Latest Drugwonks' Blog
The first is a nice omnibus article from the current edition of Nature Biotechnology, "One year in -- Obama's biotech scorecard."
The next piece is from the Belgian medical publication Le Generaliste. It's focus is on American healthcare reform. It's in French and is headlined, "USA: les soins de sante a la croisee des chemins" (US healthcare at a crossroads).
And finally a new piece in Commentary by our good pal Tevi Troy, "Health Care: A Two-Decade Blunder."
Enjoy.
As seen on the op-ed page of the Orange County Register
Cheaper medicines not always better
By PETER PITTS
President, Center for Medicine in the Public Interest, former associate FDA commissioner
Savings on drugs often result in higher costs in other areas.
The health care reform debate has been focused almost entirely on just two broad issues: the large uninsured population and the rising cost of care. But there's another problem that plagues our health system, and it's just as serious. Doctors are losing their ability to treat patients without being obstructed by outside parties.
Any discussion about improving our health system must recognize that rules which empower bureaucrats to get in the way of the doctor-patient relationship are a serious threat to the quality of medical care.
A strong, trusting relationship between doctors and patients is crucial to a well-functioning health care system. Without such a bond, serious conditions might go misdiagnosed or improperly treated, patients might give inaccurate medical histories, or doctors' orders might be ignored.
This is no small problem. Failing to follow a prescribed treatment regimen – a practice known as "nonadherence" -- costs the U.S. health system over $100 billion a year in avoidable medical costs. And, according to a study published last year in the Annals of Internal Medicine, patients who don't have a close relationship with a single doctor are less likely to receive the proper tests for preventing chronic illnesses.
Yet some policies that are gaining popularity are weakening the doctor-patient relationship by putting treatment decisions in the hands of third parties.
Chief among these schemes is "step therapy." Also called "fail first," this is a policy that is sometimes adopted by insurers and government health programs to save money on pharmaceuticals. It forces patients to try cheaper alternatives to a prescribed drug before they are permitted to get the medicine that their physicians ordered.
So even though a doctor might recommend drug A to treat a patient's hypertension, an insurance company or government program would require the patient first try cheaper drugs B and C, and only after the cheaper drugs are shown to be ineffective can the patient receive the medicine his doctor recommended.
There are several reasons such a policy hurts our health system. For one, it is an assault on the relationship between physicians and patients. When a doctor decides on a treatment, he is employing years of medical experience and weighing countless factors, like the patient's age, diet, and lifestyle. Patients, meanwhile, trust that the doctor knows best.
When it's possible for a health care bureaucrat to override the decision of a trained medical professional, this valuable association between a patient and his doctor breaks down. A patient is no longer under the care of single medical expert, but is now being treated by faceless organization looking to cut corners.
This breeds distrust among patients, but it also results in worse medical outcomes and higher overall costs.
One study of schizophrenia drugs used in Georgia's Medicaid program showed that, while step therapy saved the state close to $20 a month on drugs for every patient, the savings were more than offset by increased costs in other services. Indeed, the program saw a monthly increase of nearly $32 per patient in outpatient care.
Similar studies have shown that private plans utilizing step therapy saw hospital and emergency-room visits increase, resulting in higher overall costs.
What's troubling is that step therapy is becoming more widely adopted. In 2000, 20 percent of private insurance carriers used step therapy. By 2008, it was half.
Reformers need to recognize that policies giving health care administrators control over treatment regimes are hazardous to patient health, and actually inflate overall costs.
The deterioration of the doctor-patient relationship is a serious threat to our health system. Ending step-therapy programs is one way to fortify this relationship.
Via the Pink Sheet:
FDA could use more authority to bring negotiations over a drug's Risk Evaluation and Mitigation Strategy to a close, Principal Deputy Commissioner Joshua Sharfstein indicated during a House Energy and Commerce Health Subcommittee hearing on March 10.
The agency can require a REMS, but not specify its contents, he explained, noting the drug sponsor proposes a REMS and negotiates the final strategy with FDA.
"It's very important for us to work with companies to come up with something that works," Sharfstein said. "There's no question there's a lot we learn from the interchange with companies," but it sometimes can take a long time to come to agreement, and a level of consistency between REMS may be desirable if one approach makes sense.
The agency is open to discussions about how it can more effectively move to closure on these strategies designed to protect public health, he said.
Rep. John Shimkus, R-Ill., newly designed as the subcommittee's acting ranking member, suggested a limit on the time allowed for negotiations. Sharfstein noted that if an application is pending, companies have an incentive to finalize a plan; the problem arises particularly with drugs already on the market.
Another area that needs to be addressed, Sharfstein suggested, is the different treatment of brand and generic drugs when it comes to imposing a REMS with a communication plan for alerting health care professionals and patients about a drug's risk. Brand sponsors must implement such a plan, whereas FDA must pay for and operate a communication plan for generic drugs, he noted.
Via AP -- and something to watch:
Stricter government oversight of dietary supplements is moving closer thanks to an agreement among senators to include guidelines in a food safety bill.
Four key areas of “common ground” are outlined in a letter sent by Sens. John McCain, an Arizona Republican, and Byron Dorgan, a North Dakota Democrat, to Sen. Tom Harkin, the Iowa Democrat who chairs the Senate Health, Education, Labor and Pensions Committee.
McCain and Dorgan were co-sponsors of legislation introduced last month, the Dietary Supplement Safety Act, aimed at regulating the industry. This week’s letter to Harkin—also sent to the committee’s top Republican, Sen. Mike Enzi of Wyoming, and Sen. Orrin Hatch, a Utah Republican—talks about incorporating the “areas of agreement” into the FDA Food Safety Modernization Bill
Those provisions include:
— requiring all dietary supplement manufacturing, processing and holding facilities to register with the Secretary of Health and Human Services;
— giving the Food and Drug Administration authority to issue a mandatory recall order if a dietary supplement is adulterated or misbranded or “the use
of such supplement could cause serious adverse health consequences such as death;”
— requiring the FDA commissioner to publish guidelines on new dietary ingredients “as soon as possible;”
— mandating that the FDA notify the Drug Enforcement Administration when a new product contains a synthetic anabolic steroid.
Athletes from various sports have blamed positive drug tests on substances that turn up in dietary supplements even though they aren’t listed as ingredients on the label.
McCain has said his bill would protect professional and Olympic athletes— along with casual sports participants who use supplements.
Pro sports leagues and the U.S. Anti-Doping Agency have been supporting McCain’s efforts on supplements, and USADA chief executive officer Travis Tygart said Wednesday the letter indicates areas in which the new legislative effort “fills holes.”
“These are obviously needed improvements, and the agreement provides additional consumer protections which are certainly important to all athletes and why the pro sports and the Olympic sports came together to push for better regulation,” Tygart said in a telephone interview with The Associated Press. “It also continues to allow all consumers to have access to legitimate supplements. It’s a fair balance, and this step in the process has resulted in a quick and beneficial agreement.”
Agriculture Appropriations Subcommittee Chairman Herb Kohl, (D, WI), suggested there are "powerful interests" blocking legalization to foreign drug importation.
And he’s right – the powerful interests of the public health.
Do doctors need more and better information on how to get the right dose of the right drug to the right patient at the right time. Yes! But denying FDA approval to one drug because, in a large randomized controlled clinical trial, it may not be "as effective" as another is not only meaningless -- it's counterproductive. How can care be improved if physicians are denied access to new drugs that work? Hm.
What we DO need are more and better molecular diagnostics and more adaptive clinical trials to show what drugs work best in specific subpopulations. That's not comparative effectiveness, it's clinical effectiveness. And it's crucial to the advancement of 21st century medicine.
Here's the NPR story on the NEJM articles:
http://www.npr.org/templates/story/story.php?storyId=124516838
Watch for the issue of an FDA "third leg" to come up during the upcoming debate over PDUFA reauthorization. Watch "comparative" effectiveness morph into "cost" effectiveness. And remember -- what we need to discuss is "clinical" effectiveness.
http://www.whitehouse.gov/health-care-meeting/proposal/titleiii
Fast forward to the present. The RAND Corporation reviewed the ability of Britain's National Health Service -- the ultimate public option with centralized control over health spending, treatment practices, reimbursement levels and performance standards -- to keep people healthier and make sure qualiity of care drives all decisions. RAND looked at the NHS ability to deliver high quality care after increasing spending on health care during that time at a rate faster than spending in the US.
Here's what the study found:
A damaging rift between doctors and managers: “The GP and consultant contracts are de-professionalising, and have had the peculiar effect of simultaneously demoralising and enriching doctors. We’ve lost the volitional work of the doctors and far too many of us are now just working to rule.”
Pointless new structures. “Stop the restructurings. The only thing they generate is redundancy payments.” One body responsible for improving standards reported to five different ministers and had three different names in the space of 30 months.
A culture of fear and slavish compliance. “The risk of consequences to managers is much greater for not meeting expectations from above than for not meeting expectations of patients and families.”
http://www.timesonline.co.uk/tol/news/uk/health/article7052606.ece
RAND notes that the NHS has implemented all manner of quality commissions, practice guidelines, performance standards, value-based reimbursement contracts with doctors, etc. Sounds a lot like the Senate and House bills right? The RAND study noted that comparative effectiveness studies came in for particular criticism by everyone within the NHS:
"NICE is too focused on fiscal issues: This concern likely arises because NICE is simultaneously considering clinical effectiveness and cost effectiveness. Implementation of NICE technology appraisals is mandatory which underscores the focus on fiscal issues. Concerns were also raised about whether the thresholds are adequate (£30,000 per quality adjusted life year) and about the limited and somewhat weaker economic evidence relative to clinical evidence. This led some observers to suggest that the UK needed guidelines development that would not account for resource issues."
This concern about NICE -- the "group of doctors and health experts" President Obama refers to -- is related directly to the feeling among NHS managers and and GPs that they are simply instruments for carrying out the fiscal goals of the NHS at the expense of the needs and health of patients. And meanwhile, none of the new structures, measurements or studies have improved care. Instead, as the RAND study notes:
"Concerns have been raised about perverse incentives for treating multimorbidity patients imbedded in the financial incentives."
Or as the President said: "And to make sure that the quality of care for seniors drives all of our decisions, a group of doctors and health care experts, not Members of Congress, will be tasked with coming up with their best ideas to improve quality and reduce costs for Medicare beneficiaries."
Here’s a taste:
The firestorm over GlaxoSmithKline’s Avandia (rosiglitazone), which was rekindled two weeks ago with an incendiary Senate report, shows why people who live outside the Beltway are convinced that Washington is broken, and why they are right.
The politico-media outburst exemplifies the worst aspects of the American regulatory environment. It also exemplifies why business as usual in Washington must stop, and why the responsibility rests directly at the feet of the politicians and regulators who are failing their responsibility to put the public’s health before politics.
As with previous rounds in the Avandia circus, grandstanding members of Congress played gotcha with FDA, releasing their report to the agency and to the media simultaneously. Citing data from disgruntled FDA employees, Committee Chair Max Baucus (D-Mont.) and ranking member Chuck Grassley (R-Iowa) said in a Feb. 20 press release that “the FDA itself estimated that the drug caused approximately 83,000 excess heart attacks between 1999 and 2007.” The release gave the false impression that this represents FDA’s official position.
Unless the political leadership at HHS and FDA acts quickly and decisively, decision-making based on external political pressure and a paralyzing precautionary philosophy will become institutionalized.
Instead of being occasionally blown off course by recurring squalls, the very real risk is that permanent climate change will forever degrade FDA’s science-based over-sight process, eroding the public’s confidence in the agency and placing a stop sign on the path of biomedical innovation.
Avandia is the poster child for a culture that has evolved at FDA — encouraged by publicity-seeking politicians — in which agency employees who dislike a regulatory decision are able to keep raising the issue, and if they don’t like the results, to go outside established agency procedures for resolving scientific disputes to enlist support from members of Congress and their enabling lapdogs in the media.
There’s more. A lot more. Names are named. And punches aren’t pulled.
The complete BioCentury commentary can be found here.
The issue is – the few versus the many.
"It really isn't the average that matters to people most. For various reasons - analytical, historical and others - we use mean results. But that's not what happens to individual people. ...Of greater interest, this is true for a lot of situations, are the effects on individuals or the distribution of those effects, which we are not so used to measuring."
Even if a drug only has a 10 percent effect, Temple said, "it might have a rather large effect in a fraction of the population," so a look at distribution is useful. "We do think about that, mostly though," he conceded, "when you have context that has a safety problem or the effect is particularly important."
There are increasing opportunities in early studies to identify the responders and incorporate that information into the design of later-phase trials, Temple noted. If sponsors find a likely response predictor, "genomic or some other kind of finding, there's nothing that stops the later trials from stratifying that predictor and analyzing the groups separately or even making the result in the responder subgroup the primary analysis."
Temple's remarks came during a debate on whether clinical trials for PAH drugs should be required to show that patients in the treatment arm improve their distance by a minimum amount during the six-minute walk.
The question, he noted, is what is a big enough improvement? The answer can come from the patients themselves, in how they feel about walking or their life activities, he suggested, "We're very interested in looking more at well-validated patient reported outcomes."
Let's remember and embrace that last comment, "well-validated patient reported outcomes."
Bob’s comments, coming on the heels of the FDA’s recent documents regarding clinical trial designs (Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics), seem to reinforce the agency’s Critical Path philosophy of broadening decision-making criteria about a drug based strictly on generalized results from large scale RCTs. Trial designs that focus on patient selection for ideal response is a key tenet of the personalized medicine approach that industry and FDA are hoping to create and for which the Critical Path’s Reagan Udall Foundation was created to foster.
That being the case, perhaps we should take the Wisdom of Temple to our nation’s Temple of Wisdom and suggest to Congress that a certain percentage of PDUFA fees be used to fund the FDA’s Critical Path program.
The bill is essentially the same as the amendment, also sponsored by Welch, included in the health care reform legislation passed by the House last summer. However, a price negotiation provision is not part of the Senate bill on health reform, which has become the primary vehicle for passing health reform through Congress.
The Welch bill does not provide HHS with the authority to establish a national formulary. The Congressional Budget Office has consistently concluded that empowering HHS to negotiate prices, without also providing the secretary with authority to set up a national formulary, would not produce savings to the government.
As Stanford economists Alain Enthoven and Kyna Fong have explained, when discussing Medicare Part D, “Government price negotiation could leave people without drugs that manufacturers decide aren’t sufficiently profitable under the plan.”
That’s exactly what has happened under the health insurance program run by the Department of Veterans Affairs, which is already empowered to directly negotiate prices with drug producers.
Of the 300 most prescribed drugs among Americans 65 and older, the VA only covers 65 percent of them, according to a study from the Lewin Group. By contrast, the two most popular plans in the Medicare Part D drug benefit — where private insurers compete for customers — each cover 94 percent of those medicines.
In fact, over a third of retired veterans supplement their VA coverage by enrolling in Part D
The Part D model hasn’t sacrificed cost-savings for choice, either. The competitive pressures among participating insurers have lead to a 17 percent drop in out-of-pocket spending for seniors who enrolled in the program in 2006 — that’s equivalent to 14 extra days of medicine a year.
Moreover, Part D’s total expenses over the next decade are expected to be nearly $120 billion less than originally estimated when the program was created.
Mr. Welch and friends are hoping to have the bill move in tandem with the final push to pass health care reform legislation or be added to the package of "fixes" being planned for the Senate bill.
Add this to the growing list of reasons why “reconciliation” is both phony and dishonest.
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.
