Latest Drugwonks' Blog
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/
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.
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.”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.
“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.”
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.
BioCentury reports that the FDA has dentified eight priorities in the Strategic Plan for Regulatory Science: preclinical toxicology; personalized medicine; manufacturing; evaluation of emerging technologies; using information sciences to improve health outcomes; food safety; medical countermeasures; and communication with consumers and professionals.
The agency is seeking to improve the prediction of product safety through new animal models that consider both disease progression and co-morbidities. The plan also calls for FDA to collaborate with academia, industry and global regulatory agencies to develop and refine clinical trial designs. Such a collaboration would identify and evaluate improved endpoints and related biomarkers for areas where "optimal" endpoints are lacking, such as for osteoarthritis, gene therapy, ophthalmic indications, tumor vaccines and stem cell-derived therapies
CDRH has released two draft documents on approval process and guidance on its evolving approval process and planning clinical trials.
The first document explains the Center’s procedures for approving diagnostic and therapeutic devices -- how it weighs benefits and risks, judges the seriousness of a disease or condition, estimates how many people would use a device, and gauges availability of other devices for the same condition.
“Availability of other devices?” Really? Shouldn’t Shuren and Company stick to safety and efficacy? Where’d this third leg come from?
Sounds like insidious mission creep of the comparative effectiveness variety.
The second document explains the agency's expectations for medical device clinical trials, and suggests companies pre-submit protocols for the design of pivotal studies. Both drafts are open to public comment for 90 days, beginning August 15.
The IRS has "released what it's calling “temporary regulations” under which brand-name drugmakers must pay fees "to help fund" health-reform law provisions.
This was, as you will recall, part of “the deal.”
The regulations request payments of $2.5 billion in 2011 and are due by September 30th. Additionally, IRS calls for $2.8 billion in each of '”fee years” of 2012 and 2013; $3 billion for fee years 2014 through 2016; $4 billion for fee year 2017; $4.1 billion for fee year 2018; and $2.8 billion for fee year 2019 and thereafter.
So what’s “temporary?” And what about those who are pounding the podium about rebates for dual eligibles?
Just asking.
According to reporting in Congressional Quarterly, although pharmaceutical companies have to "pay fees," they will garner "far more sales as millions of the uninsured gain coverage."
The requirement applies to companies with annual drug sales of "$5 million or more" to Medicare "Parts B and D"; Medicaid; and Veterans Affairs, and Defense Department and TRICARE programs.
All of a sudden the Mainstream Media (highlighted by a much ballyhooed New York Times Week in Review op-ed by Zeke Emanuel) has discovered the problem of drug shortages.
Except it’s not a new problem.
According to a recent analysis, the frequency and impact of drug shortages have risen to critical levels, more than tripling since 2005, and affecting all segments of the healthcare community. In 2010, over 240 drugs were either in short supply or completely unavailable and more than 400 generic equivalents were backordered for greater than five days. In most instances, these did not progress to critical shortages, but point to instabilities in the supply chain that cause national concern. Many of the drugs identified in 2010 remain unavailable or in short supply in 2011.
Seventy-seven percent of drugs in short supply in 2010 were sterile injectable products, critical in the acute care setting. Recent media coverage highlights the plight of patients and physicians faced with shortages for cancer drugs, anesthetic agents, and critical care medications that have contributed to delays in treatment and surgery, or changes in care plans. Drug backorders cause patients to receive substitute therapies that add expense to patient care.
A major reason for shortages is quality/manufacturing issues. However there are other reasons such as production delays at the manufacturer and delays companies have experienced receiving raw materials and components from suppliers. Discontinuations are another factor contributing to shortages. (The FDA can't require a firm to keep making a drug it wants to discontinue.)
It is time not to fix the blame – but to fix the problem.
Let’s start with the FDA.
In 2010, there were 178 drug shortages reported to the FDA. Is that a solid number? Hard to say, because current regulations do not require companies to notify FDA of shortages. The only requirement is that companies inform FDA six months in advance for discontinuations of sole source, medically necessary drugs.
(In 2010, for example there were 38 shortages prevented due to companies notifying FDA voluntarily of potential issues that could lead to shortages and FDA was able to work with the company to avoid a shortage.)
Inside the FDA’s Center Drug Evaluation and Research (CDER) resides the agency’s Drug Shortage Program (DSP). The DSP was established to address potential or actual shortages of drugs that have a significant impact on public health. Through communication, facilitation and negotiation, DSP works with pharmaceutical manufacturers, review divisions, compliance and other components of FDA to manage product shortages.
For example, when the drug shortage is for a generic product (as it often is), the FDA works with other firms making the drug to help them ramp up production if they are willing to do so. Often they need new production lines approved or need new raw material sources approved to help increase supplies. FDA can and does expedite review of these to help resolve shortages of medically necessary drugs.
But the FDA can't require the other firms to increase (or commence) production.
The agency tries to do the best it can with limited authority, spare resources and shared staff. In addition to direct communication with industry, the DSP also gets reports from healthcare professionals, patients/individuals, or professional organizations using the e-mail address drugshortages@fda.hhs.gov.
But, as you can imagine, there is not a lot of e-mail traffic. And there is no social media effort to promote either its purpose or existence.
It’s a good start – but it’s not enough. And you know what they say about good intentions.
The FDA’s Drug Shortage Program is a good start. But it’s not getting the job done. The problem is getting worse. The FDA needs both more authority and greater resources. In short, more needs to happen.
The Institute for Safe Medication Practices reports that, according to survey of 1800 healthcare practitioners, more than half of respondents frequently or always encounter difficulties associated with drug shortages.
The top three problems fall squarely within the zone of appropriate FDA attention and action:
* Little or no information available about the duration of a drug shortage (85%)
* Lack of advanced warning from manufacturers or FDA to alert practitioners to an impending drug shortage and suggested alternatives (84%)
* Little or no information about the cause of the drug shortage (83%)
Survey respondents felt “unsupported by the FDA and are perplexed regarding why the US is experiencing drug shortages of epic proportion that are often associated with third-world countries.”
(More detail on the ISMP survey can be found here.)
And before it becomes a question of American lives.
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.
