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We called it.
At CMPI’s November PDUFA without the Politics conference, each panelist was asked to bring one ornament to hang on the reauthorization Christmas Tree. Tim Franson (President of the USP Convention and one of the “Fathers of PDUFA”) offered a little lifeboat.
What can we do for those with life threatening unmet needs? One thing, as was done in PDUFA IV, is adding renewal of the Best Pharmaceuticals for Children’s Act. That piece of legislation has done more for pediatric drug development than anything in the past. It’s been good for American children and it should be made permanent. I think the better policy argument is to make it permanent, because companies don’t invest in five-year cycles. (Note to reader: The Act is currently renewable every five years.)
If I’m looking for a return on an investment, I need certainty well in advance of five years on types of studies I should be conducting and I need to know the benefit I’m getting at the end. I think five years is far too short a period of time.
There’s an accumulated body of evidence, of companies fulfilling their commitments. Maybe its time we make it permanent. I think this point of holding up renewal like the Sword Damocles isn’t very persuasive even to sponsors.
Well, a bipartisan House bill now proposes to permanently reauthorize two pediatric-drug statutes that include marketing incentives and clinical-study mandates, which the drug industry supports, while also giving FDA recourse for unfinished pediatric studies and requiring drug sponsors to submit their plans to the agency earlier, measures advocated by pediatricians.
Reps. Ed Markey (D-MA), Anna Eshoo (D-CA) and Mike Rogers (R-MI) introduced the bill to permanently reauthorize the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act.
The bill would require drug makers to submit an initial pediatric-study plan by the end of phase 2 clinical trials. Currently, companies must submit the information when they submit an application or supplement to the agency. FDA must promulgate a rule and guidance on the new timing requirement, according to the bill. The plan should include an outline of proposed studies and any request for a waiver to defer the requirements.
Lawmakers backing the bill said the pediatric laws have led to pediatric studies for 180 new drugs and more than 400 updated drug labels.
“Government and private industry have worked together to fill an important void,” Eshoo said in a statement. “The laws have provided greater transparency and accountability in the programs, while improving communication between FDA and companies. I’m proud of how far we’ve come and look forward to a day when we know how all medicines will affect children.”
Now sponsors are looking for a little HELP.
In recent statements ANVISA, the Brazilian equivalent of the FDA, has put forward the proposition that (relative to raising the level of international drug quality and safety) there isn’t a way forward but rather “ways to move forward based on local situations.
While the solution must fit the problem, this poses an interesting question – can there be a floor and a ceiling for global drug safety and quality? Even as we move toward differential pricing, should we allow some countries to have lower standards than others “based on local situations?”
When it comes to the safety of pharmaceuticals and medical devices, can one man’s ceiling be another man’s floor.
And how does this fit into the debate over trade policy versus trade practice? Should medical products that are determined not to meet any given national standard be allowed to be exported to other countries? Should there be a “good enough for me, good enough for thee” standard for international trade in pharmaceuticals and medical devices?
For example, should Beijing allow substandard API -- illegal for use in the Middle Kingdom -- to be exported to, say, Nigeria?
When Paul Orhii, Director-General of Nigeria’s National Agency for Food and Drug Administration and Control, complained to the Chinese government that China was the origin of many of the counterfeit medicines in Nigeria, he was bluntly told Beijing was not responsible for the quality of medicines in Nigeria.
That’s the unfortunate distinction between trade policy and trade practice. Is this an issue for TRIPS? Should TRIPS Article 61 be amended to include punishments and penalties for those who export medicines and medical devices that do not meet a certain global standard?
While domestic standards are undeniably an issue of domestic sovereignty, shouldn’t there be transparency as to how any given nation defines safety and quality? “Market authorization” means one thing in the context of the MHRA, the FDA, the EMA, and Health Canada (to choose only a few “gold standard” examples), but how are we to judge the regulatory competencies of other national systems? Is that the responsibility of the WHO via a better-developed (and far more transparent) pre-qualification scheme? Or regional arbiters (such as PAHO)? Should there be “reference regulatory systems” as there are reference nations for pricing decisions? Should there be regulatory reciprocity?
All the more reason for “gold standard” nations to undertake a regulatory Marshall Plan to help build global systems for drug quality and safety.
The harmonization of global trade policy and practice is essential to the sinews of international medicines quality and safety.
http://www.nrdc.org/media/2012/120330.asp
FDA Rejects NRDC Call to Eliminate BPA from Food Packaging
NRDC: ‘FDA Fails to Protect Our Health and Safety’
WASHINGTON (March 30, 2012) -- The Food and Drug Administration said today it would allow bisphenol A (BPA) to remain in food packaging, an action that keeps the hormone-disrupting chemical linked to cancer, obesity and a host of other health problems in the food supply.
In rejecting a petition today from the Natural Resources Defense Council, the agency emphasized it was not making a final determination of BPA’s safety and instead will continue to examine the ongoing research of BPA’s effects on health.
The following is a statement from Dr. Sarah Janssen, senior scientist in the public health program at the Natural Resources Defense Council:
“BPA is a toxic chemical that has no place in our food supply. We believe FDA made the wrong call.
“The agency has failed to protect our health and safety - in the face of scientific studies that continue to raise disturbing questions about the long-term effects of BPA exposures, especially in fetuses, babies and young children.
“The FDA is out-of-step with scientific and medical research. This illustrates the need for a major overhaul of how the government protects us against dangerous chemicals.”
Last week, at the PMRG (Pharmaceutical Marketing Research Group) Annual National Conference, I participated on “The Coming of Age of Social Media and Healthcare” panel.
My fellow panelists were the always pugnacious Arnie Friede, UBC’s Pat Choumitsky, and Eric Schultz of QuantiaMD. The session was ably moderated by Mark Bard, the grand master of the Digital Health Coalition.
(Panel description and panelist bios can be found here.)
A few thoughts on the subject that arose from this event:
How many times have you heard (vis-à-vis social media and healthcare) that, “Pharma is different.” That’s true – but consumers are the same. They don’t think about why Pharma is absent from the conversation – and they don’t care. They assume it’s because the industry “has something to hide” or that they’re afraid of mixing it up with real people in real time.
And there’s more than a little truth to that.
Is social media about “collaborating” with consumers or “cooperating” with them? What’s the difference? Well, cooperation happens when both sides want to survive. Collaboration happens when they want to thrive. Collaboration means interacting honestly and transparently. And Pharma’s opportunity (within the context of social media) is to be the first among equals.
Otherwise social media becomes the healthcare Hunger Games.
Success for Pharma in social media will come through collaboration. And that doesn’t mean “selling.”
And may the odds be ever in your favor.
For those who want to follow this important conversation, follow the twitter feed @modernmeds for the blow-by-blow.
Who said healthcare policy was dull?
High Court Hopes
(Page 3 of 7)
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JOHN FUND
One of the least-noticed mistakes made by the Obama administration in its arguments for its health-care law this week was the claim by Solicitor General Donald Verrilli that Congress had passed Obamacare to deal with a pressing national problem only “after long study and careful deliberation.” I’m told that even liberal lawyers in the courtroom had pained looks on their faces at that preposterous assertion.
Certainly the general public doesn’t buy that argument, as indicated by the polls. I doubt many lawyers do either. Congressional leaders bullied members into voting for the measure in the dead of night, blatant payoffs such as Nebraska’s “Cornhusker Kickback” and Florida’s “Gatoraid” were handed out, and members were given almost no time to absorb the bill’s 2,700 pages (leading House speaker Nancy Pelosi to say, candidly, “we have to pass the [health-care] bill so that you can find out what is in it.”) At least one Supreme Court justice is said to be astonished that process was so sloppy that Congress left out the traditional “severability” section, which routinely allows that should the courts strike down part of a law the rest would stand.
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Indeed, the third day of oral arguments in the Supreme Court brought a mild surprise: At least five of the justices are considering repealing all of Obamacare, either because the task of figuring out which parts can work absent an individual mandate is too onerous or they don’t believe Congress would have passed the bill without the mandate. In other words, the Court just might take the extraordinary step of sending Congress a stiff rebuke: that its recklessness and sloppiness in lawmaking should have some limits.
Randy Barnett, the Georgetown law professor who has bravely pushed the legal arguments against Obamacare with bulldog tenacity for two years, summed up what has been a good week for the law’s critics: “After these arguments, if the Court strikes down the Affordable Care Act, no one in the country will be surprised.” Indeed, if it happens, a majority of Americans will be relieved. And the battle over putting back some serious curbs on Congress’s ability to run roughshod over the Constitution will then begin in earnest.
— John Fund, a New York writer, is author of Stealing Elections: How Voter Fraud Threatens Our Democracy.
This is my contribution to today's http://www.nationalreview.com on-line discussion about how the Court should rule and what should the political response to it's decision (or the decision we hope for) be.
I hope the Court rules Obamacare unconstitutional. The fact that Congress had many other ways to expand insurance pools has undermined the necessary and proper claim for the mandate. As Roger Vinson noted in his district court ruling, “While the individual mandate was clearly ‘necessary and essential’ to the Act as drafted, it is not ‘necessary and essential’ to health care reform in general.” The mandate is a way to redistribute premium revenue from the young and healthy to other groups. Under Obamacare, it is possible to pay the penalty and use the free emergency care that must still be provided under current law. I think all the judges regard Obamacare as unconstitutional and a failure.
If the Court overturns the law, Obamacare again becomes an issue. Supporters will warn that young people will lose coverage under their parents’ plan and that this will undo community rating. Opponents will be able to say the ruling stops a tax increase, blocks the indirect tax Obamacare imposed on people forced to buy the amount of health-care coverage deemed necessary by government, and pulls the plug on rationing. I hope legislators introduce a market-based reform plan that makes low-cost coverage available. If Obamacare supporters want to reintroduce the law with higher taxes and more regulation, let them.
Two important news items courtesy of BioCentury:
The first concerns the necessary and timely updating of how the FDA determines risk/benefit for medical devices.
Final FDA guidance on risk-benefit determinations for devices
FDA released final guidance that seeks to clarify how the agency determines the risk-benefit profile of a medical device during a premarket review. The guidance, which is in line with a draft released last August, says that in addition to safety and efficacy, FDA reviewers will consider criteria such as the novelty of the technology, the characterization of the disease and the availability of alternative treatments or diagnostics.
The guidance includes a worksheet that reviewers will use to make risk-benefit determinations during a review. FDA said it is developing training modules to help industry and sponsors understand how the Center for Devices and Radiological Health (CDRH) will apply the guidance. Reviewers will begin applying it to incoming submissions and to submissions already under review beginning May 1.
Of particular interest to those following the agency’s arduous journey in search of a risk/benefit “grid” is how CDRH will both develop and use its “worksheet” and whether or not that document will be public information after a regulatory decision is reached. Transparency would certainly go a long way towards holding the Center’s feet to the fire vis-à-vis regulatory predictability and consistency.
And, speaking of consistency, some news from our Regulatory Cousins across the pond.
EMA finalizes guidance on MAA transparency
The EMA and the Heads of Medicines Agencies (HMA) adopted a joint guidance for a Europe-wide approach to identify which information included in an MAA can be publicly released after approval, should a request for access to information be submitted. The guidance is part of EMA's policy to increase transparency and access to documents, which came into effect in 2010.
According to the guidance, sections of an MAA dossier that would not be released include detailed information on manufacturing processes and synthesis of the active ingredient, as well as contractual agreements. In general, reports on non-clinical and clinical trials, including efficacy and safety trials, would be made available, although exceptions may be considered when innovative study designs or analytical methods have been used. Identifiable patient information would be redacted. Scientific advice received by a sponsor on an agreed indication would be available, while advice related to new developments and formulations would remain confidential.
(For you Yanks, “MAA” = “Marketing Authorization Application.”)
This is interesting for a number of reasons, let me mention two. First, it points to the ability to harmonize across borders. Need an ocean separate us from this concept? And, second, is this at all predictive of where the FDA might go relative to “going public” with redacted Complete Response Letters?
Inquiring minds want to know.
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.
