Remember when

• 04.06.2012

BioCentury reports:

EMA backs AD biomarkers

The EMA's CHMP adopted a qualification opinion that positive/negative PET imaging of amyloid can be used as a biomarker to identify patients in clinical trials with predementia Alzheimer's disease (AD) who are at increased risk to have an underlying neuropathology. CHMP also adopted a qualification opinion that the combination of low beta amyloid (1-42) and high tau in cerebrospinal fluid (CSF), and amyloid-related PET imaging, can be used to identify patients with mild to moderate AD who are increased risk of an underlying AD neuropathology. CHMP said the biomarkers are intended to enrich recruitment in trials aimed at studying products to slow the conversion to AD or severe AD.

HELP is on the way

• 04.05.2012

The Health, Education, Labor and Pensions Committee released a discussion draft of legislation aimed at accelerating patient access to innovative medical treatments for life-threatening diseases.

The bipartisan Drug Approval and Patient Access Plan would overhaul the FDA's Accelerated Approval pathways and create a new "breakthrough" designation to provide more flexibility and certainty for developers of new medicines intended to address serious or life-threatening diseases or conditions.

The draft includes legislation introduced last week by Sen. Michael Bennet (D-Colo.) that would expedite FDA approval and provide more flexibility when a drug or treatment shows dramatic responses early in development.

Taking Pediatric Medicines Development off the 5 Year Plan

• 04.04.2012

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.

One man's ceiling is another man's floor

• 04.03.2012

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.

Is Obamacare Doomed?

• 04.02.2012

Even If It Survives the Court, the Health Care Law Is Doomed By Scott Rasmussen Media coverage now implies that the U.S. Supreme Court will determine the fate of President Obama's health care law. But nothing the court decides will keep the law alive for more than a brief period of time. There are three ways the health care law could meet its end. The first, obviously, is the Supreme Court could declare some or all of it unconstitutional in June. If it gets past that hurdle, the law also could be ended by Election 2012. If a Republican president is elected, the GOP will almost certainly also win control of the Senate and retain control of the House. While the details might take time, a Republican sweep in November would ultimately end the Obama experiment. But even if the law survives the Supreme Court and the next election, the clock will be ticking. Recent estimates suggest that the law would cause 11 million people to lose their employer-provided insurance and be forced onto a government-backed insurance plan. That's a problem because 77 percent of those who now have insurance rate their current coverage as good or excellent. Only 3 percent rate their coverage as poor. For most of the 11 million forced to change their insurance coverage then, it will be received as bad news and create a pool of vocally unhappy voters. Additionally, the cost estimates for funding the program are likely to keep going up. Eighty-one percent of voters expect it to cost more than projected, and recent Congressional Budget Office estimates indicate voters are probably right. But it's not the narrow specifics and cost estimates that guarantee the ultimate demise of the president's health care plan. It's the fact that the law runs contrary to basic American values and perceptions. This, then, is the third hurdle the law faces: Individual Americans recognize that they have more power as consumers than they do as voters. Their choices in a free market give them more control over the economic world than choosing one politician or another. Seventy-six percent think they should have the right to choose between expensive insurance plans with low deductibles and low-cost plans with higher deductibles. A similar majority believes everyone should be allowed to choose between expensive plans that cover just about every imaginable medical procedure and lower-cost plans that cover a smaller number of procedures. All such choices would be banned under the current health care law. Americans want to be empowered as health care consumers. Eighty-two percent believe that if an employer pays for health insurance, the worker should be able to use that money and select an insurance product that meets his or her individual needs. If the plan they select costs less than the company plan, most believe the worker should get to keep the change. It's not just the idea of making the choice that drives these numbers, it's the belief held by most Americans that competition will do more than government regulation to reduce the cost of health care. For something as fundamental as medical care, government policy must be consistent with deeply held American values. That's why an approach that increases consumer choice has solid support and a plan that relies on mandates and trusting the government cannot survive. COPYRIGHT 2012 SCOTT RASMUSSEN

The Supreme Court and the Individual Mandate

• 04.02.2012

A lively discussion on the US Supreme Court and health care reform on "This Week":

Drug Co-Pays and Asthma Rates

• 03.30.2012

I am quoted in this piece published in Medscape..

http://www.medscape.com/viewarticle/761255

NRDC Rejects Science

• 03.30.2012

The gap between science and the National Resources Defense Counci's fearmongering is growing at an exponential rate.  Watch as NRDC pushes a legislative BPA ban and links it to their fundraising operation... 



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.”

Pharma's Social Media Hunger Games

• 03.30.2012

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.

The other healthcare hearing

• 03.29.2012

In the midst of all the hoopla surrounding SCOTUS and the Affordable Care Act, let’s not forget today’s Senate HELP hearing on PDUFA.

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?