter (R-LA).
What's next? CMS commenting on PDUFA implementation?
BioCentury reports that Republicans on the U.S. Senate Committee on Health, Education, Labor and Pensions sent a letter to FDA Commissioner Margaret Hamburg asking "why and under what authority" the agency is publicizing new health insurance exchanges under the Affordable Care Act. According to the letter, FDA sent an email earlier this month to "families, individuals, small businesses and clinicians" promoting open enrollment in the new exchanges. The senators expressed concern that the email was an attempt by HHS to "avoid congressional limitations on implementation expenditures" for ACA, noting that the exchanges are outside of FDA's mission.
The senators are seeking information on FDA's legal authority to promote the implementation of ACA and the new health insurance exchanges and how the activities further the agency's mission, as well as details on the total costs FDA has spent during FY13 on promoting ACA implementation. The senators are seeking a response by June 26
In his mockumentary, “Sicko,” Michael Moore portrayed Great Britain’s government-funded National Health Service as a system to be admired and emulated in the United States. His extreme views didn’t take into consideration that British health providers might have something to learn from the way things are done on this side of the Atlantic.
But that’s just what 20 doctors and nurses from northwest England came to do recently in Washington.
The British contingent, part of a leadership academy with the National Health Service of Northwest England, toured the Kaiser Permanente Total Health Clinic, starting with an explanation of the company's inception in 1945. Led by the clinic's medical director, Ted Eytan, MD, MS, they landed in front of a video depicting Kaiser's vision for its future, complete with smartphone programs that allow patients to perform "virtual triage" and treat their malady without entering a clinic.
Eytan led the clinicians through a display of new technology, from a handheld, portable ultrasound to a webcam-like system for consulting doctors remotely. They stopped in between the show-and-tell to talk about the advantages of connecting patients to their doctors and nurses through regular e-mail and phone conversations.
According to Dr. Robert Stead, a pulmonologist based in East Cheshire, England, the American system seems more physician-led, which could also mean higher costs. He gave the example of how the National Health Service gave midwives more autonomy and responsibility over childbirth, rather than obstetricians.
And the idea of multiple government programs catering to different populations -- veterans, elderly, and poor -- was quite literally a foreign concept to a group raised on the universal health system.
But Dr. James Catania, a physician and medical director in Stockport, a city near Manchester, England, said the system has its own shortcomings. General practitioners only have 10 minutes to spend with their patients, discouraging them from asking more questions or spending time talking to specialists. And while the English are happy to have universal healthcare, he said they are "frustrated with the pathways" to access that care.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
