Getting schooled on drug pricing

09/04/2014 07:26 AM |

Thought provoking lead story in this week’s edition of BioCentury, Back to School Issue: Paying the piper. Here’s the challenge:

Pharma has lost its pricing power in many countries, as evidenced by reimbursement authorities' willingness to delay or outright deny access to drugs whose costs are deemed unacceptable. Now, the availability of a costly drug in the U.S. that could be given to millions of people has sparked the strongest backlash against drug pricing the industry has yet faced - in the last major market where the government has not adopted any form of drug price controls, according to the U.S. Department of Commerce.

Last year, in "Facing Reality," Back to School argued biopharma companies can no longer assume the market will support premium pricing, even for drugs that deliver meaningful and measurable improvements over the standard of care.

This year, BioCentury's 22nd Back to School essay goes on to argue that the last bastion of free pricing is crumbling, and biotech and pharma had better start experimenting with new pricing models based on value for money while they still have the chance.

And here’s the conclusion:

Back to School does not suggest drug pricing and reimbursement can be fixed easily, and certainly not by the drug industry on its own. Finding approaches that will get new and better medicines to patients sooner, that compensate companies for the health benefits their drugs provide and that don't break the bank will come only through vigorous and collaborative experimentation.

Nevertheless, unless it pursues experiments with the explicit goal of creating a win-win for payers and patients as well, the drug industry can expect controls on prices and utilization to be applied with indiscriminate force in markets worldwide.

Biopharma's brightest minds are hard at work discovering and developing breakthrough medicines. It will be a pitiful shame if patients are denied access because the industry's brightest marketing minds are not creative enough to devise models that will enable healthcare systems to pay for these transformations in healthcare.

What industry needs are brave individual first-movers to get to work on new pricing models that will preempt a cost-plus system and preserve incentives for innovation.

Back to School has described three places where drug companies can blaze the trail.

First, they should pioneer value-based approaches that wed drug prices to the patient- and payer-defined value of outcomes, rather than to the volume of drugs consumed or, in the emerging worst case scenario, the costs to develop and produce them.

Second, drug companies should take the lead on making risk-sharing a reality, not just a catch-phrase for discounts and rebates, and make investments in the kinds of enabling systems that can support appropriate use and reimbursement of medicines, as well as inform development of tomorrow's innovative drugs.

Third, drug companies should spearhead the development of payment models that enable health systems to absorb the cost of cures by enabling payment over the period in which benefits accrue, as long as the drugs continue to work.

As has been the case with Back to School's recommendations in years past, these experiments will require robust collaboration with unfamiliar and even hostile partners. Many will fail.

But the reward for taking those risks will be a menu of pricing and reimbursement options that ensure companies are compensated for the value of both incremental and breakthrough innovations, and that drive revenue and profit by extending access to a bigger pool of patients.

The entire article (and it is definitely worth a read) can be found here.

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Raising the stakes for fakes

09/03/2014 09:31 AM |

Via Fierce Pharma – and they buried the lead

EU agents seize more than €10M worth of counterfeit drugs, arrest 12

Authorities from 8 European Union countries, backed by Europol and Eurojust, have arrested a dozen suspects and seized more than €10 million worth of counterfeit drugs, mostly fakes of erectile dysfunction drugs that an organized crime group was selling online. The raid, announced Monday, came after an investigation that lasted nearly two years.

According to Europol, law enforcement folks from Austria, Belgium, Cyprus, France, Hungary, Slovakia, Spain and the United Kingdom conducted simultaneous raids. They were backed up by Europol and Eurojust. In addition to seizing several million pills, which it estimated were worth well in excess of €10 million, the agents seized cash and luxury cars and froze bank accounts that contained €7.5 million.

The investigation originated in Spain, which was able to provide information for probes in Austria, France and the U.K., Europol said in a statement. It said the fakes originated in Asia, home to many of the counterfeit drug operations.

While erectile dysfunction drugs have long been a target of counterfeiters selling online, Europe is also seeing organized crime move into stealing and faking high-priced drugs, like cancer meds. Authorities this spring warned that vials of Roche's  cancer med Herceptin that had been stolen in Italy were showing up across the continent with little or none of its active ingredient.

Because some of the organized crime rings behind counterfeits are based in Europe, the FDA has assigned one of its criminal investigation officers to Europol in The Netherlands. The FDA estimates that 40,000 to 60,000 domain names could be tied to illegal online pharmacies at any given time, and that this number is in a constant state of flux.

Not mentioned in the article – but of relevance – is that in 2013 US National Association of Boards of Pharmacy found that only 257 of 10421 online pharmacies are legally legitimate businesses (less than 2.5%).  The remainder either have bogus registration credentials or domain names that make them “suspect.”

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The ultimate in regulated speech

09/02/2014 12:34 PM |

Looking for a great job? How about Director of Communications for CDER?

Here are the details:

FDA's Center, for Drug Evaluation and Research (CDER) is searching for exceptional candidates for the position of Director of the Office of Communications (OCOMM). The Office currently has over 100 employees. OCOMM is the central hub for communication expertise, in CDER, focused on the development of consistent messaging to inform and educate the multiple audiences. The Office has a variety of responsibilities including the planning, coordination and evaluation of the policies, procedures, programs in the strategic outreach and communication about drug-related requests.

The incumbent serves as Director, Office of Communications (OCOMM) for the Center for Drug Evaluation and Research (CDER). The Director provides leadership and direction for all Center internal/external communications. The Director is responsible for the creation of a climate for cooperative work relations, and support and understanding of the CDER program objectives. Additionally, the Office of Communications Director advises and counsels the Center Director and CDER leadership on external and internal communications relative to the exchange of information and is the liaison external groups.

Qualifications:

Applicants should possess an advanced degree in Communications, Marketing, Public Relations, or Public Affairs.

Successful candidates are those that have experience working closely with highly-credentialed people. They must have substantial experience in Communications, Marketing, Public Relations, and/or Public Affairs. Knowledge of pharmaceuticals is a plus. The candidate should be persuasive, influential, and have the ability to ask the right questions.

Location: Silver Spring, Maryland

Salary: GS-15, $124,995-157,100 Salary is commensurate with qualifications and experience. A full Federal benefits package is also available including: leave, health and life insurance, retirement, long term care insurance, and Thrift Savings Plan (401K equivalent).

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FDA: Let's talk about pain

09/01/2014 11:23 AM |

Much chatter about a pending FR notice announcing an FDA public meeting on pain medications. Good idea or bad idea?

Well, as the Beltway saying goes, where you stand depends on where you sit. On the one hand there’s the side of science and the public health. Is open public debate useful? Absolutely. And timely.

On the other there are those with less than altruistic interests. The tort bar for one and ambitious politicians for another.

What does “success” look like? For the FDA and like-minded public health advocates, success means advancing safer and more safely used pain medications. For learning and accelerating applied science.

For others it means headlines and a hefty payday.

Which story is more media-friendly? If you don't know the answer to that one, do some internet research on the vaccine/autism link and the debate over SSRIs and teen suicidal ideation.

(Hint – science doesn’t win media inches.)

Who will testify at the FDA meeting? Who will serve on the expert panels? Who will the FDA participants be?

It’s good news that the session will not be an advisory committee. No votes are likely to be taken. Nor will it probably be a Part 15 affair where only “listening” is required of the agency.

It’s FDA leading – but the devil is in the details.

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Nomenclatura

08/28/2014 08:49 AM |

If you're looking for some light beach reading over the long weekend, may I recommend the lastest Policy Forum paper from the Food Drug and Law Institute (FDLI).  The title, Biosimilar Nomenclature: Can We Achieve the Truth, the WHole Truth, & Nothing but the Truth? says it all.

Enoy the paper -- and have a great Labor Day.  Read More & Comment...

Scheduling Scheduling

08/27/2014 09:10 AM |

The House is expected to vote on The Improving Regulatory Transparency for New Medical Therapies Act (H.R. 4299). (It has already been approved by the Energy & Commerce Committee.) Companion legislation is expected to be introduced in the Senate soon. The legislation would strengthen the Controlled Substances Act by requiring the DEA to “schedule” medicines 45 days after it receives the FDA’s scheduling recommendation for a new drug.

(The average time between FDA approval and DEA’s final scheduling is about eight months -- and it sometimes takes more than a year.)

The issue at hand is that patients have not been able to access a growing number of new treatments because of a lack of predictable schedule review times – and particularly long evaluation times associated with DEA scheduling decisions.

It’s time for that to change.

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In sync? Not entirely.

08/26/2014 07:00 AM |

The California Assembly just passed (by a vote of 72-0) AB2418, legislation for medication synchronization that “promotes policies designed to improve patient medication adherence.” An earlier draft of the bill included measures that would have given about one million Californians whose health plans require them to order prescriptions through the mail an option to waive that requirement -- but some strong business interests threw a fit. Fortunately, the bill does specifically addresses pharmacy synchronization, a tried and true methodology for significantly improving medication compliance. It now heads to the Governor’s desk.

It should be a no-brainer for Governor Brown -- but the Assembly’s unanimity isn’t as complete as the vote makes it look.

The mail order measure was strongly supported bya wide swath of patient groups, seniors’ organizations, provider associations, labor unions, pharmacies, and business support. Alas, it was killed by the profits over patients crowd consisting of Aetna, American’s Health Insurance Plans, Association of California Life and Health Insurance Companies, Blue Shield of California, California Association of Health Plans, Express Scripts, and the Pharmaceutical Care Management Association.

Their rationale: mail order is cheaper – at least in the short-term. The Pharmaceutical Care Management Association has launched a campaign against the bill that includes the release of a study finding that mail-order pharmacies save about 16% more than brick-and-mortar pharmacies, including $500 million in savings next year on regular prescription medications. However, the study also noted that stand-alone prescription drugs often were more costly through mail-order pharmacies.

According to the U.S. Centers for Medicare and Medicaid Services (CMS), the estimated cost of synchronization to Part D sponsors is meager $0.5million, while the savings to Part D sponsors and beneficiaries is $1.8 billion. And there will be an overwhelming reduction in overall health care costs because of improved patient outcomes due to enhanced medication adherence, which should lead to higher quality ratings for a health insurance plan.

The Assembly vote notwithstanding, Governor Brown’s signature on the bill isn’t assured. And the lobbying is intense. The health plans and PBMs mentioned above are still strongly opposed and actively lobbying for a veto

Governor Brown’s signature to AB 2418 will result in better health and lower costs.

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Facts, Damn Facts, and Opioids

08/25/2014 09:38 AM |

The New York Times reports that, The City of Chicago and two California counties are challenging the drug industry’s way of doing business, contending in two separate lawsuits that “aggressive marketing” by five companies has fueled an epidemic of addiction and cost taxpayers millions of dollars in insurance claims and other health care costs.

Do they know more than OPDP? Have they contacted the FDA? Do they have legal standing or is this a case of Federal Preemption? None of these questions are addressed in the article.

Also left unmentioned is that Purdue Pharma (mentioned at great length in the Times article as a defendant), is working with the U.S. Attorney's Office, Central District of California to help convict felonious pharmacists who operated an opioid diversion ring.

As Aldous Huxley famously said, “Facts do not cease to exist because they are ignored.”

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Opioid Upscheduling, the Precautionary Principle, and the Law of Unintended Consequences

08/22/2014 07:15 AM |

Joshua Lederberg, the Nobel Prize Laureate once observed that the failure of regulatory, legal and political institutions to integrate scientific advances into risk selection and assessment was the most important barrier to improved public health.  

Lederberg noted that in the absence of such changes,  "the precedents affecting the long-term rationale of social policy will be set, not on the basis of well-debated principles, but on the accidents of the first advertised examples."

Policies and regulations that seek to limit risk are often shaped by the immediate fear of sensational events. This perspective is commonly called "The Precautionary Principle" which in various forms asserts that unless innovators can demonstrate that a new technology is risk free, it should be not allowed into the marketplace.  Moreover, any product that could possibly be dangerous at any level should be strictly and severely regulated. 

Which brings us to yesterday’s announcement that the federal government (led by the DEA) is finalizing new restrictions on hundreds of medicines containing hydrocodone, the highly addictive painkiller that has grown into the most widely prescribed drug in the U.S.

The new rules mean that drugs like Vicodin, Lortab and other generic versions will be subject to the same prescribing rules as painkillers like codeine and oxycodone. Patients will be limited to one 90-day supply of medication and will have to see a health care professional to get a refill. Additionally, in many states prescribing authority will be limited to physicians, not nurses or physician assistants.

Will this limit abuse? That’s a theory. What's a fact is the negative impact it will have on the tens of millions of Americans who suffer from chronic pain.

Last September, CMPI held a conference on the issue of opioid pain medications. One of the panelists was Cindy Steinberg, the National Director of Policy and Advocacy for the US Pain Foundation. Here’s what she had to say about upscheduling:

That’s going to mean that people need to see their doctor at least four and sometimes 12 times a year just to obtain a script because of the very strict requirements on Schedule II medications. The hydrocodone combinations are now Schedule III and if they are upscheduled, in Massachusetts for example, where Schedule II scripts expire in 30 days and cannot be written for more than a 30 day supply, many people in my group are that are taking hydrocodone combinations medications would have to see their doctor every 30 days for a script.

They now see their doctor perhaps twice a year. So, they would have to go see their doctor 12 times per year. People have a hard time finding a pain doctor period, let alone getting an appointment.

Somebody in my group was referred to a pain specialist in January of 2012 and the first appointment she was able to get was in December of 2012. She had to wait almost an entire year to see a pain doctor. So how are we going to handle the number of people that are going to need appointments to get their medications? In Massachusetts, as I mentioned Schedule II scripts expire in 30 days and by federal law cannot be refilled. I have a person in my group that has osteonecrosis. He was an early heart transplant patient and the steroids he had to take to maintain his organ transplant resulted in osteonecrosis which has eroded every joint in his body. He’s had multiple joint replacements and lives with a very high degree of pain. His wife has to drive him to the doctor now every 30 days in order to get his Schedule II medication because he needs to get a physical script. And that’s going to happen to many people if these Schedule III medications are rescheduled. And as I mentioned, given all those extra appointments, think about what that is going to do to healthcare costs.

Upscheduling will result in tighter restrictions for patients who really need the medications, more paperwork for physicians and a heavier workload for pharmacists. Abusers and criminals rarely follow regulations.   Read More & Comment...

openFDA and Open Sesame on 21st Century Pharmacovigilance

08/20/2014 08:50 AM |

This past June, the FDA launched openFDA, a new initiative designed to make it easier for web developers, researchers and the public to access large, important public health datasets collected by the agency.

According to the agency, the initiative is the result of extensive research with internal officials and external developers to identify those datasets that are in recurrent demand and are traditionally fairly difficult to use. Based on this research, the FDA decided to phase in openFDA beginning with an initial pilot program involving the millions of reports of drug adverse events and medication errors that have been submitted to the FDA from 2004 to 2013. Previously, the data was only available through difficult to use reports or Freedom of Information Act requests.

And this week the FDA unveiled a new Application Programming Interface that offers data from the agency’s database containing medical-device adverse event reports, going back to 1992.

The crowd-sourcing of adverse event data may or may not yield interesting results, but it’s a good place to start. It represents an opportunity for the agency to begin designing a more evolved approach to 21^st century pharmacovigilance.

For the rest of the story see OpenFDA: The Key To Moving Pharmacovigilance Into the 21st Century in the new edition of PM360.
 

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Silver is the new Black

08/19/2014 07:21 AM |

Referring to the Model T, Henry Ford famously said, "Any customer can have a car painted any color that he wants so long as it is black.” That worked out fine – until there was competition. Choice is the great emancipator. The same is true when it comes to healthcare – and a lot more important.

When it comes to the Affordable Care Act, patients can access any medicine they need -- as long as it's on the exchange formulary. Sure, the ACA limits the degree to which insurers can charge higher premiums for sicker patients, but ObamaCare plans found a way around these rules: impose higher out-of-pocket costs for all or most specialty drugs. High co-pays effectively remove choice from the system for many patients.

The breakdown of Silver plans (the most popular category) is particularly revealing. In seven classes of drugs for conditions from cancer to bipolar disorder, more than a fifth of these plans require patients to shoulder 40 percent of the medicine’s cost. And 60 percent of Silver plans place all drugs for illnesses like multiple sclerosis and rheumatoid arthritis in the “formulary tier” with the highest level of cost-sharing.

Nearly every Silver plan across the country, in fact, puts at least one class of drug exclusively in the top cost-sharing tier. In effect, this leaves patients with a given condition — whether HIV or Crohn’s disease — without a single affordable treatment option. Silver is the new Black.

And those signing up for Silver plans don’t know what’s going to hit them until they access the healthcare system. It’s time, at least, for that to change. It’s time for exchange transparency.

The American Legislative Exchange Council (a forum for state legislators and private sector members to collaborate on model legislation that members can customize and introduce for debate in their own state legislatures) has drafted the “Exchange Transparency Act.” Whatever your position on ObamaCare (or, if you prefer, the Affordable Care Act), it makes a lot of sense. If there’s nothing to hide then there shouldn’t be a problem.

Exchange Transparency Act

Summary

Requires health plans offered through a state-based health exchange to provide specific information in order for consumers to draw meaningful comparisons between plans.

Model Policy

Section 1. Title. This Act shall be known as the “Exchange Transparency Act.”

Section 2. Form of Information Available to the Public and Disclosures Required of Health

Insurers. The following information about each health plan offered for sale to consumers shall be available to consumers on {insert state-based exchange website} in a clear and understandable form for use in comparing plans, plan coverage, and plan premiums:

 (1) The ability to determine whether specific types of specialists are in network and to determine whether a named physician, hospital or other health care provider is in network;

 (2) Any exclusions from coverage and any restrictions on use or quantity of covered items and services in each category of benefits;

 (3) A description of how medications will specifically be included in or excluded from the deductible, including a description of out-of-pocket costs that may not apply to the deductible for a medication;

 (4) The specific dollar amount of any copay or percentage coinsurance for each item or service;

 (5) The ability to determine whether a specific drug is available on formulary, the applicable cost-sharing requirement, whether a specific drug is covered when furnished by a physician or clinic, and any clinical prerequisites or authorization requirements for coverage of a drug;

(6) The process for a patient to obtain reversal of a health plan decision where an item or service prescribed or ordered by the treating physician has been denied; and

(7) An explanation of the amount of coverage for out of network providers or non-covered services, and any rights of appeal that exist when out of network providers or non-covered services are medically necessary.

Section 3. Enforcement. The {insert state insurance commissioner} may impose fines on any entity failing to meet the requirements of this act.

What’s the ETA of the ETA? Stay tuned.

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Leaders Lead

08/18/2014 09:05 AM |

FDA Should Lead Ongoing Opioid Debates, Former Agency Official Says

FDA Week                                                                                                                                         
By Stephanie Beasley 

Former FDA Associate Commissioner for External Relations Peter Pitts said the agency has been too quiet in escalating debates about opioid regulation and has missed recent opportunities to explain the science behind its decisions. FDA should be the lead voice on the issue of abuse deterrence and safe use of opioids but instead the conversation has been dominated by state and federal lawmakers, lawyers and advocacy groups, said Pitts, now president of the Center for Medicine in the Public Interest.

In "Who 'Lost' Opioids?" -- an article that appears in the July issue of the Journal of Commercial Biotechnology -- Pitts said FDA is losing the "struggle" to control the national conversation about opioid abuse. He said lawmakers, state attorneys general, medical groups and others have pushed FDA to reverse it's recent approval of pure hydrocodone Zohydro and for the agency to require all opioids be abuse deterrent. But the agency's scientific basis for not taking those actions has been overlooked, he said.

He notes that during testimony in the Senate earlier this year, FDA Commissioner Margaret Hamburg indicated that the agency would be reluctant to require all opioids use abuse-deterrent formulations until there is more evidence to prove they actually deter abuse. Further, he notes that while many critics of FDA's Zohydro approval have correctly cited the fact that the decision was made against the recommendation of an advisory committee, they failed to mention that the experts also affirmed there was no evidence suggesting Zohydro had greater abuse or addiction potential than other opioids.

FDA needs to bring more attention to these factors and talk about what it is doing to progress abuse deterrent opioid development, he said. "The FDA needs to continue to speak out regularly on what it is doing to help further the initiatives that it has put in place," Pitts told FDA Week. "The FDA has many, many important issues to address and opioids is only one of them, but this doesn't excuse either inaction or lack of communications. Leaders lead."

He said, for example, FDA could have spoken about progress made on abuse deterrence when it approved Purdue Pharma's abuse-deterrent oxycodone last month (see FDA Week, July 24). That was a missed opportunity to talk about the significance that kind of drug might have on the development of abuse-deterrent formulations, Pitts said. He added that FDA has also been meeting with opioid manufacturers to address issues related to abuse deterrence but has provided no information about what solutions have been proposed or when they might be implemented.

In September, FDA updated the Risk Evaluation and Mitigation Strategy for extended-release opioids to require a statement that the products were appropriate for pain severe enough to require daily and continual long-term treatment, among other changes. The agency also issued guidance on abuse deterrence last year, but has yet to release a separate guidance for abuse-deterrent generics, although agency officials have said they do not plan to require generics use the same technology as innovators.

Pitts was also critical of lawmakers that have pressed the agency to reverse its Zohydro approval. Democratic Sens. Joe Manchin (WV) and Charles Schumer (NY) have been active on the issue. Manchin introduced a bill that would reverse the approval while Schumer has urged HHS to overturn the decision. Twenty-eight state attorneys general have also called for FDA to reverse its Zohydro approval.

"Whatever your position on the issue of opioids, the proper venue for this decision is not the office of the Secretary of HHS or the halls of Congress or the courts -- but rather the office of the FDA Commissioner," Pitts said.

He also took issue with groups like Consumers Union that are weighing in on the issue but that he said were ignoring the science behind FDA policy. Last month, CU's Consumer Reports released an article warning consumers about the dangers of painkillers and specifically asking FDA to reconsider its Zohydro approval and limit acetaminophen to 325 milligrams per pill. The group further said that while 90 percent of long-term chronic pain sufferers are prescribed opioids, there is little evidence that the drugs are beneficial or safe for long-term use. It is also safer to use short-acting opioids that stay in the body for less time and avoid taking large doses of acetaminophen, according to the article.

Pitts called the article "error filled" and criticized Consumer Reports for misrepresenting the information as fact-based and non-biased. But Lisa Gill, lead editor for the article, said the group stood by its story. It is important to remember that Consumer Reports is coming at the topic from a consumer, not regulatory perspective, she said. Gill added that the report was also released in direct response to Centers for Disease Control and Prevention data identifying opioid misuse and abuse as a public health crisis.

"The story evolved out of the CDC calling opioid abuse a public health crisis," Gill said. "Because it is still an issue we wanted to cover it in a profound way."

She said the point of the article was to address common misconceptions among consumers and help them make healthcare decisions and also noted that Consumer Reports does feel FDA could be doing more to address opioid abuse, like requiring prescriber education for providers prescribing Zohydro.

Pitts also called for more education on opioid dispensing and the development of best practices. Those best practices could be developed by continuing medical education groups and prescription drug monitoring programs, he said.

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A Sheep in Wolfe's Clothing

08/15/2014 01:07 PM |

In an outrageous commentary in the British Medical Journal, Sid Wolfe cites a JAMA study that claims “new black-box warnings and safety withdrawals have increased following PDUFA’s enactment, perhaps as a result of an expedited review process that may not adequately detect serious drug safety problems in the preapproval period.”

Statistics, the saying goes, are like a bikini. What they show you is interesting, but what they conceal is essential. In the case of Dr. Wolfe, it’s a case (in fact, the latest in a series) of taking evidence and selectively using it to prove a long-held theory. In the case of Sid Wolfe, the theory is that PDUFA puts FDA in industry’s pocket. Nothing could be further from the truth.

Much has changed since the introduction of user fees in 1992 and one of the most important changes has been in the medical innovation. Since 1992 both small and large molecules have become more complex. Since 1992 these new medicines have addressed the unmet medical needs of many orphan and serious chronic diseases.

But new drugs are more than about just reward. Many of these new FDA-approved medicines have a higher risk profile. And with better data management tools, the FDA is now able to capture adverse event information in a more timely and accurate manner. This is especially important when it comes to the approval of medicines with a higher risk profile. Post-marketing pharmacovigilance, whether in the form of more targeted REMS or more sophisticated surveillance techniques allows the FDA to pursue expedited approval pathways for those medicines it feels fill a void in the therapeutic armamentarium. The voice of patients supports this approach, as does that or practitioners. And it also supports innovation.

As Paul J. Seligman, former chief of post-marketing drug surveillance at the FDA, commented back in 2005, it’s important to “develop the science for monitoring adverse events in ways that will allow us to give adequate warnings.”

No pharmaceutical company wants its product brought to market more swiftly if that will lead to a rapid recall. The fact that there are more products with boxed warnings is a direct consequence of the FDA’s efforts to better inform physicians and patients to the risk/reward ratio of these new products. It’s 21^st century safe use or, as the French refer to it, bon usage. In that respect, more product withdrawals are the natural consequence of better pharmacoviiglance – the counterweight to expedited approvals of higher risk medicines.

And nothing to do with PDUFA fees.   Read More & Comment...

Fragile Fraternity

08/14/2014 11:55 AM |

So much for regulatory fraternity …

PhRMA, BIO Open Fire On FDA's Biosimilars Guidance

By Jeff Overley

Law360, New York (August 13, 2014, 7:11 PM ET) -- Pharmaceutical Research and Manufacturers of America and the Biotechnology Industry Association are leading an attack by innovator drugmakers on the U.S. Food and Drug Administration’s most recent biosimilars guidance, saying in letters released Wednesday that it could let copycat products be sold without fully demonstrating safety and effectiveness.

In correspondence provided to Law360 following a Tuesday comment deadline, the trade groups each questioned biosimilars policies the FDA proposed in May regarding so-called clinical pharmacology data — relatively early research in small groups of human subjects.

PhRMA, for example, took issue with the FDA’s introduction of four categories of similarity — not similar, similar, highly similar, and highly similar with fingerprint-like similarity — saying that the “hierarchy is beyond the scope of the draft guidance.”

If the FDA insists on preserving the categories within the guidance, it should eliminate “vague and confusing” definitions that make it unclear how regulators will decide which classification to apply, PhRMA wrote.

BIO also voiced concerns with the four categories, specifically questioning the FDA’s statement that a product merely deemed similar — and therefore not comparable enough to qualify as a biosimilar — could be deemed highly similar if additional studies help to erase uncertainties.

That additional testing could “expose human subjects to an experimental therapy that had not met the statutory analytical threshold of ‘highly similar,’” BIO wrote.

Both organizations have members that are pursuing biosimilars, but they mainly focus on brand-name products, including branded biologics that stand to lose significant market share when biosimilars arrive on the scene. Any delays in finalizing FDA policies on biosimilars therefore may work to their advantage.

PhRMA’s letter also suggested that the guidance is murky with respect to when clinical pharmacology data is sufficient to establish biosimilarity. For example, at one point in the guidance, the FDA said that such data “may be sufficient to completely assess clinically meaningful differences between products,” PhRMA noted.

But the guidance also said that clinical pharmacology studies, “if done well,” can complement other data and guide future testing.

“Based on these passages, it is unclear to PhRMA when FDA believes that clinical pharmacology studies can constitute the full clinical assessment,” the organization wrote.

PhRMA and BIO also sounded alarms over the FDA’s approach to safety, specifically the issue of immunogenicity, which can refer to immune system responses that cause allergic reactions or alter a biologic’s effects. The FDA’s guidance said that such data may need to be supplemented by findings from post-approval studies, and both groups expressed concern. 

BIO, for example, wrote that “full evaluation of safety and immunogenicity should still be necessary before approval” and that the FDA should elaborate on the role of post-approval studies in demonstrating safety.

Also, BIO asked the FDA to better explain its suggestion that biosimilarity may be established by looking at a single, scientifically acceptable biomarker — say, blood pressure or cholesterol levels — or at a composite of multiple biomarkers that are "relevant."

“While this approach may have utility, it runs the risk of merely increasing the quantity of data without necessarily improving the quality and interpretability of the results,” BIO wrote.

Swiss drugmaker Roche AG — parent of U.S.-based Genentech Inc. and a member of BIO but not PhRMA — also submitted a letter seeking more clarity on the four categories of similarity and complaining that the guidance covers more than just clinical pharmacology data.

According to a public docket, two dozen letters have been submitted on the guidance, but as of Wednesday, the FDA had published only four of them. Those letters were from Roche, the United States Pharmacopeial Convention, an independent board established by Amgen Inc., and generics maker Apotex Inc., which posed five questions but little in the way of criticism.

Sandoz Inc., the first company to request FDA approval along the Affordable Care Act's biosimilar pathway, didn’t immediately respond to a request for a copy of any comments it submitted. Hospira Inc., which is also pursuing biosimilars, directed an inquiry to the Generic Pharmaceutical Association, which did not immediately have comments to share.


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But other than that, Mrs. Lincoln, how did you like the play?

08/13/2014 11:06 AM |

As seen in The Hill.

Wanted: Better fact checkers at Consumer Reports

The national public debate over the misuse and abuse of opioid pain medicines has been in the news long enough now for state and federal lawmakers and regulators at the Food and Drug Administration to focus on the real issues rather than the tabloid headlines.

That’s why the latest story on the subject from Consumers Report is so puzzling. Not only is it a day late and a dollar short, but it reaches all the wrong conclusions – providing a distinct disservice to its readers.

The CR cover featuring the story screams, “Deadly Pain Pills!” But the only thing deadly is the reporting which is both hyperbolic and filled with obvious errors and selective omissions.

Specifically, CR has the pain medicine Zohydro ER in its crosshairs. The article calls on the FDA to reconsider its 2013 approval of Zohydro ER and to make acetaminophen standards consistent.

Strangely the piece fails to mention that one of the benefits of Zohydro ER is that it is acetaminophen-free. It also neglects to mention that this very concern was specifically addressed by FDA Commissioner Margaret Hamburg who testified before Congress that,  “We recognize that this is a powerful drug, but we also believe that if appropriately used, it serves an important and unique niche with respect to pain medication and it meets the standards for safety and efficacy.”

The investigative tigers at CR call for the FDA to approve only opioids that are “abuse deterrent.” Well, here’s what the FDA commissioner had to say on that subject (also in open public testimony that the CR story either missed or chose to ignore), “It doesn’t do any good to label something as abuse deterrent if it isn’t actually abuse deterrent, and right now, unfortunately, the technology is poor.”

Surprisingly absent from the CR story was any mention of the most promising of the FDA’s initiatives on abuse deterrence; a study (to be conducted by the National Institute for Pharmaceutical Technology and Education) to evaluate opioid product formulations and performance characteristics for solid and oral dosages. 

Unfortunately complex systems make for bad media coverage, while simplistic, dramatic demagoguing makes for sexier headlines.

Oops.

CR also believes another reason Zohydro ER should be recalled by the FDA is that it was approved, “against the recommendation of its own panel of expert advisers.” That’s true – but it’s not the whole truth.  What the CR authors left out is that, by a vote of 11-2, the experts affirmed that there was no evidence to suggest Zohydro had greater abuse or addiction potential than any other opioid.

“Facts,” as John Adams said, “are pesky things.”

What the newshounds at CR missed completely are the issues surrounding opioid misuse – at present the poor public health stepchild of abuse. In the United States, the use of opioids as first-line treatment for chronic pain conditions doesn’t follow either label indications or guideline recommendations.

In fact, 52 percent of patients diagnosed with osteoarthritis receive an opioid pain medicine from their doctors as first-line treatment as do 43 percent of patients diagnosed with Fibromyalgia and 42 percent of patients with diabetic peripheral neuropathy.

Payers in the healthcare system often impose barriers to the use of branded, on-label non-opioid pain medicines, relegating these treatments to second line options. The result is a gateway to abuse and addiction with opioids.

Another fact conveniently missed in the CR story is that the vast majority of people who use opioids do so legally and safely. A subset, approximately four percent of patients, use these medications illegally. In fact, from 2010 to 2011, the number of Americans misusing and abusing opioid medications declined from 4.6  to 4.2 percent.

How strange that Consumer Reports missed so many facts after all this time.

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Debased Silver

08/11/2014 06:41 AM |

From today’s edition of the New York Post …

From HIV to cancer to Crone’s, ObamaCare fails the sick

By Peter J. Pitts

Melanie Thompson and HIV patient Brian Albright look over his medical bills and correspondence with his insurance company in Atlanta. Photo: AP

It turns out ObamaCare didn’t solve the problem of “pre-existing conditions” after all. It made premiums more affordable for people with chronic health conditions that are expensive to treat — but at the price of sticking them with unaffordable co-payments for their medications.

The nonprofit AIDS Institute is suing four Florida health insurers for discriminating against HIV/AIDS patients. The complaint says these patients now face prohibitive out-of-pocket drug costs. Sadly, most of the plans sold via ObamaCare all across the country have similar problems — leaving those with chronic diseases without affordable access to the specialty drugs they need.

The Affordable Care Act limits the degree to which insurers can charge higher premiums for sicker patients. But ObamaCare plans found a way around these rules: impose higher out-of-pocket costs for all or most specialty drugs.

Consider the Florida suit. Carl Schmid, the AIDS Institute deputy executive director, says the plans follow a “pattern where every single [HIV/AIDS] drug for some plans was on the highest tier, including generics.” Under these policies, drug costs for AIDS patients can exceed $1,000 a month.

And Florida’s hardly unusual. A new report from consulting group Avalere Health found that a large majority of all ObamaCare exchange plans include similarly high out-of-pocket costs for patients with certain illnesses.

The breakdown of Silver plans (the most popular category) is particularly revealing. In seven classes of drugs for conditions from cancer to bipolar disorder, more than a fifth of these plans require patients to shoulder 40 percent of the medicine’s cost.

And 60 percent of Silver plans place all drugs for illnesses like multiple sclerosis and rheumatoid arthritis in the “formulary tier” with the highest level of cost-sharing.

Nearly every Silver plan across the country, in fact, puts at least one class of drug exclusively in the top cost-sharing tier. In effect, this leaves patients with a given condition — whether HIV or Crohn’s disease — without a single affordable treatment option.

Pre-ObamaCare, about half the states had a system in place for helping people with pre-existing conditions: state high-risk pool plans, which for years offered government-subsidized coverage to patients with pre-existing conditions. But the Affordable Care Act banned those pools.

So now, with exchange plans failing them, the chronically ill have nowhere left to go.

Allies of the insurance industry blame the drug companies for the high price of certain medicines. AARP policy adviser Leigh Purvis, for instance, says cost-sharing levels “wouldn’t be so high if the prices of drugs weren’t so high.” Health-policy advocate John Rother, meanwhile, claims that “reducing price is something [drug] companies could do tomorrow.”

But drug prices aren’t arbitrary. The average biopharmaceutical therapy takes $1.2 billion and anywhere from 10 to 15 years to bring to market. Firms must charge high prices for certain brand-name drugs to make back this substantial investment with enough left over to fund research into the next generation of treatments.

And while insurers like to complain that sophisticated therapies cost too much, they tend to ignore the far higher costs of denying these medicines to patients.

Increased co-pays result in “non-adherence,” failure to take prescribed medications. And that equals increased rates of hospitalization, chronic heart failure and premature death.

And this adds to health outlays: According to a recent study in the Annals of Internal Medicine, non-adherence costs the US health system from $100 billion to $289 billion a year.

In short, by making needed medications unaffordable — and by failing to cover newer, targeted therapies — insurers are jeopardizing patient health. And far from saving money, cutting off access to specialty drugs actually increases long-term health costs.

Specialty medications need to be treated as equivalent to other essential medical services — not as some luxury that only the wealthy can afford. But the ObamaCare law has only made the problem worse than ever.

Peter J. Pitts, a former Food and Drug Administration associate commissioner, is president of the Center for Medicine in the Public Interest.

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Embracing Che

08/08/2014 09:57 AM |

Here’s something you probably don’t know – once-upon-a-time I was a stand up comic. That’s why I was so intrigued when I got a call from the Daily Show asking for an interview on … opioid abuse.

Opioid abuse?

Well, the interview happened yesterday (the "correspondent" was the charming Michael Che) and it’s still a mystery to me where the humor is in this topic. So why did I choose to participate? (1) It’s a widely watched program that acts as the main source of news for a lot of people. Sad, but true. And (2) The segment will air with or without my comments. I’d rather have the opportunity to offer some truth even in the face of … comedy?

Or in the words of Molière, The duty of comedy is to correct men by amusing them.

The segment will run either just before or just after Labor Day. Stand by for more details.

PS/ Whatever the outcome, I will certainly not be as bad as Kathleen Sebelius!
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Channeling the Passion of the Patient Voice

08/06/2014 02:00 PM |

One of the hallmark pieces of FDASIA enshrines the concept of patient-focused drug development. Per the FDA’s own website:

Patients who live with a disease have a direct stake in the outcomes of the drug review process and are in a unique position to contribute to the entire medical product development enterprise.  Under FDASIA, the FDA will increase patient participation in medical product regulation.

• Patient Participation in Medical Product Discussions under FDASIA. Sec. 1137 of the new law will assist the agency in developing and implementing strategies to solicit the views of patients during the medical product development process and consider their perspectives during regulatory discussions.  This will include:
  - Fostering participation of FDA Patient Representatives as Special Government Employees in appropriate agency meetings with medical product sponsors and investigators; and,
  - Exploring means to provide for identification of potential FDA Patient Representatives who do not have any, or have minimal, financial interest in the medical products industry.
• Patient-Focused Drug Development under PDUFA V. The PDUFA V agreement provides for a new process enhancement under a commitment that will provide a more systematic and expansive approach to obtaining the patient perspective on disease severity or the unmet medical need in a therapeutic area to benefit the drug review process.  In other words, the patient perspective will provide context in which regulatory decision-making is made, specifically the analysis of the severity of the condition treatment and the current state of the treatment armamentarium for a given disease.

But patient input needs to be more than anecdotal – it needs to be data-driven so that divisional decisions can be more scientifically-driven by the patient community. And nowhere is that more urgently needed than in discussions over risk/benefit calculations.

The Center for Medicine in the Public Interest (www.cmpi.org) weighs in on this issue with a new paper by senior fellow, Dr. John Bridges (of the Johns Hopkins Bloomberg School of Public Health). His new paper, Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study (The Patient, DOI 10.1007/s-40271-014-0081-0) provides important qualitative evidence on stakeholders’ views as to important issues associated with emerging therapies for idiopathic pulmonary fibrosis.

Bridges, et al., identifies multiple issues were identified spanning the impact of emerging therapies, including the need to document the patient experience with treatment, and factors associated with disease progression.

The paper the value of qualitative research both in understanding the benefits and risks of emerging therapies and in promoting patient-centered drug development.

Patient passion is important to share. Bridges and his colleagues go one step further and provide a pathway towards capturing that passion and channeling it into usable data that can be used to impact regulatory decision-making.   Read More & Comment...

Ian Read: LinkedIn and Locked on

08/05/2014 08:04 AM |

Ian Read (Chairman and CEO of Pfizer and the current Chairman of PhRMA) recently published a piece on LinkedIn under the title, Why Society Needs a Vibrant Pharmaceutical Industry: Improving Patients' Lives.

Towards the end, Read writes:

I recognize that there are differing views when it comes to society’s perception of the pharmaceutical industry. Many believe we are more focused on making profits rather than finding cures for patients, even though the industry has a long-standing commitment to providing patients access to needed medicines through many different programs globally. There is also a perception that we do not operate in an open and transparent manner when it comes to our clinical data and financial relationships with healthcare providers. This view lingers despite the significant steps that have been taken to increase transparency, even in the face of the current debate that rages over an individual’s right to privacy.

As an industry we are working diligently to improve our standing in society. We understand that we have a great responsibility. We are at the center of society’s desire and expectation for delivering potential cures and new lifesaving treatments. We will continue to fulfill that vital purpose.

Patients are waiting and we are working hard every day to earn their trust.

Fine sentiments and well-crafted words – but working hard alone isn’t enough to earn trust. Pharma must work hard to do the right thing. What does that mean?

Mr. Read offers the following:

Over the course of the past 50 years, this industry has tackled some of the leading causes of disease and life-threatening illnesses.

For example, today the number of people who have died from heart attacks and strokes has declined. In 2008 around 16 percent of the U.S. adult population was taking a statin to reduce cholesterol. This translated into 60,000 fewer heart attacks, 22,000 fewer strokes and 40,000 fewer deaths.

An article published in 2010 by the Journal of Health Economics found that from 1988 to 2000, improvements in cancer survival created an estimated 23 million additional life-years over this period.

And according to the World Health Organization, immunizations save an estimated 2.5 million lives every year. For every $1 the U.S. spends on childhood vaccinations, we save $10.20 in disease treatment costs.

Consider that pharmaceutical innovation has accounted for 73 percent of the total increase in life expectancy between 2000 and 2009 across 30 developing and high-income countries.

Those are, by any measure, extraordinary accomplishments. Why then is the biopharmaceutical industry so roundly pilloried in the press and so low in the general view of public opinion? Working hard, it seems, is not enough.

At  PhRMA’s 14^th annual meeting in Washington DC. this past April, Read said that industry needs to “fix the misperception gap.”

Pharma must embrace a new paradigm. Rather than focusing on traditional ROI (Return on Investment), they must now also consider Return on Integrity.

Integrity comes in many forms. Honesty. Virtue. Morality. It means not waiting to be told to do it or waiting to see what others do first. Integrity means being principled and, as my father used to say, “A principle doesn’t count until it hurts.”

Mr. Read’s article on LinkedIn is a good start – but there are many other issues that need to be addressed (such as comparative effectiveness, drug pricing, and off-label communications). Trumpeting accomplishments is both important and cathartic – but now its time for the America’s top drug honcho (and his counterparts at other companies) to move on to more contentious and complicated topics.

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Karen Ignagni: Bending the curve -- and the truth -- about the $1000 Pill

08/04/2014 11:15 AM |

Karen Ignagni's WSJ oped claims that spending on the new Hepatitis C (HCV) drug will hike health premiums and bring insurers to ruin. 

In fact, new drugs like Sovaldi will not only rescue patients, but also reduce the cost of health care.  The reason is simple. Cure are always less expensive than treating the same disease using no or halfway measures.  Would Ignagni wail about a cure for Ebola that cost $1000 a pill?

Before HIV medicines came to market, there were nearly 100000 people with HIV were hospitalized.   Today, about 38000 HIV patients are hospitalized each year.  Hospitalization rates plummeted thanks to HIV drugs. Without HIV medicines, 111000 HIV patients would be hospitalized each year.    Fewer people with HIV would be alive.

Sovaldi will produce similar savings.  Millman (a health actuarial firm) estimates 984510 with HCV will be treated between 2014 and 2020.  Assume everyone treated gets Sovaldi at $80000.  That would cost $78 billion or more accurately about $13 billion a year for six years.

Sounds dire right?  But Ignagni ignores how new HCV drugs eliminates a 48-week course of treatment for interferon (that is only effective half the time and has flu like side effects) costs about $6.12 billion year.   If hospitalization rates drop by half, it will save another $1 billion per year.  ($7 billion total.)   Over six years insurers will save $252 billion in the process.  Insurers will net savings of $174 billion.   

None of this takes into account that new HCV drugs increase life expectancy and well being so that people can work, pay taxes and cover the cost of health premiums.   Ignagni’s alarms about Sovaldi are intended to divert attention to these facts and to justify sticking more consumers with the cost of a treatment that saves lives and money. 
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