Latest Drugwonks' Blog
Research-based pharmaceutical industry launches collaborative framework to tackle NCDs
Geneva, New York, 16 June 2011 – The research-based pharmaceutical industry launches today a Framework for Action to respond to concerns that cardiovascular disease, cancer, diabetes and chronic respiratory diseases are posing mounting threats to public health as well as public and private finances worldwide. The industry’s ten-point Framework scopes out specific areas of action to tackle non-communicable diseases (NCDs) including innovation, access and affordability, prevention and health education. It also underscores the essential role of partnerships and dialogue in implementing the Framework. The NCD Framework for Action brings together the global research-based pharmaceutical industry in support of the World Health Organization’s (WHO) Action Plan on NCDs and the Moscow Ministerial Declaration. It will be presented today at the United Nations (UN) Civil Society Hearings in New York, held to prepare for the UN High Level Meeting on NCDs to take place in September, which aims to secure an action- oriented UN response to the rise of NCDs. The aim is that the NCD Framework for Action will provide a sound basis for the research-based pharmaceutical industry to partner with the people on the ground, governments and the WHO to find ways to address prevention, care and treatment for NCDs in the developing world.
NCDs are the leading cause of death and disease worldwide, killing more than 36 million people in 2008, with nearly 80% of these deaths occuring in low- and middle-income countries. NCD deaths are projected to increase by 15% globally between 2010 and 2020. In part, this is due to progress made in combating infectious diseases through economic growth, development, and better treatment options; but it is also largely linked to lifestyle choices. For most infectious diseases, rapid access to diagnosis and treatment is an imperative for patient survival. But for NCDs the “lifecycle” is different, and there may be many actions that can take place, such as prevention, before medicines or other treatments need to be prescribed.
The Framework for Action on NCDs focuses on the areas where the research-based pharmaceutical industry can make the most significant difference, such as innovation, access and affordability, but also prevention and health education. Eduardo Pisani, IFPMA Director General explains: “The framework is just the beginning; our vision is to work with others to identify what can be done in practice to help poor people to access the care and treatment they need. Together we want to find ideas for concrete actions to put on the table in the aftermath of the UN NCD Summit in September. The Framework is our roadmap for this work.” The IFPMA also pledges to report regularly on industry’s progress and share updates with stakeholders, such as the WHO and other interested agencies.
The Framework confirms the industry’s crucial role in continued investment in R&D programmes dedicated to the development of innovative medicines for the prevention and treatment of NCDs to enhance the lives of patients. There are currently over 1,500 medicines in the pipeline for major NCDs. The industry will also endeavour to address its innovation towards the specific needs of developing world populations and settings. To this end, in addition to the Framework, the pharmaceutical industry will launch a programme of research that will improve its understanding of the specific needs of developing world populations.
Actions on access and affordability of NCD treatments include promoting the right policy, regulatory and supply chain environments that secure optimal quality of care for patients and enable companies individually to implement commercially sustainable access and pricing strategies for the supply of NCD vaccines and medicines to the developing world.
It is estimated that half of deaths caused each year by NCDs are preventable[1], and lifestyle choices that often accompany improvements in living standards, such as unhealthy diet and tobacco use, are part of the explanation. The Framework for Action highlights the importance of prevention so that individuals can make informed lifestyle choices. Industry is also working with the World Health Professions Alliance to develop an NCD scorecard that will be shared with over 26 million health care professionals in more than 130 countries to help encourage patients to identify and prevent risky behaviors. Reducing disability or deaths through increased investment in prevention programs will contribute to higher economic growth and allow limited resources to be focused efficiently on patients most in need.
Eduardo Pisani, IFPMA Director General explains: “This NCD Framework for Action represents a paradigm shift for the research-based pharmaceutical industry. It puts our industry’s collective global health responsibilities firmly at the forefront of how we see our role in the global health community. Let’s be clear: it is not about altruism, but rather about revolutionising our relationship to others. Times are tough for governments, business and patients. In order to tackle the rise of NCDs, and stay the course, we need to look at sustainable new approaches to global health which have prevention at its core.”
Now Avorn and part of his crew, Aaron Kesselheim and Jan Myers are arguing that drugs for rare diseases are not studied long enough and don't have enough people in clinical trials AND should be studied in randomized controlled trials.
Avorn, Kesselheim and Myers to kids dying of rare disease: Drop dead while we study you as long as we deem it appropriate. Note to Kesselheim and the rest of the Kevorkian Corp: orphan drug trials are designed to reflect the size and needs of patients, not the use of CER as a rationale for rationing.
Orphan drugs add more years to life than any other class of treatments. Two-thirds of the people who benefit are kids who can now live longer instead of dying painfully. But CER advocates like Avorn and Kesslehim want longer and more complicated trials because they know, as with Avastin , average results will reduce the number of treatments. CER will be a death sentence to many children. People like Kesselheim and Avorn raise the bloody shirt of drug safety to justify their position. Here's my view: let them say no to orphan drugs when it might save the life of their loved ones out of safety concerns and leave the rest of us alone. Please.
Thankfully, Congress is putting it’s foot down in support of innovation and personalized medicine. Sen. Robert Casey, D-Penn., has introduced legislation last March to support this research. The Creating Hope Act of 2011, will provide incentives “to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions." Meanwhile, Senators Jon Kyl R-TX and Mitch McConnell R-KY are sponsoring The Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2011. The PATIENTS Act would bar the federal government from using “comparative effectiveness research” to deny or delay coverage of a health-care treatment….”
Today medical innovatons can use the understanding of how we get sick to prevent disease or death. Such advances lead to longer, better lives, enriching us all. CER, by design, censors these biological insights. It deliberately delays progress by demanding studies that, by ignoring individual differences, conclude no one benefits from medical progress. It is used to justify rationing, not make individuals more sustainable. To save ourselves and children dying of rare diseases we have to pull the plug on CER and it’s adherents. Starting with Kessleheim, Myers and Avorn -- Harvard's Kevorkian Krew -- is a great place to start.
[1] http://jama.ama-assn.org/content/305/22/2320.short
From today's edition of The Washington Times:
Protect inventors with strong patent reform:
By Peter J. Pitts
While there are many problems with the U.S. health care system, it's undeniable that our country leads the world in producing the most advanced medical techniques, drugs and lifesaving technology.
Today, our leadership in this field is being threatened - not just by taxes, regulations or the new health care reform law - but also by a patent system that is increasingly ill-equipped to do its job of rewarding inventors and innovation.
Now that we are tantalizingly close to reforms that will modernize and strengthen the U.S. patent system, a small group of misguided ideologues is threatening to derail the effort.
There's no question that innovation is the lifeblood of our economy and strong patents are innovation's backbone. But over the years, the U.S. patent system has failed to keep pace with changes in the economy that demand stronger patents, faster approvals and patents that are recognized by our key trading partners.
There are numerous problems. For starters, the Patent and Trademark Office (PTO) faces an astonishingly large backlog of 700,000 patent applications, and inventors typically wait three years before getting a patent. Inventors then face the prospect of lawsuits challenging their patents, which cost an average $400,000 to defend.
Because the U.S. patent system is different from those of most other industrialized countries, inventors can find themselves with patent rights in the United States, but not abroad, cutting the value of their patents.
As Rep. Lamar Smith, Texas Republican, put it: "The current patent system is outdated and bogged down by frivolous lawsuits and uncertainty regarding patent ownership."
Legislation pending in the House would go far to fix these problems. It would, for example, let the PTO set its own fees so it has the funds needed to do its job, ending congressional raids on the PTO's budget while protecting small, independent inventors with heavily discounted fees.
It would more effectively weed out wrongful patents and settle disputes through a vastly improved "supplemental examination" process.
But most important, the legislation would shift the United States from a "first-to-invent" system to a "first-to-file" system.
As things stand, if an inventor is the first to file a patent, he still can be challenged by someone who claims to have invented the same thing earlier but failed to file it with the PTO. That results in too much uncertainty and far too much litigation over patent rights, which is why so many other countries moved to a first-to-file system. Under that system, the first to file with the Patent Office is the rightful patent holder, making patents easier and less costly to defend.
Most lawmakers in the United States realize the need to make these changes, and earlier this year, the Senate voted 95-5 to pass the America Invents Act. A similar bill passed the House Judiciary Committee by an equally strong 32-3 vote.
But with the finish line in sight, a small group of conservative lawmakers want to derail the bill, claiming it is an unconstitutional attempt to subvert our patent system in deference to European and Asian governments. Phyllis Schlafly has called it an "un-American" effort by liberals to "put us on the road to a borderless patent system." Those arguments are misguided at best.
First, the fact that the reforms so far have received overwhelming support from GOP lawmakers shows that it's not some liberal conspiracy. The House bill's sponsor, Texas' Mr. Smith, has a lifetime American Conservative Union rating of 92 out of 100.
Second, the measure is clearly constitutional. In fact, Michael Mukasey, who was President George W. Bush's attorney general from 2007 to 2009, reviewed the bill and concluded that it was "both constitutional and wise."
Third, the change from first-to-invent to first-to-file isn't as revolutionary as critics make it seem. PTO Director David Kappos notes that of the 3 million patents filed in the past seven years, just 25 were granted to inventors who weren't the first to invent, with just one granted to an independent inventor.
"In the last seven years," he told a congressional panel in March, "only one independent inventor's filing would have received a different outcome under the first-inventor-to-file system. That's one in 3 million."
What these reforms would do is make the patent system stronger, more transparent, fairer, faster, less costly and more in line with our major trading partners. Does that seem un-American?
In the past, our patent system served our inventors and our country as well. Now we have the opportunity to renew our patent system so it can effectively encourage, protect and reward inventors and keep the United States on the cutting edge of health care well into the next century.
Let's not blow it.
tinyurl.com/3rkxvkp
Psychopharmacology in crisis
Researchers warn of 'withdrawal of hope' as funding shrivels.
Patients face a "withdrawal of hope" as neuropsychopharmacology funding falters.SOVEREIGN, ISM/SCIENCE PHOTO LIBRARYMany people affected by mental illness are facing a bleak future as drug companies abandon research into the area and other funding providers fail to take up the slack, according to a new report.
Produced for the European College of Neuropsychopharmacology (ECNP), the report warns that "research in new treatments for brain disorders is under threat". With current treatments inadequate for many patients, it says, "withdrawal of research resources is a withdrawal of hope for patients and their families"1.
A number of formerly big players in neuroscience have all but abandoned the area recently as the pharmaceutical industry has undergone massive restructuring. AstraZeneca and GlaxoSmithKline have both cut research funding and closed down entire teams dedicated to developing drugs for psychiatric disorders.
“These are dark days for brain science.”
Although some of the problems faced by the field also apply to other sections of the pharmaceutical industry, many are specific to researchers trying to hit targets in the brain.
David Nutt and Guy Goodwin, who authored the report following a recent ECNP meeting on the topic, note that it can take much longer to develop medicines for psychiatric disorders than for better-understood conditions such as cancer, and that potential drugs for psychiatric conditions have higher failure rates. These failures sometimes become apparent only late in the development process, making neuroscience an expensive and risky prospect for industry.
The coming crisis
Nutt, a neuropsychopharmacologist at Imperial College London, told reporters at a press conference in London on 13 June that "these are dark days for brain science".
Both authors add that, in addition to the dearth of pharmaceutical funding, there is still a stigma surrounding conditions such as depression. This feeds through into the money donated to advocacy groups. "Almost nothing" comes from charity groups for mental-health research, compared with huge charity funding in areas such as cancer, notes Goodwin, head of psychiatry at the University of Oxford, UK.
He warns of a "generational crisis" in terms of both training and capacity to develop new drugs for conditions such as depression and dementia, unless the withdrawal of pharmaceutical funding is addressed.
This warning is echoed by an editorial published last week in the British Journal of Clinical Pharmacology (BJCP). 'Vanishing clinical psychopharmacology', written by Joop van Gerven and Adam Cohen of the Leiden University Medical Centre in the Netherlands, outlines the perilous state of the field2. Over the past year, the authors write, the BJCP has published only five papers in this area, none of which involved novel drugs.
At the 2011 meeting of the American Society for Clinical Pharmacology and Therapeutics, only 13 of 300 abstracts related to psychopharmacology and, again, none related to novel drugs. This situation mirrored that at the 2010 Collegium Internationale Neuro-Psychopharmacologicum, where 8 of 870 abstracts were on human psychopharmacology and four were on "new or relatively new mechanisms of action", they report.
The road ahead
Cohen says that much of the problem derives from a failure to develop the underlying science. Depression is a complex disease, yet to assess whether drugs work, researchers have to rely on crude tools such as questionnaires.
"People have not paid enough attention to how to measure depression, how to measure psychosis," he says. "When we develop new drugs, we still measure on these basic scales."
Developing new ways of assessing brain function and disease will reduce the risks in developing new drugs, van Gerven and Cohen argue in their editorial.
Nutt and Goodwin also suggest a number of ways forward. Patents could be longer-lived for drugs that take longer to develop, such as those for brain disorders, to encourage companies to work in the area. And researchers should lobby for European funding — such as that available from the Framework programme initiative — to set brain research as a priority.
Academia could also develop more creative relationships with industry in order to fill the gap in drug development, they argue. The ECNP is pushing the idea of a 'medicines chest', to which companies can assign compounds they are no longer actively developing to be taken forward by researchers in academia or elsewhere. Nutt says that several companies have already expressed an interest in this idea.
If the current research base is allowed to evaporate, Nutt warns, it will be decades before it can be built up to start again.
FDA NEWS RELEASE
For Immediate Release: June 10, 2011
Media Inquiries: Morgan Liscinsky, 301-796-0379, morgan.liscinsky@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA
FDA approves redesigned labels for some Merck drugs
Changes developed under Merck’s Label Standardization Project
The U.S. Food and Drug Administration today is announcing the approval of Merck’s redesigned drug container labels that include a new standardized format to improve readability and provide better information on product and strength differentiation.
Merck’s Label Standardization Project includes the revision of 34 container labels for 16 solid oral drug products regulated by the FDA’s Center for Drug Evaluation and Research (CDER). Drugs affected by the revisions include: Cozaar, Crixivan, Hyzaar, Isentress, Janumet, Januvia, Mevacor, Noroxin, Prinivil, Prinizide, Propecia, Proscar, Singulair, Zocor, and Zolinza.
“We commend Merck for their efforts,” said Janet Woodcock, M.D., director of CDER. “This was no small undertaking, and we are hopeful that Merck’s new standardized labels will aid in reducing pharmacy selection errors.”
Merck's project included evaluating the proposed label content and layout, selecting new packaging design, and obtaining regulatory approval to implement the new packaging design. The Label Standardization Project process included:
- a scientific approach to label design through Human Factors Engineering and Usability Studies
- incorporation of feedback received from the FDA and from label surveys
- a bundled supplement regulatory approach to ensure that labels were acceptable across CDER’s eight clinical divisions.
To better access the impact of these label changes, the FDA encourages health care providers to report medication errors related to the products included in Merck's Label Standardization Project to MedWatch, the FDA’s adverse event reporting program.
The bad news is that safest place to be in Washington these days is between Chuck Schumer and the facts about Part D. Safe -- because it’s the path less traveled. Bad news because he has recently begun trumpeting the need for government price controls and extended rebates for duel eligibles as the “solution” to increased Medicare spending.
New York’s senior senator is living in talking point fantasyland.
The good news is that accumulating evidence shows that Part D is succeeding beyond all expectations, delivering needed prescription drugs to Medicare beneficiaries for less money than anyone expected—driven by strong competition among plans.
Consider:
Current CBO Estimates Show Part D Is Costing Far Less than Initial Estimates
* The 2011 CBO Medicare Part D baseline forecasts and actual recorded spending show costs for Part D benefit payments have declined by 46%, for the 2004 to 2013 period compared with initial estimates of the 10-year cost projections for those years.[i]
* Also this year, CBO reduced its baseline 10-year spending projections for all of Medicare by $186 billion, mostly due to lowered drug spending forecasts. CBO cites that “[a]pproximately two-thirds of the change comes from reducing the projected growth rate for Part D (prescription drug) spending per enrollee on the basis of an updated analysis of national trends in spending for prescription drugs.”[ii]
Part D Plan Bids Declined, Even as the Value of the Benefit Increased
The Affordable Care Act (ACA) significantly enhanced the value of Part D benefits by providing:
* a one-time $250 tax free check for beneficiaries with any spending in the gap in 2010
* a 50% discount on branded drugs while in the coverage gap beginning in 2011; and
* a phased-in closing of the remaining coverage gap by 2020 for both brand and generic drugs.
Despite these significant enhancements to the value of the benefit, Part D plan bids, for which plans need to be accurate since they are at risk, decreased for the 2011 plan year. According to CMS, the Part D National Average Monthly Bid Amount for 2011 is $87.05, a decrease of $1.28 (or -1.4%) compared with the 2010ii] Further, the Medicare Trustees estimate that “[f]or 2011 and beyond, the bids are projected to ultimately converge to between 1 and 2 percent lower than actual spending due to aggressive plan bidding.v]
Medicare Trustees Find Continued Robust Negotiation of Drug Rebates by Part D Plans
In analyzing Part D spending, the Medicare Trustees cite significant levels of rebates across the universe of drugs in the program, including brands and generics. In 2011 the Trustees reported, “rebates for 2009 were approximately 11.1 percent of total prescription drug costs, which was somewhat higher than the plans estimated in their bid submissions. However, some of the drugs with the highest Part D rebate amounts will be losing patent protection in the next several years. As a result, rebates are projected to decrease from 10.7 percent in 2010 to 9.7 percent in 2020.”[v]
The Trustees clarify that the reported rebate levels “are average rebate percentages across all prescription drugs. Generic drugs, which represent about 72 percent of all Part D drug use in 2009, typically do not carry manufacturer rebates. Many brand-name prescription drugs carry substantial rebates, often as much as 20-30 percent.”[vi]
Recent Research Shows that Competition among Part D Plans Lowers Drug Spending in the Private Sector
Economists from the University of Southern California and Boston University have found that because Part D resulted in more people being covered by private insurance plans, the plans’ negotiating powers were increased such that “[o]n average, Part D lowered retail prices for commercial insureds by 5.8% to 8.5%. The cost-savings to the commercial market amount to $3 billion per year, which approximates the total annual savings experienced by Part D beneficiaries who previously lacked drug coverage.”[vii]
Average Beneficiary Part D Premiums in 2011 Are 43% below Original Projections
The average monthly beneficiary premium for Part D coverage will be $30 in 2011, far below the $53 forecast originally, and an increase of only $1 over the 2010 average premium of $29. According to CMS Administrator Don Berwick, “[t]hese very modest increases in premiums, along with the new discounts...are going to make medications more affordable to Medicare beneficiaries”[viii] CMS officials report that in 2011, over 99% of Part D enrollees will have access to a plan with a premium that is the same or lower than their 2010 premium.[ix]
Beneficiaries Are Highly Satisfied with Part D
Recently released polls show that Medicare Part D enrollees are overwhelmingly satisfied with their Part D coverage. It shows 84 percent of Part D enrollees are satisfied with their coverage, and 95 percent say their coverage works well. Additionally, vulnerable beneficiaries who are dually eligible for both Medicaid and Medicare exhibited the highest satisfaction.[x]
Senator Schumer should check the facts before he points the finger.
[i] See CBO Medicare baselines for 2005 through 2011 available at www.cbo.gov
[ii] CBO, “Preliminary Analysis of the President’s Budget for 2012,” March 18, 2011, p. 12. http://www.cbo.gov/ftpdocs/121xx/doc12103/2011-03-18-APB-PreliminaryReport.pdf
[iii] See: CMS Office of the Actuary memo, “Release of the 2010 Part D National Average Monthly Bid Amount,” August 13, 2009, CMS Office of the Actuary memo, “Release of the 2011 Part D National Average Monthly Bid Amount,” August 18, 2010. Both memos available at found at http://www.cms.gov/MedicareAdvtgSpecRateStats/RSD/list.asp.
[iv] 2011 Medicare Trustees Report, p. 184.
[v] 2011 Medicare Trustees Report, p. 183.
[vi] 2011 Medicare Trustees Report, p. 183. Footnote 76.
[vii] Lakdawala, D. and Yin, W. “Insurers’ Negotiating Leverage and the External Effects of Medicare Part D” NBER Working Paper 16251. August 2010, www.nber.org/papers/w16251.
[viii] CMS Press Release, “Medicare Prescription Drug Plan Premiums to Increase Slightly Medicare Beneficiaries May Need to Enroll in New Plans,” August 13, 2009.
[ix] MedPage Today, “Medicare Part D Premiums Going Up by $1 in 2011,” Emily P. Walker, August 19, 2010.
[x] [x] KRC Survey for Medicare Today, “Seniors’ Opinions About Medicare Rx: Fifth Year Update” September 2010.
From the pages of the New York Times:
Letter
Warning Labels on Drugs
June 10, 2011
“Side Effects? These Drugs Have a Few” (Week in Review, June 5) rightly ridiculed the growing number of warning labels on prescription drugs. But it’s the Food and Drug Administration’s job to promote the safe and effective use of medications and medical technology.
Unfortunately, the current liability system encourages well-financed tort lawyers to view new product warnings or withdrawal decisions as signals to file lawsuits in hopes of securing a quick payday. Such irresponsible litigation doesn’t make America’s health care system safer — it just helps a small number of lawyers get rich.
PETER PITTS
New York, June 6, 2011
The writer is president of the Center for Medicine in the Public Interest and a former F.D.A. associate commissioner.
According to Politico, “Consumer advocates are blasting the FDA for the process it has used to renegotiate the terms of the drug approval process following news that a deal has been reached with industry on proposed changes to the process.”
The FDA had been holding two sets of meetings in working out an agreement on the Prescription Drug User Fee Act, which Congress must reauthorize in 2012. One set has been held with representatives of PhRMA and the Biotechnology Industry Organization, the other with stakeholders including patient groups, consumer advocates and provider organizations.
According to an email obtained by POLITICO, industry accepted an FDA proposal “shortly before Memorial Day weekend” and forwarded an agreement to HHS last week. But stakeholders did not receive details on the deal until Wednesday morning. The email, which was forwarded to POLITICO by a source, was sent by Theresa Mullin, the Director of the Office of Planning and Informatics in the FDA Center for Drug Evaluation and Research.
“I was not surprised that we were left in the dark,” said one rueful consumer advocate participating in the stakeholders’ meetings on background. “We went through the motions but the real deal was over there with industry and the process was opaque.”
FDA spokeswomen Karen Riley declined to comment on any of the specific details of this process.
"The package of proposed recommendations is currently undergoing further administration review and that we cannot comment on the details of the package," Riley wrote in an email back.
That’s one question we can be sure Jenny McCarthy and other anti-vaccine adherents will never answer.
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.
