Latest Drugwonks' Blog
Here's what Representative Henry Waxman (D, CA) told the American Medical Association the other day: "We all know that we have to get costs under control, but the way to do that is not to tell physicians what they can and cannot do or put them in a position where they cannot put the needs of their patients first.”
Mr. Waxman said this publicly at the AMA’s National Advocacy Conference.
Further:
“Don’t let anyone tell you that what I’m interested in is socialized medicine. I flatly tell you that is not the case … I am not interested in trying to put a public plan in place that would drive out competition.”
Thanks for that – but just what does Mr. Waxman define as “socialized medicine?”
We’ll see soon enough.
Good quotes from Mr. Waxman – but the winner of the drugwonks healthcare reform quote of the week (yes, even though it's only Wednesday) goes to Senator Ron Wyden (D, OR):
"Nobody disputes the fact that there's going to be some startup costs."
Thank you Senator Wyden.
A nice piece on comparative effectiveness by Ceci Connolly of the Washington Post. No pun intended. Nice in that it underscores the limits of comparative effectiveness and it's link to consumer choice rather than top down directives.
However, there are limits that Connolly's piece did not discuss and which likely will be silence by the IOM panel which is stacked with people who believe that the one size fits all, literature review, take it or leave approach of comparative effectiveness is the key to universal coverage. This (with some rare and sensible exceptions) is a panel of patronage appointees intended to suppress responsible, science based alternatives to evaluating treatment effects. I will have much, much more on this subject in a forthcoming post. But for instance, don't expect the IOM panel, which is stacked to simply implement an AHRQ agenda that they have developed and receive millions from to address the following issues:
Which drug or treatment works best for an individual based on a variety of factors including genetic variation, co-morbidity, life style preference, stage of life?
Moreover, will comparative effectiveness force additional studies before a product is paid for? If so, that will inevitably delay access, raise prices or reduce rates of innovation. And if trials have to be randomized for each subpopulation the cost goes higher still.
The drug effectiveness review studies conducted by DERP fail to control for subpopulations and the literature they look at themselves exclude adjustments for severity of illness, genetic variation, etc. So most "studies" show no difference in drugs for a particular illness when in fact there are substantial variations, particularly in the areas of depression, schizophrenia, hypertension.
Therefore, shouldn't money be spent on tools for personalized medicine instead of one-size fits all guidelines?
However, there are limits that Connolly's piece did not discuss and which likely will be silence by the IOM panel which is stacked with people who believe that the one size fits all, literature review, take it or leave approach of comparative effectiveness is the key to universal coverage. This (with some rare and sensible exceptions) is a panel of patronage appointees intended to suppress responsible, science based alternatives to evaluating treatment effects. I will have much, much more on this subject in a forthcoming post. But for instance, don't expect the IOM panel, which is stacked to simply implement an AHRQ agenda that they have developed and receive millions from to address the following issues:
Which drug or treatment works best for an individual based on a variety of factors including genetic variation, co-morbidity, life style preference, stage of life?
Moreover, will comparative effectiveness force additional studies before a product is paid for? If so, that will inevitably delay access, raise prices or reduce rates of innovation. And if trials have to be randomized for each subpopulation the cost goes higher still.
The drug effectiveness review studies conducted by DERP fail to control for subpopulations and the literature they look at themselves exclude adjustments for severity of illness, genetic variation, etc. So most "studies" show no difference in drugs for a particular illness when in fact there are substantial variations, particularly in the areas of depression, schizophrenia, hypertension.
Therefore, shouldn't money be spent on tools for personalized medicine instead of one-size fits all guidelines?
Sources inside the Obama administration assure me that they have no plans to bifurcate food/tobacco from drugs/devices. In other words, Commissioner-designate Hamburg and proposed Principal Deputy Commissioner Sharfstein will act as a team in addressing the direction of the FDA as a totality.
That being said, the issue of whether or not food safety and security remains inside the FDA or becomes its own agency within HHS remains as contentious as ever, with Representative Waxman and Representative DeLauro agreeing to disagree.
Mr. Waxman (D-CA) believes that the "first step on the legislative path" should be shoring up food safety and finding ways to prevent further incidents. According to the Pink Sheet, “Waxman did suggest that he would entertain the concept of a bifurcated agency in the future. Once reforms are passed and implemented, he suggested Congress could consider "whether a reorganization is necessary" for food safety enforcement.”
But Representative Rosa DeLauro, (D-CT), the chair of the House Agricultural Appropriations Subcommittee which oversees FDA's budget, has introduced the Food Safety Modernization Act, which would move some divisions out of FDA and form a Department of Health and Human Services agency for food safety. The bill, H.R. 875, has 40 co-sponsors.
That being said, a dangerous bifurcation that must be avoided at all costs is a differentiation in the vision for the future of the FDA between appointees and career staff. Drs. Hamburg and Sharfstein should listen and learn from the agency’s senior staff – most specifically as to how the agency can be both regulator and colleague with the industries it regulates. A fine line to walk -- but a journey worth the effort.
Exhibit A: The Reagan/Udall Foundation and the Critical Path Initiative.
After all, the FDA must work to both protect and advance the public health.
A shared vision, crafted together will succeed.
Unity, yes. Bifurcation, no.
Yes we can.
That being said, the issue of whether or not food safety and security remains inside the FDA or becomes its own agency within HHS remains as contentious as ever, with Representative Waxman and Representative DeLauro agreeing to disagree.
Mr. Waxman (D-CA) believes that the "first step on the legislative path" should be shoring up food safety and finding ways to prevent further incidents. According to the Pink Sheet, “Waxman did suggest that he would entertain the concept of a bifurcated agency in the future. Once reforms are passed and implemented, he suggested Congress could consider "whether a reorganization is necessary" for food safety enforcement.”
But Representative Rosa DeLauro, (D-CT), the chair of the House Agricultural Appropriations Subcommittee which oversees FDA's budget, has introduced the Food Safety Modernization Act, which would move some divisions out of FDA and form a Department of Health and Human Services agency for food safety. The bill, H.R. 875, has 40 co-sponsors.
That being said, a dangerous bifurcation that must be avoided at all costs is a differentiation in the vision for the future of the FDA between appointees and career staff. Drs. Hamburg and Sharfstein should listen and learn from the agency’s senior staff – most specifically as to how the agency can be both regulator and colleague with the industries it regulates. A fine line to walk -- but a journey worth the effort.
Exhibit A: The Reagan/Udall Foundation and the Critical Path Initiative.
After all, the FDA must work to both protect and advance the public health.
A shared vision, crafted together will succeed.
Unity, yes. Bifurcation, no.
Yes we can.
Last week, Congressman Pete Stark (R-CA), Chairman of the House Ways and Means Health Subcommittee, held a hearing on 2lst century healthcare reform. In his remarks Congressman Stark said, “Comprehensive health reform is needed to ensure affordable health coverage for the 46 million uninsured. Today’s experts made clear that the option of a public health insurance plan is the key to slowing the health spending trend and improving quality.”
Representative Pete Stark’s view of the public plan stands in stark contrast to Representative Louie Gohmert’s view.
CMPI recently interviewed Congressman Gohmert (R-TX) and he was pointed in his belief that a public health plan is merely an effort to nationalize the healthcare system.
In our interview with Congressman Gohmert he said of a public health plan, “This is a straw dog you build up and say, oh no, we’ll allow private insurance companies to compete knowing the government will be able to put them out of business even if it runs at a massive deficit.”
We have already had a hint of this scheme with the comparative effectiveness research contained in the Stimulus bill.
Congressman Stark recently expressed his support for this research: “The new research will eventually save money and lives, although it may very well shorten the lifespan of some senior citizens who would not be allowed to receive some treatments even if they volunteer to pay for them themselves.”
According to the Association of American Physicians and Surgeons, the February 15, 2009 New York Times article did not include this entire quote from Stark AAPS states “this version was taken down, and the quotation was truncated after ‘money and lives.’”
Asked in December of last year about any concerns he had in bringing private insurers to the table for an agreement on health reform, Stark said of insurance companies: “They’re the easiest to roll because no one likes insurance companies. I guess someone has to be the bad guys.”
If the goal is to reduce premiums, why not support an end to community rating, guaranteed issue, insurance mandates, etc., which all works to drive up insurance premiums and make plans less responsive to the individual medical needs of patients?
During the election, candidate Obama called candidate McCain’s proposal to tax employer-provided health benefits “the largest middle-class tax increase in history.” Today President Obama is sending signals that he would support the same idea.
Representative Pete Stark (D, CA) says it's a "stupid idea."
According to the New York Times, such a tax could raise $3.6 trillion over a decade to help “expand coverage to the 46 million uninsured Americans.”
(The complete New York Times article – worth a read – can be found here.)
Except when you take an honest look at who is being inappropriately included in that 46 million (4.1 million now eligible for government benefits via expanded SCHIP + 12 million already eligible for government programs but not signed up + 10 million illegal aliens, + 10 million Americans “opting out” = 32 million) it really comes out to about 14 million uninsured Americans.
$3.6 billion (over 10 years) + the President’s proposed $634 billion “down payment” (over 10 years) divided by 14 million uninsured. Talk about healthcare inflation!
(And this isn't counting the dollars allocated via the stimulus package that provides unemployment insurance-eligible Americans and their families with government health insurance as part of their benefits.)
For more on miscounting the uninsured, see “Sasquatch Care.”
Another thing the candidate Obama was against but now supports seems to be drug importation. Can support of government price controls be far behind?
Representative Pete Stark (D, CA) says it's a "stupid idea."
According to the New York Times, such a tax could raise $3.6 trillion over a decade to help “expand coverage to the 46 million uninsured Americans.”
(The complete New York Times article – worth a read – can be found here.)
Except when you take an honest look at who is being inappropriately included in that 46 million (4.1 million now eligible for government benefits via expanded SCHIP + 12 million already eligible for government programs but not signed up + 10 million illegal aliens, + 10 million Americans “opting out” = 32 million) it really comes out to about 14 million uninsured Americans.
$3.6 billion (over 10 years) + the President’s proposed $634 billion “down payment” (over 10 years) divided by 14 million uninsured. Talk about healthcare inflation!
(And this isn't counting the dollars allocated via the stimulus package that provides unemployment insurance-eligible Americans and their families with government health insurance as part of their benefits.)
For more on miscounting the uninsured, see “Sasquatch Care.”
Another thing the candidate Obama was against but now supports seems to be drug importation. Can support of government price controls be far behind?
According to Douglas Schoen (formerly a pollster for President Bill Clinton) and Scott Rasmussen (president of Rasmussen Reports, an independent national polling company), “It is simply wrong for commentators to continue to focus on President Barack Obama's high levels of popularity, and to conclude that these are indicative of high levels of public confidence in the work of his administration”
Relative to healthcare reform, they find that a majority of Americans aren’t so keen after all to have their Uncle Sam become Uncle Sam, MD:
“There is no real appetite for increasing taxes to pay for an expanded health-insurance program. Less than half would support such an idea, which is 17% less than the percentage that supported government health insurance when Bill Clinton first considered it in March of 1993.”
(Their complete opinion piece in today’s edition of the Wall Street Journal can be found here.)
The conclusions reached by Schoen and Rasmussen are completely in line with a poll fielded by the Center for Medicine in the Public Interest last October.
The CMPI survey was of “Millennial” voters (18-28 years old) -- often portrayed as the new “driving force” in American politics. When millennial voters were engaged on the issue of health care reform they had some very strong – and often contradictory – opinions a -- particularly when it comes to issue of “universal care.”
The national public opinion poll of young voters (the result of 1001completed interviews with adults 18-28 years of age who are registered to vote) shows limited acceptance for the potential consequences of greater government control over health care.
While millennial voters report to strongly support the need for reform and the concept of “universal care,” when asked if they are willing to pay higher taxes to pay for a government-run health care system, their level of support swiftly turns in the opposite direction.
Millennial voters are strongly against government-care that results in longer wait times to see a health care provider, limits to the types of treatments and medicines they can access, and the potential for the government to interfere in the decision making and relationship between doctor and patient.
Some germane findings:
* A majority (51 percent) were not in support of any health care reforms that could raise their personal tax burden;
* Sixty-two percent said they would not support any health care reforms that could increase wait-times to see a doctor or the availability of treatments and medicines; and,
* Millennial voters were also equally unsupportive (62 percent) of health care reforms that would increase the role of the government regulation and oversight in doctor-patient decision-making.
(For further details on the CMPI poll, see “The Young and the Restless.”)
The CMPI poll and the findings of Schoen and Rasmussen demonstrate that all sides of the debate need to do a better job educating Americans about how various reform plans will impact the future of our healthcare system.
Nobody said it was going to be easy.
Relative to healthcare reform, they find that a majority of Americans aren’t so keen after all to have their Uncle Sam become Uncle Sam, MD:
“There is no real appetite for increasing taxes to pay for an expanded health-insurance program. Less than half would support such an idea, which is 17% less than the percentage that supported government health insurance when Bill Clinton first considered it in March of 1993.”
(Their complete opinion piece in today’s edition of the Wall Street Journal can be found here.)
The conclusions reached by Schoen and Rasmussen are completely in line with a poll fielded by the Center for Medicine in the Public Interest last October.
The CMPI survey was of “Millennial” voters (18-28 years old) -- often portrayed as the new “driving force” in American politics. When millennial voters were engaged on the issue of health care reform they had some very strong – and often contradictory – opinions a -- particularly when it comes to issue of “universal care.”
The national public opinion poll of young voters (the result of 1001completed interviews with adults 18-28 years of age who are registered to vote) shows limited acceptance for the potential consequences of greater government control over health care.
While millennial voters report to strongly support the need for reform and the concept of “universal care,” when asked if they are willing to pay higher taxes to pay for a government-run health care system, their level of support swiftly turns in the opposite direction.
Millennial voters are strongly against government-care that results in longer wait times to see a health care provider, limits to the types of treatments and medicines they can access, and the potential for the government to interfere in the decision making and relationship between doctor and patient.
Some germane findings:
* A majority (51 percent) were not in support of any health care reforms that could raise their personal tax burden;
* Sixty-two percent said they would not support any health care reforms that could increase wait-times to see a doctor or the availability of treatments and medicines; and,
* Millennial voters were also equally unsupportive (62 percent) of health care reforms that would increase the role of the government regulation and oversight in doctor-patient decision-making.
(For further details on the CMPI poll, see “The Young and the Restless.”)
The CMPI poll and the findings of Schoen and Rasmussen demonstrate that all sides of the debate need to do a better job educating Americans about how various reform plans will impact the future of our healthcare system.
Nobody said it was going to be easy.
Meghan, Clyne, a former speechwriter for President George W. Bush, has an edifying piece in the New York Post about President Obama’s announcement that the federal government will be funding embryonic stem cell research. Of particular interest, though, are the connections high-ranking Obama Administration science officials have with groups that stand to benefit handsomely from the new policy.
It is interesting how in cases such as this silence suddenly overtakes the usual troublemakers who incessantly whine about research funded by the pharmaceutical industry being tainted.
This week CMPI president Peter Pitts also touched upon this gross double-standard in his Op-Ed in The Journal of Life Sciences. Read that article here.
Here are the relevant excerpts from Meghan Clyne’s piece:
Problem is, the administration's preening about its own scientific purity just isn't justified.
Consider, for instance, Eric Lander and Harold Varmus - the co-chairmen of the President's Council of Advisers on Science and Technology, which helps steer decisions on issues like the stem-cell matter.
Lander is a renowned stem-cell researcher at MIT, a world-class university that stands to get even more federal funding, thanks to Obama's stem-cell move. An MIT spokeswoman says the university takes conflict-of-interest precautions when its faculty serve in government positions - but added that it won't recuse itself from funding opportunities related to Obama's decision.
More of the public-relations legwork on the Obama stem-cell decision has been done by the advisory council's oth- er co-chairman, Harold Varmus.
What of Varmus? The president lauded him Monday as an example of the type of "outstanding scientists" who "will guide us in the years to come." He's a Nobel-prize winning scientist who now serves as president of the Memorial Sloan Kettering Cancer 20Center.
And a spokeswoman says Sloane Kettering's Stem-Cell Research Facility didn't qualify for federal funds under the Bush policy, because it uses tissues that violate the Bush ethical guidelines. Now that Obama has reversed that policy, the federal funding floodgates are open.
According to its most recent IRS 990 form, the hospital is already pretty good at working the public-sector pump: For 2007, it reported $120 million in revenue from government grants. Documents show that in 2006, Varmus earned more than $250,000 worth of his salary for "fund-raising."
Read full Op-Ed here:
CMPI recently visited the Conservative Political Action Conference in Washington, DC and had the opportunity to speak with Governor Tim Pawlenty of Minnesota. As some might recall, Governor Pawlenty was one of the leading contenders for Senator McCain’s VP running-mate.
Governor Pawlenty has been one of the more active governors in the country in advancing free market based healthcare initiatives.
He has been an ardent proponent of electronic health records and co-authored the following Op-Ed last year with Former Speaker of the House Newt Gingrich:
Read Op-Ed here.
Read Op-Ed here.
We asked Governor Pawlenty if he had any advice to offer President Obama as his administration tackles healthcare reform. Governor Pawlenty said, “I’m afraid that President Obama and the Congress are headed in a very different direction” from free-market healthcare reforms.
He noted, “I think we should get consumers involved” and “let them decide in the marketplace how to best manage their healthcare.”
Let’s hope Administration officials are listening.
SUTENT SIGNIFICANTLY INCREASES PROGRESSION FREE SURVIVAL FOR PATIENTS WITH ADVANCED PANCREATIC ISLET CELL TUMORS,
STUDY STOPPED EARLY
NEW YORK, NY, March 12 -- Pfizer Inc announced today that a phase 3 clinical trial of Sutent (sunitinib malate) has been stopped early after the drug showed significant benefit in patients with advanced pancreatic islet cell tumors, also known as pancreatic neuroendocrine tumors.
An independent Data Monitoring Committee (DMC) recommended halting the trial after concluding that Sutent demonstrated greater progression-free survival compared to placebo plus best supportive care in patients with pancreatic islet cell tumors.
“We are delighted by these findings which demonstrate that Sutent provides a benefit for patients with advanced, well-differentiated pancreatic islet cell tumors — a rare cancer with limited treatment options,” said Dr. Mace Rothenberg, senior vice president of medical development and clinical affairs for Pfizer’s Oncology Business Unit. “These and previously reported phase 2 data contribute to the growing body of evidence indicating activity with sunitinib in patients with pancreatic islet cell tumors.”
Pfizer has notified clinical trial investigators involved in the trial and regulatory agencies of the DMC recommendations. All patients in the trial will have the option to continue taking Sutent or be switched from placebo to Sutent. The full data set from this trial is being analyzed and more details will be presented at an upcoming scientific meeting.
This phase III trial of sunitinib in patients with advanced pancreatic islet cell tumors was initiated based on the results of a earlier phase II trial published in the Journal of Clinical Oncology(July 2008).
In contrast to exocrine pancreatic adenocarcinoma, pancreatic islet cell tumors are rare, indolent tumors of the endocrine pancreas with an incidence of 5-10 per million worldwide annually. Pancreatic islet cell tumors include insulinomas, glucagonomas and gastrinomas. Current treatment options are limited.
Sutent is currently approved for both advanced renal cell carcinoma (RCC) and second-line gastrointestinal stromal tumor (GIST), based on efficacy and safety data from large, randomized Phase 3 clinical trials. Sutent has played an important role in reshaping the treatment landscape for these two difficult-to-treat cancers. To date, more than 38,000 patients globally have been treated with Sutent in the clinical setting and trials.
This is the second phase III Sutent trial Pfizer has stopped early on the recommendation of an independent data monitoring committee due to benefit. In January 2005, a phase III trial in GIST was unblinded early when a planned interim analysis showed significantly longer time to tumor progression with Sutent compared to placebo.
STUDY STOPPED EARLY
NEW YORK, NY, March 12 -- Pfizer Inc announced today that a phase 3 clinical trial of Sutent (sunitinib malate) has been stopped early after the drug showed significant benefit in patients with advanced pancreatic islet cell tumors, also known as pancreatic neuroendocrine tumors.
An independent Data Monitoring Committee (DMC) recommended halting the trial after concluding that Sutent demonstrated greater progression-free survival compared to placebo plus best supportive care in patients with pancreatic islet cell tumors.
“We are delighted by these findings which demonstrate that Sutent provides a benefit for patients with advanced, well-differentiated pancreatic islet cell tumors — a rare cancer with limited treatment options,” said Dr. Mace Rothenberg, senior vice president of medical development and clinical affairs for Pfizer’s Oncology Business Unit. “These and previously reported phase 2 data contribute to the growing body of evidence indicating activity with sunitinib in patients with pancreatic islet cell tumors.”
Pfizer has notified clinical trial investigators involved in the trial and regulatory agencies of the DMC recommendations. All patients in the trial will have the option to continue taking Sutent or be switched from placebo to Sutent. The full data set from this trial is being analyzed and more details will be presented at an upcoming scientific meeting.
This phase III trial of sunitinib in patients with advanced pancreatic islet cell tumors was initiated based on the results of a earlier phase II trial published in the Journal of Clinical Oncology(July 2008).
In contrast to exocrine pancreatic adenocarcinoma, pancreatic islet cell tumors are rare, indolent tumors of the endocrine pancreas with an incidence of 5-10 per million worldwide annually. Pancreatic islet cell tumors include insulinomas, glucagonomas and gastrinomas. Current treatment options are limited.
Sutent is currently approved for both advanced renal cell carcinoma (RCC) and second-line gastrointestinal stromal tumor (GIST), based on efficacy and safety data from large, randomized Phase 3 clinical trials. Sutent has played an important role in reshaping the treatment landscape for these two difficult-to-treat cancers. To date, more than 38,000 patients globally have been treated with Sutent in the clinical setting and trials.
This is the second phase III Sutent trial Pfizer has stopped early on the recommendation of an independent data monitoring committee due to benefit. In January 2005, a phase III trial in GIST was unblinded early when a planned interim analysis showed significantly longer time to tumor progression with Sutent compared to placebo.
Commissioner Hamburg and Deputy Commissioner Sharfstein. Does this mean a partnership where one addresses the FDA’s food-side mission (Hamburg) and the other drugs and devices (Sharfstein)? Does it mean that the FDA will be under the thumb of Representative Henry Waxman? (Dr. Sharfstein was a former Waxman staffer.) Or does it mean that the FDA now has the political cover to remove the knife from its back and get back to work?
Will Dr. Sharfstein’s strong support of more robust OTC labeling help or hinder the FDA’s move towards a BTC (Behind-the-Counter) regulatory paradigm?
How will Dr. Hamburg address the dire state of food safety and security at an agency that gets dinged by our elected representatives – but for which Congress is slow to write a bigger check? Will Dr. Hamburg fight for an FDA food safety and security stimulus package? She certainly has the bona fides to do so.
And what about DTC? Will we see more letters (a perennial Hanukah wish from Mr. Waxman) or a move towards brighter lines from DDMAC?
And then there’s the question of REMS plans and complete response letters.
And biomarkers.
And guidance on off-label promotion.
And follow-on biologics.
And bioequivalence for generics.
And drug importation.
And comparative effectiveness for NDA submissions.
And companion diagnostics.
And 510(k) reform.
And greater international harmonization.
And the working relationship with the senior career staff at FDA.
And working relationships with industry and academia.
And, and, and …
Many questions. Few answers.
Here are seven things for Dr. Hamburg and Dr. Sharfstein to consider:
1. A Strong, Science-Based FDA
Everybody benefits from an FDA that leads. This means the agency has to be out in front of every issue for which it is responsible. Every specific action the agency takes is an opportunity to speak to a larger public health issue. Vioxx, for example, was a missed occasion for the FDA to seize the day on the issue of drug safety. When the FDA confidently leads, other stakeholders follow with their expertise, resources and sense of duty. This is not a people-intensive proposition. But it does require the commitment and the skill to do it – and do it right and regularly.
2. The Reagan/Udall Foundation: A Partnership of Unequals
The FDA must be both regulator in protecting the public health and colleague in helping to advance it. This is a delicate balance and in the current political climate the agency gets no merit points for being seen as collaborating with those it regulates. The best way to do this is via the Reagan/Udall Foundation. Now that the election is over an immediate first step should be for the new FDA Commissioner to meet with Representative DeLauro and issue a joint announcement on moving forward with the work of the agency’s Critical Path program to develop the tools necessary for 21st Century pharmaceutical and medical device development and regulation. But FDA must be seen as leading rather than simply participating in the process.
3. Clarity vs. Ambiguity
Regulators often love ambiguity – because ambiguity is power. The problem is that such a philosophy can lead to regulatory dissonance – ranging from completed Phase III trials supported by the agency at advisory committee meetings and then derided by a division afterwards, to warning letters sent to companies over marketing materials that have been “pre-cleared” by DDMAC. If people want the various industries regulated by the FDA to follow the rules, there need to be as many bright lines as possible – and they need to apply to everyone equally. While a high degree of pragmatism will always be required, this is not an excuse for “I know it when I see it” regulation.
4. Information Management
The FDA’s information management system is dysfunctional. The FDA sits at the crossroads of vast amounts of information that is of vital use to both protecting and advancing the public health. –yet most of it is unusable. The new Commissioner should immediately appoint an IT Czar who, as her first order of business, should audit existing systems and draw up an information technology roadmap for the agency. A solid “taskforce of talent” is needed to address this tremendous opportunity. Solid information management systems will allow the agency to do its job better, faster, and less expensively. A solid public health triple play.
5. Food Safety and Security
The agency’s programs on food safety and security are failing. Resources at CFSAN (the Center for Food Safety and Applied Nutrition) are stretched drum tight. More money is certainly needed – but there is also a crisis in confidence that the agency knows what it’s doing – or that it cares. The issue of BPA (bisphenol A) is a good example. The FDA was purely reactive in its call for a review by its panel of experts. As a result, the agency was destined to have its judgment called into question whatever the decision. And this is precisely what happened. This further cements the general perception that the FDA doesn’t care and/or is beholden to the industries it regulates. The BPA issue was out there for a long time in a visible way. And the agency didn’t do anything. It didn’t lead, it followed. And the consequences shouldn’t have surprised anyone. A similar situation is brewing with melamine in baby formula. Further, there is a growing sense that CFSAN should be moved to its own independent status within HHS. Perhaps. But for this to proceed, serious thinking needs to go into two issues: (1) DSHEA and the regulation of dietary supplements as foods, and (2) the increasingly important issue of nutriceuticals and qualified health claims. Both are clearly FDA issues and should remain so.
6. Risk Communications
Rather than assuming the mantle of responsibility and proactively stepping forward with more regular and transparent risk communications programs, the FDA was driven by the winds of crisis. Today the agency has implemented certain programs (some required by FDAAA) that provide risk information – but without any context, rhyme or reason. The result is confusion among patients and physicians and a field day for the media. The unintended consequences have swamped the public health benefit. Senior agency leadership knows it – but what are they doing to address it? The answer is not clear. A good beginning would be for the FDA’s Risk Communications Advisory to look into the matter. This problem needs to be fixed as no one (not industry, not doctors, not patients,) is happy with the current state of affairs.
7. The Drug Label and the “Safe Use” of Drugs
Wyeth v. Levine notwithstanding, the drug label is the single most important piece of communications material the agency issues – and it isn’t working as well as it should. The New Physician Labeling Rule (January 2006) has had minimal impact for three main reasons: (1) It has not been widely adopted for products licensed prior to the rule (not a requirement, but an option), (2) There has been little agency out-reach to physicians and, (3) There has been no broader agency program on the issue of “safe use.” This last point will change in January when the FDA (via CDER – the Center for Drug Evaluation and Research) launches a more comprehensive “safe use” initiative. The agency must consider not just safety, efficacy and quality – but safe use as well. Not just drug safety, but patient safety. This makes perfect sense and gives the FDA the opportunity to speak not only to physicians, but to consumers as well. The program should be expanded to also include medical devices. It is a timely, important, and urgent opportunity and must be done with determination, creativity, and relentless passion. It must be the FDA on the offense for the public health. And the offense must never stop.
Good luck Peggy. Good luck Josh.
Let’s get to work.
Will Dr. Sharfstein’s strong support of more robust OTC labeling help or hinder the FDA’s move towards a BTC (Behind-the-Counter) regulatory paradigm?
How will Dr. Hamburg address the dire state of food safety and security at an agency that gets dinged by our elected representatives – but for which Congress is slow to write a bigger check? Will Dr. Hamburg fight for an FDA food safety and security stimulus package? She certainly has the bona fides to do so.
And what about DTC? Will we see more letters (a perennial Hanukah wish from Mr. Waxman) or a move towards brighter lines from DDMAC?
And then there’s the question of REMS plans and complete response letters.
And biomarkers.
And guidance on off-label promotion.
And follow-on biologics.
And bioequivalence for generics.
And drug importation.
And comparative effectiveness for NDA submissions.
And companion diagnostics.
And 510(k) reform.
And greater international harmonization.
And the working relationship with the senior career staff at FDA.
And working relationships with industry and academia.
And, and, and …
Many questions. Few answers.
Here are seven things for Dr. Hamburg and Dr. Sharfstein to consider:
1. A Strong, Science-Based FDA
Everybody benefits from an FDA that leads. This means the agency has to be out in front of every issue for which it is responsible. Every specific action the agency takes is an opportunity to speak to a larger public health issue. Vioxx, for example, was a missed occasion for the FDA to seize the day on the issue of drug safety. When the FDA confidently leads, other stakeholders follow with their expertise, resources and sense of duty. This is not a people-intensive proposition. But it does require the commitment and the skill to do it – and do it right and regularly.
2. The Reagan/Udall Foundation: A Partnership of Unequals
The FDA must be both regulator in protecting the public health and colleague in helping to advance it. This is a delicate balance and in the current political climate the agency gets no merit points for being seen as collaborating with those it regulates. The best way to do this is via the Reagan/Udall Foundation. Now that the election is over an immediate first step should be for the new FDA Commissioner to meet with Representative DeLauro and issue a joint announcement on moving forward with the work of the agency’s Critical Path program to develop the tools necessary for 21st Century pharmaceutical and medical device development and regulation. But FDA must be seen as leading rather than simply participating in the process.
3. Clarity vs. Ambiguity
Regulators often love ambiguity – because ambiguity is power. The problem is that such a philosophy can lead to regulatory dissonance – ranging from completed Phase III trials supported by the agency at advisory committee meetings and then derided by a division afterwards, to warning letters sent to companies over marketing materials that have been “pre-cleared” by DDMAC. If people want the various industries regulated by the FDA to follow the rules, there need to be as many bright lines as possible – and they need to apply to everyone equally. While a high degree of pragmatism will always be required, this is not an excuse for “I know it when I see it” regulation.
4. Information Management
The FDA’s information management system is dysfunctional. The FDA sits at the crossroads of vast amounts of information that is of vital use to both protecting and advancing the public health. –yet most of it is unusable. The new Commissioner should immediately appoint an IT Czar who, as her first order of business, should audit existing systems and draw up an information technology roadmap for the agency. A solid “taskforce of talent” is needed to address this tremendous opportunity. Solid information management systems will allow the agency to do its job better, faster, and less expensively. A solid public health triple play.
5. Food Safety and Security
The agency’s programs on food safety and security are failing. Resources at CFSAN (the Center for Food Safety and Applied Nutrition) are stretched drum tight. More money is certainly needed – but there is also a crisis in confidence that the agency knows what it’s doing – or that it cares. The issue of BPA (bisphenol A) is a good example. The FDA was purely reactive in its call for a review by its panel of experts. As a result, the agency was destined to have its judgment called into question whatever the decision. And this is precisely what happened. This further cements the general perception that the FDA doesn’t care and/or is beholden to the industries it regulates. The BPA issue was out there for a long time in a visible way. And the agency didn’t do anything. It didn’t lead, it followed. And the consequences shouldn’t have surprised anyone. A similar situation is brewing with melamine in baby formula. Further, there is a growing sense that CFSAN should be moved to its own independent status within HHS. Perhaps. But for this to proceed, serious thinking needs to go into two issues: (1) DSHEA and the regulation of dietary supplements as foods, and (2) the increasingly important issue of nutriceuticals and qualified health claims. Both are clearly FDA issues and should remain so.
6. Risk Communications
Rather than assuming the mantle of responsibility and proactively stepping forward with more regular and transparent risk communications programs, the FDA was driven by the winds of crisis. Today the agency has implemented certain programs (some required by FDAAA) that provide risk information – but without any context, rhyme or reason. The result is confusion among patients and physicians and a field day for the media. The unintended consequences have swamped the public health benefit. Senior agency leadership knows it – but what are they doing to address it? The answer is not clear. A good beginning would be for the FDA’s Risk Communications Advisory to look into the matter. This problem needs to be fixed as no one (not industry, not doctors, not patients,) is happy with the current state of affairs.
7. The Drug Label and the “Safe Use” of Drugs
Wyeth v. Levine notwithstanding, the drug label is the single most important piece of communications material the agency issues – and it isn’t working as well as it should. The New Physician Labeling Rule (January 2006) has had minimal impact for three main reasons: (1) It has not been widely adopted for products licensed prior to the rule (not a requirement, but an option), (2) There has been little agency out-reach to physicians and, (3) There has been no broader agency program on the issue of “safe use.” This last point will change in January when the FDA (via CDER – the Center for Drug Evaluation and Research) launches a more comprehensive “safe use” initiative. The agency must consider not just safety, efficacy and quality – but safe use as well. Not just drug safety, but patient safety. This makes perfect sense and gives the FDA the opportunity to speak not only to physicians, but to consumers as well. The program should be expanded to also include medical devices. It is a timely, important, and urgent opportunity and must be done with determination, creativity, and relentless passion. It must be the FDA on the offense for the public health. And the offense must never stop.
Good luck Peggy. Good luck Josh.
Let’s get to work.
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.
