Latest Drugwonks' Blog
I’ve just returned from Dubai (no, I didn’t do any shopping) where I was honored to speak at the Economist’s conference on Health Care in the Middle East: Evolution and Reform. My talk was on “Enhancing Public Health Through Innovation.”
One of my main points is that innovation doesn’t happen in a vacuum. People make it happen – and it’s an ecosystem: academics, manufacturers, physicians, patients, pharmacists, hospitals, caregivers, and government. If we allow ourselves to believe that innovation happens in bursts of “eureka” moments, we do a disservice to those who advance progress through a lifetime of work and through important failures. Innovation happens incrementally – and it’s expensive. The myth of “eureka innovation” is dangerous science fiction.
In the majority, the role that government plays in advancing innovation isn’t, as many think, the science that comes from institutions like the National Institutes of Health (although the NIH makes important contributions), it’s in being a public health partner across the board – and that includes medicine regulatory policy.
So, what is the role of regulators in advancing healthcare innovation? Regulators can be partners in innovation three ways: Through robust oversight. Through active collaboration. And, most importantly, by being an innovation enabler
And regulatory predictability is Step 1 in being an innovation enabler. And one aspect of predictability is an even playing field when it comes to quality. There can be only one level of quality for medicines – whether they be of the innovator or generic variety.
There must also be a dedication across the healthcare ecosystem to safety and quality in the post-market environment – more robust and actionable pharmacovigilance.
There must be the recognition that new medicines enhance, extend, and save lives and, as such, should be reviewed and licensed with all due speed.
There should be a recognition that medicines regulation must never be an arm of domestic industrial policy.
And, finally, that government’s role as an innovation enabler must go beyond words to deeds.
In the words of the American poet, John Andrew Holmes, “Speech is conveniently located midway between thought and action, where it often substitutes for both.”
The most important role government can play in supporting and advancing innovation is to enable action.
In addition to the complicated question of FDA regulation of Non-Biologic Complex Drugs (NBCDs), there's the legal morass of patent legislation. Per the latest on that front, BioCentury reports thatU.S. Supreme Court justices on Wednesday voiced skepticism about arguments from both sides in the petition by Teva to overturn an appeals court decision that invalidated the company’s process patent for multiple sclerosis blockbuster drug Copaxone.
The high court will decide whether the U.S. Court of Appeals for the Federal Circuit (CAFC) overreached by reviewing de novo certain questions of fact in Teva's lawsuit against the Sandoz International GmbH generics unit of Novartis AG, rather than deferring to the findings of the U.S. District Court for the Southern District of New York.
Teva’s suit alleges Sandoz’s ANDA for generic Copaxone infringed on Teva's patents. The district court ruled in favor of Teva in 2012. In 2013, the CAFC reviewed the facts of the case and instead invalidated the process patent for Teva's daily formulation of Copaxone, set to expire on Sept. 1, 2015.
In Wednesday’s arguments, Justice Stephen Breyer repeatedly challenged Sandoz representative Carter Phillips to explain why the case should provide an exception to civil procedure rule 52(a) that “fact-finding of the district court should be overturned only for clear error.”
Justice Samuel Alito suggested to Teva representative William Jay that the company was asking CAFC to “to struggle to determine” which factual questions have resulted in clear error, and “is it worthwhile as a practical matter?”
Justice Sonia Sotomayor also asked: "Why don't we defer, as has been done now forever, to the Federal Circuit and let them review these things de novo?"
The SCOTUS decision is due by the end of June 2015. Any generic competitor entering the market before the patent expires would have to pay damages to Teva if it is upheld.
We are not afraid of competing against mail-order pharmacies': Portland firm thrives in specialty medication market
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By Darren Fishell, Bangor Daily News, Maine |
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McClatchy-Tribune Information Services |
It's rare to hear a business praise its state's regulations, but a Portland pharmacy is relying in part on Maine law to gain a stronger foothold in the growing market for specialty medication.
By volume, the market is dominated by major national mail-order pharmacies, but the founders at Apothecary By Design see things shifting in their direction, making Maine one battleground between regional and national pharmacies selling specialty pharmaceuticals.
"The model of what is the right way to manage specialty pharmacy... that's a story that's not played itself out yet," said Mark McAuliffe, one of the pharmacy's five founders. "We would argue that it's critical to have a regional presence."
Specialty pharmacy is to retail pharmacy as the surgeon is to primary care. It's the fastest-growing segment of the pharmaceutical industry, driven by new treatments for conditions like Hepatitis C, cancer and multiple sclerosis.
That's part of what's driven Apothecary to grow more than threefold, to 64 employees, and increased revenue more than 21-fold since opening in 2008, the year it began with a focus on filling retail prescriptions in Portland'sEast Bayside neighborhood.
"We want to own specialty pharmacy in Maine," said Catherine Cloudman, another of the pharmacy's founders.
The company sees itself as part of a shift away from large mail-order pharmacies, whose promise of savings are a matter of ongoing public health policy debate at the national level.
The debate deals with the largely behind-the-scenes decision-makers who determine which patients on which health plans can qualify for coverage for which drugs. The discussion involves issues of health care cost, quality of care and choice for patients and doctors.
For patients, what's at stake is the range of choices a doctor has for treatments. That is, which drugs a given insurance plan will cover. But another aspect of choice comes in which pharmacies can provide drugs for a certain insurance plan.
Maine law requires that any certified pharmacy willing to meet an insurer's terms can fill a prescription.
"That's not true in a lot of other states," McAuliffe said. "We're not afraid of competing against mail-order pharmacies, we are afraid of not being allowed to compete."
But hurdles for getting into that market for specialty pharmacy are a little higher.
That's part of why, in August, Apothecary got accreditation from the group URAC and next spring will pursue approval for its specialty pharmacy from the Accreditation Commission for Health Care. The company's principals said the accreditation is a badge of honor in itself, but the signs of approval will also satisfy an all-important group of decision-makers in the pharmacy world: pharmacy benefit managers, or PBMs.
Generally, PBMs act as middlemen between health insurers and pharmacies, negotiating over which drugs an insurance plan will include, pricing and other details that make up what's called a formulary. Some of the largest PBMs, like Express Scripts, want two certifications.
With those certifications in hand, the integrated pharmacy expects to grow its share of the estimated $400 million market for specialty drugs in Maine. Last year, Apothecary invested $800,000 in an 8,700-square-foot expansion, growing its specialty pharmacy and adding a compounding pharmacy across the street from its first location.
The company's founders project they'll bring in around $85 million in revenue this year, up more than 160 percent from last year. About 95 percent of that will come from specialty drugs.
Still, the company expects to face continued challenges from PBMs apparently unaware of Maine law limiting how narrow a PBM can make its network. Cloudman said the company earlier this year spent time correcting letters sent out by insurers, telling patients that they were not able to cover prescriptions filled at Apothecary and instead need to use their affiliated mail-order pharmacy.
"We have to stay hypervigilant on that all the time," Cloudman said.
It's something Peter Pitts, president of the nonprofit Center for Medicine in the Public Interest, said happens often because of the structure of the system.
"The large PBMs are both judge and jury," Pitts said. "By denying a claim, they are often benefitting their own bottom line."
National pressure
In recent years, consolidation of PBMs and mergers between them and pharmacies and drug manufacturers have raised antitrust concerns at the national level.
The issue received particular scrutiny in 2012 with the merger of Express Scripts and Medco Health Solutions, creating a PBM the Federal Trade Commission said would control at least 40 percent of the market. Opponents of the deal, including principals at Apothecary, said that number is closer to 60 percent, giving the PBM strong influence over pharmacies and drug manufacturers.
That influence became clear this year. The New York Times reported in June that more PBMs have started to exclude from their formularies certain expensive specialty drugs to treat conditions such as cancer and multiple sclerosis in an effort to curb rising costs.
PBMs manage drug benefits for about 210 million people in the United States, and that market is dominated almost entirely by the three largest PBMs that also operate mail-order pharmacies to fill prescriptions.
Advocates of that system argue that centralizing drug purchasing through PBMs lowers costs. Critics say it has economic consequences for small pharmacies and health consequences for patients.
David Balto, a Washington, D.C.-based attorney and former policy director at the Federal Trade Commission, has argued a number of cases raising concerns about what he's called one of the "least regulated and least understood aspects of the health care delivery system."
"PBMs were formed to negotiate the best arrangements for insurance companies and employers with pharmacies and drug manufacturers," Balto said. "But when a pharmacy is owned by a PBM or a drug manufacturer is owned by a PBM, there is a conflict of interest and there's really a fox guarding the henhouse."
Principals at Apothecary said they share the concern that such joint ownership opens the door to conflicts of interest, though there is supposed to be a firewall in place to prevent collusion between PBMs and affiliated pharmacies.
Pitts, at the Center for Health Care in the Public Interest, contests that the PBMs provide cost savings in the health care system, arguing that limiting treatment options for doctors and patients incurs costs elsewhere for patient treatment.
"When you centralize distribution and when you remove choice, bad things happen around the margins and you have to keep a close eye on those," Pitts said. "There's a difference between medicines and commodities like air conditioners: certain [medications] work better for some people than others."
While smaller pharmacies like Apothecary have been concerned about being elbowed out of the game, those larger PBMs have recently started facing new pressures in their own market as well.
The Wisconsin-based Navitus Health Solutions is one of many so-called "transparent" PBMs, handling prescriptions for about 4 million people.
Instead of making a profit margin on each prescription filled, PBMs like Navitus charge insurers a flat fee and "pass-through" the cost of drugs directly from the manufacturer.
It is the PBM for Maine Community Health Options, the insurer for the majority of people getting coverage through the Affordable Care Act in Maine that recently announced that it will open plans to all of New Hampshire. Apothecary has contracts for specialty drugs with Navitus.
Brent Eberle, a vice president at Navitus, said that there's greater transparency in pricing with introduction of the Affordable Care Act and some employer groups looking to know more about the cost of drugs, especially for specialty pharmacy where the average cost of a prescription is about $2,000. And some of the larger PBMs are starting to offer transparent pricing options as well, he said.
In the end, pharmacies like Apothecary hope the growing demand for specialty pharmacy and the greater attention that requires gives them a niche for bringing pharmacy back down to a smaller scale while becoming a bigger part of the health care system.
"Even in the eyes of physicians, pharmacists are viewed as a commodity," Cloudman said. "We're having a lot of success changing that."
The facts:
NIH budgets have increased since 2001 except for 2011.
The NIH - through the National Institutes for Allergies and Infectious Diseases -- has received $11 billion for research and development to find diagnostics, drug and vaccines for Ebola and other lethal pathogens. In fact in 2010, NIAID received an additional $304 million when Congress diverted money for buying vaccines from the Bioshield procurement fund. And each year the budget was never cut, except for 2011.
If you go to clinicaltrials.gov and type in Ebola, Marburg or other viral hemorrhagic fevers and limit your search to NIH sponsored trials here's what you get.
The only study completed that actually reported results was a test of yellow fever vaccine on people with excema
The NIH developed 7 Ebola vaccines.. None of which worked. $11 billion. 7 vaccines. No results.
There are several drugs that do work. TKM-Ebola and three drugs from a company called Sarepta. AVI-7537 has already shown effectiveness in non-human primates against the Zaire Ebolavirus, the species implicated in the current outbreak. Moreover, Sarepta had been conducting Phase 1 safety trials with the drug alone and together another Ebola-directed PMOplus molecule (AVI-7539, with the combination called AVI-6002). Sarepta's work was being funded by the Department of Defense, NOT NIH. The administration and Congress could have transferred the money from Bioshield to the DOD work under a cooperative research agreement but it did not. The dough went to NIH.
According to a recent article in Forbes, Sarepta " does indeed have clinical trial-quality drug on hand. The Marburg program that has continued “uses the same backbone chemistry,” so the safety studies that are continuing with that drug at higher human doses could be applied to AVI-7537.
The company's CEO Chris Garabedian told Forbes: “We’re here to raise awareness that we do have a technology that might be helpful, and that we do have drug substance on hand if we received a request from a government agency,” said Garabedian. “We, of course, would have to get the appropriate waivers and approvals from the Department of Defense who supported the development of this compound as well as the FDA in terms of an emergency use authorization.”
Actually it's the small biotech startups and medical innovation that would be screwed if Towns has his way
My late colleague John Vernon Jr. explains why cutting IP protections to biologics will hurt Americans (from is article EXPLORATION OF POTENTIAL ECONOMICS OF FOLLOW-ON BIOLOGICS AND IMPLICATIONS FOR DATA EXCLUSIVITY PERIODS FOR BIOLOGICS. )
"A minimum of 17 years of data exclusivity or data protection is required to provide the necessary incentives for continued biotech R&D investments...evidence suggests that these incentives are in fact inadequate for pharmaceuticals in light of the social rate
of return to both pharmaceutical and medical R&D."
Vernon concluded that at present, the U.S. is under-investing in new medicines. "One study suggests that for every $1,345 invested in pharmaceutical R&D, a U.S. life year is gained, on the average. "
This return could be even higher if FDA regulation did not ratchet up the cost and complexity of drug approvals. We can find common ground on this important point!
In 1992, Congress created a program -- known as "340B" -- to help caregivers serving disproportionately large numbers of low-income beneficiaries and uninsured patients. Under 340B, drug manufacturers are required to sell their products at a discount to such institutions. The discounted prescriptions are dispensed either through the caregiver's in-house pharmacy or through a contractual arrangement with an outside pharmacy.
340B has a noble cause. And many of the medications discounted through 340B do in fact go to clinics, hospitals, and medical facilities providing care almost exclusively to uninsured and poor patients.
However, some 340B participants are exploiting the program and the vulnerable patient populations 340B was intended to help are often still stuck struggling to gain access to affordable pharmaceuticals.
To wit, Pharmaceutical Research and Manufacturers of America (PhRMA) Executive Vice President and General Counsel, Mit Spears has issued the following statement:
“PhRMA strongly supports the 340B program, which is intended to help vulnerable and uninsured patients obtain access to life-saving medicines. We are committed to working with all stakeholders to improve the program to ensure it benefits those it was intended to help. To achieve this important objective, it is critical the program operate in a manner consistent with the clear direction of Congress.
“At issue is the Health Resources and Services Administration’s (HRSA) interpretation of the 340B orphan drug exemption, enacted as part of the Affordable Care Act (ACA). The ACA significantly expanded the type of entities that can access 340B discounts for prescription drugs. To preserve incentives to invest in research and development of new treatments for rare diseases, the ACA expressly exempts manufacturers from having to provide these discounts on orphan drugs to newly eligible providers.
“After the Federal District Court of the District of Columbia vacated the HRSA July 23, 2013 rulemaking regarding the 340B orphan drug exemption, in July 2014, the agency issued the exact same rule, but labeled it ‘interpretive.’ HRSA’s action in this regard is unlawful.
“While we value the hard work and efforts of all agencies, it is important federal agencies recognize and work within the bounds set by Congress. PhRMA is therefore filing suit against the U.S. Department of Health and Human Services to challenge its second attempt to issue a rule conflicting with the plain language of the statute.
Go get’em, Mit!
In case you missed the FDA’s Viewpoint commentary in JAMA, here it is:
Addressing Prescription Opioid Overdose: Data Support a Comprehensive Policy Approach
And it’s not about banning Zohydro:
“The risk in singling out a single drug like Zohydro ER in this complex, multidrug epidemic is that resulting policy is unlikely to have an effect on the underlying causes—the abuse of multiple opioids and other drugs and inappropriate prescribing.”
The first sentence of the concluding paragraph says it all, “The problem of opioid overdose demands well-informed policies.”
Not knee-jerk. Not political. Not bowing to threats. Well-informed policies.
Attention must be paid.
Leading medical innovation think tank claims CBS is ‘dead wrong’
New York, NY (PRWEB) October 08, 2014
The Center for Medicine in the Public Interest (CMPI.org– a non-profit research organization specializing in promoting medical innovation) criticized CBS’ “60 Minutes” segment on cancer treatment prices as not just distorted but ‘dead wrong.’ “‘60 Minutes’ ignores the contribution new medicines make in reducing health care costs, improving wellbeing and saving lives,” said Robert Goldberg, Vice President for Research of CMPI. “Featuring physicians that argue that treatment prices are too high does not change the fact that the cost of cancer drugs are about 12 percent of what health insurers spend on cancer or that new cancer drugs actually save health insurers money. Yet insurers are increasing oral therapy cost-sharing requirements to actively encourage patients to use infusible products.
While a Milliman study found that shifting to co-insurance would only add about $2 per member per month in private health plans, 60 Minutes never discusses this solution. Instead, 60 Minutes remains silent about the cost shifting and has never advocated for co-pay reforms that could relieve the burden on patients.
Spending on cancer treatments has climbed from $24 billion in 2004 to about $40 billion today. But such treatments represent only 0.6 percent of U.S. healthcare spending, and that proportion has been fairly consistent for the last several decades.
Such medical innovations were largely responsible for a 40 percent increase in cancer survivors (from 9.8 million to 13.6 million) since 2004. Since 2004 the use of new therapies saved $188 billion on hospitalizations, and avoided nearly $100 billion in lost productivity each year. Since 1990, new cancer drugs have helped generate 51 additional years of life, worth nearly $5 trillion.
A doubling in the use of new-targeted therapies will raise the amount spent on medicines as a percentage of total health care spending. But that increase must always be compared to what would be spent on healthcare, disability, unemployment and rehabilitation in the absence of medical innovation.
Peter Pitts, the President and Co-Founder of CMPI noted, “New medicines almost always reduce the cost of living longer and healthier lives and increase the value of such improvements. It is disappointing that “60 Minutes” failed to investigate why some health plans have deliberately made these innovations unaffordable by requiring patients to pay up to half their costs. And it is disheartening to know that doctors are more interested in attacking innovators than in defending patients from such cost shifting.”
Pitts also remarked, “We are happy to talk about the prices of cancer therapies, but only in the context of value and the rising cost and prices of other healthcare services that constitute a substantially bigger portion of total spending. In addition, the discussion should only be in the context of what can and should be done to cut the cost of developing new therapies through smarter, faster regulation.”
CMPI has a website—valueofmedicalinnovation.org—that provides a balanced view of the role new medicines play in our lives.
The Center for Medicine in the Public Interest is honored to join the US Pain Foundation and other patient-centered organizations in offering our support to the FDA in general and Commissioner Hamburg specifically for savvy and strategic management of pain medicine regulation.
Why now? Well for starters because of the unfair, unbalanced, ad hominem and just plain erroneous attacks on the agency’s efforts.
It’s all spelled out in this letter of support just sent to HHS Secretary Burwell. It’s designed for impact.
Have a look and please share. This issue is too important to allow the lunatic fringe to drive the agenda.
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.
