Some interesting tidbits from the FDA’s June 24^th meeting on the issue of transparency.

 “FDA should make it obvious, to those who are interested, how scientific data leads to its approval decisions.” -- PhRMA Assistant General Counsel Jeffrey Francer. As an example, Francer pointed to FDA approval decisions that do not track with advisory committee recommendations. "That's often confusing, not only to those of us in industry, but people generally."

Absolutely.  But, since transparency cuts both ways, wouldn’t it be equally beneficial for pharmaceutical companies to proactively release complete response letters received from the agency once a final action has been taken?

Transparency thrives in an atmosphere of clarity.  And that was also a topic of conversation.

BIO’s Director for Science and Regulatory Affairs Andrew Emmett, commented that the FDA does not meet and communicate with firms on a consistent basis early in the development and review process. He urged, and appropriately so, that the agency work with industry to minimize barriers to such meetings.

Emmett also called for the agency to provide more information so industry can understand how FDA makes decisions. There needs to be "regulatory transparency and clear articulation of FDA's policies and expectations."

Since it can take several years for FDA to finalize guidance, "This leaves companies to ascertain FDA policy by interpreting the agency's regulatory decisions and enforcement actions, which is an inefficient way for industry to understand and meet the agency's regulatory expectations."

If we’re going to go “open kimono” on this, then the real 800 pound gorilla in the room is the fact that many inside the FDA prefer to leave guidances in draft form – because that ambiguity gives them almost unlimited and perpetual power.  That’s why interpretation of FDA actions is such a vibrant cottage industry. Industry, on the other hand, seeks clarity. They want bright lines. They want to know the rules. They want predictability. This may sound simple, but it has proven to be a fractious bureaucratic kulturkampf within the FDA.

Regulators change industry behavior by changing the rules of the game. But changing the minds of regulators, having them embrace bright lines rather than broad definitions, is a distinctly more challenging proposition, because changed minds must begin with change agents within the agency itself.

FDA transparency cannot be achieved without FDA predictability.  Predictability is power in pursuit of the public health.

On a related note (and at a different venue – the annual DIA meeting in San Diego), Sandy Kweder (Deputy Director, FDA Office of New Drugs) and Gerald Dal Pan (Director, Office of Surveillance and Epidemiology) discussed the need to begin REMS conversations as early as Phase II. Dal Pan commented that "REMS are as new for us] as they are for all of you. It's not that we hold some secret and we're just not telling anybody about it. We're all navigating through this and trying to figure it out.”   

Looks like the FDA is getting down to some serious social science

According to the Pink Sheet, a study proposed by FDA to evaluate how benefit information is conveyed through direct to consumer advertisements could end up showing drug marketers how to describe "high" and "low" efficacy drugs.

The proposed study will evaluate how consumers' interpretations of a drug's efficacy after viewing DTC ads compare to the efficacy data in the product's label.

The study will be conducted in two concurrent parts - one looking at print ads and one at TV ads - and examine three factors: drug efficacy, visual format, and type of statistic.

The agency defines drug efficacy as a quantifiable, objective metric that can be conveyed in graphical representations of the drug versus placebo, with "high efficacy" meaning "noticeably better than placebo" and "low efficacy" meaning "minimally better than placebo."

The agency traditionally has discouraged companies from making such qualitative claims - or even making too much of quantitative numbers - in its advertising. But the proposed study endpoints suggest a growing FDA comfort with, and even an interest in, consumers receiving this kind of information.

Testing Pfizer-like pictographs

The study will examine graphs, pictographs and pie charts as ways to visually present that efficacy information, and whether statistical information is best conveyed as frequency, relative frequency, or percentage.

The test product will be for a cholesterol treatment and modeled on an actual drug, such as Pfizer's Lipitor (atorvastatin), with labeling used as the reference for defining efficacy levels and the objective metrics for clinical performances.

The study will include 4,500 participants, 2,250 in each group (television or print). The subjects will read or view one advertisement version, and then make a series of judgments about the drug in a 20-minute interview.

FDA first will test whether, within each format, the participants were able to distinguish between low- and high-efficacy drugs. Then, it will look at whether the participants' efficacy estimates differ across formats and how accurate the estimates are.

Comments on the proposed study, called "Experimental Study of Presentation of Quantitative Effectiveness Information to Consumers in Direct-to-Consumer Television and Print Advertisement for Prescription Drugs," are due by Aug. 21.

All to the good.  And it sure beats the ignorant "drug facts box" – the a la nutrition facts panel idea,that’s being kept alive via legislation introduced by Senators Jack Reed (D, RI) and Barbara Mikulski, (D, MD) which, BTW, would also require comparative effectiveness information to be included in product labeling.

Vey izmir.

Roche is leaving the Pharmaceutical Research & Manufacturers of America trade association effective June 30.

Roche describes the action as a "business decision" by "the newly combined Genentech-Roche US commercial organization."

According to the Pink Sheet, "As a practical matter, the departure will add to the strain on PhRMA for resources as it works on all fronts to build alliances and promote its reform agenda. The pending mergers of Pfizer/Wyeth and Merck/Schering-Plough will further reduce the group's core member base. The association traditionally adjusts dues following periods of consolidation."

I recently participated in the 9^th France/USA AGIPHARM Conference.  This year’s event was held in Geneva at the Globe of Science and Innovation, CERN (the European Organization for Nuclear Research).  CERN is best known these days as the location of the particle accelerator used for the creation of the fictional “antimatter bomb” featured in Angels and Demons.

 There were no angels or demons – but there was a fair amount of intellectual acceleration.  Here are a few highlights:

* Edouard Croufer (Director, Healthcare & Chemical Practice, Arthur D. Little), declared that the pharmaceutical industry needed a new model – that Pharma needs to move from “find a molecule and market it” to “identify a need and find a solution.”  When I pointed out to him that the latter was precisely what the industry is currently doing, he … agreed!  Nothing like having the strength of yor convictions. As Henri Poincaré said, “To doubt everything, or, to believe everything, are two equally convenient solutions; both dispense with the necessity of reflection." Croufer also pointed out that 21^st success for medicines would be realized through “speed and intensity.” Amen.

* Francois Meyer (Director, Haute Autorité De Santé) made a rather revolutionary statement for the head of a national reimbursement authority when he stated that “we must move beyond clinical trials” to make true 21^st century healthcare technology assessments.  He also spoke to the need to include outcomes data in reimbursement decisions – or rather in the revisiting of  already-decided reimbursement decisions.  Or as he put it, “comparative efficacy in real life.” Heady stuff.

* Relative to the development of public/private partnerships, Patrice Jaillon (President of GIP CENGEPS – the French National Trial Management Center for Healthcare Products) called for a “climate of confidence and trust.”

* All well and good until Noel Renaudin (President of the CEM – the comité économique du medicament), the Grand Fromage of healthcare reimbursement in France, remarked that the CEM needed to keep industry “on a short leash.”

Particle accelerators are, after all, about high-speed collisions.

Officials said Saturday that the White House, lawmakers and the pharmaceutical industry had struck a deal to have drug companies pay $80 billion to help narrow a gap in coverage in the prescription drug program for Medicare. The officials said that the agreement would have drug companies pay part of the cost of brand name drugs for lower and middle-income older people in the "doughnut hole." That term refers to a feature of the current drug program that requires beneficiaries to pay the entire cost of prescriptions after initial coverage is exhausted but before catastrophic coverage begins.

Obama seized on the deal as evidence that his efforts are gaining momentum.

"Key sectors of the health care industry acknowledge what American families and businesses already know — that the status quo is no longer sustainable," the president said, and noted: "We are at a turning point in America's journey toward health care reform."

So drug and biotech companies are finally being permitted what they wanted to do all along but were banned from doing -- giving seniors a discount to give them a seamless drug benefit.  That makes sense since the decision about what drug to take should be what works, not the price or cost.  At least that is what the evidence says.  And making that decision on clinical evidence leads to value based benefit designs which focus on overall health outcomes...

All to the good.  And the rationale, in part, to Medicare Advantage pay more for prescription drugs. 

But what is Obama proposing to fill the nearly $2 trillion gap to shove 25 million people into  public health plans.

"Obama has proposed cuts in Medicare spending on hospitals, prescription drugs and other services, but a political consensus is far from certain."

He has proposed eliminating Medicare advantage, which has primarliy benefited the chronically ill and minorities...  Leaving them with what?  

So just as the private sector provides seniors with a solution that moves us towards a preventive approach to health care Obama is pillaging the one part of Medicare that provides just that to prop up a health plan that cannot even support paying for preventive care....

The cynicism is breathtaking and the mainstream media is too busy fawning to take note. Real health care reform is within reach but not the way the President wants to take us...

Read article here.