Latest Drugwonks' Blog
Some interesting new regulatory tea leaves in the debate over biosimilar nomenclature.
According to the USP, if Sandoz’s biosimilar (Zarxio – or filgrastim-sndz) meets the filgrastim monograph, it should carry the same nonproprietary name as Neupogen. But the FDA says it’s already determined the monograph does not apply. According to Dr. John Jenkins, director of FDA's Office of New Drugs the naming of this particular drug does not necessarily mean this will become the general policy for naming biosimilars.
An important point to remember is that the USP is not responsible for testing and enforcement of its standards, including comparing products to existing monographs. That’s an FDA responsibility.
While the FDA agrees that the USP has a role “regarding the nonproprietary names of drugs in certain circumstances,” they do not believe that the nonproprietary names FDA assigns for drugs and biologics are temporary until the USP reached a decision.
On March 18 (just under two weeks after the FDA approved Zarxio) USP issued a statement to the effect that it had “just begun a dialog with FDA regarding Zarxio and it is too early to comment further.”
So watch the regualtory fur fly and keep those tea leaves steeping.
Dr. Kantarjian and Dr. Rajkumar use a combination of misstatements and unsupported claims in demonstrating that they are more concerned with protecting the profits of health insurers than they are with the lives and well-being of their patients.
To learn more go to http://bit.ly/1GYiDeE
First Big Pharma was advised to adopt free or differential pricing to developing nations – now the mask is off and it’s all about middle-income countries after all. But wasn’t this entirely predictable, that companies would be criticized for doing what the activists had asked for in the first place?
In healthcare, as in life – no good deed goes unpunished.
Merck and the Medicines Patent Pool (MPP) signed a licensing agreement last for pediatric formulations of raltegravir, one of the few HIV drugs approved for children younger than three. Using the new license, drug manufacturers can make cheaper versions of raltegravir because they do not have to pay royalties to Merck.
The MPP was founded in 2010 by UNITAID, a UN-backed initiative aiming to use innovative financing to increase access to HIV, tuberculosis and malaria medicines in developing nations. The pool lets drug manufacturers in developing countries produce and sell low-cost HIV treatments through voluntary licensing and patent pooling.
Good news right? Not for some. Critics have told SciDev.Net (hardly a pharma-friendly media outlet) that it is "a false solution to a real problem.”
Intellectual property “expert” Othoman Mellouk is critical of the agreement. Mellouk, who works at the International Treatment Preparedness Coalition, a well-known anti-IP organization, says he thinks the agreement looks like a PR exercise that will not deliver improved access to medicines where they are most needed.
"The MPP mechanism ensures originator companies are in control and determine the parameters of agreements,” says Mellouk. He then adds that voluntary licenses such as those agreed through MPP actually undermine a country's negotiation power and ability to use TRIPS flexibilities.
He notes that the 92 licensed nations are mostly low-income countries with little pharmaceutical industry, meaning it would be easy for monopolies on drug production to form. He points out that middle-income nations including China and India - which have more competitive pharmaceutical industries - are excluded.
"The MPP mechanism ensures originator companies are in control and determine the parameters of agreements.” Owners of intellectual property, per Mr. Mellouk, shouldn’t be in charge of their … intellectual property.
The bottom line is that he’s living in a fantasyland where working with the globally applauded Medicines Patent Pool is not just a bad idea – but a nefarious scheme. Note to Mr. Mellouk – Wake up. The reality of global healthcare isn’t good versus evil but about allies working together to improve patient care. Alas, that is not a potent fund raising message for his (and like-minded) organizations, but it’s good news for patients in the developing world.
Martina Penazzato, pediatric advisor for the WHO's HIV department, is more positive about the agreement: "What we need for children with HIV is better drugs, better formulations, and we need to have them developed much more quickly."
Amen.
Departing FDA Commissioner Peggy Hamburg isn’t going to take “blame the FDA” as an excuse any more.
Bravo.
According to a report in BioCentury, Senator Lamar Alexander (R/TN) is leading the Senate down a narrower, slower path to 21st Century Cures legislation than the House is taking. Speaking at a hearing to launch legislation intended as a companion to the House Energy & Commerce Committee’s 21st Century Cures initiative, Alexander asked FDA Commissioner Margaret Hamburg to identify two or three top priorities for legislation and said he didn’t want to “waste time” discussing a large number of ideas. The focus on a slim bill is a stark contrast to the 393-page discussion draft of 21st Century Cures legislation released by the Energy & Commerce Committee.
Responding to Alexander’s request for top legislative priorities, Hamburg called for greater investment in “regulatory science to develop the knowledge, tools and strategies that allow us to assess in an efficient way the safety, efficacy and performance of medical products.” Taking aim at a white paper Alexander and Senator Richard Burr (R/NC) released in January outlining the committee's goals for legislation to promote medical innovation, Hamburg argued against assuming that "FDA regulation is the principal obstacle to the development of innovative treatments, and that FDA’s authorities and procedures should be fundamentally reconsidered." She also cautioned "against solutions that seek to lower the safety and effectiveness standards for approval of the medical products on which Americans rely."
Like Gogol says, "It is no use to blame the looking glass if your face is awry."
“Blame the FDA” is a simplistic view of a complex problem. Unfortunately, it’s a sure-fire media sound-bite and it removes all responsibility for correcting many important problems from everyone else. How convenient. It’s time to grow up.
The reality is that FDA is at the center of the “21st century cures” innovation ecosystem. So rather than continuing to listen to the facile and ignorant drumbeat of “FDA as the problem,” emanating from certain quarters, groups ranging from the pharmaceutical industry, to patients groups, forward-thinking members of Congress, academia, and healthcare practitioners are beginning to realize (some faster than others) that FDA is a crucial partner – indeed a senior partner in advancing the public health.
Blame the FDA? The fault, dear Brutus …
PDUFA VI anyone?
"Some states are taking action against insurers’ use of high-cost sharing for specialty medicines. Specialty medicines are typically used by patients with severe or rare health conditions, generally less than 5 percent of patients in the U.S., but are often subject to greater cost sharing and utilization management techniques. Policymakers in several states have passed legislation to limit the amount that patients pay for specialty medicines.
For example, Delaware, Maryland and Louisiana have passed laws that cap copays at $150 per month for a 30-day supply of a specialty medicine. New York State passed a law that prohibits insurers from requiring higher cost sharing for specialty medicines, which treat diseases like cancer and HIV, than they do for other brand name medications. Several more states are considering legislation this year."
Here's a list of states that have limited cost discrimination:
And to find out what is going on in your state go to the National Patient Advocacy Foundation website or click here:
http://www.npaf.org/whats-happening-your-state
Los Angeles Business Journal
Shopping Site Looks to Bottle Up Prescriptions INTERNET: GoodRx teams with 4D to reach corporate customers, health plans.
By MARNI USHEROFF
People routinely look around for the best deal on a car, a TV or a nice pair of jeans. But they likely won’t shop at all for their monthly refill of Lipitor.
GoodRx Inc. has been trying to change that. The Santa Monica company runs a website allowing consumers to comparison shop for the best price on prescriptions at local pharmacies or by mail order.
The company, co-founded in 2011 by former Facebook Inc. and Yahoo Inc. employees, makes money from ads, referral fees from links to websites like Amazon.com for over-the-counter drugs, and minor administrative fees when consumers print coupons and present them at pharmacies.
But as of the first of the year, GoodRx took on a larger market, partnering with 4D Pharmacy Management Systems Inc., an established pharmacy benefits manager, to bring its shopping platform to health plans and corporate clients.
Rather than charge an annual contracted fee like other benefits managers, 4D and GoodRx are paid an administrative fee for each claim paid. The partnership has already signed San Antonio’s iHeartMedia Inc. as a client, allowing its employees to find the cheapest place to fill a prescription online, through an app or by calling an 800 number.
“Traditional pharmacy benefits managers are mysterious,” said Doug Hirsch, co-chief executive of GoodRx. “We’re going to pass through, show you exactly how much a drug costs. We’ll take a small administrative fee and show your employee exactly how much it costs.”
Jeff Polter, a 4D vice president for business development and account management, credits GoodRx co-founders and their tech background with improving the user experience.
“We are not technologists. ... They’re not (benefits) people,” he said. “You put the two together and we challenge each other.”
Yahooligan
Hirsch, 44, was inspired to take on the prescription market as a result of his own experience trying to fill an order for medication. He said he shopped around and found a prescription he needed that cost $250 at one drugstore and $300 at a supermarket, where the pharmacist chased him into the parking lot and offered to negotiate down the price.
“There’s all kinds of crazy discounts people can have with and without insurance,” Hirsch said, noting charges can vary wildly from pharmacy to pharmacy depending on dose, quantity and whether one is getting a capsule or tablet.
Hirsch, a veteran tech executive, used his experience to take on the new challenge.
His career started at Yahoo, which he joined in 1996 as one of the then-two-year-old company’s first product managers. Working at its Sunnyvale headquarters, he helped build such products as Yahoo Mail; Message Boards; and Yahooligans, a content site for kids. He later moved his family to Los Angeles, his hometown, and helped build out Yahoo Entertainment as its general manager.
He left in 2005 for a short stint as head of product at Facebook, leaving after six months to found DailyStrength, a health-focused social network where people could upload photos, write journals, support each other and share information about their treatments.
The company raised $4 million in venture capital and had 3 million visitors a month at its peak. Hirsch sold the company in 2008 to HSW International Inc. for $3.13 million in cash, with the opportunity to earn an additional $3.53 million if certain benchmarks were met, according to regulatory filings.
Hirsch worked on incubating startups until 2011, when he reconnected with Scott Marlette, a Georgia Institute of Technology-trained programmer with whom he worked at Facebook, and Trevor Bezdek, a programmer from Stanford University. Together, they co-founded GoodRx.
The company now has 27 employees and is profitable, said Hirsch, though he declined to disclose revenue. He did say that after an initial $1.5 million fundraise in 2011 from a combination of venture capital and angel investors, including Santa Monica’s UpFront Ventures, the company hasn’t had to go back for another round.
New approach
Hirsch said his team explored getting into pharmacy benefits management after being contacted by companies whose employees asked for the system to be integrated into their own prescription drug program.
The market is dominated by companies such as Express Scripts Inc., which racked up net income of $2 billion on revenue of $100 billion last year, and CVS/caremark, which acts as a benefits manager and has a nationwide network of pharmacies.
GoodRx ultimately went with 4D, a Troy, Mich., company with revenue in the “hundreds of millions,” according to Polter, that serves clients throughout the country, including government programs, commercial groups, cities and states.
Polter said clients must opt in to the program, which started serving clients Jan. 1 and has so far enrolled more than 100,000 users. The venture has about a dozen clients, including media company iHeartMedia, formerly Clear Channel Communications.
He said the platform is popular with clients whose employees are on high-deductible health insurance plans and must pay a lot out of pocket before greater reimbursements kick in.
That makes sense to Peter J. Pitts, a former associate commissioner of the Food and Drug Administration who is now president of Center for Medicine in the Public Interest, a New York think tank.
“It’s a great thing to know you can (comparison shop), especially for high-deductible plans,” Pitts said, provided consumers have transportation to get to the less expensive pharmacy.
He said that while pharmacy benefits managers are supposed to reduce costs for employers, their interests aren’t always aligned with those of consumers because many tack on a fee that’s a percentage of a drug’s price.
“A lot of times, kind of perversely, a pharmacy benefits manager will prefer a more expensive medicine because their markup will be more,” Pitts said. “The tightrope pharmacy benefits managers walk is weighing corporate profitability versus the best interest of the patient. And sometimes that goes hand in glove, other times there’s a conflict.”
He noted that pharmacy prices don’t just represent the manufacturer’s charge, but also how people all up and down the line make their money.
From a consumer’s perspective, the more transparent, the better, he said.
Hirsch doesn’t see his company walking such a fine line.
“We’re capturing profitability on a per-claim basis, not getting more on drug X versus drug Y,” he said. “Many pharmacy benefits managers have in-house mail order and are not only adjudicating claims but picking up money as an actual seller of drugs, maximizing profitability by sending people to themselves. We don’t offer that because we think that’s a conflict.”
The Payers Pity Party
AHIP (America’s Health Insurance Plans – the trade organization for the insurance industry) is holding it’s annual conference this week in Washington, DC. This year one of the issues they’ll be highlighting (not necessarily “debating”) is “high-priced” pharmaceuticals. Well, it’s their party – and they can cry if they want to.
It’s an interesting point of departure to note that the word “innovation” appears exactly once in the conference program. Since this is an event for payers it isn’t surprising but it is disappointing. Sins of omission are seldom fun – especially when those sins weigh the profits of payers over the needs of patients.
The only panel to discuss the value of innovation does so in a more than slightly slanted manner. Consider the title of the session, “Can We Afford New Advances in Biomedical Innovation?” And if the title doesn’t give you a clue as to the position of the organizers, consider the balance of the speakers – an EVP from CVS, a lobbyist from AARP, a thought leader who believes that “innovation “is too expensive,” and a representative from the biopharmaceutical industry, the industry that actually is primarily responsible for discovering, developing and funding innovation in US and across the globe.
Ignoring the value of innovation may be convenient, but it’s neither honest nor helpful to the broader public policy debate. Karen Ignagni, the President of AHIP, recently lead the charge against Sovaldi (an innovative medicine for Hepatitis C), accusing the drug’s developer — Gilead Sciences Inc. — of exploitative pricing. “The company in this case is asking for a blank check,” she said. “It will blow up family budgets, state Medicaid budgets, employer costs and wreak havoc on the federal debt” – a good and oft-repeated sound bite that is now widely acknowledged to be 100% wrong.
New, better medications are actually the best and swiftest way for this country to cut down on our health-care expenses. By more effectively combating disease and improving patients’ lives, drugs reduce long-term medical costs and bolster the overall economy.
Consider one pre-Sovaldi “best practice” treatment for Hepatitis C, the drug Pegasys. This requires one injection a week for 48 weeks — and very few patients see the treatment through to completion, so much of that treatment, both physician time and drug cost, is wasted. Nor is it that much cheaper: At about $7,000/month, the full course of treatment is over $70,000 — barely less than cost of the three months needed for Sovaldi to work a cure. And the price of not using Sovaldi is very high. One in three patients with the Hepatitis C virus eventually develops liver cirrhosis, and managing these patients is costly. A “routine” liver transplant (where the liver is from a cadaver) costs close to $300,000; a “living donor” transplant is even more expensive. But why let the facts get in the way.
It’s easy to point to “high-priced” drugs. It’s a savvy media strategy when your memberships’ focus is on short term savings and quarterly profits versus long-term patient outcomes (data recently published by the PwC Health Research Institute shows that the use of Sovaldi will actually drive down overall spending within a decade). It’s “savvy” because AHIP plays to an easy to articulate argument that the media and the general public is ready to accept, “healthcare costs are driven expensive drugs.” But facts are stubborn things. Consider that pharmaceuticals represent only 11.5% of our national healthcare costs – with on-patent “innovative” medicines representing only 8.5% of our healthcare resources. And their benefits are manifold, returning many times their cost via patient value.
Yet these and many other facts backing pharmaceuticals as a sound healthcare investment have been twisted to suit the agendas of politicians, pundits, and other competing stakeholders. It goes relatively unreported that insurance companies continue to increase their monthly premiums without really explaining why. The industry claims its costs are increasing because prescription drug costs are busting their budgets. But prescription drugs account for only a small part of monthly insurance-premium hikes. From 1998 to 2003, insurance companies increased premiums by an average of $104.62 per person. During that same period, drug costs rose by $22.48.
According to CMS-published data, patients pay less out-of-pocket for pharmaceuticals today than at any time in the past 40 years. This is the result of the proliferation of generic drugs following a strong period of innovation in the 2000’s. However, this is probably news to those patients who are seriously ill and require therapies that do not have a generic alternative. In those cases, patients are often asked to pay a significant amount of their own money for their medicines. If they find that they eventually can’t afford those out-of-pocket costs, they don’t take their medicine, leading to higher health costs elsewhere in the system. But treatment for a severe illness is exactly what should be covered as an insured benefit. Part of the solution to the out-of-pocket cost dilemma may be a more rational insurance design where patient cost sharing for various health services is based on the value of the intervention rather than price, as formularies should help patients and doctors make the best treatment choices, not forgo treatment all together.
Should we blame “Big Insurance”? Out-of-control out-of-pocket expenses cause many patients to stop using prescription drugs for controllable chronic conditions. The unfortunate result is that visits to the ER have jumped by 17 percent and hospital stays have risen 10 percent. And a new Integrated Benefits Institute study shows that when employers shift too much of their healthcare costs to employees, the companies lose more than they save, through absenteeism and lost productivity.
Having one person defend the value of innovation at a high profile event such as the annual AHIP event is stacking the deck against the pharmaceutical industry to be sure – but what’s worse (and far less forgivable) is the disservice it does to patients.
As Harvard University health economist (and health care advisor to President Obama) David Cutler has noted: “The average person aged 45 will live three years longer than he used to solely because medical care for cardiovascular disease has improved. Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost.”
Healthcare innovation saves lives, saves money, promotes economic growth, and provides hope for hundreds of millions of people (both patients and care-givers) in the United States and around the world. But innovation isn’t easy – and the task of defending it against a cognitively closed audience is certainly a challenging one.
Pharmaceutical innovation faces many roadblocks. Beyond the daunting scientific and regulatory hurdles, the complicated and conflicting dynamics of politics, perspectives on healthcare economics, of friction between payers, providers, and politicians, the battle for better patient education, and the need for a more forceful and factual debate over the value of innovation all create the need for a more balanced and robust debate.
Alas, the AHIP conference will not advance this agenda.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest
Instead of calling Zarxio simply filgrastim -- the desire of those who believe differential nomenclature is the devil incarnate, the FDA has dubbed the new product filgrastim-sndz, the suffix standing for Sandoz.
According to Dr. John Jenkins, director of FDA's Office of New Drugs the naming of this particular drug does not necessarily mean this will become the general policy for naming biosimilars.
Stay tuned and attuned.
Welcome Zarxio, the Sandoz version of filgrastim. While nothing is “easy” when it comes to drug development, many view this initial Sandoz experience as forecasting clear skies and fair winds for every biosimilar put in front of the agency for consideration.
If you believe that, there’s a bridge in Brooklyn you might find of interest.
The filgrastim example is an n of 1 and shouldn’t be viewed as predictive of anything other than the FDA approval for one product.
As surely anyone inside the agency or at Sandoz will tell you, the pathway to approval has been anything but easy. But that hasn’t stopped the sale of rose-colored glasses to many. So be it.
More important to consider is how the agency will continue to study global biosimilar experiences – and especially in the EU.
Case in point: Remicaide (infliximab), certainly a product on the radar screens of many as the next big biosimilar marketing opportunity – and regulatory challenge.
The Remicaide road has been a rocky one in the EU. A new poster presentation at the European Crohn’s and Colitis Organisation, titled, “Biosimilar but not the same,” offers some timely and important real-world data on the differences between originator biologics and their biosimilar cousins.
The study, from Mercy University Hospital, University College Cork, Centre for Gastroenterology, Mercy University Hospital, Cork, Ireland, studied the clinical impact of both the innovator product (Remicade) and it’s EMA-approved biosimilar (Inflectra). The findings are important. Specifically, the rates of surgery in Infliximab and Inflectra groups were significantly different.
80% of the Inflectra group required hospital readmission versus 5% of the infliximab (Remicade) group. (p=0.00004). 60% of patients in the Inflectra group needed steroid augmentation of standard steroid tapering protocol with 50% requiring multiple increases in steroid dose versus 8% of patients in the Infliximab (p-value = 0.0007). Over the course of 8 weeks, 93% of patients in the Inflectra group had an increase in CRP with 7% remaining unchanged whereas 100% of patients in the infliximab group had a decrease in CRP (p=<0.001).
The conclusion is not ambiguous, “Our results suggest that biosimilars may not be as efficacious as the reference medicine. The results found reflect the ECCO statement position that the use of most biosimilars in IBD will require testing in this particular patient population and cannot be extrapolated from other disease populations."
And now the British Society of Rheumatology (BSR) is calling for clinicians to register patients with its biologics registers to track the efficacy and long-term safety of new biosimilar products coming on to the UK market.
The BSR – the professional association for health professionals involved in rheumatology and muscular skeletal disease – maintains two biologics patient registers. One is for people being treated for rheumatoid arthritis, the other for patients with ankylosing spondylitis.
The BSR has released a position statement on biosimilars and says that it supports their introduction but wants to make sure that a system is in place to monitor their safety and efficacy in order to reassure health professionals and the public.
“Widening the range of treatments available to our patients at a lower cost to the NHS is clearly a positive step, but there are still a few gaps in the evidence for the safety and efficacy of these products, which will need to be explored further,” says Alex MacGregor, chair of BSR Biologics Registers.
“For example, the clinical trials of these drugs for people of rheumatological or autoimmune diseases were with new users only, and no trials have explored the effects of substitution from a reference drug.”
The release of the BSR’s position statement comes as biosimilar versions of Janssen Biologics’s Remicade — a medicine that contains the monoclonal antibody infliximab and is used for a variety of inflammatory conditions including rheumatoid arthritis — are being launched in the UK.
These First World data points about a product from a respected manufacturer (Hospira) cannot be ignored and must be used to inform the policy debate over nomenclature, interchangeability, label extrapolations, and overall pharmacovigilance practices. Surprising? Not if you understand that the decision tree for biosimilars is quite different from the Hatch-Waxman pathway. And that for the foreseeable future, each biosimilar application is going to be an important learning experience for FDA reviewers and senior division staff.
In case you missed ...
The Food and Drug Administration has finalized its guidance clarifying the agency’s hands-off approach to regulating mobile devices and apps. The agency has issued two documents outlining its position on medical device data systems and mobile medical applications, including those on smart phones. The documents reiterate the agency’s position that it won’t oversee low-risk medical devices, or will regulate them under a lower-risk classification. “Through smart regulation we can better facilitate innovation and at the same time protect patients,” the agency said.
The agency will, however, regulate mobile apps or devices intended to detect life-threatening conditions. “FDA’s oversight approach to mobile apps is focused on their functionality, just as we focus on the functionality of conventional devices,” the agency said. “Our oversight is not determined by the platform.”
(The documents are largely unchanged from draft versions.)
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
