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The following from Dr. Michael Weber, Chairman of the Center for Medicine in the Pubic Interest and a founding father of the Association of Clinical Researchers and Educators (ACRE).
Most medical progress in the last few decades has come from the combined efforts of medical practitioners and the pharmaceutical industry, the medical device industry and the makers of other innovative therapeutic and diagnostic products.
Physicians, particularly those in academic settings, are uniquely able to identify unmet needs, design research to address these needs, and ultimately interpret and disseminate the results of that work to the benefit of their colleagues and patients in the community. Industry has the talent and resources to undertake the detailed research and development to complement the work of their physician partners and to deal with the highly complex scientific, regulatory and financial hurdles that must be crossed before new products can be made available.
Sadly, this highly productive collaboration has come under attack. The reasons are nothing to do with scientific or ethical concerns, but rather are driven by the fear of health care insurers and some politicians and media writers that medical progress comes at a financial cost that could threaten the profits of commercial insurers and the budgets of government agencies.
While understandable, these attempts to stifle medical research and education -- for instance, such tools as the Sunshine Act within the Affordable Care Act -- create an even higher cost, for they will have the effect of preventing access of people with medical needs to new clinical developments that could increase the length and quality of their lives.
The Association of Clinical Researchers and Educators (ACRE), in response to these dangerous constraints being placed on physicians, has gone back to basics and written a statement – in essence, a set of guidelines – on how physicians should interact with medical industries to fully preserve the high ethical standards, scientific integrity and clinical productivity that have characterized this vital work.
The guidelines (as published in Endocrine Practice) can be accessed here.
Read More & Comment...An ode to healthcare in 2013
IPAB, PCORI (of which neither I’m keen).
The FDA trying to do the right thing
While the Doc Fix sends physicians to pawn all their bling.
New state exchanges will make us all guess
And there’s dual eligible coding down at CMS.
What will become of the tax on devices
Will it come down to a Congressional role of the dices?
Obamacare will result in rule-making galore
And who knows what Kathleen Sebelius might have in store?
What health benefits will be seen as “essential”
And how will they be viewed in the Manse Presidential?
What of the pharmacists' expanding new role
Will academic detailing be the price of their soul?
Will innovation be daunted via less exclusivity
Or will clearer minds win the day with lucidity?
Will we move closer to embracing effectiveness that’s comparative
Or will we discover a more personalized narrative?
All this and more in the New Year, my friends
Let’s hope that it centers on a patient’s best ends.
Read More & Comment...From the pages of the Washington Examiner:
Obama's fiscal cliff drug 'reform' idea will backfire
As part of the fiscal cliff negotiations, the White House has trotted out a deeply misguided healthcare idea. The President has his allies want to loosen "intellectual property" protection for an advanced class of pharmaceuticals called "biologics."
This is a mistake. Including this plan in any federal budget deal would inevitably undermine drug innovation, compromise care for millions of American patients, and -- ironically -- drive up long-term healthcare costs.
Unlike traditional chemical drugs, biologics are derived from living organisms. This makes them incredibly complex -- and uniquely effective. Biologics are one of the most promising modern healthcare technologies. Drug firms have already created breakthrough biologic treatments for some of the country's most prominent diseases, including HIV/AIDS, Alzheimer's and various forms of cancer.
A report from the research firm Credit Agricole predicts that six of the top twenty best-selling drugs next year will be biologics. Within five years, it's estimated biologics will comprise roughly half of the top 100 bestselling drugs in the country.
There are knock-off equivalents to biologics. They're called "biosimilars" or "follow-on biologics." However, unlike, say, that aspirin in your bathroom cabinet, a biologic can't be perfectly replicated. It's just too complex. A "follow-on" biologic will have some differences from the original.
It is possible to create a version of an innovator biologic that's close enough to the original to have basically the same therapeutic effects. Given this fact, traditional patent protections aren't sufficient to foster innovation.
This is why biologics need an additional layer of intellectual property protection. And that's where data exclusivity comes in. This statute prevents outside firms from accessing the research data behind a new biologic for a preset period of time. This way, the original inventor has a limited market monopoly, during which they can try to recoup their development costs in sales. The 12 years of exclusivity granted under the Affordable Care Act, while still less than the 14 allowed in Europe, is a good compromise.
Mounds of research shows that data exclusivity should be set at around 12 years to give the average biologic a chance to at least break even. The White House wants to scale back that period.
A recent deficit deal crafted by the administration included a provision that would scale back the period of data exclusivity granted to biologics from 12 to 7 years. The idea is that allowing lower priced biosimilars to flow into the market sooner will drive down costs for public insurance programs and generate major savings for the federal treasury.
While shortening biologic data exclusivity could generate some short-term financial gains, it will ultimately reduce the rate of medical innovation and deprive American patients of life-saving new treatments.
The brute reality is that if data exclusivity drops to seven years, many biologic innovators won't have enough time to make back their original investment before biosimilars flood the market and siphon away their profits.
After all, creating a biologic is an incredibly expensive, time-consuming, and risky endeavor. The average biologic costs well over a billion dollars to develop. For every 10,000 compounds tested in the lab, only one will wind up getting federal approval and making it to market. And simply making it to market is no guarantee of profit. Only about three out of every ten new pharmaceutical medicines will ever recoup back its development costs in sales.
If the government reduces the exclusivity window for biologics, many drug firms will actually end up losing money on that massive investment. That leaves less capital to pour into new drug research operations. And outside investors will be less willing to underwrite new research initiatives given that their chance of even breaking even on a new treatment has dropped significantly.
Ultimately, that means fewer new drugs for American patients.
The Obama administration and its allies need to drop this push to scale back data exclusivity protections for biologic drugs. These treatments have proven to be highly effective at improving patient health and saving lives. Diluting the intellectual property rights protecting biologics will slow down the rate of innovation and choke off the flow of new treatments.
Peter J. Pitts, a former FDA Associate Commissioner, is the president of the Center for Medicine in the Public Interest.
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http://www.youtube.com/watch?feature=player_embedded&v=JWA1LyTNq78
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Subject: Leadership Changes in the Office of Regulatory Policy
CDER Staff:
I would like to make you aware of an upcoming important transition in CDER. Effective January 6, 2013, Jane Axelrad will be leaving her post as head of the Office of Regulatory Policy (ORP) to assume a new role, on detail, as senior advisor to me.
In this new role, Jane will serve as the Agency lead on managing policy, surveillance and enforcement, legislative, and oversight issues related to pharmacy compounding arising out of the meningitis outbreak. As time permits, she will also manage various other high-priority projects that cut across office lines.
Denise Esposito will serve as acting director of ORP until at least February 1, 2013, following which she will be leaving ORP to assume a management position elsewhere in the Agency. While Denise is serving as acting director of ORP, Nancy Hayes will serve as acting deputy director. When Denise leaves for her new position, Nancy Hayes will serve as acting director until the position is filled permanently.
As you know, Jane has been the associate director for policy, CDER, for more than 20 years, and she has led ORP for more than 15 years. Jane’s expertise on various legislative and policy issues is well recognized, and she is much sought after on a variety of matters.
Because her involvement in pharmacy compounding will take up most of her time, she has expressed the desire to step out of the management of ORP so that she will be able to devote the necessary time to compounding and other policy projects. With Denise’s impending departure, I want to establish a plan during this transition to ensure the continued stability of ORP, which performs so many vital functions for the Center.
Denise has been with CDER for two and a half years, and she has made important contributions on a number of different issues, as well as serving as Jane’s deputy during this time of intense activity in CDER. We are grateful that we will still have the benefit of her skills and expertise at FDA.
Janet Woodcock
Read More & Comment...The number of new medicines approved or pending approval is on the rise on both sides of the Atlantic, painting an encouraging picture for the global drugs industry as it emerges from a wave of patent expiries.
Yesterday, European regulators said they expect an increase in new drug applications to about 54 in 2013. Here at home, a total of 34 new drugs have been approved for sale so far in 2012 - the highest level in eight years.
The FDA still has just over a week to add more approvals to this year's tally - and there are signs that the number will increase further. The agency is expected to announce decisions on an additional four drugs before the end of the year.
The European Medicines Agency painted a different picture of improving productivity by announcing that its work program for the year ahead included a forecast for 54 new drug applications, up from 52 in 2012, 48 in 2011 and 34 in 2010. And these figures exclude medicines designated for orphan diseases.
The desire to take medicine is perhaps the greatest feature which distinguishes man from animals. – Sir William Osler
Read More & Comment...The PCORI projects are politically safe and scientifically unsensational. Chloform in print as Mark Twain would say. In it's effort to avoid political controversy PCORI has exposed itself to be financially wasteful and clinically irrelevant.
http://www.pcori.org/assets/PFA_Awards_Dec_2012_FINAL.pdf
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Bob Temple is having nightmares – and it’s not because of too much coffee before bedtime.
Bob envisions a nightmare situation if the Caronia ruling enables companies to promote drugs for uses that are not supported by well-controlled clinical trials.
Temple, deputy director for clinical science in the Center for Drug Evaluation and Research, discussed the decision at the FDA/CMS Summit in Washington Dec. 11. The U.S. Court of Appeals for the Second Circuit ruled in United States v. Caronia that the government cannot prosecute pharmaceutical manufacturers or their representatives under the Food, Drug, and Cosmetic Act for speech promoting the lawful, off-label use of an approved drug.
“What is worth talking about is what the consequences would be if people could promote uses that they hadn’t established, hadn’t bothered to get through the system,” Temple stated. “I’m horrified by that.”
Temple expressed concern that the Caronia ruling could undermine the 1962 amendments to the FDCA, which require that drugs be proven to be effective as well as safe. He said that prior to 1962 people did not know whether drugs worked. “If this [ruling] were to change the situation so that yes, people had to get their first claim in with adequate and well-controlled studies, but after that they didn’t have to bother anymore, and if that made them not bother anymore, that would be a nightmare.”
“There are all kinds of things that if you didn’t know the answer [it] would be terrible, and having people promote those uses is frankly terrifying,” said Temple.
“First of all, things that are wonderful would never be approved,” he stated, noting that most of the known cardiovascular outcomes were secondary outcomes. “There are all kinds of things that if you didn’t know the answer [it] would be terrible, and having people promote those uses is frankly terrifying.”
The unintended (but entirely predictable) consequences of the dangerous idiots of vaccine denial continue to spread.
A group of prominent doctors and public health experts warns in articles to be published today in the journal Pediatrics that banning thimerosal, a mercury compound used as a preservative in vaccines, would devastate public health efforts in developing countries.
Banning it would require switching to single-dose vials for vaccines, which would cost far more and require new networks of cold storage facilities and additional capacity for waste disposal, the authors of the articles said.
“The result would be millions of people, predominantly in low- and middle-income countries, with significantly restricted access to lifesaving vaccines for many years,” they wrote.
Fears of autism? Entirely bogus. The Gray Lady reports that “the evidence is overwhelming that thimerosal is not harmful …” And according to Louis Z. Cooper, former president of the American Academy of Pediatrics, “Science clearly documented that we can’t find hazards from thimerosal in vaccines. The preservative plays a critical role in distribution of vaccine to the global community.”
According to Dr. Heidi Larson, senior lecturer at the London School of Hygiene and Tropical Medicine “You can’t just pull the plug on something without having a plan for an alternative.”
The full New York Times article can be found here.
Read More & Comment...According to Reuters, “Medical providers have begun to think more about cost, as well as safety and effectiveness, when they decide on cancer treatments.”
That’s a pretty common statement these days – but it’s important to consider and discuss that “cost” is as relative a concept as “safety and effectiveness.”
Doctors at New York's Memorial Sloan-Kettering Cancer Center decided in November not to use Zaltrap, (an $11,000 a month medicine for colon cancer) because it has only a "modest" impact on survival, works no better than Avastin, a similar but cheaper competitor, and has worse side effects.
"In cases where there are co-pays, they really do effect the consumer," says Mark Mynhier of PricewaterhouseCoopers. "Patients are saying 'I can't afford to pay 10 or 20 percent of a $100,000 therapy.”
That is true for many patients – and perhaps for most patients. But what about patients for whom Zaltrap works better than Avastin?
Reuters: “Linking value to patient outcomes … is particularly important in oncology, where treatment costs can total tens of thousands of dollars a year.
Certainly. But outcomes data must also be used to learn about specific patient responsiveness – and lead to the development of better companion diagnostics.
Reuters: “As scientists unravel the biological underpinnings of cancer cells, new targeted therapies are being developed, but the process is expensive.”
Alas, Dr. Leonard Saltz, chief of Memorial Sloan-Kettering's gastrointestinal oncology service said the solution might just be to walk away from drugs with small, incremental benefits.
"We simply can't afford to pay these very, very large amounts for drugs that offer most people very small benefit," Dr. Saltz said. "We haven't figured out how to rein it in."
Dr. Saltz’s comment, alas, reinforces the fact that physicians rarely understand the drug development process. There are few discontinuous “eureka!” moments in drug development. Progress comes step by step, one incremental innovation at a time. But physicians (and particularly oncologists such as Dr. Saltz) should appreciate the importance of incremental innovation.
Rather than a wholesale denial of a product such as Zaltrap (and minus robust outcomes data or a companion diagnostic tool) an interim strategy might be to adopt a risk-sharing scheme wherein pharmas and federal programs establish a measure of success for a therapy and companies reimburse the government when their products fail to meet that standard.
In other words, let’s measure and reward for clinical effectiveness and ensure that medicine remains patient-centric rather than cost-obsessed.
Day Two of the Social Media, Mobile & Gaming for Pharma Conference brought new insights and raised some important questions.
The question most on the minds of the audience was just what do practicing medical professionals know about the regulated environment of healthcare speech? The answer, it seems, is very little – and that which doctors and nurses do know … is wrong.
That is not an asset in the forward march of pharmaceutical companies towards a more regular and robust use of social media. In fact – it’s a deterrent. Perhaps its time for HCPs to learn not only “where drugs come from” (they generally do not understand the roles of pharmaceutical companies and the FDA), but also what the rules are for pharmaceutical industry communications.
As my grandmother used to say, “It couldn’t hurt.”
John Mack (aka, “Pharmaguy”) bemoaned the lack of more regular pharma participation in social media. When confronted with the issues of internal review and control he opined, “How can you use social media if your company doesn’t trust you?
Good question.
And then there was the inevitable debate over the value of “Big Data.” Is more information better?
As Francis Collins recently said, “We are living in an awkward interval where our ability to capture information often exceeds our ability to know what to do with it.”
Collins’ comment was directed at the complete human genome sequence – but is equally germane to an equally complex human proposition – social media.
Read More & Comment...Some interesting take-aways from Day One of the Social Media, Mobile & Gaming for Pharma Conference:
The significant investment (in both FTE and dollars) required to run social media programs led Eileen O’Brien (Siren Interactive) to comment that, “Social media is free like having a puppy is free.” As Elvis Costello says, “Accidents will happen.”
Katie White (Lundbeck) shared that part of her job is to “hold the line against marketing” relative to what social media should and should not be doing. Respect is earned. Katie, bar the door.
Laying claim to “the largest social media site on gout,” Gretchen Goller (PRA International) discussed the need for CROS to do more on social media to advance patient recruitment for both orphan products as well as for adaptive clinical trials for more common conditions. One question raised was how to make clinicaltrials.gov more of a social media proposition. Hm.
Crohn’s Disease activist Michael Weiss suggested that pharma companies send MSLs to participate on disease-specific social media sites. If these medical professionals are available to speak with physicians – why not with patients as well? Perhaps this is the first-ever composition for the Caronia Philharmonia.
After all, according to Claude Debussy, music is between the notes.
Read More & Comment...Brain Cells Made from Urine
Human excreta could be a powerful source of cells to study disease, bypassing some of the problems of using stem cells, such as the risk of developing tumors and difficulties in obtaining blood samples from children
By Monya Baker and Nature magazine | Monday, December 10, 2012 | 1
Getting neurons from cells discarded in urine, may one day help develop therapies for neurodegenerative diseases like Parkinson's disease.
Image: Lihui Wang
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Some of the waste that humans flush away every day could become a powerful source of brain cells to study disease, and may even one day be used in therapies for neurodegenerative diseases. Scientists have found a relatively straightforward way to persuade the cells discarded in human urine to turn into valuable neurons.
The technique, described online in a study in Nature Methods this week, does not involve embryonic stem cells. These come with serious drawbacks when transplanted, such as the risk of developing tumors. Instead, the method uses ordinary cells present in urine, and transforms them into neural progenitor cells — the precursors of brain cells. These precursor cells could help researchers to produce cells tailored to individuals more quickly and from more patients than current methods.
Researchers routinely reprogram cultured skin and blood cells into induced pluripotent stem (iPS) cells, which can go on to form any cell in the body. But urine is a much more accessible source.
Stem-cell biologist Duanqing Pei and his colleagues at China's Guangzhou Institutes of Biomedicine and Health, part of the Chinese Academy of Sciences, had previously shown that kidney epithelial cells in urine could be reprogrammed into iPS cells.
However, in that study the team used retroviruses to insert pluripotency genes into cells — a common technique in cell reprogramming. This alters the genetic make-up of cells and can make them less predictable, so in this study, Pei and his colleagues introduced the genes using vectors which did not integrate in the cellular genome.
One of their experiments produced round colonies of reprogrammed cells from urine that resembled pluripotent stem cells after only 12 days — about half the time usually required to produce iPS cells. When cultured further, the colonies took on the rosette shape common to neural stem cells.
Tumor-free
Pei and his colleagues transferred the cells to a growth medium used for neurons, and found that these reprogrammed cells went on to form functional neurons in the lab.
When the team repeated the experiment and transplanted the cells into newborn rat brains, the cells did not form tumors. Instead, when the brains were examined four weeks later, the cells had taken on the shape and molecular markers of neurons.
Neural progenitors proliferate in culture, so researchers can produce plenty of cells for their experiments. Getting enough cells has previously been a problem for such 'direct reprogramming' techniques, which produce neurons more quickly than producing and differentiating iPS cells.
“This could definitely speed things up,” says James Ellis, a medical geneticist at Toronto's Hospital for Sick Children in Ontario, Canada, who makes patient-specific iPS cells to study autism spectrum disorders.
The benefit of sourcing cells in this way is that urine can be collected from nearly any patient, says geneticist Marc Lalande, who creates iPS cells to study neurogenetic diseases at the University of Connecticut Health Center in Farmington, and is particularly intrigued by the possibility of making iPS cells and neural progenitors from the same patient.
“We work on childhood disorders,” he says. “And it's easier to get a child to give a urine sample than to prick them for blood.”
This article is reproduced with permission from the magazine Nature. The article was first published on December 9, 2012. Read More & Comment...
I am not one of those who in expressing opinions confine themselves to facts.
-- Mark Twain
What? Off-label communication is protected free speech?
Cry havoc and let slip the dogs of war!
Not so fast.
As Steve Usdin of BioCentury so aptly opines, the ruling is “far less consequential than media coverage suggests.”
According to Usdin:
… companies and individuals who take the decision as a signal that the rules of the road have changed and they are now free to promote off-label indications put themselves in great legal and economic peril, attorneys who helped persuade the court to overturn Caronia's conviction told BioCentury.
At the same time, the decision by one of the country's most influential and respected courts to overturn a criminal conviction on First Amendment grounds is persuasive evidence that, in the long term, FDA will have to change some of the assumptions underpinning its regulation of medical products.
FDA, which now has lost a string of First Amendment cases, cannot forever hold on to the notion that it is empowered to prohibit drug companies and their employees from saying things that anyone else is free to say. Sooner or later, according to legal experts, the agency will have to reconcile itself with the idea that industry has the right to truthful, non-misleading speech.
While change is inevitable, the pace of change is uncertain. It is also not clear who will shape that change - FDA employees, judges, or members of Congress.
In short the Caronia ruling didn’t really answer any questions so much as it opened up the conversation. And that alone is worth the price of admission.
What will it mean on the ground? In the short term, not much. But it’s not about instant gratification for aggressive marketers.
This ruling isn’t the beginning of the end of FDA warning letters on off-label communication -- but it very well may be the end of the beginning of such speech being off-limits to prudent corporate compliance officers.
Equally important, what will it mean for off-label conversations with patients and caregivers? What will it mean for regulated speech on social media?
As William Safire once said, “Is sloppiness in speech caused by ignorance or apathy? I don’t know and I don’t care.”
Usdin’s complete story, Free speech, in theory, can be found here.
Read More & Comment...TransCelerate Appoints Dalvir Gill as CEO
TransCelerate has named Dalvir Gill, PhD, as its Chief Executive Officer, effective January 1, 2013. Dr. Gill will lead the independent non-profit entity, which was formed by ten healthcare and pharmaceutical companies in September to improve the quality of clinical studies and bring new medicines to patients faster by facilitating the collaboration required to solve common challenges encountered during the clinical trial process. Dr. Gill will succeed Garry Neil, MD, who has served as interim CEO. Dr. Neil will remain as Chairman of the Board of Directors.
Dr. Gill has more than 25 years of drug development experience. Prior to his appointment as CEO of TransCelerate, he was the President of Phase II-IV Drug Development at PharmaNet-i3, an organization specializing in drug development services. In this role, Dr. Gill was responsible for a global business spanning nearly 40 countries.
Read More & Comment...Nature Biotechnology reports:
Having Hamburg, who is a “tough cookie,” remain at the FDA provides something of a “public health bulwark against political intrusions,” says Pitts.
The full article, Cuts loom over research and industry following Obama re-election, can be found here.
Read More & Comment...Yesterday I participated on the Pennsylvania BIO-sponsored panel, “Election Aftermath: The Fiscal Cliff and the Future of Healthcare Reform.”
You might have heard of these issues.
My fellow panelists were Senator Bob Casey (D, PA), Congressman Jim Gerlach (R, PA) and Gary Karr (Executive VP, AdvaMed). The panel was expertly moderated by Chris Satullo (Executive Director of News and Civic Dialogue at WHYY).
Much of the evening’s focus was on the collision of healthcare innovation and national healthcare policy – and not in a good way.
Specific items of discussion were IPAB – Uncle Sam dictating (for tens of millions of Americans) what treatments will be available for physicians and patients; PCORI – the government instructing physicians as to how to practice medicine, FDA reform (specifically surrounding the issue of predictability), waste in Medicare and Medicaid, the medical device tax (both Casey and Gerlach are for repeal of the tax), the President’s call to roll back biologics exclusivity from 12 to 7 years (both Casey and Gerlach are for preserving 12) and, of course, the issues surrounding state exchanges.
Since states are the laboratories of innovation, governors should be given wide authority to design programs that best fit the needs of their particular situations. Ultimately states will learn from each other. HHS waivers should be broad. We’ll see.
The worst scenario is a one-size-fits-all top-down approach that fits everyone poorly. Alas – that’s a pathway preferred by many inside the Beltway. A key issue here is that of essential health benefits, what are they and who makes the call.
Congressman Gerlach was especially effusive (and appropriately so) of the free-market design of Medicare Part D. Lower than expected costs, high utilization and user satisfaction. And he strongly agreed this is a model that can work in the design and implementation of state exchanges.
Jim Dandy.
Senator Casey mentioned his strong support of NIH funding. I suggested to the Senator (politely) that perhaps more than a small slice of that largesse should be redirected to the FDA.
Does White Oak have a friend in Pennsylvania?
Nature reports:
FDA under pressure to relax drug rules
Industry says antibiotic pipeline is being blocked by overly stringent clinical-trial requirements for new treatments.
The latest skirmish in the battle between human and microbe played out on 29 November in a hotel conference room in Silver Spring, Maryland. There, an assembly of scientists and clinicians debated the merits of an experimental antibiotic. For some, the coveted prize was not just an endorsement of the drug itself, but a sign that the US Food and Drug Administration (FDA) is finally ready to rethink its clinical-trial requirements for antibiotics — requirements that the drug industry says are unrealistic.
The number of FDA approvals of new antibiotics has dropped even as multi-drug-resistant strains of bacteria have proliferated. FDA advisers at last week’s meeting did recommend approval of telavancin (Vibativ) — a derivative of vancomycin — for the treatment of hospital-acquired pneumonia when alternative drugs are not suitable. But that vote came nearly two years after the FDA had rejected the drug for a second time because clinical data did not measure up to the agency’s guidelines.
“The agency has painted itself into a statistical corner,” says Scott Hopkins, chief medical officer of Rib-X, a drug company in New Haven, Connecticut, focused on antibiotics. “While the infectious-disease community was crying out for new antibiotics, the FDA seemed to be going in the opposite direction.”
Many trace the agency’s tougher stance to the scandal surrounding telithromycin (Ketek), an antibiotic approved by the FDA in 2004 and later linked to liver failure. In 2007, the US Congress launched an investigation into whether the FDA had ignored staff concerns about Ketek’s safety. The following year, the agency convened its advisers to discuss antibiotics then under review. “Four drugs representing over a billion dollars of investment went into that week and only one came out alive,” recalls Mark Leuchtenberger, president of Rib-X.
Telavancin was caught in the changing tides. When Theravance, the company in South San Francisco, California, that developed the drug, designed the large phase III clinical trials needed for approval, the FDA simply required a demonstration that the drug eliminated symptoms of infection as reliably as the approved antibiotic vancomycin. But, after Theravance submitted its second application on 30 June 2010, the FDA decided instead that applicants needed to show that patients were no more likely to die — of any cause — within 28 days of treatment with a new drug.
Theravance scrambled to gather the data, hunting down medical records for 1,419 out of the 1,503 patients scattered across dozens of countries that were enrolled in the telavancin trials. But the FDA determined that the study lacked statistical power and asked for new clinical studies. Theravance refused, and a stalemate followed.
Strict clinical guidelines for antibiotics have dogged the industry ever since, with pneumonia providing a good illustration. Patients who contract pneumonia in hospital are already ill, making it hard to know if the treatment under review played a part in their death. That means trials have to be larger to capture enough deaths to have any statistical meaning. This, combined with the relative rarity of infections that warrant the use of new antibiotics and the further FDA requirement that patients not receive other antibiotics before they get the experimental drug, has set the goal out of reach, argues David Shlaes, who runs Anti-Infectives Consulting in Stonington, Connecticut. Not a single new antibiotic for hospital-acquired pneumonia has been submitted for approval since the new guidelines were put in place. “The trial simply cannot be done,” says Shlaes. “Whoever was writing these guidance documents doesn’t live on the same planet that I do.”
However, since the Ketek scandal, the political winds have reversed. This summer, Congress passed a set of measures to encourage antibiotic development. In May, Janet Woodcock, head of the FDA’s Center for Drug Evaluation and Research, pledged to “reboot” the antibiotic-approval process. And in September, the FDA told Theravance that its advisers would take another look at telavancin, resulting in last week’s vote.
Investors have also noticed these changes, says Leuchtenberger. Last week, Rib-X announced that it had raised US$18.7 million to help the company start phase III trials of an antibiotic that could target skin infections. “Without some of these positive developments this year, you’re looking at a number of companies that might not be around any more,” he says.
Shlaes, however, emerged from the telavancin meeting still doubtful, noting a continued focus on all-cause mortality. He says that, for now, he will continue to advise his clients to apply for approval in Europe first, where regulators have not been as demanding as the FDA. “I’ve been very optimistic about the whole thing,” he says. “But the FDA has to do something to show that it is actually rebooting.”
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