Latest Drugwonks' Blog
"We spent as much money as we could, and got as little for it as people could make up their minds to give us. We were always more or less miserable, and most of our acquaintance were in the same condition.” -- Great Expectations
In terms of “learning” about healthcare reform from our British cousins, here’s lesson #1: Don’t believe everything (or anything) you heard on SiCKO. Lesson #2 is seeing CMS Administrator Dr. Donald Berwick in the role of Pip.
According to an investigation in the Daily Telegraph, at least 10 primary care trusts (PCTs) have told hospitals to increase the length of time before they see patients in order to save money.
In one case a manager said the policy keeps patients in line as “short waiting times also create more demand for treatment due to the expectations this raises."
In some areas, patients endured delays of 12 or 15 weeks after GPs decided they needed surgery, even though hospitals could have seen them sooner.
The maximum permitted time between referral and treatment is 18 weeks.
It comes after an NHS watchdog suggested that if patients are forced to wait a long time, they will remove themselves from lists “either by dying or by paying for their own treatment."
Andrew Lansley, the Health Secretary, said: “This practice is simply unacceptable and one of the many reasons we need to modernise the NHS and put patients’ interests first.
The complete article from The Telegraph can be found here.
“Pause you who read this, and think for a moment of the long chain of iron or gold, of thorns or flowers, that would never have bound you, but for the formation of the first link on one memorable day." -- Great Expectations
Hat tip to Helen Evans at Nurses for Reform.
A sudden bold and unexpected question doth many times surprise a man and lay him open. – Francis Bacon
The official PDUFA V technical letter contains no surprises. There are no “Christmas Tree items.” It is a document full of incremental improvements. It is, in a word, “clean.” But don’t let that fool you. There’s a battle ahead.
(The technical letter can be found here.)
Unlike past reauthorization, when industry and agency reached agreement and Congress rubber-stamped it, this year there are going to be questions. Some relevant (predictability and responsibility), some not (greater regulation of consumer marketing practices), some thorny (should biosimilar reviews be covered under PDUFA through fiscal year 2017). It’s a long list.
For many involved in the reauthorization process, one statement that keeps coming up is -- ”The FDA is broken.” But what does that mean? Rather than making blanket statements that cause friction and promote areas of disagreement, one thing everyone can agree to is that the FDA’s must be both ally and accelerator in the advancement of innovation.
Can that be accomplished within the confines of PDUFA V?
PDUFA IV expires Sept. 30, 2012. Senator Tom Harkin (D, IA -- Chairman of the Health, Education, Labor and Pensions Committee) said he hopes PDUFA V will reach Congress by year-end, so that his committee can mark up the legislation in the spring. Representative Joe Pitts (R, PA -- Chairman of the House Energy and Commerce Committee's health subcommittee.) said he hopes to have PDUFA V enacted by June 30, 2012.
Let the battle begin.
For another interesting view on the “deal on the table,” have a look at what Robert Metcalf, Vice President, Global Regulatory Affairs at Eli Lilly & Company, has to say. His comments can be found here.
Life imitates art. In Franz Kafka’s unfinished story, “The Trial,” a man is arrested and prosecuted by a remote, inaccessible authority, with the nature of his crime revealed neither to him nor the reader.
Well – it’s déjà vu all over again -- new Russian regulations making it harder for the Rodina to become an international destination center for clinical trial excellence. And nobody knows why.
As Dr. Meir Pugatch discusses in a new Journal of Commercial Biotechnology article, “The overall, the strength of national pharmaceutical IP environments provide a good estimate of the level of clinical trials taking place in these countries. Accordingly, countries with a more robust level of pharmaceutical IP protection tend to enjoy a greater level of clinical trial activity by multinational research-based companies. In other words, by choosing to improve their level of protection of pharmaceutical IPRs (together with other factors), developing countries may also be exposed to higher levels of biomedical FDI, not least in the field of clinical trials.”
(More on Dr. Pugatch’s article can be found here.)
This is bad news for Russia.
New data shows that clinical trial activity in Russia is still on the decline following the introduction of new regulations last year. The total number of trials approved in the first half of 2011 fell by 36% to just 200, with trials conducted locally by domestic sponsors most affected, falling by around 80%, according to the Russian clinical trials organization, ACTO.
The slowdown is mainly due to the law on circulation of medicines, which came into effect in September last year. Among other things, the law incorporated local clinical trials into the drug registration process, introduced new trial approval and insurance procedures, and imposed a ban on the import of registered products for use in clinical trials.
Not only is this bad news for international investment – but it also has the unfortunate side effects of making it more difficult for Russian companies to conduct clinical trials in their own country. Russian companies wanting to conduct local trials (as opposed to Russian arms of multinational studies) can do so only as part of a drug registration procedure, not as freestanding studies.
According to ACTO Executive Director Svetlana Zavidova, she did not know why this distinction had been made, but it was possible that those drafting the law had simply forgotten to state that local clinical trials were also a freestanding kind of study.
Kafkaesque – and with unfortunate results.
The number of local safety and efficacy studies approved in Russia fell by 78.4% to 19 in the first half of 2011, while bioequivalence studies were down by 84.4% to just seven. According to ACTO, the fall in bioequivalence studies is probably due to two factors: there are no approved requirements for such studies, and in some cases generic manufacturers cannot refer to the originator's pre-clinical trial results and are having to carry out their own tests, thereby delaying the bioequivalence study.
These developments are somewhat worrying given that the government has made much of its plans to build up the Russian pharmaceutical industry and reduce the country's reliance on imported medicines. "It is remarkable that the implementation of the law had a particularly tough impact specifically on the innovation activity of Russian companies, which is in direct contradiction with the governmental desire for import substitution in pharmaceuticals," ACTO commented.
“Remarkable?” Not really. Predictable? Absolutely.
On the positive side, sponsors of international trials fared better than their Russian counterparts in the first half of 2011, gaining 163 trial approvals compared with 160 in the 2010 period. Nonetheless, they had their own problems, including the lack of new rules on insurance, which should have been in place on 1 January this year, and the ban on importing registered drugs (for use as comparators in clinical trials of experimental drugs).
Bad News: The import ban affected not only new studies but also those already under way where stocks of the approved drug were running short, says ACTO, which notes that some new studies were cancelled and transferred to other countries.
Good News: The ban was finally lifted in June this year when the health ministry took over responsibility for the importation of both approved and unapproved medicines, while the insurance question was dealt with by a May decree implementing the amended rules.
Bad News: Another factor dampening trial activity has been the fact that the authorities are not adhering to the new, shorter timeframes for trial approvals stipulated in the new law.
In fact, according to ACTO, things are even worse than they were before the law came into force: in the first six months of 2011, the total time taken to gain approval for a trial and obtain import/export permits amounted to an average of 160 days. This is "twice as long as the maximum period allowed by law and 30.5% longer than the all-time worst results.”
Other problems remain, says ACTO, including the fact that foreign companies still have to conduct a local trial (free-standing or as part of an IMCT) when seeking new drug registration in Russia. "Unfortunately, this problem is still on the front burner for us", ACTO’s Zavidova commented.
That must be some big front burner.
Considering the importance of clinical trials as a leading indicator of international pharmaceutical investment, perhaps Moscow should seek expert advice on the subject. After all, as the Russian proverb says, “With a helper a thousand things are possible.”
Is 4% a lot or a little? Well – it depends whether you’re one of the 4%.
As the AP reports, “Pfizer Inc.'s just-approved drug Xalkori, the first new medicine in more than six years for deadly lung cancer, proves the value of precisely targeting rare diseases linked to gene variants
The drug was approved Friday in the U.S. along with a companion diagnostic test for just a small subset of lung cancer patients. It is the poster-child for the 21st century strategy of developing medicines for relatively few patients to replace (as the AP puts it) “blockbusters for the masses.”
"This is a paradigm shift," Dr. Paul A. Bunn Jr., a University of Colorado professor and cancer researcher involved in testing Xalkori, told journalists during a conference call hosted by Pfizer. "It used to be that everybody with cancer was treated the same," with surgery and chemotherapy.
Xalkori, a pill with relatively minor side effects compared to the hair loss and nausea that chemotherapy can cause, was approved for the roughly 4 percent of patients with advanced nonsmall cell lung cancer who have what's called the ALK fusion gene.
About 6,000 Americans a year develop this cancer, Pfizer said. Those patients, called ALK positive, now can be identified with a $250 molecular diagnostic test developed by Pfizer's partner, Abbott Molecular Oncology. The test was also approved Friday.
According to Dr. Mark Kris, head of the thoracic oncology service at Memorial Sloan-Kettering Cancer Center in New York, "You're going to be sparing individuals side effects (and) the waste of resources, time and drug that really isn't going to help them."
Cost efficient and patient-centric. Bingo.
Personalized medicine, targeted therapy – call it what you will. It’s real. And it’s a giant step towards achieving the 4 rights – the right medicine in the right dose for the right patient at the right time.
For those of you who missed Latin class that day, memento mori means, “remember your mortality.”
It seems that the American Medical Association has (albeit a little late in the game) woken up to that fact and gotten religion.
The AMA has invited other healthcare groups to sign onto a letter to the Patient Centered Outcomes Research Institute (PCORI) on the types of research that should and should not be conducted under ObamaCare. The AMA is concerned about its proposal to "investigate ... optimizing outcomes while addressing burden to individuals, resources, and other stakeholder perspectives."
They should have read the bill before they so aggressively supported its passage.
Why the turnaround? Could it be that America’s physicians worry that including cost in the equation will open the door to rationing down the line?
"We seek further clarification toward the Board's intentions regarding this last component and whether this includes cost analysis," states a sign-on letter scheduled to be sent later this week to PCORI executive director Joseph Selby. "If that is the case, we do not believe that it is consistent with the PCORI's enabling statute."
The issue, at its core, is physician disempowerment. With first insurance companies and now Uncle Sam telling doctors how to practice medicine (step therapy, restrictive formularies, more strident and cumbersome preauthorization requirements, and academic detailing), it’s no wonder that physicians are mad and that the AMA is doing a volte-face on PCORI and comparative effectiveness.
My good friend, Professor Meir Pugatch of the University of Haifa, is one of the world’s most respected experts in the role that robust intellectual protection plays in economic development.
In a new paper in the Journal of Commercial Biotechnology, Dr. Pugatch asks a crucial question -- To what extent does the strengthening of intellectual property (IP) environments in developing countries lead to greater in-flows of technology transfer in these countries?
According to Dr. Pugatch, there is a growing body of statistical evidence suggesting that a stronger IP environment does contribute to an enhanced level of foreign direct investment (FDI) and technology transfer in developing countries.
For example, an OECD study ï¬nds that in developing countries an increase of 1 per cent in the strength of patent rights resulted in 1.7 per cent increase in FDI flows, which in turn resulted in the transfer of know-how, that is innovative capabilities. Quite an attractive multiplier.
Here’s the official abstract:
The strength of pharmaceutical IPRs vis-à-vis foreign direct investment in clinical research: Preliminary findings
This article examines the effect of the intellectual property (IP) environment in developing countries on the level of foreign direct investment (FDI) and technology transfer occurring in the biopharmaceutical field in these countries. In particular, it considers the correlation between the strength of IP protection in several developing countries (using the Pharmaceutical IP Index) and the number of clinical trials taking place in these countries (as a proxy of biomedical FDI). The article finds that overall, the strength of national pharmaceutical IP environments provide a good estimate of the level of clinical trials taking place in these countries. Accordingly, countries with a more robust level of pharmaceutical IP protection tend to enjoy a greater level of clinical trial activity by multinational research-based companies. In other words, by choosing to improve their level of protection of pharmaceutical IPRs (together with other factors), developing countries may also be exposed to higher levels of biomedical FDI, not least in the field of clinical trials.
The full article can be accessed here.
As the recent Institute of Medicine report concludes the scientific evidence about vaccines benefits and microscopic risks is clear and convincing. Report: Vaccines Are Safe, Hazards Few And Far Between
Yet, politicians of all stripes have indirectly fed the the unfounded fear of parents who refuse to vaccinate, thereby endangering the lives of others. the Obama administration has ducked the issue of vaccine safety instead of addressing the issue clearly.. It has missed several opportunities to promote vaccine safety and have, in the past, caved into anti-vaccine forces. It has given money to anti-vaccine groups. At the same time it was unfortunate that Rick Perry backtracked on the issue of requiring HPV vaccination, not because mandatory vaccination is appropriate (it should be used sparingly) but because in doing so he did not reaffirm the importance of the vaccine in eradicating many forms of cancer that are increasing in prevalence. And Washington State, where parents are refusing to immunize their kids in record numbers, has buckled under pressure for anti-vaccine forces and failed to enact a law making it tougher to bail out of vaccinations. www.kvewtv.com/article/2011/aug/01/wa-leads-nation-parents-opting-out-immunizations/
For many reasons lots of politicians don't want to say they support a government requirement of any form. (The battle over the individual health mandate is a case in point.) And they don't want to confront parents who insist vaccines cause autism. But requiring people to be immunized is a way of protecting the freedom not to be exposed to vaccine preventable illnesses. Many, if not most, of those who are infected by vaccine choicers and their kids, are infants or immunocompromised children who rely upon others to protect them against disease and death.
Freedom is not the unfettered right to avoid the dangers dancing in your head at the expense of the health and life of others. Living in and benefitting from a free society requires not just meeting obligations but also abiding by norms of thought and collective action that comport with reason and science. Thomas Jefferson observed: "The value of science to a republican people, the security it gives to liberty by enlightening the minds of its citizens, the protection it affords against foreign power, the virtue it inculcates, the just emulation of the distinction it confers on nations foremost in it; in short, its identification with power, morals, order and happiness (which merits to it premiums of encouragement rather than repressive taxes), are considerations [that should] always [be] present and [bear] with their just weight."
Politicians and public health officials should stand up for medical science and not contribute to what the National Vaccine Advisory Committee calls "the culture of refusal."
They single out any relationship involving any resource or transaction that "may" have a monetary value exchanged between drug or device companies and academics.
But health IT companies, HMOs, trial lawyers, hospitals, government bureaucrats who hand out dough for comparative effectiveness research are exempt from oversight. Apparently there is no possibility for any undue influence from such entities.
The NIH are defensive about the impact these regs will have on innovation. And they should be.
"We want to emphasize that the revisions are not designed to prevent or hinder relationships among government, academia, and industry. Rather,
the revisions are aimed at facilitating such relationships by increasing transparency and accountability so that the resulting research is considered objective and in the interest of the public."
Given that most scientific misconduct and publication bias is a product of career climbing having nothing to do with industry, the emphasis on industry seems misplaced.
Moreover, who said that people are concerned about objectivity as defined by the Obama administration? One person's objectivity is another person's bias. And the new regs simple open up the process of biomedical innovation to more questions about conflict that have nothing to do with quality of the research -- ultimately measured by whether we can prevent or control diseases more effectively -- but everything to do with the cultural and ideological belief that commercialization inherently benefits large companies at the expense of the rest of us and that the commercialization process is inherently corrupt and corrupting.
The advocates of the regs claim that more oversight and investigation is needed because industry support for research has increased. Is it the industry support itself or the magnitude? Where is the evidence that more money means more corruption? Isn't the opposite outcome as likely: That is, people with a financial stake in the outcome of a research are more likely to be more scrupulous about research to protect their investment? Loss of reputation is a huge price to pay for fudging data for starters. Do the NIH reg advocates believe that industry funded scientists are more likely to lie about the safety and effectiveness of new products even at the expense of harming people?
The answer is yes. This culture of mistrust would have ensnared and persecuted the following individuals who had -- under the new guidelines -- the potential or actual presence of a financial conflict:
Louis Pasteur -- wine and beer maker and recipient of wine industry money
Gertrude Elion
George Hitchings
Sir James Black
Craig Zello
Phil Sharp
Joseph Goldstein
SIr Peter Mansfield
Judah Folkman
Most are Nobel Prize winners. All used industry money to commercialize devices and medicines that have extended and improved life. But according to the conflict of interest priesthood, the research they produced was tainted and therefore the results could be not trusted. They would be required to "report the name of the company or entity in which there’s a conflict of interest, the value of the interest, why it’s a conflict and “some key elements” of how the institution plans to manage the conflict."
blogs.wsj.com/health/2011/08/23/new-nih-conflict-of-interest-rules-better-than-the-old-rules/
Note there is no room for arguing there is no conflict. It's a confict. Acting as if it isn't is a grounds for investigation. Your only option is to tell the world just how guilty are are or appear to be. This makes a forced confession easy by comparison.
Meanwhile Andrew Wakefield, who received cash from trial lawyers and his own patients, would be exempt not only from the new NIH rules but the suspicion of the COI kapos.
Ditto the groups who pushed for this sort of witchhunt. Money from George Soros, the IOM (a conduit for ideologically tilting foundations) and others are exempt too.
The new rules are the culmination of an ideological assault by those who believe commercialization is inherently corrupt and corrupting and who ignore the fact that as a result of such innovation, life expectancy and death rates from cancer, heart disease and other illnesses have declined in direct relation to the industrial investment in treatments for death and disease.
Google Inc. has agreed to pay $500 million for carrying advertisements by online Canadian pharmacies targeting consumers in the United States, according to the U.S. Justice Department.
The ads resulted in the illegal importation of prescription drugs, the Justice Department said.
That's a big oops.
The $500 million represents the money Google made from selling the drug ads, plus the revenue earned by Canadian pharmacies from sales to American customers.
As an FDA Associate Commissioner said back in 2003 (on the front page of the Washington Post), "You can't blame them (Google) for accepting commerce. But they really haven't understood the consequences."
They do now.
Who was that prescient FDA guy?
For the answer to that question and further background on this story, see here.
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.
