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You mean the ends doesn’t justify the means?
According to a Reuter’s report, “The healthcare spending watchdog NICE said it was reviewing how it values new technologies, a week after an industry report called for such a move.” NICE's announcement followed an industry report published last week calling for an enquiry to assess the long-term impact of NICE on the cost and the uptake of drugs, along with a series of tax breaks and other measures to support the crisis-hit biotechnology industry
The study (due this July) will be led by Ian Kennedy, Emeritus Professor of Health Law, Ethics and Policy at University College London. He’s an academic lawyer who, for the past few decades, has lectured on the ethics of medicine. A long-standing member of the General Medical Council, he is a former president of the Centre of Medical Laws and Ethics, which he founded in 1978.
On a releated note, the Pink Sheet reports that, “Third-party payer policies appear to have an effect on clinical trial participation, but the impact "is difficult" to quantify, according to a draft report by Duke Evidence-Based Practice Center researchers prepared for the Agency for Healthcare Research and Quality.”
The draft report, "Horizon Scan: To What Extent Do Changes In Third-Party Payment Affect Clinical Trials and the Evidence Base?" was posted on AHRQ's technology assessment Web site. Comments on the draft are due Jan. 23. The topic is of interest because there is no consensus on financial responsibility for clinical trial-related health care costs, resulting in uneven reimbursement policies. A lack of adequate coverage for those costs may discourage patients from participating in trials, reducing the body of available clinical evidence.
A 2000 survey of nearly 6,000 cancer patients who were aware of clinical trial availability revealed that about 75 percent chose not to participate, with 20 percent of that group citing uncertainty about insurance coverage as the reason for declining participation. The top responses given for not entering into a clinical trial were: standard treatment was believed to be better (37 percent) and fear of receiving a placebo (31 percent).
Yet another unintended consequence of cost-based versus patient-centric reimbursement policies.
What’s better than 1 FDA? How about 50.
New evidence of a healthy change in public opinion may be found in a survey released this week by our colleagues at Timbro. A positive result of the breakdown of the Swedish monopolistic health care system is that the young generation (especially age 16-29) looks with favour on private options in social services.
Try this for size:
- three out of ten people think that private financing may need to increase for health and elderly care
- about half of this population consider this a positive development, and the younger are the most positive: 55 per cent of people age 16-29, and 49 per cent of the age group 30-44 think this "rather or very good".
Perhaps more surprising is the figure for the population age 60 and above: 48 per cent (the same as for the overall population) of the oldest Swedes are rather or very positive (as a proportion of those who replied that private provision of welfare services will increase in at least one area).
Last but not least, 51 per cent also think it largely positive if citizens were able to access private insurance for welfare services, beyond what the state provides. (There was an ominous attempt in 2008 by the supposedly centre-right government to abolish this option.) And again, the strongest supporters are found among the 16-29 year-olds.
There is room for optimism in the home country of cradle-to-grave socialism.
Stupak food safety investigation expanded to include Salmonella in peanut butter
WASHINGTON - U.S. Congressman Bart Stupak (D-Menominee), chairman of the House energy and commerce subcommittee on oversight and investigations, and full Committee Chairman Henry Waxman (D-California), yesterday expanded the subcommittee’s food safety investigation to include the recent salmonella outbreak attributed to peanut butter.
The subcommittee on oversight and investigations held 16 hearings on food and drug safety over the past two years, including two involving a 2007 outbreak of salmonella from peanut butter.
Stupak issued the following statement:
“My subcommittee’s two-year investigation into the safety of our nation’s food supply essentially began when peanut butter contaminated with salmonella was discovered in February 2007,” Stupak said. “Today our investigation has been expanded to include the latest outbreak involving salmonella in peanut butter, which has sickened at least 448 people nationwide including 25 in Michigan.‬‪
“We have held 16 hearings over the past two years on food and drug safety, and have drafted legislation to provide the FDA the regulatory and financial tools to protect the American people. Food safety will remain a top priority for the Subcommittee and I remain committed to advancing legislation to address the weaknesses that allow 76 million Americans to be sickened by food borne illness every year.”
Good thing he is on the case. And good thing he has requested more money for the FDA in this latest disgorgement of tax dollars.
By the way salmonella happens all the time. It's not the result of FDA oversight or lack thereof now or in the past...The idea that food borne illneses be prevented by putting more FDA cops in facilities is absurd. And in terms of value per dollar spent how about investing in the Critical Path to pursue personalized medicine instead of spreading fear about peanut butter.
No doubt there is much yet to be written about the Pfizer/Wyeth deal. But going forward, it's good to see that the key word of the day isn't "profits" or "patents" or "promotion" or "PhRMA" (which will now have one less dues-paying member) -- but "patients."
Here's the verbiage straight from the press materials:
For Patients Today – Broad Range of Health Care Solutions and Treatments: The new company will offer customers and patients a broad range of products for every stage of life. Unique and valuable insights will be gleaned from a portfolio that spans wellness and preventive care, such as vitamins and vaccines, as well as therapies for a wide range of illnesses and diseases, such as Alzheimer’s disease and cancer. We will leverage research across our portfolio and input from an extensive network of customer, physician and stakeholder relationships to accelerate, improve and expand the health solutions and treatments we offer.
For Patients Tomorrow – Robust Discovery and Development Program: The new company will have more resources to invest in research and development than any other biopharmaceutical company. We will have access to all leading scientific technology platforms – enhancing the opportunity to produce significant breakthroughs in key disease areas. As a result, we will be better able to help patients and invest in pursuing multiple avenues to address a wide range of unmet health needs.
At All Times – A Patient-Centric Business: We will operate small, distinct business units tailored to patients and customers that also benefit from being part of a premier global organization. Each business unit will oversee product development from early stage research to clinical trials to commercialization. This approach will allow for more customer input into the development process, rapid decision making and a better use of resources. As a result, we will have the ability to invest in long-term opportunities while optimizing near-term patient access to existing products.
"Patients Today." "Patients Tomorrow." "Patient-Centric." That's what I call real p-value.
The words are right. Now let's see what happens.
That is sort of like suing the Wright Brothers for every airplane crash.
Here is a blog that puts this awful decision in perspective. As the blog notes: "55 years of product liability law out the window."
And that's just the beginning. Ultimately, it's all about dinging the drug companies on labeling.
There is one bit of reportage that does, however, need to be corrected. Here’s what was written:
“Peter Pitts, president of the Center for Medicine in the Public Interest (CMPI) and former FDA staffer, confirmed those rumors, both on DrugWonks.com -- CMPI's blog -- and when I called him yesterday. Pitts, who has advised the Obama transition team, said that Sharfstein and Califf were the only two names receiving serious consideration.”
Almost right. What I told Grant was that I thought the two most serious candidates for the job are Sharfstein and Califf. Whether or not the Obama team is considering others is above my pay grade.
To view the complete interview in The Scientist, see here.
(For more on the Mexican conference, see here: Hecho en Mexico
He makes some good points -- but misses one key one: what about new types of evidence? Pharmacogenomic evidence to be precise. Another interesting point is that at the end of the editorial he notes that he does work both for NICE and for pharmaceutical companies. Good for him. The point here is that being paid by the government to do healthcare technology assessments is as much of a conflict as doing work for private industry.
Nuff said.
Here's the complete editorial:
Rationing new medicines in the UK
In England and Wales the National Institute for Health and Clinical Excellence (NICE) issues guidance on the appropriate use of medicines that is based on an assessment of evidence submitted by the manufacturer. The scope of the assessment depends on whether the appraisal concerned is a single technology appraisal or a multiple technology appraisal. NICE recently terminated four single technology appraisals of cancer drugs because it did not receive submissions from drug companies that met the institute’s specification of evidence (1).As a result, NICE was unable to recommend the use of the products for the clinical indications for which they were licensed, but it stated that, after considering the reasons for the lack of guidance, NHS organisations could still use the drugs. In contrast, the Scottish Medicines Consortium approves medicines only if drug companies submit evidence, so non-submission results in a recommendation not to use the drugs concerned in the Scottish NHS (2).
This situation is one consequence of NICE’s switch to undertaking more single technology appraisals, the main advantage of which is a shorter time between the drug’s marketing approval and a preliminary decision. However, in shortening the time allowed for the appraisal, NICE is largely reliant on information provided by the manufacturer, whereas under the original (multiple) technology appraisal process, the independent review group contracted by NICE also undertook an analysis.
One concern is that, in the future, companies could terminate an appraisal by failing to submit data if they thought the chance of a positive NICE recommendation was small. Clinicians or patient organisations could then bring pressure to bear on local decision makers, whereas this would not be possible after a negative NICE appraisal. In most jurisdictions that use an evidence based approach to drug use, this situation cannot arise because a formal application must be made by the manufacturer for inclusion on the national formulary or "positive list." (3) In the United Kingdom, however, most licensed drugs are automatically available for prescribing on the NHS, unless guidance from NICE, the Scottish Medicines Consortium, or the All Wales Medicines Strategy Group limits their use. If terminated appraisals effectively delegate decisions to the local level, this could exacerbate the "postcode lottery" that NICE was created to tackle (4).
So what could be done? Moving towards a comprehensive approach for evaluating the clinical effectiveness and cost effectiveness of all new drugs, linked to listing for reimbursement, raises a wide range of questions, not least that of whether NICE could cope with the workload. Certainly, without substantial extra resources it would have to simplify its procedures greatly. In particular, it would need to limit stakeholder involvement and perhaps be less rigorous with its reviews, thereby increasing its reliance on manufacturers’ submissions.
Alternatively, NICE could follow the approach used by the Scottish Medicines Consortium and, in the absence of a submission, rule that the drug is not recommended for use. This approach would remove the incentive not to submit. However, this equates absence of evidence with evidence of absence (of clinical effectiveness and cost effectiveness), and it may deny patients access to drugs that might be cost effective. A third option would be for NICE to negotiate a "coverage with evidence" agreement with the manufacturer. Under this scenario, the drug would be available for use in NHS patients, but access would be conditional on a commitment by the manufacturer to provide evidence on outcomes and costs at a set date in the future when the NICE decision would be reviewed. This approach may be useful if non-submission reflects an absence of evidence for the relevant patient group (for example, the terminated appraisals on carmustine implants for recurrent glioma (TA149) and cetuximab for colorectal cancer (TA150)). However, it would be of little use if the drug company chose not to submit because the limited available evidence indicates that the drug is unlikely to be cost effective when assessed against NICE’s cost per quality adjusted life year threshold (for example, bevacizumab for breast cancer (TA147)). In such situations a coverage with evidence approach could provide a perverse incentive for companies to claim that no data exist, to increase the chances of market access for their product.
A fourth possibility, arguably more in keeping with NICE’s ethos, would be to commit to convert a single technology appraisal to a multiple technology appraisal if and when it becomes clear that the manufacturer is not intending to make a submission in accordance with the institute’s specification. This might lengthen the appraisal process and may be unsatisfactory if the manufacturer fails to give access to unpublished data. However, it is more likely to encourage submissions from manufacturers wherever possible, because the incentive to the manufacturer would be to ensure that its point of view was adequately reflected in the appraisal.
None of these strategies is without its drawbacks. Nevertheless, simply terminating appraisals runs the risk that the NHS in England and Wales will have to make difficult decisions in the context of an absence of evidence. The fourth option, of converting single technology appraisals to multiple technology appraisals when the manufacturer fails to make a submission, would be the best way forward.
Cite this as: BMJ 2009;338:a3182
Michael Drummond, professor of health economics, Anne Mason, research fellow in health economics
Centre for Health Economics, University of York, York YO10 5DD
Competing interests: MD and AM have worked on technology assessment reviews, for which the Centre for Health Economics at The University of York receives funding from NICE. They have also received funding for research from, and undertaken consultancy projects for, several drug companies. MD is chair of one of NICE’s guideline review panels.
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.
