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Thanks to President Barack Obama’s landmark healthcare law, insurance companies are no longer allowed to turn away patients with pre-existing health problems. For millions of sick Americans, this “guaranteed issue” mandate has been transformative, ensuring they can secure the coverage they need to afford vital medications.
Unfortunately, insurers have discovered a sneaky way to undermine this requirement. They’re structuring plans to heap huge costs and bureaucratic burdens onto high-risk patients. Technically, such patients are insured. But they don’t actually have access to medical care.
Federal officials must halt such discriminatory practices. Fortunately, Reps. David McKinley, R-W. Va., and Lois Capps, D-Calif., recently introduced the Patients’ Access to Treatments Act (PATA) (HR-1600) to do just that. Lawmakers should pass it immediately.
Insurers strap sick patients with big bills by putting expensive medications into the highest “tier.” Insurers typically divide their drug benefits into different tiers, with the lowest providing the most financial support and the highest providing the least. The higher the tier, the higher the patient’s out-of-pocket expenses.
For example, in the state-level insurance exchanges established by Obamacare, more than half of the popular “Silver” plans place all multiple sclerosis drugs in the top tier. Patients suffering from this devastating conditions are getting hit with huge costs. Many are forced to forego needed treatments.
Making matters worse, the slice of Silver plans requiring enrollees to pay 30 percent or more of the cost for top-tier drugs has jumped dramatically since last year, from 27 percent to over 40 percent.
PATA ensures that patients can afford these life-saving medications. The bill prohibits insurers from grouping specialty drugs in higher cost sharing tiers than the ones used for regular medicines.
Many health insurers also have a “fail first” policy. Patients must first take drugs that are less effective and often less safe — and only when these fail can they receive needed medicines.
In other words, insurers force people to get sicker before offering them lifesaving treatments.
Consider the plight of Shima Andre, a hepatitis C patient in Los Angeles. Shima’s insurance company refused to cover Harvoni — a costly yet highly effective hepatitis drug — because her liver wasn’t damaged enough. Even after repeated pleas from her doctor, the insurer wouldn’t budge.
Technically, Shima is insured. But that coverage means little, since she can’t access the drugs she needs. Rather, she must take older medicines that the FDA warns have a high risk of serious and even fatal side effects.
A new Harvard study suggests insurers are deliberately offering thin coverage for high-cost therapies to dissuade chronically ill patients from signing up in the first place. When people who need costly drugs see treatments’ price tags, they look elsewhere for coverage — which is what insurers want.
Insurers justify such discrimination by claiming it helps contain healthcare costs, keeping premiums affordable. After all, those with serious illnesses disproportionately rely on expensive, specialty medicines. They’re the 1 percent of patients — often referred to as “super spenders” — that account for more than a fifth of the nation’s annual health expenditures.
But this argument is misleading. Expanding access to prescription drugs actually helps bring down long-term healthcare costs — and denying sick patients needed medications drives costs up.
Cancer drugs, for instance, help keep patients healthy and out of the hospital. As a proportion of total cancer treatment costs, drug spending nearly tripled from 2001 to 2011. Over that same time, the share of total costs spent on hospital stays dropped by 25 percent. Spending more on better medications has improved cancer patient health and saved money.
Restricting drug access to trim healthcare expenses usually backfires. The average patient will skip prescribed medications if her monthly out-of-pocket costs exceed $200. Such “non-adherence” typically causes a patient’s condition to worsen to the point where she requires much more expensive medical interventions.
America already spends over a $100 billion a year on avoidable emergency room visits and hospital stays because patients abandon their medication regimens. This will drive up non-adherence even higher and exacerbate these problems.
One of Obamacare’s central promises was that sick patients would no longer suffer from discrimination. Insurers are breaking that promise by strapping vulnerable patients with huge costs and forcing them to fail on less effective treatments. Lawmakers must stop these abuses — and the Patients’ Access to Treatments Act is a good place to start.
Robert Goldberg is vice president of the Center for Medicine in the Public Interest. Read More & Comment...
From FDA News:
The FDA is taking steps toward getting more patient input during the drug development process, a move that puts the agency more in line with its EU counterparts, agency officials said at the DIA annual meeting.
The FDA has met with patients from over 20 different disease areas to hear their perspectives on how their treatments are working and how their diseases impact their lives as part of the PDUFA reauthorization process. The agency wants to glean information they don’t get from clinical trials, said Robert Temple, deputy director for clinical science in CDER, during a session at the DIA annual meeting in Washington, D.C.
Temple said patients’ chief complaints aren’t being factored into drug development or clinical trials, and they should be. Trial designs for new drugs should aim to identify the three most irritating symptoms patients with a particular disease have, so that drugmakers can focus on solving those issues. “Any endpoint that affects a patient is an endpoint that needs to be included in a trial,” he said.
Theresa Mullin, director of CDER’s Office of Strategic Programs, echoed that statement, saying the motivator for the FDA to include more patient input is the need to develop better endpoints. The agency can’t properly assess medicines without fully understanding the value, benefit and effect on patients, she said.
In the EU, patient participation is grounded in legislation, with patients having input at all levels of a clinical trial, said Enrica Alteria, head of human medicines evaluation at the European Medicines Agency. The EMA holds meetings with patient advisory and advocacy groups four times a year, and patients view labeling information and leaflets, she added.
As part of that patient discussion, Alteria said the agency consults with health technology assessment bodies early in the drug development process to gather additional patient evidence. In this respect, the EMA takes a broader lifespan approach than the FDA, she said.
The FDA is precluded by law from taking cost into account when it decides to approve or disapprove a drug. Temple said the agency wants people to have choices. “We try hard not to think about the economics.”Read More & Comment...
This week the American Society of Clinical Oncology released a tool that it claims will help oncologists measure the value of drugs and discuss their costs with patients. The Task Force that developed this so-called value framework proclaims that the perspective of the patient is of central importance in defining value. It notes that “patient perception of value is highly individualized, can be subjective, may change over time and include convenience and quality of life as well as cost. “
Yet value framework – which will be used to develop an app -- does not take into account any measure of the value of individual responses to treatments. It admits that it doesn’t measure the costs saved by taking newer medicines or the productivity gains generated by treatments that require quitting a job and spending months in the hospital.
The app only measures value by more or less estimating how much would be spent on cancer drugs for the additional survival the medicine has shown in clinical trials. (“Bonus” points are added if studies show the drug to generate fewer side effects than a placebo). In addition, the app uses the out of pocket cost to the patient as the benchmark.
There are several defects with this approach that will likely reduce the number of lives saved from cancer.
First, the app uses average response obtained in clinical trials to measure value. For example, most news accounts have made a big deal about how treating people with advanced non small cell lung cancer (NSCLS) with Avastin with two older drugs costs $10000 to add 2 months of survival compared to using the older drugs alone for $800.
But average response is virtually meaningless in treating cancer. Yet the comparison does not take into account lung cancer patients with specific genetic mutations who are likely to live much beyond that average. It can't measure the relative value of "regimens..not directly compared in clinical trials. "That’s just about 90 percent of cancer treatment combinations approved for use. Combining Tarceva with Avastin in lung cancer patients with specific mutations adds 4 months to people who would otherwise die.
Multiply the number of treatments by the number of genetic mutations shaping each and every tumor and then combine these targeted medicines with therapies that teach the immune system to shut down tumor growth. Waiting for randomized clinical trials that compare each and every of these combinations would deny thousands of people potentially life saving and life enhancing care.
Further, the Task Force claims there is no clinical benefit to increasing overall survival by less than 20 percent. That would eliminate the use of a whole host of treatments for pancreatic, brain, lung, stomach and cancers in use or being studied today. By way of comparison, the increase in average life expectancy for people with AIDS was less than 20 percent a year between 1987 and 2000. The ASCO app would have completely rejected these increases, which in turn would have denied life to thousands of people alive today.
Indeed, the Task Force asserts – without evidence – that the use of new drugs is being driven by “sometimes unrealistic patient and family expectations that lead clinicians to offer or recommend some of these services, despite the lack of supporting evidence of utility or benefit.” For all it’s lofty rhetoric about the primacy of patient value, the Task Force sneers that cancer patients “overestimate the benefits of treatments that sometimes extend life by only weeks or months or not at all. “ In fact the evidence suggests just the opposite.
As a result, the app does not calculate the value of hope as measured by the significant progress made in reducing the death and cost associated with cancer. Since 2000, new cancer drugs have been responsible for nearly 90 percent of the decline in cancer death rates. The number of cancer survivors has increased from about 9.8 million to nearly 14 million today. Over that time we have added 26 million additional life-years worth over $2 trillion in better, healthier life.
As the number (and price) of targeted treatments have increased the percent of health care dollars devoted to cancer spending has remained at 4.6 percent. How? New cancer drugs reduce spending on more expensive medical services. In 2001, 64 percent of cancer care went to hospitals and only 3.6 percent to drugs. By 2012 drug spending ‘skyrocketed’ to 11.3 percent of cancer costs but hospitalizations dropped to 38 percent of care. That’s why a government study concluded, “The net value of (cancer) treatment has grown substantially, consistent with medical technology improving over time and leading to better health outcomes at a lower cost per patient.”
All these benefits have been generated by new medicines that are only .7 percent of health spending. The Task Force acknowledges that new medicines are a small fraction of total costs but then asserts that use of new medicines is driving up premiums, reducing wages, etc., when the evidence clearly concludes the opposite is the case.
If the app completely disregards the evidence of patient preferences, cumulative life years saved from small gains in survival, the reduction in treatment costs and increase in the economic value of health, what it’s good for?
It turns out that the app will be very helpful to health insurers who want to
“evaluate the relative value of new treatments” as they develop “benefit structures, adjustment of insurance premiums, and implementation of clinical pathways and administrative controls.”
The task force acknowledges that one important effect of these benefit structures is “patients will find themselves increasingly responsible for a greater proportion of the cost of their health care. Cost shifting or sharing can occur through the increased use of high-deductible policies and larger copayments. “ Which appears to be acceptable to ASCO since it does not take into account evidence of how such practices, by reducing the cost of drugs, drives up the number of deaths and the cost of care.
Health plans are forcing cancer patients to pay thousands of dollars out of pocket for new medicine. And they are requiring them to ‘fail first’ on less costly treatments that insurers want to claim are as, on average, effective as new therapies. Conveniently insurers are already using the ASCO calculation to restrict access.
Meanwhile, the Department of Health & Human Services has warned “placing most or all drugs that treat a specific condition on the highest cost tiers discourages enrollment by individuals based on age or based on health conditions, in effect (is) making those plan designs discriminatory.”
If the app is used by health plans as ASCO hopes – to support the development of pathways and administrative controls – it will be powerful tool for trampling on the civil rights of cancer patients.
ASCO has asserted that oncologists should be aware of the value of an intervention in terms of societal cost when making treatment decisions. It assures patients that this does not conflict with the Hippocratic oath. It doesn’t just conflict; it out right violates that solemn pledge.
Read More & Comment...
The Patent Trial and Appeal Board of the U.S. Patent and Trademark Office has accepted a request from Celgene to consider sanctions against the Coalition for Affordable Drugs, an entity controlled by hedge fund manager Kyle Bass.
Celgene filed a motion seeking sanctions as part of its response to an inter partes review (IPR) petition the Coalition filed that attempts to invalidate some of Celgene’s patents on Thalomid thalidomide. In a document filed with the PTAB, Celgene accused the Coalition of using the IPR process to affect the value of public companies. "This is not the purpose for which the IPR process was designed,” the company wrote. Celgene also alleged that before the IPRs were filed, someone associated with the Coalition “threatened to file IPRs against the challenged patents unless Celgene met their demands.”
Celgene wrote that when it did not pay, the IPRs were filed. Celgene described the alleged attempt to obtain payment to forestall IPR challenges as an “abuse of process and misuse of the IPR proceedings.” In granting Celgene’s request to file a sanctions motion to dismiss the petitions, the PTAB said its decision “is not a decision on the merits of Patent Owner’s allegation of abuse of process.” The board set a July 30 deadline for Celgene to submit a brief supporting its request.
Per a report in BioCentury, the board’s decision is likely to be announced three months after the Celgene brief is submitted, according to Matthew Kreeger, a partner at Morrison & Foerster who is an expert in patent law. The Celgene request puts the PTAB and the IPR process “in uncharted waters,” Kreeger told BioCentury. He noted that if the decision hinges on a demand for payment made prior to filing the IPR, then it might not affect other IPRs filed by Bass or other investors.Read More & Comment...
Did somebody say “PDUFA VI?”
With the upcoming PDUFA renewal, 21st Century Cures uncertainty, and a leadership transition at FDA, it is a dynamic and uncertain time in the biopharma industry. The outcomes of these key policy debates, along with others, will impact how industry pursues drug development and works with patients and regulators to improve public health.
To advance the conversation and propose practical ways forward, a panel of regulatory experts, including former industry and FDA leaders, got together in Boston on May 20, 2015 to share their best thinking on pressing regulatory policy issues. And there was also a fair degree of appropriate (and useful) venting.
The panel was chaired by Tim Franson, MD (Chief Medical Officer for YourEncore, Board Member for the Critical Path Institute, and current President of the USP Convention. Tim was a key contributor to PDUFA V, particularly as it relates to rare disease incentives, and is generally considered one of the “founding fathers” of the PDUFA concept.
Panelist #2 was Joe Lamendola, Ph.D., the former VP of U.S. Regulatory Sciences for Bristol-Myers Squibb, responsible for over 20 global approvals across 10+ therapeutic areas. Over a 25+ year career, Joe also led the Regulatory Policy and Intelligence Organization for BMS, which was responsible for assessing and influencing regulatory policies, including PDUFA V and multiple therapeutic guidances.
And rounding out the panel was yours truly – the junior associate.
It’s called a regulatory RANT (Relevant Assessment and New Trends) and is a great read to get ready for PDUFA season. The full document can be found here. It's an entertaining and informative read with some relevant assessments and aggressive suggestions
Get ready. Get set. Go!Read More & Comment...
At the recent BIO confab in Philly I was honored to moderate the panel on “Public Sector Biotech Initiatives in Middle East and North Africa.” I was joined with public officials and regional experts (Marwan Abdulaziz Janahi, Executive Director of Dubai Biotechnology and Research Park, DuBiotech, Samir Khalil, Executive Director of Middle East & Africa, PhRMA, Tarek Salman, Assistant Minister of Health and Population for Pharmaceutical Affairs in Egypt, David Torstensson, Senior Consultant, Pugatch Consilium, and Jeffrey Kemprecos, Executive Director, Emerging Markets Public Policy, Merck).
A key take-away was that one of the key drivers of biotech investment in the region (and, indeed, any region) is sound regulatory policy. This was most directly addressed by Vice Minister Salman who discussed how the Egyptian regulatory authority had upgraded both its processes and procedures to reward innovation with a review pathway that is more predictable and timely.
As the panel’s moderator, I had the opportunity to present opening remarks. Here’s what I had to say:
When it comes to biotech incentives in the MENA region, there are many languages, priorities, pressures, and impediments (social, political, cultural) to consider.
In April 2015 I spent three fascinating days in Sharm El Sheikh, Egypt at the Second Arab Conference on Food & Drugs.
Delegates from the Levant to Morocco had a lot to say and share. The fundamental take-away was that the Arab world is serious about coordinating their efforts in healthcare in general and in regulatory affairs specifically. “Convergence” and “harmonization” were the two key words of the event.
(The Middle East/North Africa Region – MENA – consists of 22 nations – but just 2% of global pharmaceutical sales.)
Biotech initiatives are a global opportunity, but they take many local forms --because public health is a global fraternity with national priority, local impact and global implications.
Biotech Initiatives take many forms:
- Investment programs
- Clinical trial incentives
- Reinvention of medicine and device regulation
- A high regard for Intellectual Property Rights
- An embrace of the concept of both price and value
- Transparency of laws and regulations
- And the rule of law
Most importantly, national biotech initiatives rest on the foundation of the importance and urgency of healthcare innovation.
But Biotech Initiatives mustn’t falter under the false banner of Biotech Imperialism. Initiatives must benefit all parties, transnational, national – and local.
That means “doing the right thing.” It’s about “Nazaha” – Integrity.Read More & Comment...
This happens everytime a bunch of new medicines hit the market. But how about putting the amount into context or discussing value. As any baseball fan (or FBI agent investigating the St. Louis Cardinals hack of the Houston Astros scouting database) knows context is everything.
So for instance, the Pharmalot blog posted: How Much?! Global Prescription Drug Sales Forecast to Reach $987B by 2020
But the headline obscures and deflects attention from the fact that between 2015-2020 Rx spending will actually decline as a total share of global health spending.
The same designed neglect applies to the breathless discussion of "skyrocketing drug prices or drug costs".
You wouldn't know that drug spending as a percent of total health spending in the US will remain FLAT between now and 2025 even as specialty drug spending becomes half of total Rx expenditures.
Is it too much to ask for some context. It took me, a C minus math student, five minutes to figure out percentages. Why can't journalists and policy types who are a lot better at this stuff than I am run the same numbers?
Read More & Comment...
Per a report in BioCentury, PatientsLikeMe Inc. has granted FDA access to its patient-reported data, which the agency said it will use to identify risks and benefits of drugs. The agency said PatientsLikeMe's adverse event data have the potential to supplement existing sources, including data that sponsors are obligated to report and data from FDA's Adverse Event Reporting System (FAERS), to address safety issues that arise postmarket. PatientsLikeMe said FDA will have access to more than 110,000 adverse event reports on 1,000 different drugs at no cost. The data include information on drug tolerance, adherence and quality of life. PatientsLikeMe President Ben Heywood told BioCentury the collaboration "may lead to FDA-sponsored research projects designed to understand how patient-reported data might be used to enhance post-market surveillance, support regulatory decision making and inform regulatory science." FDA also operates the Sentinel system, with which it can query electronic health records to identify and monitor postmarket safety concerns.Read More & Comment...
From the pages of the Houston Chronicle:
Pitts: Obama's drug crusade comes at high price
Medicare Part D program, which provides affordable drug insurance, needs to be emulated, not reformed
A new federal report misleadingly estimates the cost of the Medicare Part D program at $103 billion. That figure is bunk. Nonetheless, it will give President Barack Obama more ammunition for his assault on Part D - a program that provides affordable prescription drug insurance to more than 2 million Texas seniors.
Already, the president has pushed for government controls on drug prices in Part D - a "reform" he endorsed in his 2016 budget.
But Part D doesn't need to be reformed - it needs to be emulated. The program is a historic success. Federal interference in Part D drug pricing would destroy that success and drive up the cost of medications, reduce enrollee choice, and harm patients.
Part D drug coverage is affordable for seniors and taxpayers alike. The average monthly premium for a Part D plan is around $32 - and hasn't changed much over the last five years.
Nearly a decade after Part D was created, the program's overall costs are almost $350 billion below initial estimates, according to the Congressional Budget Office.
Unsurprisingly, the program is enormously popular. Among beneficiaries, Part D's satisfaction rate is roughly 90 percent.
Yet the Obama administration wants to tamper with the program by bargaining directly with drug manufacturers for rebates on Medicare prescriptions.
Currently, the law that established Medicare Part D prohibits federal officials from interfering "with the negotiations between drug manufacturers and pharmacies and [prescription drug plan] sponsors."
The Obama administration views this lack of government negotiation as a shortcoming that boosts Part D's cost. But non-interference is actually essential to the program's success.
Indeed, the reason the program is able to deliver satisfactory coverage at such a reasonable cost is its competitive structure. Under Part D, Texas beneficiaries are free to choose from 32 different private coverage plans.
In order to win customers, plan providers compete with each other to offer the best policies at the lowest cost, driving down prices in the process.
This arrangement works because private insurers are able to secure their own drug rebates through private negotiations with pharmaceutical firms. These rebates frequently amount to a 20 or 30 percent discount. And those savings are passed on to beneficiaries.
If the federal government intrudes on private Part D negotiations, the competition driving the program's success will collapse. Drug prices will go up. Quality of plans will go down.
Worse, federal interference in the negotiations wouldn't result in lower drug prices, as the Obama administration claims. Researchers from the Congressional Budget Office have concluded that government officials "would be unable to negotiate prices across the broad range of covered Part D drugs that are more favorable" than the prices achieved by private insurers.
Unfortunately, the administration's rhetoric against Part D ignores these existing discounts. The $103 billion figure, which comes from a Center for Medicare and Medicaid Services report, doesn't account for the discounts either. CMS simply tallied up the market price of all drugs dispensed through Part D.
Factoring in those privately negotiated rebates paints a more accurate picture of Part D annual costs, which are closer to $62 billion.
Federal intervention won't lower Part D's total cost, but it will make it harder for seniors to get the medicines they need. According to Douglas Elmendorf, the former head of the Congressional Budget Office, negotiating for a lower drug price on a given drug carries "the threat of not allowing that drug to be prescribed." Beneficiaries could loss access to treatments they now depend on to stay healthy.
Sadly, none of these facts have stopped the Obama administration from trying to interfere in Part D price negotiations. But it's important to ask: What does the president hope to accomplish with his proposal?
If the president's aim is to expand access to affordable prescription drugs, he should be celebrating Part D - not trying to destroy it.
Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.Read More & Comment...
To that point, a new op-ed from today's edition of the Wall Street Journal.
The Patent Trial and Appeal Board was supposed to make the system better. It hasn’t.
BY: Peter J. Pitts
When Ultratec, a manufacturer of closed-captioned phones for the deaf, realized that a rival had created a knockoff using its patented technology, the company filed a patent-infringement lawsuit. A Wisconsin federal jury ruled for Ultratec in October, ordering rival Sorenson Communications to pay $44 million in damages.
But Ultratec may never receive a cent. In March a little-known but hugely powerful federal body called the Patent Trial and Appeal Board (PTAB) invalidated Ultratec’s patents, on grounds that the designs were too obvious to be patentable.
The PTAB, created by the 2011 America Invents Act, was intended to strengthen the patent system. Lawmakers hoped to avoid the need for patent lawsuits by giving patent holders and challengers a quick and inexpensive way to resolve disputes as an alternative to the courts.
But the board uses looser standards than a federal court to evaluate a patent’s legitimacy. Courts assume that a patent is valid until a challenger provides “clear and convincing” evidence to the contrary. The PTAB requires only that challengers show that it’s more likely than not (i.e., a “preponderance of the evidence”) that a patent is too broad.
In recent months the board has overturned patents on a computer memory technology, a popular videogame, and a system for monitoring car tires. The PTAB has invalidated at least one “claim”—or part—in almost 80% of the patents it has ruled on, according to a study in the University of Chicago Law Review. Some patent experts such as Randall Rader, former chief judge at the U.S. Court of Appeals for the Federal Circuit, have referred to the 300-odd administrative judges, attorneys and legal aids on the board as “patent death squads.”
Patent challengers have jumped at the chance to exploit the board’s lax standards. Since it began to operate in September 2012, the PTAB has received more than 2,600 patent challenge requests—three times more than it expected.
Many of these challenges—such as one against Combigan, an eye-drop medicine that prevents blindness in patients with glaucoma—seek to overturn patents that district courts have already upheld. In many other cases, the patents have also been challenged in federal courts—but courts have stayed the litigation until the PTAB has ruled. The patent may be invalidated without facing a court’s stricter standard.
The PTAB could devastate innovation-intensive industries. Consider pharmaceutical developers, which spend about $51 billion a year researching new treatments. But less than 12% of drugs that reach clinical trials ever make it to market.
Patents give firms the financial incentive to fund challenging research and development projects. In the event that a huge upfront investment results in a popular new product, the developer can recoup its costs in sales.
The PTAB jeopardizes this process. Since an overturned patent means that rival companies could create knockoff products, firms will lose the confidence that they’ll reap the rewards of innovation.
Some financiers have started using the PTAB to make a quick buck. Kyle Bass is a hedge-fund manager, not a pharmaceutical developer, but he recently challenged six drug patents. His strategy, which has been widely reported, is to bet that the challenges would drive down the patent owners’ stock prices.
The strategy is working. Early this year Mr. Bass challenged Acorda Therapeutics ’ patent on Ampyra, a medicine that uses a re-engineered bird poison to help multiple sclerosis patients walk. The claim: Medical experts would have been able to deduce the effectiveness and proper dosing of the re-engineered molecule. The challenge caused the company’s stock price to drop almost 10%.
If hedge funds and copycats continue to take advantage of the PTAB’s bias against patent holders, it will choke off funding for lifesaving medicines.
The Patent Trial and Appeal Board will make it harder to create the products that improve lives and fuel the economy. To avoid this dangerous outcome, Congress has to reform the PTAB so that it operates under the same standards as a regular court.
Mr. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest. Read More & Comment...
(Some animals are more equal than others.)
It's an important, timely, fun -- and a must read pre BIO and DIA.
Enjoy. Read More & Comment...
What does the FDA think of Amarin's plan for off-label communication?
Here's the letter the agency sent them. And, from the pages of the Wall Street Journal,the latest on Amarin's lawsuit and the FDA's counter-strategy.
FDA Tries to Blunt Amarin’s Free-Speech Lawsuit Over Off-Label Info
By Ed Silverman
Last month, a small drug maker called Amarin AMRN -4.82% caused a stir by filing a lawsuit against the FDA to argue that its right to distribute information about unapproved uses of a medicine is protected by the First Amendment.
Now, in an unusual move that appears designed to blunt the impact of the lawsuit, the FDA has written a letter to Amarin saying the types of materials the drug maker would like to distribute to doctors actually would not be a problem. And the FDA suggests that Amarin might have known this if the drug maker had discussed the issue before filing its lawsuit.
The lawsuit is being closely watched for its potential to determine whether the FDA can prohibit drug makers from distributing off-label information. The issue has been widely debated after a federal appeals court in 2012 overturned a criminal conviction of a sales rep for promoting off-label uses. The court ruled his speech was protected, since the information was truthful and not misleading.
As we reported previously, Amarin wants to be able to provide doctors with information that does not directly pertain to the approved uses of its Vascepa prescription fish-oil pill. The FDA endorsed the drug to treat people with very high levels of triglycerides, a type of fat in the blood that can lead to heart disease.
But two months ago, the FDA rejected an Amarin bid to market its pill to patients with slightly lower triglyceride levels and denied its plan to add effectiveness data to the Vascepa product labeling. Amarin then filed its lawsuit, arguing it has a constitutional right to distribute the information, even though such a move would be considered by the FDA to be off-label promotion.
In its lawsuit, Amarin included a list of medical journal articles it would like to distribute to physicians. However, the FDA writes that none of these are problematic. The agency “does not have concerns with much of the information you proposed to communicate” and the FDA “would not consider the dissemination of most of that information to be false or misleading,” the FDA letter to Amarin states.
As it turns out, the FDA says that Amarin never approached the agency about the issue. In her letter, FDA official Janet Woodcock makes a point of writing that Amarin “did not ask for our views before filing” its lawsuit “as other pharmaceutical companies sometimes do.” A spokesman for Amarin says the drug maker would not comment.
Moreover, Woodcock also reiterated FDA plans to release a so-called industry guidance for governing the dissemination of off-label information. In effect, the letter amounted to a pre-emptive move that might slow the progress of the lawsuit, according to regulatory experts.
“I think Amarin is frustrated by the results of its dealings with FDA and has resorted to the courts instead of trying to maintain a constructive dialog with FDA,” says Ira Loss, senior health care analyst at Washington Analysis, a consulting firm. “Woodcock’s letter undercuts their case. Not talking to FDA before filing the suit will likely result in a summary judgment for the agency.”
Adds Peter Pitts, a former FDA associate commissioner for external affairs, who now does policy consulting for the pharmaceutical industry: “Amarin got some bad regulatory advice. The FDA has loudly signaled that it is going to act with increased regulatory discretion on off-label communications. This was either missed or ignored by Amarin.
“… A meeting with the agency would have addressed their concerns. [As for the FDA], “exercising regulatory discretion is a smart move as it takes the matter out of the hands of a judge. A free speech ruling would make things much more difficult for the agency. The FDA opted for strategic retreat rather than face a potential sledgehammer legal decision.”Read More & Comment...
Now, innovation is NOT invention. Here's Sir Harold Evans, another very smart person on this issue: innovation is bringing an invention to use (i.e. commercialising it)...a scientist will have understanding, an inventor will have a solution but an innovator will have a universal solution.
An invention without innovation is a past-time. Essentially, many inventors are hobbyists, since an MIT study has shown that fewer than 10% of patents granted have had any commercial application.
Evans says that few scientists are able to turn ideas into a commercial impact.
Which leads me to the NIH' foolish foray into drug development.
Innovation is about commercialization. Commercialization especially today requires something that is in large supply in the private sector and short supply at the NIH:
Experience in commercialization, which includes constantly learning from failure in the development of a product that one hope's will have mass appeal and consumption.
Further, as a Fast Company article notes: ln a world of rapid disruption, having a core competency—that is, an intrinsic set of skills required to thrive in certain markets—is an outmoded principle of business. Just as Google needed Android to attack mobile and Apple needed Siri to pursue search, thriving businesses need to constantly evolve, either through partnerships, new talent, acquisitions—or all three. Nike, No. 1 on Fast Company's 2013 list of Most Innovative Companies, proves this idea more than most. Last year, it launched FuelBand, a high-end electronic wristband that tracks your energy output and signaled Nike's growing strength in the digital realm. "Think about it: Nike is now included in conversations around technology—it's shifted into an adjacent industry, breaking out of apparel and into tech, data, and services," says Forrester Research analyst Sarah Rotman Epps. "That strategic shift is incredibly important to Nike's future."
NIH has been throwing little bits of money to keep up with the furious pace of evolution and the rapid shift of life science firms into related industries. It looks a little desperate but there's no choice because most of what NIH does and spends money on are discoveries and technologies developed outside it's walls.
Thus, while it was great to see that the NIH and FDA is looking for an entrepreneur in residence (for database analysis), I had to laugh since the idea of an entreprenuer locked inside government regulations and surrounded by people who's job longevity depends on avoidng risk and stifling others has the makings of a great comedy sketch.
Add to that the fact that entrepreneurs commercialize.. How can you do that when you have little experience scaling up clincal trials with safe and consistent and ample supplies of the materials you need to make something.
Ask people at the FDA what they think about the ability of NIH bureaucrats and researchers to engage in drug development. It's a good way to get them to laugh for a change.
I won't go into the demise of government run vaccine development facilties. I will mention that even before NIH contaminates samples that it is not very good at deciding what to develop or not.
Further, the NIH is horrible at clinical trial recruitment and management. Just ask cancer patients who on average wait 800 days for NIH sponsored trials to get up and running. Here's what Vince DiVita, the former head of the NCI said in 2008. Sadly, it is still relevant today.
"The requisite talent to know the right way to design and modify an ongoing study does not reside on remote review committees at the NCI or the FDA, yet those are the places where the delays are greatest. Too many cooks are spoiling the broth."
As John LaMattina points out When NIH was asking for funding for the National Center For Advancing Translational Science (NCATS) Dr. Roy Vagelos, the legendary former Merck CEO and now Chairman of the Board of Regeneron Pharmaceuticals. Testifying before the subcommittee as an adviser to the American Society for Biochemistry and Molecular Biology, he made the following point:
“Does anyone in the audience believe that there is something that NCATS is going to do that the industry thinks is critical and that they are not doing? That is incredible to think that. If you believe that you believe in fairies.”
In fact, what NIH has done has pull funding from the Molecular Libraries Program which supported has made a contribution to the development of drug discovery tools. These tools have, in the hands of Hugh Rosen, who runs a lab at Scripps, revolutionized drug discovery. Rosen's work only led to the development of biotech startup Receptos which has several drugs under development and as well as the development of a drug to control the cytokine storms that cause sudden death in people with the flu. So what did NIH do? It not only rejected future grant requests from MLP participants it pulled the money that would have supported improvements and next generation tools and it put it into the NCATS, the government's drug company. That's not what entrepreneurs do unless they want to waste money. As one advisor to the Molecular Library Program noted the kind of spinoff for which Receptos is the poster child is “something that has come from this and is probably going to disappear.”
Think of NIH as a $30 billion business that has strayed from it's core competency: funding outside the box research and ideas and supporting small groups of researchers who contribute to and interact with anyone that seems to be solving key research questions and wants to turn them into products. The way back is to democratize and de-routinize grant giving and break up the cartel of research centers that rely upon NIH money for overhead. Craig Venter's suggestion should be taken seriously:
"The academics might not like it, but peer review is like the prisoners running the prison. They're not going to vote for change. Universities like this system because it helps support the universities. We have to change it so that 25%, 30%, 40% of the money is set aside for true risk research with independent parties to do that. That's going to disrupt a lot of things. I argue that the American public should be outraged that there's not 10 times to 100 times more breakthroughs in medicine every year over what we're getting, particularly for the money that's being spent."
Congress is likely to increase NIH funding. Unfortunately, the influx of cash will go to waste without the kind of mission change Venter has suggested. Read More & Comment...
From the pages of the Orange County Register
The FDA has patients playing a dangerous name game
What's in a name? In medicine, it could be the difference between life and death.
A new class of drugs is coming onto the U.S. market. Unfortunately, Congress is standing by as the Food and Drug Administration prepares to allow marketers to sell these imitations under the same name as the original drug.
The only problem is that the drugs aren't the same. The differences between the drugs, although subtle, can have serious consequences for patients' health. Lawmakers should instruct regulators to acknowledge these differences by establishing a naming system that distinguishes between the knock-offs and the originals.
The new drugs are "biosimilars." They're inexact copies of biologics – a type of complex drugs derived from living cells. Because living cell strains can't be replicated exactly, the biosimilar drug grown from these cells is also impossible to copy exactly. They're similar, but not identical – and that's the problem.
Biosimilars produced by different manufacturers have similar properties but are not identical to the original or each other.
The medical field rightly values precision, and the ability to trace side effects back to a specific drug is a crucial patient right. To protect that right, Congress should instruct the FDA to develop a clear-cut naming system that calls different medications by different names.
A study conducted in Ireland revealed important distinctions between biosimilars and the biologics on which they were based. The study found variations when Inflectra, a biosimilar that treats rheumatoid arthritis and Crohn's disease, was tested against the biologic it tried to copy. While only 5 percent of patients who received the biologic required hospital readmission, 80 percent of the Inflectra group did.
In addition, just 8 percent of the biologic patients needed multiple bumps in steroid dosage for effective treatment, but 50 percent of Inflectra patients required them. The authors of the study concluded that biosimilars might be less effective than the original biologics.
The FDA itself noted that it's first-ever commercially approved biosimilar, Zarxio, has a lower protein content than filgrastim – the original biologic. The agency dismissed the difference as a manufacturing flaw. But because biosimilars don't undergo extensive clinical trials, drug defects or harmful side effects will be detected only after they enter the market. When side effects do occur, calling different drugs by the same name would subject patients to an impossible guessing game. Did Patient A take the original filgrastim or biosimilar Zarxio?
Fortunately, the FDA's naming system is trying to address this problem. The FDA is calling Zarxio "filgrastim-sndz."
That suffix, representing the drug's manufacturer name "Sandoz," is better than no distinction, but it's still problematic. That's because a suffix shouldn't be tied to the manufacturer's name.
If Sandoz changes its name, or merges with another drug company that makes its own knockoff version of filgrastim, the four letter suffix would lose all meaning. "Sndz" is not a clear-cut differentiator that would follow a drug from patient to patient, year to year. A constant alpha-numeric suffix like "aaa123" would be considerably more effective.
The FDA must recognize that Zarxio and filgrastim are different products and have different side effects. Common sense dictates they should have different names.
Congress and the FDA need to recognize this and implement a distinct naming system before patients get hurt. Zarxio and filgrastim are almost copies of each other. But giving them the same name is akin to almost healing a patient or finding almost the right diagnosis. It's just not good enough.
Peter J. Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest.Read More & Comment...
From FDA Law Blog:
Return of the Scarlet Letter? AbbVie Petitions FDA to Require Biosimilar Labeling to Include Disclaimers and a Description of Data Differences
It was just a few weeks ago that concerning FDA’s recently finalized guidance document on “” that the Agency removed from the of the document any requirement that the labeling of a biosimilar biological product licensed under PHS Act § 351(k) indicate that it is biosimilar to a reference product, and also to call out whether or not it is interchangeable with a reference product. As we commented then, “[w]hile such designations may not have made a biosimilar feel like Hester Prynne, it does seem that mandating such terms be present may have led to some shunning in the marketplace that is today’s town green.”
It seems that AbbVie Inc. (“AbbVie”) – which, interestingly, starts with the letter “A” – took note of the Scarlet Letter change as well. In a June 2, 2015 (Docket No. FDA-2015-P-2000) that popped up on regulations.gov earlier this afternoon (June 3rd), the company requests FDA to require the approved labeling for biological products licensed under PHS Act § 351(k) to contain (if applicable) certain statements and descriptions that would differentiate biosimilars from their reference product counterparts. Specifically, AbbVie wants biosimilar labeling to include:
A clear statement that the product is a biosimilar, that the biosimilar is licensed for fewer than all the reference product’s conditions of use (if applicable), and that the biosimilar’s licensed conditions of use were based on extrapolation (if applicable);
A clear statement that FDA has not determined that the biosimilar product is interchangeable with the reference product (if applicable); and
A concise description of the pertinent data developed to support licensure of the biosimilar, along with information adequate to enable prescribers to distinguish data derived from studies of the biosimilar from data derived from studies of the reference product.
Biosimilars, which came about with the March 23, 2010 enactment of the Biologics Price Competition and Innovation Act of 2009 (“”), “are not generic drugs and should not be labeled like generic drugs,” says AbbVie in its petition. Including in biosimilar labeling the items above “is necessary to enable rational and informed prescribing decisions regarding these complex products, to avoid potentially unsafe substitution of biosimilars and reference products, and to combat widespread misconceptions among prescribers about biosimilars and their relationship to reference products,” writes the company. Furthermore, without such differentiating statements and description, “biosimilar labeling will not reflect the unique licensure provisions established by the BPCIA and will be materially misleading in violation of the FDCA and FDA regulations.” And, in what might be a signal of future litigation, AbbVie alleges that FDA’s “about-face” reversing, without explanation, what the Agency proposed in the 2012 draft guidance is a violation of the Administrative Procedure Act (“APA”). (Though, keep in mind that the last time Abbvie – then Abbott Laboratories – petitioned FDA on the BPCIA back in 2012 – concerning Reference Product Exclusivity – we though a lawsuit might be looming – see our previous post . That didn’t happen – at least it hasn’t yet.)
Pointing to FDA’s March 6, 2015 approval of for Sandoz Inc.’s (“Sandoz’s”) ZARXIO (filgrastim-sndz), a biosimilar version of Amgen Inc.’s NEUPOGEN (filgrastim), AbbVie says that FDA long ago made the decision to apply the “same labeling” requirement under FDCA § 505(j) (applicable to ANDAs) for small-molecule generic drugs to biosimilars licensed under PHS Act § 351(k):
Publicly available materials from FDA’s review of Zarxio confirm that FDA followed a “same labeling” approach. According to the action package for Zarxio, in November 2013, Sandoz proposed and FDA agreed that the biosimilar and reference product labeling “should be essentially the same.” In February 2015, FDA provided the labeling for Neupogen to Sandoz for use “as a template” in developing the labeling for Zarxio, and instructed Sandoz to track any changes made to the Neupogen labeling and provide annotations to explain and justify any such changes. That is essentially what FDA regulations require of applicants seeking to market generic drugs under section 505(j). Indeed, in the media briefing announcing the approval of Zarxio, the Director of the Office of New Drugs in the Center for Drug Evaluation and Research (CDER) acknowledged that the “approach” taken with respect to the labeling for Zarxio was “not that different from the approach . . . taken in the past . . . for generic applications.” Consistent with that approach, the approved labeling for Zarxio is nearly identical to that of Neupogen . . . .
According to AbbVie, however, applying the FDC Act’s ANDA “same labeling” approach to to biosimilars is legally unsound. AbbVie then ticks off the reasons why such an approach doesn’t comport with the law.
“First, a ‘same labeling’ approach is flatly inconsistent with the BPCIA, which—unlike the [ANDA] provisions in section 505(j)—includes no ‘same labeling’ requirement and recognizes that biosimilars are different from their reference products. . . .” (Emphasis in original). Here, AbbVie compares and contrasts the BPCIA and the FDC Act, noting, among other things, that the BPCIA specifically amended the FDC Act (at FDC Act § 505B, concerning required pediatric studies) to provide that a non-interchangeable biosimilar biological product “shall be considered to have a new active ingredient” (at least for purposes of FDC Act § 505B).
“Second, a ‘same labeling’ approach to biosimilars would result in labeling that omits material information necessary for safe and informed prescribing, and would exacerbate, rather than dispel, misconceptions among prescribers regarding biosimilars.” Here, AbbVie argues that applying a “same labeling” approach to biosimilars violates the FDC Act’s misbranding and misleading labeling provisions at FDC Act §§ 502(a) and 201(m), respectively.
Third, AbbVie argues that “FDA has not provided the reasoned explanation required by the APA for its decision to abandon the approach taken in the Draft Scientific Guidance, which stated that the labeling of a biosimilar product should disclose that the product is a biosimilar, the scope of its approval, and whether it has been found to be interchangeable, on the ground that this information is ‘necessary’ for informed prescribing.” Going through the record of FDA meetings and comments leading up to and on the draft guidance, AbbVie asserts that “FDA’s decision to reverse course and adopt a same labeling approach for biosimilars is arbitrary and capricious.”AbbVie’s petition includes a certification under FDC Act § 505(q), which was made applicable to petitions affecting pending Section 351(k) biosimilar applications under the 2012 FDA Safety and Innovation Act. This is, by our count, the second citizen petition involving the BPCIA containing such a certification. FDA denied the first 505(q) biosimilar petition earlier this year (see our previous posts and ). If FDA tags AbbVie’s petition as a 505(q) petition – which seems probably given the likley pending status of Section 351(k) applications at FDA that could be affected by the petition – then we can expect some sort of response within 150 day Read More & Comment...
It is true that out of pocket costs discourage use and force many to choose between food and medicine. The culprits in this instance are health plans and pbms that have increased cost sharing on new drugs by thousands.
AARP -- being an insurer as well -- participates in this immoral activity. The claim that drug prices and drug costs drive up premiums -- including those of AARP -- is false. New medicines for life threatening illnesses are less than 2 percent of total health spending. A Milliman study concluded it would cost about 50 cents per person to cap copays at $250 a year.
So instead of just publishing studies about drug prices, AARP should do the right thing and close the co-pay gap.
Read More & Comment...
Going to BIO? Join me on June 16th for “Public Sector Biotech Initiatives in Middle East and North Africa. I’ll be moderating a panel of experts and government officials discussing government and public sector initiatives designed to attract biotech research and development in the MENA region. We’ll discuss tax incentives, regulatory, reimbursement and IP policies as well as government investments that key MENA countries have implemented to attract biotech companies.
And here are the details:
WHEN: June 16, 2015, 3:30 PM–4:45 PM
WHERE: Room 119A
Marwan Abdulaziz Janahi, Executive Director of Dubai Biotechnology and Research Park (DuBiotech)
Samir Khalil, Executive Director of Middle East & Africa, PhRMA
Tarek Salman, Assistant Minister of Health and Population for Pharmaceutical Affairs in Egypt
David Torstensson, Senior Consultant, Pugatch Consilium
Jeffrey Kemprecos, Executive Director,Emerging Markets Public Policy, Merck
Peter J. Pitts (Moderator), President, Center for Medicine in the Public Interest
Hope to see you there.
The WSJ gave a lot of coverage to Leonard Saltz vitriolic attack on the cost of new immunotherapies for cancer. Saltz call the prices for immunotherapy for advanced melanoma insance and immoral, some the WSJ article: High Prices for Drugs Attacked at Meeting
Cancer specialist criticizes new-treatment costs in high-profile speech
Dr. Leonard Saltz bases his fear about the unsustainability of cancer drug costs on emotion, not fact. He claims that if we treated everyone with metastatic forms of cancer with treatments costing $295000, we would spend $174 million each year and that cost would be unsustainable.
Actually, it would be a bargain. Dr. Saltz is assuming everyone who dies of cancer each year (580000) would get a combination of immune therapies. The combination in question -- nivolumab (Opdivo) and ipilimumab (Yervoy) adds about a year of life to people with advanced forms of cancer. So let’s stick with melanoma in explaining the incredible amount of value generated and money saved by such therapies.
The most recent estimate of the direct cost of treating advanced melanoma is $160,000 per person. (The direct costs of treating metastatic colorectal, breast and lung cancer are about the same.) A good portion of this cost would be eliminated by using immunotherapy because the treatments displace the use of existing medicines and hospitalization. I don’t count the amount of money saved from avoiding surgeries to remove tumor masses since the immune therapy shrinks tumors by 80 percent. But if we treat everyone with such therapies in end stage cancers, we could save $92 billion a year on less effective forms of treatment.
Further, stage IV melanoma currently has two-year survival rate of only 15 percent when treated with conventional chemotherapy. Immune therapies allow 90 percent of patients to live 2 years.
If we assume that 90 percent of people who otherwise die of cancer in a given year lived two years that would add 1.1 million life years for each group of patients. An early study estimates the productivity loss of cancer death at about $250 billion. Thus adding 1 million life years would generate $500 billion. A more conservative estimate (valuing an additional life year at $150000) would be $150 billion. And even if we assume a 60 percent response rate under either scenario, our society more than breaks even with a jump in two year survival. Of course, if 90 percent of people who live two years, wind up living 5 years, both savings in medical spending and life year value soar.
Dr. Saltz said cancer drug prices are insane and immoral. I’d say the same about someone who said – given these benefits – we shouldn’t perhaps pay even a little more. Read More & Comment...
From the pages of Politico …
21st Century Cures up against FDA’s 20th century communication
When it comes to communication, the 21st Century Cures Act is stuck in the 20th century.
The sweeping biomedical innovation bill aims to speed up development of medical treatments by taking advantage of the latest in science and technology. But provisions meant to update the FDA’s regulations on drug advertising were dropped.
The agency’s current marketing and promotion regulations don’t even mention the Internet. They focus instead on brochures, file cards and, inexplicably, lantern slides — a mid-1800s invention that was supplanted decades ago.
In the era of Facebook, Twitter and YouTube, drug and medical device makers contend a social media reboot is critical.
The first draft of the House Energy and Commerce’s bill did make an attempt. Among other changes, it would have forced the FDA to let drug companies communicate truthful “introductory” information in character-limited settings such as Twitter so long as they hyperlinked to a webpage with more safety and efficacy information.
That “one-click” concept is gone from the legislation that cleared the committee on May 21 and is now headed to the House floor. Also gone is a measure directing FDA to revise all regulations and guidances that could apply to online communications so that drug and medical device makers can use the Internet “in a meaningful way” to disseminate truthful and non-misleading information.
In Rep. Billy Long’s view, these provisions would push FDA “to update its regulatory approach to communications to keep up with today’s technology.” The Missouri Republican quickly introduced them as a separate bill after they were cut from Cures, and an E&C staffer signaled late Friday that members remain supportive and that more work could take place later this year on proposals that didn’t garner a bipartisan consensus before the committee’s vote.
The social media debate involving FDA, the pharmaceutical industry and Congress is taking place in what is quickly becoming a free speech powder keg. Companies are pushing the agency to align its policies with recent court decisions that have upheld their right to communicate certain information regardless of whether it appears on an FDA-approved product label — the regulatory standard for what is widely marketable.
Without faster change, the industry warns, FDA instead risks a complete overhaul of its advertising and promotional regulations by the courts.
Richard Samp, chief counsel at the nonprofit Washington Legal Foundation, which defends the free enterprise system as its mission, has worked for decades on these issues. Though he doesn’t foresee the courts taking over FDA’s authority to set limits on pharmaceutical marketing, he does expect continued legal challenges that could complicate the regulatory environment.
“FDA has been on a pretty long losing streak when it comes to First Amendment issues, and the longer FDA continues to do nothing, the longer it will be that they continue to have these losses and the more confusion that will be created,” he said.
The agency should see the Internet as unique from older, static forms of communication, drug makers argue.
“Right now, FDA essentially treats a web page or even a banner ad or a tweet as if it were a print vehicle, when patients and health care professionals who are online treat the Internet differently,” said Jeff Francer, vice president and senior counsel at the Pharmaceutical Research and Manufacturers of America.
Unlike a print ad, the web allows a company to constantly update information. It also lets consumers quickly jump from one page to additional product details or third-party content, and companies want to know how much they’re on the hook if a third-party site later adds information about a drug’s unapproved use.
Social media triggers other scenarios. Companies say they need more guidance on when they must report adverse drug experiences patients share with them through a posting on their Facebook page, for example.
At the same time, FDA is itself engaging online — in ways that only accentuate the gap between its lack of guidance for industry, critics say.
While the agency uses Facebook and Twitter to make short announcements on medical products, companies that do the same risk legal repercussions for not including enough risk and efficacy specifics in their post. Even retweeting the FDA’s own communication would be in violation of the agency’s advertising policies.
“The FDA uses Twitter to link its announcements to more comprehensive information. It is commonsense for the federal government to allow drug manufacturers to use these platforms in a similar fashion,” Long told POLITICO.
FDA first held a public meeting to discuss promotion of agency-regulated products on the Internet nearly two decades ago. Joy Liu, who specializes in drug advertising and promotion at the Washington law firm Ropes & Gray, remembers officials discussing the use of chat rooms and news groups, “sort of a precursor” to social media, she said.
But that 1996 meeting failed to translate into substantial policy changes. In 2009, Liu said, the agency reconvened another public meeting, asking how drug makers could fairly balance benefit and risk information for consumers, how they could embed hyperlinks and how they should correct inaccurate information on the Internet about a product.
Not until last year, however, in response to the FDA Safety and Innovation Act of 2012, did the agency release a pair of Internet-specific draft guidances.
“Is this as important as curing cancer? Maybe not, but it is I think important,” Liu said. “Getting information out about your products, understanding what are the rules of the road, have become critical to companies because they have been prosecuted for allegedly doing the wrong thing.”
Others maintain that the FDA has given industry sufficient direction. The agency’s former associate commissioner for external relations, Peter Pitts, says its current guidance on the Internet and social media is fairly good — but that industry has chosen not to pay attention.
“I think it’s a bit of an excuse for industry not to engage,” he said, adding that companies may find it “very frightening” to take part in such immediate, two-way communication with consumers and do the 24/7 monitoring that social media can require. “It takes guts and chutzpah” to engage online, Pitts said.
FDA did not respond directly to a question about its pace in adapting advertising regulations to evolving technology, instead highlighting its two recent guidances and the projected release of further regulations this year.
“FDA sees social media as an important resource for industry and is committed to developing additional guidance for drug and device manufacturers that outline the agency’s current thinking,” a spokesperson said. “We do all of this work with the best interest of patients in mind.”
The 21st Century Cures bill does expand what industry can say about its products when communicating with health insurers and asks for FDA to issue specific guidance on communication related to a drug’s off-label use.
Yet even as the 2014 draft documents make their way forward, the technology continues to evolve and trigger new questions, said Mark Senak, a lawyer who focuses on pharmaceutical and device marketing at the communications firm FleishmanHillard.
For instance, given that many people use a cell phone as their primary means for accessing the Internet, do companies have to make sure any website advertising is optimized for a mobile device? Senak says it is unclear whether a site that’s fully compliant with FDA regulations when it appears on a larger computer screen could face FDA enforcement because of how mandated information displays differently, and perhaps far less accessibly, on a small device.
One of FDA’s hurdles is that “they are an organization that’s really into understanding data,” Senak said. FDA wants to fully research an issue in a scientific manner before creating policy. “The whole process of them issuing a guidance document is just too slow for this environment.”
FDA’s drug center director, Janet Woodcock, testified at an April 30 congressional hearing on the 21st Century Cures legislation that the difference between FDA’s social media use and what industry is permitted to do stems from the different stakes each has — one is a government public health entity, the other markets drugs for profit.
What Woodcock didn’t note is how flexible FDA already is on direct-to-consumer drug advertising compared to the rest of the world. New Zealand is the only other country that allows drug companies to directly market to consumers.
There’s little chance that the U.S. public will stop using the Internet as a go-to resource on health concerns. According to a 2012 Pew survey, 72 percent of Internet users said they had looked online for health information within the past year. Much of that info is unregulated, a reality that also frustrates drug manufacturers.
Aaron Kesselheim, an associate professor of medicine at Harvard University, says FDA’s slow approach to releasing Internet and social media policy may be due to industry’s response to its past guidances on drug and device promotion.
After the agency issued guidance on print media in the 1980s, “investment in print media advertisement exploded,” Kesselheim said. The same thing happened after FDA put out guidance on broadcast media in the late 1990s, revising the parameters on the risk information companies had to include in TV ads. Virtually overnight, direct-to-consumer advertising spiked. It went from $579 million in 1996 to $1.3 billion in 1998. A decade later, it had again more than doubled to over $4 billion.
“You can understand why the FDA may be wanting to cover all of the bases … and is being deliberative about this,” Kesselheim said. “When these kinds of guidance do come out, they lead to a substantial expansion of advertising — which can have bad outcomes for patients because some drug companies take advantage of it.”Read More & Comment...
The latest post by Tracy Staton -- essentially a rewrite of an Express Scripts blog complaining about the cost of medicines -- lives up to that proud journalistic heritage. Staton once again obsesses about the amount of money spent on two or three cancer drugs without mentioning their impact. I think it’s because no matter the value of such innovative medicines, the profits and prices are too high for Staton.
"As the American Society of Clinical Oncology meeting approaches, the pharmacy benefits manager released some numbers on cancer drug spending. The top line? Spending on cancer meds grew by more than one-fifth last year, with a 9% increase in use and almost 12% increase in prices.
Prime suspects for that increase? The leukemia treatment Gleevec, from Novartis ($NVS), which boasts the biggest share of the market at 12.5%. Fellow blood cancer treatment Revlimid--the multiple myeloma therapy from Celgene ($CELG)--came in close behind with 10.8% of pharmacy benefit spending.
She will never be convinced. "
Let's take a look at those prime suspects and what they are guilty of.
All Gleevec did was launch a revolution in personalized medicine and an array of medicines that allow people with CML to live full lives. According to my colleague Tomas Philipson and his research group: The TKI drug class in CML therapy has created more than $143 billion in social value. Approximately 90% of this value is retained by patients and society, while approximately 10% is recouped by drug companies...
The introduction of TKI drugs to treat CML has generated significant social value as a result of survival gains, the vast majority of which has accrued to patients.
What about treatments for multiple myeloma developed by Celgene, Takeda and others since 1998:
Frank LIchtenberg concluded:
"(A)lmost two-thirds (0.99 years) of the 1997-2005 increase in the life expectancy of American myeloma patients was due to an increase in the number of chemotherapy regimens now preferred by specialists. Based on a back-of-the-envelope calculation, this means that the cost per US life-year gained from post-1997 chemotherapy innovation is unlikely to have exceeded $46,000. We also investigate the impact of chemotherapy innovation on the myeloma mortality rate using longitudinal country-level data on 38 countries during the period 2002-2012. Countries that had larger increases in the number of chemotherapy regimens now preferred by specialists had larger subsequent declines in myeloma mortality rates, controlling for myeloma incidence.
Put another way, people with myeloma added 360,000 life years since 1997, worth about $54 billion.
Finally, Staton ignores the contribtuion of cancer drug spending on total health expenditures.
As the amount spent on new medicines increases, the cost of treating such diseases has declined
In 2011, 11.4 percent of total cancer expenditures were for prescription medicines as compared with 3.6 percent in 2001
The proportion spent on inpatient hospital stays declined from 47 percent in 2001 to 35 percent I 2011.
Cancer spending as a percent of total health care spending and GDP actually has declined. Prescription drugs as a percent of total health care spending has remained at about 9 percent since 2000 and is projected to remain 9 percent for the next decade. All the while cancer drugs make up more and more of spending on drugs and cancer care.
So maybe the takeaway is that more spending on the newest and most expensive drugs is a good thing.. or at least not portrayed as a criminal activity by FiercePharma.
Fierce Pharma has to decide whether it has a responsibility to correct her bias by including other viewpoints or by providing greater editorial direction to include information that places the price and cost of medicines for cancer and other life threatening or disabling diseases in context.
My bet is that it will continue to demagogue an issue short on facts and chock fill of misplaced anger.
Read More & Comment...