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The Food and Drug Administration is studying whether disclosure limited only to serious side effects would improve consumer understanding, according to an agency document. To cover lesser side effects, the FDA proposed simply adding a line about “potential additional risks.”
“Our hypothesis is that, relative to inclusion of the full major statement, providing limited risk information along with the disclosure about additional risks will promote improved consumer perception and understanding of serious and actionable drug risks,” the FDA said in its document.
The FDA plans to survey 1,500 study participants about ads with varying ranges of side-effect disclosure, and then measure their understanding of risk.
The underlying problem is that risk information is hidden in plain sight by not being in plain English. Risk information is neither designed nor delivered to be user-friendly. At present it is designed to be “in compliance.” And that has to change.
When it comes to DTC print ads, the joke inside the FDA (and in many regulated industry review offices) is that the Brief Summary is like the Holy Roman Empire – it is neither brief nor a summary. So, when it comes to TV ads, hopefully the agency is asking the right questions. Fair Balance and Adequate? “Fair” for whom and “Balanced” how? Adaquate Provision? Doesn'tlook like it.
Working together, the FDA and industry can make a difference. DTC can be a more potent, precise, and persuasive tool on behalf of the public health. And rather than rubbing the lamp and wishing, we need to burn the midnight oil and work harder to make it a reality—because an educated consumer is not only a better customer, but a more compliant and adherent patient.Read More & Comment...
FDA Commissioner Peggy Hamburg visits India and confirms that honesty is the best policy. The feeling is not entirely reciprocated.
It boils down to a simple, foundational question: Can there be more than one global standard of pharmaceutical quality?
“If I have to follow U.S. standards in inspecting facilities supplying to the Indian market,” G. N. Singh, India’s top drug regulator, said in a recent interview with an Indian newspaper, “we will have to shut almost all of those.”
Does that mean US standards are too high or that Indian ones are too low? Well, where you stand depends on where you sit. And if you’re sober and sitting up straight, the answer is obvious.
According to a May 2012 article in The Lancet,
“To say that India's drug regulatory authority, the Central Drugs Standard Control Organisation (CDSCO)-whose remit includes new drug approval, licensing of manufacturing facilities, and regulation of drug trials-is not fit for purpose seems a gross understatement.”
The CDSCO has a staff of 323, about 2 percent the size of the FDA, and its authority is limited to new drugs. According to a report in the New York Times, “The making of medicines that have been on the market at least four years is overseen by state health departments, many of which are corrupt or lack the expertise to oversee a sophisticated industry. Despite the flood of counterfeit drugs, Mr. Singh, India’s top drug regulator, warned in meetings with the FDA of the risk of overregulation.”
Per the Times, “This absence of oversight, however, is a central reason India’s pharmaceutical industry has been so profitable. Drug manufacturers estimate that routine F.D.A. inspections add about 25 percent to overall costs. In the wake of the 2012 law that requires the F.D.A. for the first time to equalize oversight of domestic and foreign plants, India’s cost advantage could shrink significantly.”
This profits-over-patients philosophy is entirely consistent with earlier “passing the rupee” comments of India’s Deputy Drug Controller, S. Eshwar Reddy.
When asked if Indian manufacturers -- which produce more than 40 per cent of the API used in the US and Europe -- should be more sympathetic with Western guidelines and regulations, Reddy said the opposite should be true.
He said any additional requirements made are the sole responsibility of the authority that issues them.
“If the importing country has specific GMP requirements, that is their responsibility to audit the facilities. It is the responsibility of the importing country not the exporting country.”
Per the Times, “The unease culminated Tuesday when a top executive at Ranbaxy — which has repeatedly been caught lying to the F.D.A. and found to have conditions such as flies “too numerous to count” in critical plant areas — pleaded with Dr. Hamburg at a private meeting with other drug executives to allow his products into the United States so that the company could more easily pay for fixes. She politely declined.”
How do you say chutzpah in Hindi?
Or Chinese? Or Arabic? Or Russian? Or Zulu?Read More & Comment...
New research conducted by CVS Caremark and Brigham and Women's Hospital (and published online in Health Affairs) identifies five key features of popular Value-Based Insurance Design (VBID) plans that are associated with the greatest impact on medication adherence. The study, which will also appear in the journal's March issue, was funded by a grant from the Robert Wood Johnson Foundation's Changes in Health Care Financing and Organization (HCFO) Initiative.
The philosophy is sound – but the factors are a bit fuzzy. And this isn’t surprising as the VBID model is still in in its design infancy.
Rather than encouraging patients to actively consider and bear the cost of prescription medications via copayments, co-insurance and deductibles, VBID plans reduce the cost to the patient for medications that offer higher clinical benefit.
The theory is that using medication that actually improves health outcomes will reduce overall health care spending. Correct! For example, patients in a VBID plan who have a chronic disease such as high blood pressure would have their out-of-pocket costs (e.g., copay) significantly reduced or eliminated for essential medications to treat their condition.
This is important – but it’s not new. As I wrote almost exactly four years ago in an op-ed:
Over the past several years, insurance companies have become increasingly reluctant to foot the bill for brand-name medications. Indeed, since 2000, co-pays have increased four times faster than prescription drug prices.
Patients respond to higher co-pays by skipping their meds more often. In 2003, researchers at the University of Oregon studied the effects of introducing a $2 to $3 co-pay for prescription meds among 17,000 patients. Adherence to treatment dropped by 17 percent.
Some insurers are even refusing to cover new prescription drugs. According to a study from Wolter Kluwer Health, insurers’ denial rate for brand-name meds was 10.8 percent at the end of 2008 — a 21 percent jump from the year before.
Abandoning treatment — a practice known as "non-adherence” — has serious consequences for patient health. For instance, people with hypertension who neglect their meds are more than five times more likely to experience a poor clinical outcome than those who don’t. Heart disease patients are 1.5 times more likely.
It also results in higher medical costs, as patients who go off their meds often end up in the hospital. Minor conditions that might have been controlled by inexpensive medications can sometimes balloon into life-threatening illnesses that require surgery or other costly treatments.
This makes sense. After all, a daily cholesterol-lowering drug is far less expensive than emergency heart surgery.
(My complete op-ed can be found here.)
Now, as to the “factors.”
According to Niteesh Choudhry, MD, PhD, associate physician, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital and associate professor, Harvard Medical School and the lead author of the study, "The results show that several specific features can improve adherence from between two to five percentage points and this information can help influence how future copayment reduction plans are structured for optimal benefit."
The researchers evaluated 76 VBID plans provided by CVS Caremark to 33 unique plan sponsors and involving more than 274,000 patients. Based on the analysis, five key features were found to have a greater impact on adherence. These included:
- More generous VBID plans (e.g., those plans that had no cost-sharing for generic drugs and low monthly copayments of < or = $10 or co-insurance rates of < or = $15 for brand-name medications),
- Plans that targeted high-risk patients,
- Plans that had concurrent wellness programs,
- Plans that did not have concurrent disease management programs, and
- Plans that made the benefit available only by mail order, offering 90 day prescriptions.
That’s a lot of work by a lot of smart folks to develop some pretty fuzzy factors. But it’s a good start.
As for the future, your task is not to foresee it, but to enable it.
-- Antoine de Saint-Exupery
Conflict of Interest (COI)? Only for thee but not for PCORI.
From the pages of Current Medicine:
The Patient Centered Outcome Research Institute (PCORI) was created by the PPACA “ObamaCare” law in 2010. Well-funded with approximately $3 Billion over ten years, the mission was, among other things, supposed to be to conduct comparative effectiveness research (CER) that would determine whether costly therapies are any better than cheaper alternatives.
The rise of CER has been one of the most feared developments by the drug and device industries. To avoid powerful lobbying efforts that could have resulted in de-funding and the death of PCORI before it got started, the institute steered away from even hinting at conducting CER. Now, almost four years later, PCORI is finally funding CER research. However, critics, such as former White House Director of Office of Management and Budget, Peter Orzag, say that the money spent by PCORI on CER is still not enough.
Meanwhile, a leading doctor in charge of PCORI research strategies, Harlan Krumholz, MD, a cardiologist at Yale, helped create The Yale University Open Data Access project, or “YODA”, and is the Principal Investigator. The YODA mission is to make clinical trial data more open and accessible to researchers.
Medtronic was one of the first corporations to pay YODA to analyze clinical data on the spine fusion growth factor product called InFuse. Recently, Johnson and Johnson (JNJ) signed up as the next large corporate client of YODA.
The drug and device industries have long been adept at muting the regulatory influences of healthcare agencies, such as the FDA, AHRQ, and CMS, using a variety of tactics. The “golden revolving door” hires former government regulators into lucrative jobs at JNJ, etc. Also, academic medical center “institutes” designed to keep the industry honest then become corrupted by accepting industry funding.
Since Dr. Krumholz is the senior clinical decisionmaker for both the Yale YODA, and the ObamaCare-created PCORI, conflicts of interest would arise if the industry began funding YODA. For examples, PCORI could be conducting CER on the various hip implant products and conclude that the JNJ DePuy device is no better than a less costly hip implant, or a Medtronic coronary stent might be found by PCORI research to be harmful and ineffective. But if Medtronic and JNJ began to pay millions of dollars to Yale’s YODA, then Dr. Krumholz would have a significant biasing force against initiating any CER at his other job, PCORI, that might harm sales of JNJ or Medtronic products.
In a brief interview with Dr. Krumholz about how he is managing these conflicts of interest, he replied, “Right – (there is) no direct connection (between YODA and PCORI research)– just a potential COI. All I can say is that it is disclosed. And I note that PCORI has people with different relationships. And that most of what PCORI does is in public view so people can assess for themselves.”.
Dr. Krumholz is correct in pointing out that many other members of the PCORI executive team and board have reported conflicts of interest accepting financial support from drug and device companies. We will have more on that in future reports.The Executive Director of PCORI, Joe Selby, MD PhD, did not reply to our emails in time for this publication. Read More & Comment...
Think governments can set prices and interfere in markets with no consequences? Think again.
According to a just released analysis by the United States Government Accountability Office, the FDA is doing a better job preventing drug shortages.
Per CDER Deputy Director, Doug Throckmorton’s Congressional testimony, new shortages declined in 2012 for the first time in a number of years and 2013 data indicated a similar downward trend. He said the agency’s new authorities (granted under a 2012 law) have allowed the FDA to manage shortages more aggressively.
In addition to new actions, the FDA has been more willing to demonstrate regulatory flexibility.
For example, in some cases where particles were found to be contaminating a drug that was in short supply, the agency allowed the company to filter the drug to avoid disrupting supplies instead of shutting down the production line altogether.
But, according to the New York Times,
Economic factors are also a contributing factor. Narrow profit margins are making some drug companies reluctant to invest in fixing old production facilities. Changes in Medicare reimbursement and the role of group purchasing organizations, which buy drugs on behalf of hospitals, could also be contributing, by further reducing prices that producers get for the drugs.
(This is almost a direct citation from the June 2012 Center for Medicine in the Public Interest, "Fixing Drug Shortages.")
While the FDA’s new authorities are both timely and important, there are many pieces to the drug shortages problem – not the least of which is that (when it comes to hospital injectables) 30% of manufacturing capacity is off-line due to FDA inspection issues.
That’s a lot of capacity. In fact, according to the agency, 43% of reported potential shortages were due to manufacturing problems. Safety is non-negotiable and alleviating a shortage by shorting GMPs is a bad and dangerous pathway. Expediency causes as many problems as it solves.
That being said, regulatory discretion must be part of the solution – and per Throckmorton, it is. With 30% of production capacity off-line because of FDA issues, the agency must continue to work with manufacturers to find creative, science-based solutions. If you create a "science- and risk-based action plan," industry can often address quality issues without disrupting supplies of essential drugs.
But who inspects the inspectors? Per that 30% of manufacturing capacity off-line due to FDA issues, perhaps the FDA should undertake an agency audit to see why there’s been such a jump in GMP issues. It’s hard to believe that year-over-year, production quality control has suffered such a significant lapse. Is there something wrong in the way FDA inspectors (many of them still wet behind the ears and eager to please) are doing their jobs? It’s a question worth asking – and answering.
But the real headline (alluded to but not directly addressed in the New York Times article) is that artificially low prices are the major cause drug shortages.
Most of the drug shortages that occur in the U.S. arise in the generics market, where profitability is fairly low For many of these drugs the market can only sustain a handful of manufacturers -- sometimes just one or two. So, when supply disruptions occur -- caused by manufacturing violations, production delays, shipping problems or ingredient issues -- there aren't a lot (or in many cases any) additional producers in the market to pick up the slack.
But the key factors behind drug shortages are perverse economic incentives. Consider the October 2011report by the US Department of Health & Human Services, Economic Analysis of the Causes of Drug Shortages.
HHS (the Obama HHS) mostly blames a dysfunctional marketplace for drug shortages. In fact the HHS report does more than blame a 'dysfunctional market', it explains what is behind the problem:
"...drugs that subsequently experienced a shortage are those in which the volume of sales was declining in the 2006-2008 period prior to the shortages."
It goes on to note that, "Analysis of average sales prices shows that shows that oncology sterile injectable drugs that experienced shortages since 2008 decreased in price from $56.17 per unit in Q1 2006 to $37.88 per unit in Q1 2011. Oncology sterile injectable drugs that have not experienced shortages have had relatively stable prices over this period."
In plain English: artificially low prices caused the manufacturing decline of the drugs that are in shortage and a variety of perverse government regulations (ranging from Medicaid reimbursement rates to the benighted 340B program) are causational.
Where there is still a profit left, you rarely see shortages.
Congrats to the FDA for a job well begun. But the issue of drug shortages (and particularly the economic issues that must be addressed) mustn’t be left half done.Read More & Comment...
Excellent cover story in today’s edition of BioCentury on the February 4th FTC hearing on competitiveness issues surrounding biosimilars.
Steve Usdin calls it like he sees it – and he sees it pretty clearly.
Agendas. Agendas. Agendas.
The headline reads, Biosimilar schism.
Some choice quotes:
For years, innovator biologics manufacturers battled companies that wanted to create an American biosimilars industry, skirmishing over the scientific and legal standards for demonstrating similarity.
Now, however, the biggest conflicts are among biosimilars developers. The growing ranks of biosimilars developers and manufacturers have split roughly into two camps. One is pursuing a business model based on branded biosimilars that will require substantial investment in marketing and sales, and the other is hoping to create interchangeable biologics that could be substituted for innovator products with little or no marketing.
While supporters of the principles for state substitution laws portray them as applying equally to innovator and interchangeable products, that view was rebuffed by companies advocating for a biosimilars market that would allow them to avoid marketing costs and compete primarily on cost.
Bruce Leicher, SVP and general counsel at Momenta Pharmaceuticals Inc., told FTC that the substitution principles are an effort to ask “states to join in a commercial marketing campaign to disparage interchangeable biologics.”
Notification requirements would “restrict substitution and provide notice to doctors to intervene and be concerned about FDA approved biologics,” he said. Leicher accused supporters of the state legislation proposal of attempting to blur the distinction between biosimilars and interchangeable biologics. “The notification provisions are really designed to make the point that interchangeable biologics really aren’t interchangeable, they’re different,” he said.
What Leicher doesn’t seem to understand (or what he understands but wants to purposely obfuscate) is that interchangeable biologics are different from their innovator priogenitors – but bioequivalent enough to be therapeutically interchangeable (as per the FDA).
That's not a "scare tactic," that’s just a fact. Another fact is there can be more than one winner – the most important being the patient. The FTC hearing often sounded like the Biosimilar Hunger Games.
Here’s a link to Usdin’s complete Biosimilar schism.
For more on the FTC hearing, see The Sic et Non of Biosimilars and be sure to tune in to BioCentury This Week, where Usdin will interview with Geoff Eich, Amgen’s Executive Director of R&D Policy and Craig Wheeler, CEO of Momenta. The show will be broadcast and available on the web on SundayRead More & Comment...
Dr. Michael Weber, Chairman of the Center for Medicine in the Public Interest (among other appointments), is the chief author of new guidelines for the treatment of hypertension, perhaps the most common life-threatening risk factor around the World. The guideline is an official statement of the American Society of Hypertension and the International Society of Hypertension.
Dr. Rick Turner, senior fellow at CMPI (the least of his many accomplishments), has penned (along with Philip Galtry is Vice President and Cardiovascular Therapeutic Strategy Head, Cardiovascular and Metabolic Therapeutic Delivery Unit, Quintiles) a primer to the ASH/ISH guidelines. It appears in the current issue of the Journal for Clinical Studies.
Turner and Galtry’s commentary can be found here.
The ASH/ISH guidelines can be found here.
The Center for Medicine in the Public Interest has heart and is in the heart of the battle for better, more personalized CVD diagnosis and treatment, and outcomes.Read More & Comment...
Representative Rosa DeLauro once again demonstrates her ignorance.
According to a press release, “Congresswoman Rosa DeLauro (D-CT) today outlined her concerns over accelerated drug approval in a letter to Food and Drug Administration (FDA) Commissioner Margaret Hamburg. She also asked Hamburg for details on device safety to ensure that women and families can rely on safe and effective drugs and medical devices that improve health and wellness. DeLauro is a former chairwoman of the subcommittee responsible for funding the FDA.”
Didn’t she vote for PDUFA V, which put into place many of the accelerated approval pathways she is now criticizing?
If Representative DeLauro believes no drug should be introduced into the marketplace until it is completely understood then there would be no drugs in the marketplace. When it comes to serious and life-threatening diseases, that is nothing short of a death sentence for tens of thousands of Americans.
It should also be noted that patient groups tend to be the most ardent supporters of the accelerated approval pathways and patients with more dangerous diseases are more willing to accept taking treatments that may have higher risks
The good news is that Peggy Hamburg once again demonstrates both her knowledge and her ability to share it in a politically palatable manner.
Why FDA Supports a Flexible Approach to Drug Development
By: Margaret A. Hamburg, M.D.
We all know that just as every person is different, so too is every disease and every drug.
so we weren’t surprised by the results of a new study published in the Journal of the American Medical Association. The study found that FDA used a range of clinical trial evidence when approving 188 novel therapeutic drugs for 208 indications (uses) between 2005 and 2012. These results are entirely consistent with our regulatory mandate. We believe varying approaches to clinical studies to support drug approval is good news, not bad.
Data to support the approvals studied were based on a median of two pivotal trials per indication. A pivotal trial presents the most important data used by FDA to decide whether to approve a drug.
But when the authors looked more closely, they found that more than a third of these drugs were approved on the basis of a single pivotal clinical trial, while still other trials involved only small groups of patients for shorter durations. Of the approvals studied, the new drug was compared with existing drugs on the market only about 40 percent of the time.
The authors concluded that, based on these results, the ways in which FDA arrived at those approvals “vary widely in their thoroughness.” Or, in the words of one study author, “Not all FDA approvals are created equally.” Although I don’t think it was actually the author’s intent, a number of commentators framed this as criticism. But I would be more troubled if FDA used a rigid, “one size fits all” approach.
People with serious or life-threatening illnesses, particularly those who lack good alternatives, have told us repeatedly that they are willing to make some trade-offs in order to gain access.
And, of course, “thoroughness,” such as whether a clinical trial is large enough, is in the eyes of the beholder. There is no reason to expect drugs to be tested on similar numbers of patients, regardless of the disease.
Variation in approach to clinical studies demonstrates FDA’s innovative and flexible approach to drug development and approvals. Such an approach was specifically adopted by Congress in the Food and Drug Administration Modernization Act in 1997 and, most recently, in the Food and Drug Administration Safety and Innovation Act in 2012.
The FDA of today works with sponsors of new drugs to design a development and review pathway for each drug that best reflects the disease and patients it is intended to treat, the drug itself, and other treatment options. Some of the factors that enter into our calculus include whether the drug treats a rare or serious disease or addresses an unmet need and any previous knowledge we might have about the drug.
Thus, for example, FDA approved Imbruvica (ibrutinib), a treatment for mantle cell lymphoma, last year based on an “open-label, single-arm trial,” which means that every patient received the treatment and both patients and researchers knew they were receiving it. The results were compared to how well the 111 participating patients had responded to previous treatment for their disease.
And Elelyso (taliglucerase alfa) – for Gaucher disease – was an orphan drug approved in 2012 based on two trials with 56 patients.
In contrast, some trials require large numbers of patients to demonstrate a drug’s effects. This is often the case in studies in patients with a chronic condition such as cardiovascular disease, where larger populations are studied to capture treatment effects.
No matter what clinical trial design is chosen, the Agency always applies the same statutory approval standards of safety and efficacy to all drugs seeking to be marketed in the United States.
Increased flexibility does not mean abandoning standards, and it certainly does not mean abandoning science. Just the opposite. We need to employ the best science in ways that will increase efficiency, productivity and our shared ability to find creative solutions to the challenges that confront us.
At the end of the day, that is just smart regulation – ensuring that patients can more rapidly have access to the best that science has to offer.
Margaret A. Hamburg, M.D., is the Commissioner of the Food and Drug AdministrationRead More & Comment...
Expectations for the FTC’s February 4th hearing on biosimilars were low and, by that measure, the meeting didn’t disappoint.
What was surprising was the degree of dissimulation and desperation portrayed by certain speakers. Also surprising (and not in a good way) was the unexpected naivety of the so many so-called “experts.” The term “dangerous idiots” comes to mind – not to put too fine a point on it. What was disappointing (but, alas, not surprising) was the one-sided nature of the presentations.
This really shouldn’t come as a surprise considering that the FTC’s excuse for holding this hearing was to focus on issues that might constrain competition. Unfortunately, too many of the presenters viewed safety and patient outcomes as a constraint to competition. Exactly zero FDA officials or physician organizations testified. Draw your own conclusions.
And, in a sort of twisted way safety and outcomes are a constraint to competition insofar as they have a direct impact on physician prescribing decisions. Nowhere was this more fiercely debated than in discussions of various examples of state legislation and the issue of biosimilar naming.
Let’s take each in turn.
On the state legislation front, more than a few speakers made the point that physician notification was anti-competitive because it somehow besmirches the reputation of generic drugs (and would do the same to biosimilars). Bruce Leicher (Senior Vice President & General Counsel for Momenta Pharmaceuticals) called physician notification a “tactic” to scare physicians. And Krystalyn Weaver (Director of Policy and State Relations, National Alliance of State Pharmacy Associations), pointed specifically at a Tennessee law that requires physician notification for pharmacy-based switching of epilepsy medications. She cited data that showed this requirement results in increased state spending for epilepsy medications (translation: increased physician insistence on innovator products). What she did not discus was the fact that epilepsy drugs fall into the category of Narrow Therapeutic Index medications.
If there had been an FDA speaker, there might have been appropriate comments about the FDA's Pharmaceutical Science and Clinical Pharmacology Advisory Committee that debated and determined that the bioequivalence specifications should be tightened for, among other categories, generic versions of epilepsy medications – and that FDA officials presenting at that adcomm signaled strong agency support for the move.
Anti-competitive or pro-patient? You be the judge.
Steven Miller (Senior Vice President & Chief Medical Officer, Express Scripts), said he had research showing that physicians don’t want information from pharmacists telling them which patients have filled a prescription – one of the key stumbling blocks to addressing the quagmire of adherence. (Miller was unable to cite the source of this data point.) Thus, according to Miller’s logic, physicians will not care to be notified about a pharmacy-level switch of an innovator biologic to a biosimilar (interchangeable or otherwise).
Well, there are a lot of things “physicians don’t want” – like having to adjust to the world of electronic health records – but that doesn’t mean things can’t (indeed must!) change.
It’s also important to mention that, in it’s 1979 report on generic drug substitution, the FTC concluded, “increased communication (as well as lower prices) may explain why most pharmacists report that product selection laws have had a positive effect on their relations with patients”
(Or as Sumant Ramachandra, Senior Vice President & Chief Scientific Officer, Hospira, commented, “Communications fosters confidence.”)
Anti-competitive or pro-patient? You be the judge.
As Geoffrey Eich (Executive Director, R&D Policy, Amgen) commented, “Hubris is for the uninitiated.”
Eich was one of the few voices for patient safety as a tool for competitiveness. He warned against “overly narrow or false choices” and asked:
* Must we choose between increased access or the ability to monitor specific medicines reliably?
* Must we choose between lower cost medicines or for patients to have a complete and accurate medical record?
* Must we choose between vigorous competition or enabling manufacturers to voluntarily stand accountable to the patients we serve?”
Why not transparently label biosimilars to engender patient and physician confidence?
Why not ensure accurate patient medical records that clearly identify specific products?
Why not select distinguishable non-proprietary names for distinguishable drug substances?
Increased access to biologic medicines can and should include policies appropriate for these classes of medicines. We believe in “and” not “or.”
Let’s be clear – an inability to identify specific biologic medicines reliably jeopardizes all biologic programs equally. Such a preventable lapse would constitute an inexcusable, unsustainable, anti-consumer approach to policy.
Momenta’s Leicher’s called out Eich’s comments as representative of the “campaign against biologics” being used to “scare” physicians away from biosimilars. Even though Amgen is going to be in the biosimilars business?
Leigh Purvis (Senior Strategic Policy Advisor, AARP) pointed to both state legislation requiring physician notification of biosimilars as well as differential naming systems as “roadblocks to competition.”
Does the AARP really believe that safety and, well, knowledge are roadblocks to competition?
Amgen’s Eich spoke to “and not or.” The philosophical father of this concept is the “sic et non” (“yes and no”) of the 12th Century theologian Pierre Abélard. Abélard outlined rules for reconciling contradictions, the most important of which is noting the multiple significations of a single word.
In the case of biosimilars and the FTC, that word is “safety.”
Those who view physician notification and distinct naming as anti-competitive are addressing these issues through a single dimension.
And it just ain’t that simple.
As BioWorld Today reports,
In the end, the FDA will base its decision on what’s best for public health – not on commercial interests, Pitts said. “It isn’t a question of tactics or strategies trying to squash biosimilars in the womb,” he added, responding to some of the comments raised at the FTC workshop that suggested concerns about pharmacovigilance were simply an effort by brand companies to protect their market.Read More & Comment...
ObamaCare and mandatory contraception coverage is a hot button issue. But flying well below the national media’s radar is the other side of the coin – how insurance companies are denying care to pregnant Americans.
According to WebMD, “Nothing can blast the euphoria of discovering you're pregnant faster than morning sickness.” Perhaps a close second is knowing there’s an FDA-approved treatment for this condition – and that your insurance company refuses to pay for it. As my wife commented, “If men got morning sickness, this wouldn’t be a problem.”
In a study based on a survey recently published in the medical journal Obstetrical and Gynecology Survey, more than two-thirds of all survey respondents reported that “morning sickness” diminished their general enjoyment of pregnancy, and some women, especially those with moderate/severe symptoms, reported anxiety about the health of the baby and lacked confidence that they were doing the best they could for their unborn child.
The repercussions of choosing short-term savings over long-term results, of cost-based choices over patient-centric care, or “fail first” policies over the right treatment for the right patient at the right time – are pernicious to both the public purse and the public health.
Consider Diclegis, a medication specifically developed and recently approved by the FDA to treat Nausea and Vomiting of Pregnancy (aka, “Morning Sickness”), a condition experienced by 70-85 percent of pregnant women. (Diclegis is the first and only treatment for nausea and vomiting of pregnancy approved by the FDA in more than 30 years, and the only treatment granted a Pregnancy Category A rating by the FDA, the highest level of pregnancy product safety.)
Not only are some insurers (such as Aetna) restricting access to this FDA-approved treatment, they’re requiring health-care providers to first prescribe off-label medications, or a combination of over-the-counter treatments neither developed nor approved for use during pregnancy. It’s only after a pregnant woman has failed to control her morning sickness through trial and error with these untested-in-pregnancy propositions before being allowed access to the only medication tested and FDA-approved as safe and effective for pregnant women who suffer from this condition.
Importantly, for women who have morning sickness, it's critical to get treatment early to reduce their complications – and that means getting the best treatment as soon as possible, not “failing” their way up the therapeutic ladder.
Aetna’s decision to deny cutting-edge care for America’s Moms also illustrates the danger to the development of important new treatments for a host of other diseases. According to Tufts University it costs about one billion dollars to bring a new medicine to market. That's one billion per drug -- and those are the ones that make it through the clinical and regulatory processes. The innovation ecosystem is a fragile wetland and investment dollars are harder to come by than ever before. Why would a pharmaceutical company invest in high risk R&D if there is likely to be no insurance company willing to pay for the therapeutic benefits it can deliver?
Pregnancy is a difficult journey– but its travails are worth the result. That’s also true for healthcare innovation. Today, it takes about 10,000 new molecules to produce one FDA-approved medicine. And only three in 10 new medicines earn back their research and development costs. And here's the kicker: Unlike other R&D-intensive industries, biopharmaceutical investments generally must be sustained for more than two decades before the few that make it can generate a profit. But ask any patient whose life has been saved or prolonged through the miracles of modern medicine, whether or not the investment has been worthwhile.
Protecting “sustainable innovation” is crucial for both the public health and to maintain America’s lead as the global hub for biotechnology.
Harvard University health economist (and Obama healthcare advisor) David Cutler has noted that: "Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost." To borrow an over-used adjective from the world of global climate change -- we must protect "sustainable" innovation.
America’s mothers-to-be are on the front lines. A “pregnant pause” in appropriate access is not acceptable.Read More & Comment...
Some tidbits from Salvatore J. Giorgianni, Jr.’s docket submission to the FTC hearing on biosimilar naming, (Giorgianni, PharmD, BSc, CMHE, is Chair of the American Public Health Association Caucus On Men’s Health and President, Consultant Pharmacist, Griffon Consulting Group, Inc.)
Patients across the US and in every other country in the world will be best served if distinguishable names are required for all biologics and their biosimilar follow on compounds. Providing clarity of information with distinguishable names or codes that are transparently traceable back to the point of bio-manufacture and ending at the point of dispensing/administration to the consumer is essential to keeping a secure process that insures patient safety. As history shows, the integrity of drug and biologic supply and the ability to properly manage clinical care require clear unique product identification. Such unique naming/coding also continues FTC’s long and valued tradition of advocating for full and transparent disclosure of product information for the consumer
The importance of providing for specific and precise product identification is of heightened importance with biosimilar products and their presumed biotherapeutic equivalents because of the large proportion of immunocompromised, frail and vulnerable populations with complex and life-threatening medical conditions that are most likely to receive these products.
In the truest sense of the word, the assertion of biotherapeutic equivalency by some in support of non-unique product designations is by any convention a scientific hypothesis that needs to be validated.
As a practicing pharmacist and compounding pharmacist for 30 years and former pharmacy educator I feel strongly that the ability to precisely know and rapidly trace back product components and identity is fundamental to protecting the health of patients. Having distinguishable product identification for biosimilar products, in my view, seeks to provide the most direct route back to the root of an issue if an adverse event occurs and being able to do this is part of the standard of practice for all pharmacists.
Salvatore J. Giorgianni, Jr., PharmD, BSc, CMHE
Chair, American Public Health Association Caucus On Men’s Health
and President, Consultant Pharmacist, Griffon Consulting Group, Inc.
Giorgiann’s complete docket submission can be found here.Read More & Comment...
Witness the support of the GPhA to Florida’s pending legislation on biosimilars.
FDA News reports that, GPhA has jumped into the biosimilars substitution debate, saying it prefers that doctors not be notified when a pharmacist substitutes a biosimilar for a name-brand biologic, and it is supporting legislative language that would implement that approach in states throughout the U.S.
GPhA and other critics believe the physician notification provisions of the compromise will deter pharmacists from making substitutions.
Making sure that a patient gets the best treatment should never be viewed as "impeding access." That's a canard and shows the venality of a certain approach to biosimilars.
This bill should be called "The Biosimilars Don't Ask, Don't Tell Act." Rather than placing the burden of knowledge on physicians and pharmacists, this bill forces a patient (often a very ill patient) to demonstrate an advanced level of pharmaceutical sophistication. Is it plausible that patients are educated enough to know what a biosimilar is, let alone ask whether or not they are getting a biologic or a biosimilar? This is clearly not the case with small molecule generics – a much less complicated proposition. The fact that physicians have the ability to use "prescribe as directed" is good. But it is not enough.
A more practical Washington State bill offers a better, holistic and appropriate approach, specifically the language that reads:
If a biological product is dispensed, the pharmacist or the pharmacist's designee shall within a reasonable time but not to exceed ten days following the dispensing, record the name and manufacturer of the product dispensed in an interoperable health records system shared with the prescribing practitioner, to the extent such a system is available; or, in the case that an interoperable electronic health records system is not in place, communicate to the prescribing practitioner the name and the manufacturer of the biological product dispensed to the patient. No communication to the prescribing practitioner is required under this subsection where there is no interchangeable biological product for the prescribed biological product, or for a refill prescription that is not changed from the product originally dispensed.
This makes it much better legislation than the Florida version and a superior piece of "model legislation."Read More & Comment...
As the February 4th FTC workshop on follow-on biologics approaches, here’s another VIP (Very Important Perspective) that the Commission has chosen not to bring to the public. Today’s VIP is a physician with experience prescribing and treating patients with biologics. Dr. Bert Petersen, a surgeon, is director of the Breast Surgery Clinic of St. Barnabas Hospital in New York City and an adjunct associate professor of surgery at New York University School of Medicine. Dr. Petersen is an advocate for the elimination of health disparities, particularly in terms of cancer and chronic diseases. Here are his thoughts on why the biologics naming issues matters for both him and his patients.
Q: What role do biologics play today in treating patients?
A: In my field, cancer – specifically breast cancer, we’ve seen great success in treatment for early and advanced stages with biologics. As we move toward more targeted therapies for chronic disease, they play an increasingly important role.
Q: Do you think there may be certain populations who are more at risk to an immunologic response from a biologic?
A: Yes. Any populations that may have a compromised immune system—specifically, many patients with chronic disease—can be impacted. These include at risk populations such as the elderly, immune deficient and chronic renal disease patients, etc. Additionally, at-risk populations tend to be patient populations that may lack quality insurance or access to healthcare. Furthermore, many of these chronic diseases disproportionately impact the poor. This makes access to biosimilars even more important for this population
Q: What value could biosimilars offer patients?
A: Two of the biggest reasons to look at biosimilars are cost and access. Can we offer the same effective treatment while controlling cost? My biggest concern is how we increase equal access to quality health care. We want to increase our reach in expanding healthcare, but it must be quality health care. Biosimilars offer a chance to meet the goals of affordable and quality treatment options.
Q: What is your view on the best approach FDA could take on biologics naming and how does distinguishable naming help keep our biologic supply safe?
A: Unlike any other field, medical decisions must be met with great scrutiny and thoughtfulness because any mistakes or missteps can be fatal. Patient safety should be the FDA’s overarching principal when it comes to approving biosimilars and any other drug.
In terms of distinguishable naming, I believe that biosimilars definitely should have different names, so you can determine if drugs are equal in their effectiveness. In my opinion, it’s unethical to treat patients with something pretending to be something else when it may or may not be. It’s also unsafe. I have a real problem with this as a practicing physician who treats patients with life threatening illnesses.
Q: Why is it important for patients and doctors to know what biologic is being, and has been put into, a patient’s body?
A: Much of how we practice is based on evidence-derived medicine. This is how we gather our evidence to know what is effective and what is not. Understanding which biologics patients have used will help us as we move toward the future to make any modifications that are found necessary.
Q: What impact would distinct naming have on trust in biosimilars?
A: I think if we could distinguish drugs, providers would have less hesitation in prescribing them. If providers are more educated and they have a clear pathway to report adverse effects—they would be more motivated to trust and prescribe biosimilars.Thank you, Bert Read More & Comment...
WASHINGTON (AP) — It seems to be something of an occupational hazard for President Barack Obama: When he talks about his health care law, he's bound to hit a fact bump sooner or later.
OBAMA: "More than 9 million Americans have signed up for private health insurance or Medicaid coverage."
THE FACTS: That's not to say 9 million more Americans have gained insurance under the law.
The administration says about 6 million people have been determined to be eligible for Medicaid since Oct. 1 and an additional 3 million roughly have signed up for private health insurance through the new markets created by the health care law. That's where Obama's number of 9 million comes from. But it's unclear how many in the Medicaid group were already eligible for the program or renewing existing coverage.
Likewise, it's not known how many of those who signed up for private coverage were previously insured. A large survey released last week suggests the numbers of uninsured gaining coverage may be smaller. The Gallup-Healthways Well-Being Index found that the uninsured rate for U.S. adults dropped by 1.2 percentage points in January, to 16.1 percent. That would translate to roughly 2 million to 3 million newly insured people since the law's coverage expansion started Jan. 1.
OBAMA: "Because of this (health care) law, no American can ever again be dropped or denied coverage for a preexisting condition like asthma, back pain or cancer. No woman can ever be charged more just because she's a woman. And we did all this while adding years to Medicare's finances, keeping Medicare premiums flat, and lowering prescription costs for millions of seniors."
THE FACTS: He's right that insurers can no longer turn people down because of medical problems, and they can't charge higher premiums to women because of their sex. The law also lowered costs for seniors with high prescription drug bills. But Medicare's monthly premium for outpatient care has gone up in recent years.
Although the basic premium remained the same this year at $104.90, it increased by $5 a month in 2013, up from $99.90 in 2012. Obama's health care law also raised Medicare premiums for upper-income beneficiaries, and both the president and Republicans have proposed to expand that.
Finally, the degree to which the health care law improved Medicare finances is hotly debated. On paper, the program's giant trust fund for inpatient care gained more than a decade of solvency because of cuts to service providers required under the health law. But in practice those savings cannot simultaneously be used to expand coverage for the uninsured and shore up Medicare.Read More & Comment...
Although FDA is tasked with creating naming policy for biosimilars before they enter the U.S. market, FTC is trying to force its misguided view on the issue in the hopes of building advocates for non-unique names. During a workshop scheduled for February 4 will delve into the biosimilars naming issue, but somehow they forgot to include a number of VIPs (Very Important Perspectives). Amazingly, patient advocates are nowhere to be found on the agenda; also absent are physicians who prescribe biologics and health system pharmacists.
Because of FTC’s slight – presumably so they could through the workshop arrive at near consensus on the need for biosimilars to share the same name as the innovators to which they related, and in doing so muddy the central facts that distinguishable naming is the right approach for efficient adverse event reporting, patient safety and even promoting uptake of and competition among biosimilars.
Today’s VIP on the issue is that of the first person – the actual long-time and life-long biologics user. Donna Cryer is really a patient-plus though – in addition to using a mix of biologics and synthetic medicines for rheumatoid arthritis, inflammatory bowel disease, to preserve a transplanted liver she received nearly 20 years ago, and to deal with kidney issues that impair her body’s ability to make red blood cells, Donna is a Harvard-trained health policy lawyer, a patient representative on an FDA advisory committee and the first patient to serve as Chairman of the American Liver Foundation. Here’s Donna’s perspective on why the right naming policy for biosimilars and all biologics matters for her and the millions of other biologics users like her.
Q: What role do biologics play in treating patients?
A: Biologics play an incredibly important role in treating patients, like me, who have multiple autoimmune conditions. My life really depends on biologic medications. And for so many thousands of other patients, our health, our productivity, our ability to work and be with our families all are because we have access to biologic medications.
Q: What value could biosimilars offer to patients like yourself?
A: Biosimilars often offer lower cost options, so that can provide more access to medications for more patients.
Q: Why is it important for patients and doctors to know which biologic is being and has been put into a patient's body?
A: It is essential that doctors and patients know exactly which medication, particularly with biologics, they are prescribing and using. Being able to manage a disease based on the reactions of your immune system is really tricky. You want to make sure that you are not suppressing the immune system so much that you are open to every infection, every cold, as well as more serious conditions like tuberculosis. Knowing exactly which biologic medication you're taking is absolutely vital because if there is a side effect, an adverse event, or just a change in your condition and your body's response, you want to be able to track it back to exactly the drug that you were prescribed, exactly the drug that you took.
Q: Since biologics are more complex than normal, chemical prescription medicines, how does that alter the conversation and relationship you have with your doctor?
A: The doctor/patient relationship is based on trust. In fact, the patient relationship within the entire healthcare system is based on trust, and a high degree of confidence, that what we're being prescribed, what we rely on for our very lives, is safe and effective. We want to be able to know, and have confidence that our biosimilars and biologic medications are distinguishable, so that we can know what we're taking, how we're taking it, how it differs.
Q: From your view as a patient, what would be the best approach the FDA could take when creating a naming policy for biosimilars?
A: Well, the issue of biosimilars naming is really important, because unless FDA ensures that unique distinguishable names for biosimilars are given, patients and doctors really will be left without any recourse to track back and understand what medication might have caused their adverse event or their side effect. We want to be able to track back if there is an issue, a side effect, a serious adverse event, or just a change in our condition. We want to be able to know. We deserve the right to know what we have taken so that we can have recourse, if need be, about what has happened and what is happening to our bodies. As a patient, I'm not really sure why there is even an argument about having a distinguishable name for a biosimilar: it's such a simple solution to have a distinguishable name.
Thank you, Donna.
The Wall Street Journal reports:
NEW DELHI—Workers at a Ranbaxy Ltd. drug plant repeatedly fudged test results to make it appear that raw materials and active pharmaceutical ingredients met required standards when they didn't, according to a report by inspectors from the Food and Drug Administration.
FDA officials visited Ranbaxy's Toansa factory in the northwestern Indian state of Punjab early this month and said they discovered workers retesting "until acceptable results are obtained" and deleting evidence of failed tests.
The FDA inspectors also noted that analytical and microbiological laboratories at the plant were in "significant disrepair," with windows that couldn't close and a sample-preparation room with flies that were "too numerous to count."
In one lab, the FDA report said, inspectors "identified the presence of numerous sticky notes" that were found to "contain instructions for corrections to be made to the raw data" for testing. The inspectors also wrote that they had observed a worker in the quality-control lab backdating a log entry.
"We immediately questioned this analyst regarding the reason for backdating his record, who responded that he had only entered '2014,' despite our visual observation of him entering a signature and full date entry a few moments earlier," the report said.Read More & Comment...
On January 6th, CMS issued a proposed rule that would result in foundational changes to Medicare Part D and negatively impact America’s seniors, and other constituencies. Most disturbingly (if not surprisingly) it reveals the Administration’s authentic view of Part D by attempting an unprecedented level of government interference with what was intended to be a competitive, market-based proposition. Specifically, the proposed rule:
· Interprets the statutory non-interference provision for the first time since the MMA passed in 2003.
· Imposes new, non-statutory restrictions on sponsor contracts and plan bids, limiting sponsors to no more than two plans per region.
· Intervenes in private market contracting between Part D plans and network pharmacies.
· Further encourages the displacement of employer-provided coverage that the MMA intended to preserve.
· Introduces enormous uncertainty into the market for plan sponsors as they prepare for the 2015 bid cycle.
· Places new restrictions on access to medicines in classes of clinical concern, including mental health drugs and drugs to prevent organ transplant rejection.
Why this rule and why now? One explanation is that it shows the continuing cognitive dissonance of the administration (and career CMS staff) that anything driven by the free-market could possibly work –- and that seniors are not capable of making their own healthcare choices. And it doesn’t matter that both of these almost religious beliefs fly in the face of all facts and figures to the contrary.
This rule is designed to solve a problem that doesn’t exist – but which its authors believe should exist. The proposed rule creates a Bizarro Part D wherein the role of free-market forces and the imperative of citizen choice are replaced by the heavy hand of the infallible Uncle Sam, MD.
Think about it: This proposed rule would limit competitive bidding, limit patient choice, and stifle innovation in plan design. In short, it would gut the philosophical framework of Part D –- a framework that has consistently resulting in government spending coming in under budget with 90%+ user satisfaction.
All this and the proposed rule also revises long-standing prior agency policy on the “six protected classes” policy. Specifically targeted are medications for mental illness. The proposed rule revises long-standing prior agency policy that required Part D plans to include on their formularies “all or substantially all” drugs within six classes: anti-depressants, antipsychotics, anticonvulsants, antineoplastics, and immunosuppressants. This policy has been in effect since the inception of Part D, and has strong congressional support.Why? Why fix something that isn’t broken? Maybe it’s all a “fix” of a different kind. Maybe this proposed rule is designed to ruin Part D so the argument can be made for direct government interference and, ultimately, a move to a single payer system.
Remember, you’re not paranoid if they’re really out to get you. Read More & Comment...
And just wait until they start exporting biosimilars.
WASHINGTON (AP) -- U.S. health regulators said Thursday they are barring imported drugs from an overseas factory operated by Ranbaxy Laboratories, India's largest drugmaker, due to quality control violations.
The Food and Drug Administration ban effectively stops the company from shipping drugs and raw ingredients from its Toansa plant in the Punjab province. A Jan. 11 inspection by FDA staffers uncovered factory workers retesting drug ingredients that had failed quality testing, in an apparent effort to return positive results. Those practices and others found at the plant violate manufacturing standards for drugmakers that do business in the U.S.
"The FDA is committed to ensuring that the drugs American consumers receive - no matter where they are produced - meet quality standards and are safe and effective," said FDA compliance director Carol Bennett.
Ranbaxy did not immediately respond to requests for comment Thursday.
Ranbaxy will be required to hire an outside inspector to review the plant and certify that it is meeting U.S. quality standards before the ban can be lifted.
In September the FDA placed a similar hold on imports from Ranbaxy's Mohali facility. Both actions were taken under a 2012 legal settlement with the FDA, which subjects Ranbaxy to extra scrutiny and inspections to improve its drug production.
With annual revenue of more than $2 billion, Ranbaxy is the leading drugmaker in India's $26 billion generic pharmaceutical industry, but it has faced penalties from U.S. regulators for years.
In May, the company's American subsidiary agreed to pay $500 million in fines and penalties for selling adulterated drugs and lying to federal regulators, the largest financial penalty against a generic drug company for violations of the Federal Food, Drug and Cosmetic Act, which prohibits the sale of impure drugs.Read More & Comment...
Yesterday 28 Republican lawmakers from both the House and Senate (led by Senate HELP ranking member Lamar Alexander and House Energy and Commerce Chairman Fred Upton) sent a letter to the FDA voicing their concern over the agency’s proposed rule that would allow generic drug companies to change the safety information on the labels of their products. Their fear is that “equivalent products could temporarily” end up showing “different safety information” before “the FDA determines if the changes are warranted.”
Dr. Janet Woodcock, the F.D.A.’s head of drug evaluation and research, said the proposed change would create better parity between brand-name drug manufacturers and generic companies, which is especially important given that more than 80 percent of prescriptions in the United States are currently dispensed as generic drugs. “Now, with the generic industry having grown up, most people are taking generic drugs,” she said. “It’s really time to level the playing field.”
But, methinks, the 28 signatories have been pretty heavily lobbied, because there’s another issue here that’s not safety related.
The proposed rule would also pave the way for lawsuits from patients who could now claim that generic companies did not sufficiently warn them of a drug’s dangers. In 2011, the Supreme Court ruled that such lawsuits were not valid because generic companies were required to use the same label warnings as brand-name manufacturers and thus could not be blamed for failing to warn patients about the risks of taking their drugs.
It’s interesting to note that Senator Alexander has also been front and center in lobbying the FDA on the biosimilar INN issue. He does not believe in differentiation.
Why does his concern about safety and clarity apply only to small molecules? Hm.
For more on this issue, see “Generic Originals.”Read More & Comment...
Sometimes statements of the obvious are useful. Here’s one from Yale University’s Nicholas Downing, a third year medical student at Yale University School of Medicine and lead author of a new study, which examined nearly 200 new drug approvals between 2005 and 2012:
“Not all FDA approvals are created equally.”
According to the Washington Post, “Downing and his team found broad differences in the data it took to get a thumbs up from FDA. For instance, the agency required that many new drugs prove themselves in large, high-quality clinical trials. But about a third won approval on the basis of a single clinical trial, and many other trials involved small groups of patients and shorter durations. Only about 40 percent of approvals included trials in which the new drug was compared with existing drugs on the market.”
“There was a lack of uniformity in the level of evidence the FDA used,” Joseph Ross, assistant professor of internal medicine at Yale and a senior author of the study, said in announcing the findings.
The Yale study, funded by the Pew Charitable Trusts and published Tuesday in JAMA, acknowledges that the FDA has valid reasons for its varied requirements.
“Such regulatory flexibility allows for a customized approach to approval,” it states, “including the ability to rapidly approve potentially effective therapies for life-threatening diseases, such as certain cancers, or those diseases for which there is no existing effective treatment, such as orphan diseases.”
Downing, the study’s lead author, said the analysis is not intended to suggest that the FDA should use a one-size-fits-all approval process or that it somehow has approved the wrong drugs. Rather, he said, it was meant to educate people who assume that all new drugs have cleared the same amount of safety testing before ending up on the market.
“It’s very understandable that regulators have a flexible standard for approval, but patients and doctors need to be aware the standard is flexible,” he said. “It’s important for doctors and patients to have a discussion about how much we know about the potential benefits or risks of taking a drug before they take it . . . [and] it underscores the need to continue to study these drugs after approval.”
Yep. Not new – but worth repeating.
Missing from the Yale study is the relevant fact that, per PDUFA V, the FDA is designing a five-item grid as a management tool to explain its risk-benefit decisions in a more concise format. The model that it has created as a working template confirms a truism about its drug approval tendencies that industry has suspected for years: the baseline for FDA approval is a high rating of the severity of the disease being treated and the medical need for the product.
The agency is developing a grid of the five basic factors that need to be addressed in any decision on the commercial availability of a drug. The top two are the seriousness of the condition addressed and the need for a new treatment of the condition. Then comes the traditional heart of the NDA package: analyses of clinical data on the benefits of the drug and the risks associated with its use.
The fifth fundamental factor is explicitly the level of risk management associated with the product. The FDA is going to take it into consideration in every decision; sponsors who ignore or underplay the identification of who should use the product and who might use it will have a gap in their filings.
The grid proposal does not call for a fixed mathematical formula behind each approval. The agency has not tried to reduce the judgments in an approval decision to a rigid calculation.
Judgment? You mean FDA decisions aren’t black and white?
In the words of John Jenkins, director of the Office of New Drugs at the Center for Drug Evaluation and Research, disagreement “happens a lot in the decisions that we have to make. Very few of the decisions that we make on drugs are easy. Very few of the drugs we see have a dramatic overwhelming benefit with relatively no risk. We see that most drugs have marginal to moderate benefits on a population basis and they have general safety but they have the risks of serious toxicities at some low levels." In other words, every decision is very complex.
The unspoken danger is that regulators love ambiguity because ambiguity is power. That’s different than regulatory flexibility – which is a good thing. But it’s a fine line and the distinction is often in the eyes of the beholder. But it is certainly a distinction with a difference.Read More & Comment...