Latest Drugwonks' Blog
http://www.youtube.com/watch?feature=player_embedded&v=JWA1LyTNq78
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
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
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
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.
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.
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.
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.
