Latest Drugwonks' Blog
PMLive reports:
Pharmaceutical companies may have enthusiastically embraced Twitter, at least at a corporate level, but local European regulators have proved somewhat more reticent.
The European Medicines Agency (EMA) led the way by signing up to the social network in 2010, but few local bodies have followed suit.
One of those that did was the Medical and Healthcare products Regulatory Agency (MHRA), and the UK regulator has just expanded its use of Twitter and launched three new accounts on the social network.
One of these is a corporate account, the use of which tends be pharma’s first step into social media.
The MHRA says its new corporate Twitter presence, @MHRAgovuk, will be used to promote general information and updates, while two other accounts will focus on safety alerts and information about medical devices and medicines.
The regulator has been cautious in its approach to Twitter to date, launching dedicated accounts in 2011 for first herbal remedies and then its press office, the account names of which have been simplified as part of its Twitter expansion.
It remains to be seen how well the use of separate accounts for press and corporate purposes will work, given the potential for subject overlap between the two.
But the use of new, separate accounts for medicines and medical devices seems logical, given the differences in audience for a recall of Merck Sharp & Dohme’s Tredaptive versus that for a mass spectrometer.
• Links to all of the MHRA's Twitter accounts, and many more from regulators, industry associations and pharma companies, can be found in the Twitter section of the Digital Handbook’s pharma social media directory
The Drug Enforcement Administration has once again asked the FDA to consider tightening prescribing and other rules on hydrocodone drugs.
Proving, once again, that the DEA doesn’t understand the problem. Denying access to patients in pain (the unintended but obvious consequence of up-scheduling) does nothing to stop those who provide hydrocodone – via inappropriate prescribing or illegal means.
The FDA will hold hearings tomorrow and Thursday to consider whether to move hydrocodone products from schedule III to the more highly regulated schedule II class under which narcotics such as OxyContin (oxycodone) fall. In a briefing document released as background material, FDA scientists said that although "patients taking hydrocodone products might develop moderate or low physical dependence, they would not be expected to develop addiction."
This is regulatory-speak for not wanting to change the classification.
CDER Deputy Director Dr. Doug Throckmorton said an analysis conducted internally by scientists at the agency does not reflect the FDA's "official stance." He said the agency will refrain from making recommendations until after it has reviewed testimony and received a recommendation from the independent advisory committee, which scheduled the two-day hearing.
This is regulatory-speak for, “we know what we want to do – but we want to solicit informed opinion."
Please step up to the microphone and state your name.
FDA said in a statement that the government decided not to seek further review of a December decision that found that the federal government could not prosecute a sales representative for speech promoting the legal, off-label use of an FDA-approved drug. According to the statement, the agency does not believe the 2-1 decision from the U.S. Court of Appeals for the Second Circuit in United States v. Caronia "will significantly affect the agency's enforcement of the drug misbranding provisions" of the Food, Drug and Cosmetic Act (FDCA). FDA added that the decision "does not strike down any provision [of FDCA] or its implementing regulations, nor does it find a conflict between the act's misbranding provisions and the First Amendment or call into question the validity of the act's drug approval framework"
Surprised? Don’t be. Whenever the FDA goes to court on First Amendment issues it either loses or gets a stern rebuke for its unpredictable, ambiguous and sometimes capricious application of off-label speech constraints.
Regardless of the FDA’s decision, Caronia will impact the way FDA views off-label promotion within the context of the free-and-fair dissemination of scientific data. I believe a new (and hopefully more enlightened) FDA view based on intent will arise. Alas, that will not assuage any of the ambiguity that is currently driving FDA (OPDP) communications oversight. That being said, any revisitation and discussion is for the better.
Some believe that, if Caronia stands, pharmaceutical companies will no longer feel obligated to seek FDA approval for new indications, since they can openly "promote" them without fear of prosecution. This is a flawed argument. Indications of the on-label variety have many benefits—not the least of which is reimbursement. But such negative unintended consequences are important to discuss and consider. IMHO, any company that chose this route would be acting in a highly irresponsible manner, putting promotion before the public health.
“See you in court?” Not likely.
From: A Message from the Commissioner
Sent: Tuesday, January 22, 2013 10:54 AM
To: FDA-Wide
Subject: Personnel Announcement for the Office of Medical Products and Tobacco
Dear FDA Colleagues:
I wanted to make you aware of the upcoming departure of Dr. Stephen Spielberg, Deputy Commissioner for Medical Products and Tobacco. Due to a family medical issue, Dr. Spielberg will be leaving FDA on February 15 to return home to the Philadelphia area. In March, he will assume the role of editor-in-chief of “Therapeutic Innovation and Regulatory Science”, a new journal launched by the Drug Information Association.
For the past year and a half, Dr. Spielberg has been a terrific spokesperson for the Agency and a key member of my senior management team, supporting the invaluable work performed across the Centers for drug, biologics, medical devices, and tobacco products and the Office of Special Medical Programs. He has been an important partner in helping the agency toward a more integrated approach to medical product evaluation across the Directorate, in working together with Center leadership to address the rapid changes in human biology and therapeutic interventions, and in helping optimize FDA’s approach to evolving areas such as personalized medicine.
We will miss the leadership and wise counsel he has provided during his time at the agency.
I am pleased to announce Leona Brenner-Gati, MD, has graciously agreed to serve as Acting Deputy Commissioner for Medical Products and Tobacco. Dr. Brenner-Gati is currently the Associate Commissioner for Medical Products and Tobacco and comes with twenty years of experience in the private healthcare sector, where she has focused on emerging technologies and complex product development. She has extensive experience leading multi-disciplinary, cross-functional teams spanning pharmaceuticals, devices, diagnostics and consumer sectors, and has applied highly integrative scientific and administrative expertise to foster the development of groundbreaking medical products.
Dr. Brenner-Gati spent seventeen years at Johnson & Johnson, where she held senior leadership positions in Science and Technology, Clinical Research and Development, Global Project Management, Medical Affairs, and Business Development in the Pharmaceuticals and Medical Devices & Diagnostics Groups and Corporate Headquarters As a champion of Regenerative Medicine product development and initiatives, she founded J&J’s cell therapy for diabetes program, and chaired the Scientific Advisory Board and served on the Management Board. She was also Vice-Chair of J&J’s Human Pluripotent Stem Cell Committee, helped found and served as an officer and board member of the Alliance for Regenerative Medicine, and was a member of the FDA/California Institute of Regenerative Medicine Roundtable on Regenerative Medicine. Prior to J&J, Dr. Brenner-Gati led the Internal Medicine Clinical Research Therapeutic Area at Sandoz Pharmaceuticals and achieved successful registration of several products.
Dr. Brenner-Gati graduated from Princeton University, magna cum laude in Biology, Phi Beta Kappa, and received her medical degree from Harvard Medical School. She obtained her postgraduate medical training at New York University/Bellevue Hospital Medical Center, and held academic appointments at Cornell University Medical Center/New York Hospital, where she performed basic science and clinical research, taught medical students and had a medical practice. While at Cornell, Dr. Brenner-Gati was a clinical investigator for seven years in the landmark NIH Diabetes Control and Complications Trial. She is board certified in Internal Medicine and Endocrinology & Metabolism and holds medical licenses in New York and New Jersey.
Please join me in extending a warm welcome to Dr. Brenner-Gati as she takes on this new role, and please join me in thanking Dr. Spielberg for his significant contributions to our public health mission.
Sincerely,
Margaret Hamburg, MD
Commissioner for Food and Drugs
Year, n. A period of 365 disappointments. – Ambrose Bierce
What role will FDA Commissioner Peggy Hamburg play in 2013?
Perhaps a better question is what role will the White House permit her to play?
Since the Plan B controversy things have been pretty quiet over at White Oak. But, post-election, things seem to be heating up – in a good way.
Enrichment trials. New review pathways. A record number of approvals. Yes, 2013 could be a very good for the FDA … if Peggy is allowed to set her own course and stick to it.
And then there’s the question (often posed but rarely addressed) of the many senior players at the agency nearing retirement.
Succession plans, anyone?
The good news is that, with a firm hand at the wheel and the authority to make tough and noble decisions, good things can happen.
Future, n. That period of time in which our affairs prosper, our friends are true and our happiness is assured. – Ambrose Bierce
"There is good news and bad news about enrolling patients in clincal trials. First, the good news – 89 percent of all trials meet their enrollment goals. Now, the bad news – 48 percent of the trial sites miss enrollment targets and study timelines often slip, causing extensions that are nearly double the original duration in order to meeting enrollment levels for all therapeutic areas."
Fewer patients and longer studies increases development costs without advancing science. It's like feeding a parking meter but never leaving your car...
But there is a solution....
FDA gives go ahead for Phase 2 trial using remote monitoring and crowd sourcing
Posted By Stephanie Baum On December 18, 2012 @ 8:02 am In Health IT,MedCity News
It looks like the U.S. Food and Drug Administration is feeling growing pressure to make clinical trial design more flexible. A newly approved Phase 2 clinical trial is the first to use both crowdsourcing and remote monitoring. The regulator has granted New York-based biopharmaceutical startup Transparency Life Sciences [1] a greenlight to study the effectiveness of a blood pressure drug lisinopril [2] in multiple sclerosis patients, after clearing the company’s Investigational New Drug application. [3]Telemonitoring company AMC Health will work with Transparency Life Science to do remote monitoring for the trial. For the 12-month study, data will be collected from the 180 participants’ homes, using wearable monitors to collect vital signs. Secure video interaction between investigators and trial participants will facilitate communication between them.In the runup to the FDA’s clearance of the IND, Transparency Life Sciences had obtained insights from patients and and healthcare professionals to strengthen the protocol for the Phase 2 trial, such as primary and secondary endpoints, inclusion and exclusion criteria and remote monitoring strategies.
It’s a significant milestone for the FDA and for the pharmaceutical industry. The study is estimated to cost $1.5 million. Using a more traditional clinical study model, with frequent site visits, the one year study could have cost as much as $5 million, John Holland, senior vice president for research and business development for AMC Health told MedCity News in a phone interview. Another benefit is that it will reduce the geographical limitations of clinical trials.
AMC Health is also working with an unnamed pharmaceutical company in a Phase 1 study. The New York telemonitoring business has worked with pharmaceutical companies before, mostly on pilot studies evaluating the use of telemonitoring for clinical trials. It has also worked with Geisinger Center for Health Research [4] in Danville, Pennsylvania to reduce readmissions by improving post discharge patient follow up.“This will increase the quality and quantity of data, and reduce the burden on patients. Frequent data collection at home is also expected to increase patient safety, because if a side effect were to appear, researchers will know about it sooner, ” Holland said. “A lot of people are collecting electronic patient-reported outcomes so that’s not unique but I have not seen anyone else doing the biometric, medication adherence monitoring that we are doing.
”Telemonitoring is not a perfect system either. Information provided by monitors tracking vital signs can generate false alarms. Scalability is also a problem when you are looking at a clinical trial of thousands of people.
Transparency Life Sciences has been a proponent of telemonitoring [5] as an effective way to carry out clinical trials and ensure data is uniform. In an interview with MedCity News earlier this year, Marc Foster, Transparency’s chief operating officer said: “One of the things you see a lot these days is researcher bias, where a doctor or clinician is taking data in a clinical setting their involvement can bias the results of a clinical trial. If people are at home in their most natural setting, the ability to take their data remotely we think would be more uniform.”Industry insiders from pharmaceutical companies to health IT companies to patient advocates have been pushing for the FDA to be more innovative [6] in its approach to clinical trials. Pfizer’s attempt to use social media to carry out the tricky job of patient recruitment [7], marked a critical step in how Big Pharma view alternative approaches to clinical trial design. Pharmaceutical companies have been conducting pilot studies [8] for years to ascertain factors that could impact the accuracy of these studies. The evolution in the sophistication of devices that can monitor vital signs and transmit that data remotely without the patient keying it in makes this kind of clinical trial design more feasible. Proponents argue that they can better track participants’ health than regular site visits.
From the pages of Food Safety News:
Common Ground Exists in Drug Resistance Debate: Response to Pew Op-Ed
To some, the ongoing debate over the use of antibiotics in animals raised for food is like the sign post in the Wizard of Oz — pointing in opposite directions. But from reading “Antibiotics in Food Animal Production: Pew’s Response to Raymond Op-Ed,” Jan.11, there is violent agreement between PEW and the animal health industry.
Gail Hansen writes that Pew “opposes the use of antibiotics for growth promotion, but strongly supports their use to treat sick animals.” That’s radically similar to the position the animal health industry has taken on FDA’s timely and thoughtful effort to phase out growth promotion claims on medically important antibiotics and phase in veterinary oversight of these compounds. This policy harmonizes antibiotics use in animals and humans and eliminates the use of antibiotics as growth promoters, similar to the European ban on antibiotic growth promoters.
FDA’s action ensures that medicines will now be used in animal health much the same way as they are used by humans – administered only to address disease and under the direction of a licensed medical professional. This policy is the net result of what a number of public health advocacy groups, including Pew, called for in a 2009 letter to the White House.
On the issue of the amount of antibiotics used in animals, Hansen also acknowledges something the animal health industry has pointed out: about 40 percent of the total compounds used in animal agriculture aren’t used in human medicine and don’t figure into the debate over antibiotic resistance. The focus on the amount used in animals vs. humans, according to Dr. Raymond, is a “distraction from the real truth.” I agree.
While the Danish ban on growth promoters – known as the Danish Experiment –has reduced some instances of resistance in animals, reductions and changes in use of antibiotics in animals have had no effect on rates of antibiotic resistance in humans. For example, after certain antibiotics in animal feed were banned in Denmark, a 2011 GAO Report stated, “Danish officials told us that Denmark’s resistance data have not shown a decrease in antibiotic resistance in humans after implementation of the various Danish policies, except for a few limited examples.” Again, this appears to be another area where the animal health industry, Hansen and the GAO all agree. Wonderful!
Fact: Simply banning uses of antibiotics used to keep food animals healthy does not address the unrelated problem of antibiotic resistance in humans, but will certainly add to animal disease and suffering. The issue of antibiotic resistance is one that is both important and scientifically complex, and reliance on simplistic solutions will not solve the problem and may make it worse.
It does matter which direction we go and how we get there. I look forward to contributing to this importance discussion on the nexus between human and animal health.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest and a member of the Animal Health Institute’s Board of Scientific Advisors
From the pages of Drug Industry Daily:
New FDA Drug Approval Pathway Aims to Promote, Ease Enrichment Trials
The FDA is considering a new drug approval pathway intended to boost development of treatments targeting small subgroups of patients with serious or life-threatening conditions.
The pathway would establish a new, speedier process for studying a new drug or biological product in a smaller subpopulation of patients with more serious manifestations of a disease.
Labels for drugs approved under the new paradigm would make clear the drug is narrowly indicated for use in limited, well-defined subpopulations in which the drug's benefits have been shown to outweigh the risks, the agency said in a Federal Register notice.
The FDA plans to hold a public hearing on the potential new approval pathway on Feb. 4 and 5.
The agency is allowed to "exercise its scientific judgment" under existing regulations to determine the level of data a sponsor must provide in support of a particular candidate. Such flexibility has spurred the advance of a wide range of orphan drugs for rare diseases.
But traditional drug development programs typically require very large study populations, which put subpopulations of patients with more severe conditions at a disadvantage in the clinical setting - a situation that often results in less timely availability of new drugs for those patients.
The new approval pathway "could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations," the FDA said.
The pathway proposal comes on the heels of new draft agency guidance on strategies for developing enrichment trials, which prospectively apply a patient characteristic to select a subpopulation more likely to benefit from a drug.
"The two announcements coming together signal to the industry that the blockbuster model is slowly being replaced by the personalized medicine model," Peter Pitts, president of the Center for Medicine in the Public Interest, said.
The message to the investment community is that drug failures are often due to trial design difficulties and not just heavy-handed regulation, he added. The proposed approval pathway is not likely to allow for less-robust evidence, just a different kind of evidence, Pitts cautioned.
Public Hearing Questions:
The meeting at the FDA's White Oak Campus will address such issues as:
The pathway's necessity considering the existence of other expedited approval pathways;
The conditions and patient subpopulations most likely to benefit from the pathway;
The degree to which regulators should monitor drugs approved under the pathway; and
Whether the pathway should be used to help reduce antibiotic-resistant bacteria by limiting the drugs' use to those patients in whom use is "appropriate and necessary."
Comments are due to docket no. FDA-2012-N-1248 by March 1. View the Federal Register notice at
www.fdanews.com/ext/files/01-14-13 DrugApprovalPathwayMeetingNotice.pdf.
“Human action can be modified to some extent, but human nature cannot be changed.” Those are the words of Abraham Lincoln (16th President of the United States and Academy Award nominee). And they pretty well sum up a major issue in American healthcare – adherence/compliance.
I’ve just returned from the speaker’s podium at the Optimizing Pharmaceutical Patient Adherence conference. There’s a lot to be done. There are a lot of good ideas. There seems to be a lot of commitment. The better angels of our nature were on display.
Now it’s time for action.
Zig Ziggler once said, “If what you’re doing isn’t working, try something else. If what you’re doing is working, try anything else.” While there are certainly success stories and validated methodologies in the battle for better adherence/compliance, we’re losing the war. It’s time to reconsider what we’re doing.
And the first thing to admit is that we can’t relegate the conversation to healthcare policy. While the conversations are fascinating – another white paper, another committee, another conference – isn’t going to move the needle. We need to act.
As I see it, there are six issues we are trying to impact – and they are linked:
1. Sub-optimal patient outcomes (the Big Kahuna).
2. Sub-optimal physician pay-for-performance metrics. (More important today than ever and back at the top of strategies to control costs. Alas – one of the unintended consequences of pay-for-performance is that some physicians will try to game the system by not seeing those patients who they see at high risk for non adherence/compliance,)
3. Lower healthcare costs for payers. Not surprisingly, all of the big private payers are in the adherence/compliance game with both feet.
4. Sub-optimal profits for pharmaceutical companies. (The sale doesn’t end once the script is written.)
5. Impact on safe-use. The way to make drugs “safer” is to ensure they are used appropriately. Safe use begins with adherence/compliance. (Hear that FDA?)
6. Lower healthcare costs for society. (You might have heard of this issue – it’s been in the news a lot.)
Alas, there are no magic bullets in the fight to improve adherence/compliance. News articles feature talking pillboxes that offer bells and whistles, rings, buzzes, and flashes, and that’s all to the good – but they only combat forgetfulness (purposeful or otherwise). It’s a part of the solution – but, just as in the battle against counterfeit medicines, it’s only a piece of the puzzle
Some think that (as with REMS), the FDA should insist that new drugs have adherence/compliance plans that can be monitored and improved through iterative learning. Should sales reps (or, better yet, MSLs) “detail adherence/compliance programs and share validated tools for adherence/compliance “triage?” The only thing that’s currently on the table is that the FDA has promised to make MedGuides more user-friendly. (We can do better.)
Others talk about behavior modification through gamification – and that too is a useful pathway. We talk about carrots – but what about sticks to address bad patient behavior (particularly sticks of the financial variety)?
All of these are important. But talking pillboxes and better MedGuides are only making existing tools better. And trying to “regulate” adherence/compliance is a slippery slope indeed. To really make a difference, to change the game, what we really need are solutions that impact social conditioning and address patient responsibility – and that means using innovative platforms such as social media and, specifically, apps.
Not apps that are medical devices (although those play an important role in 21st century healthcare), but apps that remind, cajole, educate, praise, incentivize, and assist patients in their quest for better health. Apps are at the nexus of sage use, treatment outcomes, and patient satisfaction. And it’s not science fiction.
At present, there are some 17,828 healthcare and fitness apps and 14,558 that can be deemed “medical.” While some are better than others, these numbers tell us one thing – this is not a fad or a trend. It is reality.
And as Philip K. Dick wrote, “Reality is that which, when you stop believing in it, doesn’t go away.”
Will our socio-economic “technology gap” lead to a more pronounced “adherence/compliance gap?” It’s an important question. That’s why it’s crucial we remember there is no one-size-fits all solution. But that’s mustn’t mean we disregard the reality of the growth and pervasiveness of apps, mobile apps. Let’s face it, when it comes to mobile phones, any gap is rather narrow.
Apps for adherence/compliance are “safe use” apps. Apps that can be “prescribed” by physicians to their patients are the wave of the present. Adherence/compliance “app-ens” and patients, physicians, payers, pharmaceutical companies – and society benefit.As Walter O’Malley (the man who moved the Dodgers from Brooklyn to Los Angeles) famously opined, “The future is just one damn thing after another.”
FDA will hold a public hearing on Feb. 4-5 to obtain input on creating a new approval pathway for therapeutics for limited, well-defined subpopulations of patients with serious or life-threatening conditions, including infections caused by antibiotic-resistant bacteria. According to the agency, such a pathway could allow companies to evaluate safety and efficacy in subpopulations of patients with more serious manifestations of a condition. The result, said FDA, could be smaller, more rapid clinical trials than studies in a broader group of patients with a wide range of clinical manifestations. The agency said a narrow indication of therapeutics approved via such a pathway could be expanded based on additional data demonstrating efficacy and safety in a broader population.
FDA is seeking input on the need for and feasibility of such a pathway, as well as its potential advantages and disadvantages. The agency is asking stakeholders to discuss which diseases or conditions would benefit from such an approval pathway, as well as whether the pathway would increase the number of therapeutic options in the context of the agency's existing expedited processes, including accelerated approval and the Fast Track program. FDA also is seeking input on how the agency could monitor use of drugs approved under the pathway. Comments are due March 1.
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.
