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When I was at the FDA, we made a conscious effort to stop referring to “compassionate use” because it made it sound like “noblesse oblige.” We began to call it what it should be – expanded access.
In a house editorial, the New York Times misses the mark not only on this point (where they refer to “humanitarian use” – a nonsensical misnomer), but also as it relates to “comparative effectiveness.”
The Gray Lady opines, “This case raises the question of whether the F.D.A. should demand more rigorous trials before a device is granted a humanitarian exemption. It clearly shows the value of conducting rigorous controlled studies with enough patients to provide meaningful results. This is just the kind of comparative effectiveness research that the national health care reforms seek to promote.”
Well, yes and no.
Sure, the FDA should always strive to better understand where expanded access programs may lead. (And, it’s important to note, many patient groups – such as the Abigail Alliance – believe the FDA is already too slow and stingy with such protocols.) But even the best folks at the FDA are only so prescient. I think it wise to give the folks at White Oak the benefit of the doubt. Adoption of the Precautionary Principle (where nothing is done until everything is known) only leads to nothing being done and the death of innovation.
And as far as the “comparative effectiveness” statement is concerned, this situation has nothing to do with it whatsoever. A larger scale trial of these stents uncovered the safety problem -- precisely what such trials are designed to do. It has nothing to do with "comparative effectiveness." When it comes to the FDA -- it's about safety and efficacy.
But when you’ve got a hammer, every problem looks like a nail.
Read More & Comment...First it was gun slinging against Forest Labs, now the HHS OIG is gunning for price controls.
According to BioCentury, the OIG has recommended that the Obama administration seek authority from Congress to more effectively control Part B drug and biological expenditures. The recommendation came in a report released Wednesday documenting the difference in acquisition cost for Lucentis ranibizumab from Genentech Inc. for wet age-related macular degeneration and Genentech's Avastin bevacizumab, which is used off-label in the indication as a cheaper alternative.
The report said using Avastin instead of Lucentis for wet AMD would have saved Medicare Part B $1.1 billion and beneficiaries $275 million in copayments in 2008-09. In those years, HHS OIG said the average sales price for a dose of Lucentis, a mAb fragment against VEGF-A, was about $1,915 compared to about $7 for an intravitreal dose of cancer drug Avastin, a humanized mAb against VEGF.
The report noted that CMS, which reimburses for both drugs for AMD, does not have the authority to require price concessions or rebates for products covered under Part B. In a written response including in the report, CMS said it is "evaluating our current authorities and will seek additional authorities as necessary."
Genentech said in a statement it will not comment until it finishes reviewing the report. The company added "we do not believe that cost should be the only factor considered when choosing a medicine."
Read More & Comment...In fact, he made the most sense science-wise of all the candidates.
1. He did not back down on the need or requirement to have children receive a vaccine that can eliminate many forms of cancer. I thought it was a courageous and principled stand when he first called for HPV immunizations for all 12 year old girls. By contrast other presidential candidates -- Bachman, Santorum and Ron Paul in particular -- seem to suggest that parental rights trump immunization requirements in every case. If that is so, then we need to ask these candidates if they oppose immunization requirements for children and if they believe vaccines cause autism. Then we will see who is anti-science.
2. Perry did more to advance medical science in Texas than other governors running for President have done. Not only did he lead in the establishment of Cancer Prevention and Research Institute of Texas (CPRIT), a $3 billion, 10-year cancer research fund, Texas in one. Just recently CPRIT recruited a leading stem cell researcher to establish a pediatric cancer initiative at University of Texas (UT) Southwestern Medical Center at Dallas. The researcher, Sean Morrison, said this about Texas:
While I have been spending the last five to six years arguing with the Legislature about what kind of research would be permitted in the state, in Texas they were looking for ways to invest billions of dollars into medical research..."Texas is clearly an environment that's more supportive generally of research innovation. Three billion [dollars] for cancer research is going to change the landscape."
PS. Perry has never supported proposals to ban stem cell research in Texas either.
Ignoring these aspects of Governor Perry's record while questioning his position that the 'science' behind predictions of global disaster and requires massive government intervention in the economy -- which is what the debate is all about -- is anti-science is simply a tactic to silence that individual. Or make him or her look stupid.
I still would like to know if the candidates who criticized Perry are opposed to mandatory vaccination in any case.
Read More & Comment...
Read More & Comment...
"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.”
Read More & Comment...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.
Read More & Comment...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.
Read More & Comment...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."
Read More & Comment...
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.
Read More & Comment...
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.
Read More & Comment...
Guidances are okay but they are no substitute for less regulation and more scienced-based common sense, as evidenced by this account of how farmers may soon have to regulate...dust.
blog.heritage.org/2011/08/20/president-obamas-regulatory-dust-up/
Read More & Comment...
Call it Obamacare's Final Destination Tour. While President Obama rests up at Martha’s Vineyard, people whose lives should be saved by new and existing cancer drugs are driving from hospital to hospital in search of medicines in short supply thanks in part to Obamacare’s implementation.
Over the past two years shortages have developed for over 180 drugs, including cancer treatments. The shortfall is the result stricter FDA regulation, government price controls on already discounted but complex drugs, and policies that discourage the use of new medications. Companies, facing lower prices, tighter regulation and increasing government control over what drugs will be used when, are exiting the US market and investing in product development in china and India where, sadly, it is easier and cheaper to produce next generation medicines than in America.
Stockpiling will only add to people's suffering by replacing market reforms with government micromanagement. Government planners require months, if not years, to produce regulations, bids and supply estimates that are usually overgenerous to compensate for paltry prices. Government bungling was behind the failure of the smallpox and H1N1 vaccine program and responsible for billions of dollars in flu vaccines and antibiotics being dumped.
The same forces pushing stockpiling also believe commercializing medical discoveries is evil. It’s part of a larger effort to nationalize the development of medicines that under Obamacare is become institutionalized
Indeed, the drug shortage is a product of a more troubling trend: At a time when medical research could yield breakthroughs in the treatment of obesity, Alzheimer's, diabetes, stroke among others, innovation has all but dried up. Most of the medicines being used today were developed 30 years ago. Most of them have generic competition. They have contributed greatly to increased wellbeing but as the return on generic drugs fall, price controls and regulation have created shortages.
Obamacare is making the commercialization of newer drugs and devices more difficult.
Though new and faster methods to determine a technology's safety and effectiveness exist, Obama’s FDA still demands evidence collected with science and statistical methods developed in the 19th century. To be sure, in the last two years new medicines for AIDS, cancer, lupus and hepatitis have been developed. Yet, these products should have been available sooner if not for FDA nitpicking.
And now that they are finally approved, patients are finding it next to impossible to access several new drugs and genetic tests that would transform the quality of life and extend survival for such illnesses as lupus, prostate cancer and organ transplantation.
Provenge, the first cancer vaccine, stalled at the FDA for years. Once approved, it faced 18 months of additional delay while the Obama administration figured out whether to pay for it. The gauntlet cancer patients face with Provenge is being extended to everyone waiting for a medical breakthrough under Obamacare: Before a medical innovation can be used or paid for the government will now demand additional research demonstrating that a new product will be more effective and cheaper than existing technologies. Since most new products come from small start-ups with limited cash, such a requirement means live saving innovations will not be available at all.
Similar regulations have been used to delay and deny access to cancer drugs in England, Canada and Australia. Drugs such as Avastin, Revlimid and Herceptin are barely used in Britain's cancer wards because government decided they were not valuable. The people who could not use them are dead. Those -- mostly in America -- that did are alive.
But now this de facto rationing is coming to America. Before the cancer drug shortage there was the decision that women under 50 should not get mammograms. Both Provenge and Benlysta, new treatments for prostate cancer and lupus respectively, are hard to come by because of uncertainty about reimbursement by health plans and government. .
The death and suffering flowing from such delays are the result of policies promoted by those who want to use the FDA and increased government control over medicine to slash access to new technologies. In their mind, rationing of cancer drugs frees up money to expand the welfare state. The shortage of old drugs is simply one side effect of this malevolent strategy.
It would be simpler to claw back regulations and let markets work. But stockpiling is part of a larger effort to centralize the development and use of medical services. The administration has gone silent on the wonders of obamacare even as it issues regulations and hires bureaucrats to replace free choice with government edict. But it will emerge as a campaign and social issue. In America no one should go without medicine because they can't afford it. And it shouldn’t be a nation where people are denied such treatments because their government makes medicine impossible to produce or obtain.
Read More & Comment...I am currently attending (and speaking at) the Technology Policy Institute’s annual Aspen Forum. It’s refreshing to listen to presentations on issues other than healthcare. But, on the other hand, there are a lot of parallels.
One disturbing similarity was brought to mind by a talk given by FCC Commissioner Robert McDowell. The topic was regulation of the Internet. McDowell commented that he wasn’t sure when the question of “Should the Internet be regulated?” morphed into “How should the Internet be regulated?
And he was very clear that this was a disturbing “iterative change.”
Sound familiar? We should be asking ourselves the same thing about comparative effectiveness. When did “Should we adopt a national comparative effectiveness approach to healthcare?” morph into “How should we adopt a national approach to comparative effectiveness?”
There’s still time and opportunity to robustly debate the former so that we can avoid the latter.
We can, in the words of Commissioner McDowell, “avoid a race to the bottom.” Read More & Comment...The Generic Pharmaceutical Association never misses an opportunity to miss an opportunity. They are balking at continuing to negotiate the design of a biosimilar user fee program that is independent of other user fees and has its own baseline appropriations trigger.
The disagreement stems from a proposal by GPhA and a representative of potential biosimilar sponsor Momenta Pharmaceuticals that biosimilar reviews remain part of the Prescription Drug User Fee program through fiscal year 2017, the end of the first biosimilar program cycle, according to minutes of a July 18 meeting.
The tactic could be a way to lower the cost of a biosimilar application. If the agency is forced to include biosimilars in the PDUFA program, it likely would have a more difficult time arguing it needed a strong funding base to ensure the program functions properly.
GPhA said it could not continue “detailed negotiations regarding volumes and metrics without agreement to parity with the PDUFA user fee program.”
The position would seem to reverse one taken during a July 11 negotiation. Minutes indicated “industry stakeholders” and FDA agreed to support a flat product development fee of about 10% of the marketing application fee for the fiscal year while a biosimilar product was in the IND stage.
It is unclear whether FDA could send a commitment letter to Capitol Hill for approval without support from a major trade group involved in the negotiations.
If FDA went ahead with a commitment letter for a separate biosimilar user fee program and GPhA wanted to press the issue, the generic group would presumably need to convince legislators that its interpretation of the 2007 bill creating the new approval pathway was correct.
Read More & Comment...“Facts are stubborn things” – John Adams
Médecins Sans Frontières (MSF) should be called Médecins Sans Facts.
MSF reports (correctly) that “while countries are rolling out new tests that will enable them to diagnose more patients with drug-resistant tuberculosis (DR-TB), a worldwide shortage of the drugs to treat these patients is likely.”
Not quite.
The news item generating this story comes from South Africa, which has the world's fifth-largest burden of multi-drug resistant (MDR) TB cases. According to Norbert Ndjeka, director of DR-TB, TB and HIV at the South African National Department of Health, the government will replace all microscope-based TB diagnoses with faster, more sensitive GeneXpert testing within two years, making it the world's largest user of the machine.
Good news. But here’s the MSF spin:
But while more patients may get diagnosed, their access to treatment remains precarious as a limited number of approved drug producers keep many DR-TB prices high and supply uncertain, according to Dr Eric Goemaere, MSF's senior regional adviser.
It’s the familiar refrain from our friends at MSF: Blame industry! Blame international intellectual property (IP) protection! Convenient and consistent – and untrue. Médecins Sans Facts.
The truth of the matter is that the fault for shortages lies with all too familiar misguided government policies – in this case the government of South Africa and their myopic focus on lowest price. Combined with a concerning lack of transparency, utter unpredictability and the exploding processing costs of their regulatory system, it all adds up to delays and drug shortages of not just medicines for TB – but for many other critical healthcare products.
Mr. Ndjeka has admitted that South Africa's regulatory Medicines Control Council (MCC) has been laboring for years under a backlog in drug registrations, including those of some fixed-dose antiretrovirals long available in other nations. South Africa now pays a private company to import the drug through a special application to the MCC.
So what does MSF recommend doing beyond their criticism of IP and the innovator pharmaceutical industry? Ignoring Economics 101, they recommend lowest cost "access to essential drugs" -- which just further exacerbates the medicines supply problem.
Médecins Sans Facts continues to call for countries to avert the looming crisis by “improving drug forecasting, negotiating better prices and accelerating national medicines registrations.”
As Mark Twain said, “Get your facts first, then you can distort them as you please.”
It would be helpful if groups like MSF took a longer term view -- encouraging multiple suppliers of medicines through more stable and predictable pricing versus their usual and customary (and wrong) focus on lowest cost. If MSF really wants to be part of the solution, they should consider combining efforts with industry to help streamline the complicated and costly regulatory process in South Africa (and in many other nations of the Developing World). Harmonizing regulatory practices to align with evolving global standards (as called for recently by US FDA Commissioner Dr. Margaret Hamburg) and pressing domestic regulatory officials to respect timelines and transparent performance criteria, would make a huge difference for patients in countries like South Africa.
Why can't MSF advocate new drug approvals in places like South Africa within 90 or 180 days for products already approved leading regulatory agencies in Europe, Japan, and North America? Should South African patients really wait 2-5 years for access to promising new medicines that are already licensed for use in dozens of other countries? Where is the activist outrage about these inexcusable regulatory delays?
Perhaps a better question (at least for starters) is why won’t MSF and their fellow travelers opt for a more collegial and logical agenda?
Perhaps the answer to that question was best addressed by George Orwell who wrote that “All political thinking for years past has been vitiated in the same way. People can foresee the future only when it coincides with their own wishes, and the most grossly obvious facts can be ignored when they are unwelcome.”
Read More & Comment...When I served at FDA we signed an MOU with PhRMA that required its member companies to notify the agency when instances of counterfeit medicines came to the attention of manufacturers.
It got the job done – minus additional federal regulation.
Today we face a different problem – drug shortages – but a similar decision tree: regulation or public-spirited public health cooperation.
Minus the latter, we will get the former.
Senators Amy Klobuchar (D, MN) and Bob Casey (D, PA) have introduced legislation to require drug manufacturers to give “proper prior notification” to FDA when there is going to be a drug shortage.
Further, Klobuchar (D, MN) and Richard Blumenthal (D, CT) have formed a bipartisan group to work on the issue of drug shortages.
At their request, the FDA will hold a hearing on the matter September 21, and the General Accounting Office will review the problem.
It’s time for FDA and PhRMA to get the job done.
Legislation takes longer and leads to mission creep.
Read More & Comment...
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