The year ahead: A Capitol bit of crystal ball-gazing

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MM&M asked three healthcare policy experts what 2013 holds in store. Their edited responses follow.

John Kamp
executive director,
Coalition for Healthcare Communication

What provisions of the Affordable Care Act will cause the most disruption—positive or negative?
JK:
Outside of the Sunshine provisions, the ACA has little direct impact on the marketing of medicines. But it intensifies and magnifies all the existing and emerging stresses on our businesses. Most important, it's no longer enough to prove that the drug works well enough to meet FDA standards of safety and effectiveness. Companies must also demonstrate the economic value to both individual patients and to payers to find marketplace success.

Considering the hazy regulations, what will it take for pharma to engage in more digital dialogue with patients?
JK: FDA is just one barrier to aggressive engagement with patients and professionals using internet and social media, but those barriers are breaking down fast. While marketing in the medicines industry often lags behind consumer product marketing in the use of new media and methods, pharma, bio and device companies are learning quickly now.  FDA, too, is smelling the coffee and is likely to weigh in soon with incremental but helpful guidance that will increase the velocity of change.  Most important, professionals and patients are in the space, leaving our industries no choice but to be there or become irrelevant. Meanwhile, let's give a little credit to the daring companies that have both created effective, compliant programs and supported the coalition efforts to move the industry forward.  

Will FDA keep inching the drug approvals window open with the economy improving and executive leadership no longer in doubt?
JK:
One of the lesser known stories of the past 18 months is that the drug approval numbers are moving in the right direction. New drugs and new indications are happening. But, the innovator drugs are coming from smaller companies and have narrower uses than the great blockbusters of earlier days. So, the days of big budgets and broad selling are giving way to more limited budgets aimed at a smaller sector of doctors and patients.

Will the federal appeals court's decision in US vs. Alfred Caronia result in thin-to-no regulation of off-label marketing?
JK:
While Caronia, if upheld, undermines the FDA's ability to declare all off-label claims as per se false and misleading, the decision clearly upholds FDA regulation of false claims. It would be unwise for a regulated company to make any claim it cannot substantiate with significant scientific evidence. Or as one industry leader has said, "In God We Trust, All Others Bring Data!"

Wayne Pines
president of healthcare and regulatory services,
APCO Worldwide, and and a former FDA associate commissioner

What provisions of the Affordable Care Act will cause the most disruption—positive or negative?
WP: I think that transitioning to a new and broadened healthcare system itself will inevitably cause temporary disruption. One specific issue I am concerned about is assuring that the states and private insurance systems do not restrict reimbursement for medically appropriate, off-label uses of treatments. We already are seeing some evidence that restrictions are being imposed. In a different area, as a patient I think we must train enough new physicians to assure ready access to medical care.

Overall, it will be challenging to maintain the quality of health care that we all want and expect as the system adopts new delivery and payment systems. Both patients and the medical community will need to adapt.

Considering the hazy regulations, what will it take for pharma to engage in more digital dialogue with patients?
WP:
I am enthused about the new FDA patient initiative which will bring patients and patient organizations into more direct contact with FDA and pharma, and will bring patient considerations more aggressively into benefit-risk decisions. I think this process will open up a greater dialogue between pharma and patients on serious disease issues. The issue is not whether the dialogue is digital. The industry needs to focus more on content, specifically on disease education.

Will the year bring greater regulatory clarity? Or will FDA, CMS, et al. kick the can down the road again on Sunshine and social media, etc.?
WP: I would anticipate much greater clarity on the Sunshine Act as the government issues regulations  I believe that broader implementation of the law will cause some physicians to limit their interactions with pharma companies.

On social media,  FDA has said it will issue new guidances, but the underlying policies themselves will not change from what they are now. The same basic rules will apply to social media as to other forms of promotion.  Anyone in industry who still expects significant changes in FDA policy will be disappointed. But clarity is absolutely essential. FDA must be much clearer as to what it expects generally in the promotional area.

Will FDA keep inching the drug approvals window open with the economy improving and executive leadership no longer in doubt?
WP: I think the FDA Safety and Innovation Act will add some measure of certainty to the drug development and approval process. I especially look forward to efforts by FDA to enhance communications with pharma during the approval process. FDASIA calls for an initiative in this regard.

The notion that FDA had to play a greater role in de-risking drug development is an important one. The agency needs to provide greater predictability so that investors will feel more comfortable with the process.

Will we see big movement on online privacy regulations? Or elsewhere impacting advertising and promotion?
WP:
I don't see any radical changes coming in 2013 in these areas. Protecting medical privacy remains a universal goal but one that is very challenging.

Regarding advertising and promotion regulation, I also see no major changes in policy or enforcement, as the same people at FDA and, in the case of the False Claims Act settlements, in the Department of Justice will continue in charge. 

Will the federal appeals court's decision in US vs. Alfred Caronia result in thin-to-no regulation of off-label marketing?
WP: We have not yet seen any reaction from the government to the 2nd circuit decision. I think we need to see whether the government appeals. For now, I think companies should not change their usual compliance practices.

Peter Pitts
president, Center for Medicine in the Public Interest,
and a former FDA associate commissioner

What provisions of the Affordable Care Act will cause the most disruption—positive or negative?
PP:
The ACA greases the skids toward government price controls through both the IPAB and the Patient-Centered Outcomes Research Institute (PCORI). Through these two new bodies, Uncle Sam is morphing into not only Uncle Sam MD, but also Uncle Sam as our nation's largest payer, as funder of comparative effectiveness research, as “academic” detailer of what's best for patients, and as determiner of Essential Health Benefits. What's also interesting is what President Obama wants to remove from the ACA—specifically, the 12 years of exclusivity for biologics. Will the President continue to push to roll that number back to seven years? Will he have the chutzpah to claim he is pro-innovation if he does?

Considering the hazy regulations, what will it take for pharma to engage in more digital dialogue with patients?
PP:
Social media for regulated industry is a wonderful green field of opportunity. But to maximize the opportunity, we must accommodate the reality of a messier world. Social media, almost by definition, is messy—and the regulatory framework (or lack thereof) is equally so. And it's not likely to get much better. Get used to it. Embracing social media means embracing regulatory ambiguity. And that's a paradigm shift for an industry that has (in a post-Vioxx world) been going in precisely the opposite direction. It's not going to be easy, or risk-free, or inexpensive. And whatever social media “marketing models” companies build will have to be elastic—just like the media environment in which they are designed to operate. Pharma, guide thyself!

Will the year bring greater regulatory clarity? Or will FDA, CMS, et al. kick the can down the road again on Sunshine and social media, etc.?
PP
: Per social media, see above. The Sunshine Act is a mess. First, HHS will have to determine how various state laws will impact one federal statute. Will the administration invoke federal preemption? And, if so, what will they put in its place? Also, why aren't payments to physicians from insurance companions included in the Sunshine Act? This will be debated and will further delay (and appropriately so) swift implementation. When it comes to the Sunshine Act, it's best to measure it twice and cut it once.

Will FDA keep inching the drug approvals window open with the economy improving and executive leadership no longer in doubt?
PP:
Will she stay or will she go?  I predict that Commissioner Hamburg will stay. That's a good thing because what moves drug approvals ahead is predictability and that starts at the top. The solution is to make the regulatory process more predictable.

A quarter-century ago, the success rate for a new drug used was about 14%. Today, a new medicinal compound entering Phase I testing—often after more than a decade of preclinical screening and evaluation—is estimated to have only an 8% chance of reaching the market. For very innovative and unproven technologies, the probability of a product's ability to make it to the market is even lower. We must work together to turn that around. When Thomas Edison was asked why he was so successful, he responded, “Because I fail so much faster than everyone else.” Consider the implications if the FDA could help companies fail faster.

Using the lower end of the Tufts University estimate of the average pre-tax cost of new drug development, $802 million:
•    A 10% improvement in predicting failure before clinical trials could save $100 million in development costs.
•    Shifting 5% of clinical failures to Phase I, the earliest stage, from Phase III, the latest stage, reduces out-of-pocket costs for developers by $15-$20 million.
•    Shifting of failures to Phase I from Phase II, the middle stage, would reduce their out-of-pocket costs by $12-$21 million.

All of these dollars could then be reinvested in other innovative development programs for new life-saving medicines.

For all that modern science has to offer, developing new treatments is still very much an art, in which hunches, intuition, and luck play a critical role. The odds are long. But for more medicine that is affordable and innovative, we need up-to-date regulations that complement the drug trial process in order to take these chances, which is precisely the mission of the FDA's Reagan-Udall Foundation.

Will we see big movement on online privacy regulations? Or elsewhere impacting advertising and promotion?
PP:
Just because DDMAC got a “promotion” to OPDP doesn't mean that (1) they have any more resources (they do not) or authority. The FDA must still abide by the First Amendment, and they are acutely aware of the limitations that that places on their regulation of industry speech. That's one of the reasons they have been so prudent in their statements and guidances regarding social media. They will continue to address advertising and promotion with one major goal in mind—to make sure industry efforts are neither false or misleading. Alas—the truth is that the definition of those terms is often in the eyes of the beholder.

Will the federal appeals court's decision in US vs. Alfred Caronia result in thin-to-no regulation of off-label marketing?
PP:
When it comes to Caronia, where you stand depends on where you sit.

For industry, the decision opens up tremendous potential for enhanced (but restrained and responsible) sharing of important scientific data. For the FDA (understandably) the decision was the Nightmare Before Christmas. The question is, do the opportunities outweigh the risks? There are a few ways to approach this.

There's the First Amendment question. While I can't predict which way a Federal Appeals court will rule, I will certainly predict that 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.

Another way to look at it is 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.

The third, most important and most contentious angle is to consider whether or not such relaxed communications approaches would actually advance the public health. Would industry (on one side) and the FDA (on the other) answer this differently? I'm not so sure.

All that being said, whether or not the Appeals Court overrules Caronia, it is likely to offer a stern rebuke to the FDA for its unpredictable, ambiguous and sometimes capricious application of off-label speech constraints.

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