The Fable of Off-Label

12/11/2012 06:57 AM |

I am not one of those who in expressing opinions confine themselves to facts.

-- Mark Twain

What? Off-label communication is protected free speech?

Cry havoc and let slip the dogs of war!

Not so fast.

As Steve Usdin of BioCentury so aptly opines, the ruling is “far less consequential than media coverage suggests.”

According to Usdin:

… companies and individuals who take the decision as a signal that the rules of the road have changed and they are now free to promote off-label indications put themselves in great legal and economic peril, attorneys who helped persuade the court to overturn Caronia's conviction told BioCentury.

At the same time, the decision by one of the country's most influential and respected courts to overturn a criminal conviction on First Amendment grounds is persuasive evidence that, in the long term, FDA will have to change some of the assumptions underpinning its regulation of medical products.

FDA, which now has lost a string of First Amendment cases, cannot forever hold on to the notion that it is empowered to prohibit drug companies and their employees from saying things that anyone else is free to say. Sooner or later, according to legal experts, the agency will have to reconcile itself with the idea that industry has the right to truthful, non-misleading speech.

While change is inevitable, the pace of change is uncertain. It is also not clear who will shape that change - FDA employees, judges, or members of Congress.

In short the Caronia ruling didn’t really answer any questions so much as it opened up the conversation. And that alone is worth the price of admission.

What will it mean on the ground? In the short term, not much. But it’s not about instant gratification for aggressive marketers.

This ruling isn’t the beginning of the end of FDA warning letters on off-label communication -- but it very well may be the end of the beginning of such speech being off-limits to prudent corporate compliance officers.

Equally important, what will it mean for off-label conversations with patients and caregivers? What will it mean for regulated speech on social media?

As William Safire once said, “Is sloppiness in speech caused by ignorance or apathy? I don’t know and I don’t care.”

Usdin’s complete story, Free speech, in theory, can be found here.

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Transfer at TransCelerate

12/10/2012 10:41 AM |

TransCelerate Appoints Dalvir Gill as CEO

TransCelerate has named Dalvir Gill, PhD, as its Chief Executive Officer, effective January 1, 2013. Dr. Gill will lead the independent non-profit entity, which was formed by ten healthcare and pharmaceutical companies in September to improve the quality of clinical studies and bring new medicines to patients faster by facilitating the collaboration required to solve common challenges encountered during the clinical trial process. Dr. Gill will succeed Garry Neil, MD, who has served as interim CEO. Dr. Neil will remain as Chairman of the Board of Directors.

Dr. Gill has more than 25 years of drug development experience. Prior to his appointment as CEO of TransCelerate, he was the President of Phase II-IV Drug Development at PharmaNet-i3, an organization specializing in drug development services. In this role, Dr. Gill was responsible for a global business spanning nearly 40 countries.

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Tough Cookie for Tough Times

12/10/2012 07:47 AM |

Nature Biotechnology reports:

Having Hamburg, who is a “tough cookie,” remain at the FDA provides something of a “public health bulwark against political intrusions,” says Pitts.

The full article, Cuts loom over research and industry following Obama re-election, can be found here.

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Healthcliff!

12/07/2012 08:44 AM |

Yesterday I participated on the Pennsylvania BIO-sponsored panel, “Election Aftermath: The Fiscal Cliff and the Future of Healthcare Reform.”

You might have heard of these issues.

My fellow panelists were Senator Bob Casey (D, PA), Congressman Jim Gerlach (R, PA) and Gary Karr  (Executive VP, AdvaMed). The panel was expertly moderated by Chris Satullo (Executive Director of News and Civic Dialogue at WHYY).

Much of the evening’s focus was on the collision of healthcare innovation and national healthcare policy – and not in a good way.

Specific items of discussion were IPAB – Uncle Sam dictating (for tens of millions of Americans) what treatments will be available for physicians and patients; PCORI – the government instructing physicians as to how to practice medicine, FDA reform (specifically surrounding the issue of predictability), waste in Medicare and Medicaid, the medical device tax (both Casey and Gerlach are for repeal of the tax), the President’s call to roll back biologics exclusivity from 12 to 7 years (both Casey and Gerlach are for preserving 12) and, of course, the issues surrounding state exchanges.

Since states are the laboratories of innovation, governors should be given wide authority to design programs that best fit the needs of their particular situations. Ultimately states will learn from each other. HHS waivers should be broad. We’ll see.

The worst scenario is a one-size-fits-all top-down approach that fits everyone poorly. Alas – that’s a pathway preferred by many inside the Beltway.  A key issue here is that of essential health benefits, what are they and who makes the call.

Congressman Gerlach was especially effusive (and appropriately so) of the free-market design of Medicare Part D. Lower than expected costs, high utilization and user satisfaction. And he strongly agreed this is a model that can work in the design and implementation of state exchanges.

Jim Dandy.

Senator Casey mentioned his strong support of NIH funding. I suggested to the Senator (politely) that perhaps more than a small slice of that largesse should be redirected to the FDA.

Does White Oak have a friend in Pennsylvania?

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Fear of Non-Inferiority

12/06/2012 09:54 PM |

Nature reports:

FDA under pressure to relax drug rules

Industry says antibiotic pipeline is being blocked by overly stringent clinical-trial requirements for new treatments.

The latest skirmish in the battle between human and microbe played out on 29 November in a hotel conference room in Silver Spring, Maryland. There, an assembly of scientists and clinicians debated the merits of an experimental antibiotic. For some, the coveted prize was not just an endorsement of the drug itself, but a sign that the US Food and Drug Administration (FDA) is finally ready to rethink its clinical-trial requirements for antibiotics — requirements that the drug industry says are unrealistic.

The number of FDA approvals of new antibiotics has dropped even as multi-drug-resistant strains of bacteria have proliferated. FDA advisers at last week’s meeting did recommend approval of telavancin (Vibativ) — a derivative of vanco­mycin — for the treatment of hospital-acquired pneumonia when alternative drugs are not suitable. But that vote came nearly two years after the FDA had rejected the drug for a second time because clinical data did not measure up to the agency’s guidelines.

“The agency has painted itself into a statistical corner,” says Scott Hopkins, chief medical officer of Rib-X, a drug company in New Haven, Connecticut, focused on antibiotics. “While the infectious-disease community was crying out for new antibiotics, the FDA seemed to be going in the opposite direction.”

Many trace the agency’s tougher stance to the scandal surrounding telithromycin (Ketek), an antibiotic approved by the FDA in 2004 and later linked to liver failure. In 2007, the US Congress launched an investigation into whether the FDA had ignored staff concerns about Ketek’s safety. The following year, the agency convened its advisers to discuss antibiotics then under review. “Four drugs representing over a billion dollars of investment went into that week and only one came out alive,” recalls Mark Leuchtenberger, president of Rib-X.

Telavancin was caught in the changing tides. When Theravance, the company in South San Francisco, California, that developed the drug, designed the large phase III clinical trials needed for approval, the FDA simply required a demonstration that the drug eliminated symptoms of infection as reliably as the approved antibiotic vancomycin. But, after Theravance submitted its second application on 30 June 2010, the FDA decided instead that applicants needed to show that patients were no more likely to die — of any cause — within 28 days of treatment with a new drug.

Theravance scrambled to gather the data, hunting down medical records for 1,419 out of the 1,503 patients scattered across dozens of countries that were enrolled in the telavancin trials. But the FDA determined that the study lacked statistical power and asked for new clinical studies. Theravance refused, and a stalemate followed.

Strict clinical guidelines for antibiotics have dogged the industry ever since, with pneumonia providing a good illustration. Patients who contract pneumonia in hospital are already ill, making it hard to know if the treatment under review played a part in their death. That means trials have to be larger to capture enough deaths to have any statistical meaning. This, combined with the relative rarity of infections that warrant the use of new antibiotics and the further FDA requirement that patients not receive other antibiotics before they get the experimental drug, has set the goal out of reach, argues David Shlaes, who runs Anti-Infectives Consulting in Stonington, Connecticut. Not a single new antibiotic for hospital-acquired pneumonia has been submitted for approval since the new guidelines were put in place. “The trial simply cannot be done,” says Shlaes. “Whoever was writing these guidance documents doesn’t live on the same planet that I do.”

However, since the Ketek scandal, the political winds have reversed. This summer, Congress passed a set of measures to encourage antibiotic development. In May, Janet Woodcock, head of the FDA’s Center for Drug Evaluation and Research, pledged to “reboot” the antibiotic-approval process. And in September, the FDA told Theravance that its advisers would take another look at telavancin, resulting in last week’s vote.

Investors have also noticed these changes, says Leuchtenberger. Last week, Rib-X announced that it had raised US$18.7 million to help the company start phase III trials of an antibiotic that could target skin infections. “Without some of these positive developments this year, you’re looking at a number of companies that might not be around any more,” he says.

Shlaes, however, emerged from the telavancin meeting still doubtful, noting a continued focus on all-cause mortality. He says that, for now, he will continue to advise his clients to apply for approval in Europe first, where regulators have not been as demanding as the FDA. “I’ve been very optimistic about the whole thing,” he says. “But the FDA has to do something to show that it is actually rebooting.”

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Freedom of Speech Upheld In Healthcare

12/06/2012 03:07 PM |

"The First Amendment directs us to be especially skeptical of regulations that seek to keep people in the dark for what the government perceives to be their own good. " A US Appeals Court ruled that sharing scientific information about off-label uses of prescription drugs is protected by the First Amendment. Note to opponents of off-label information: you will still have plenty of chances to sue, attack, etc., since the Appeals court stated that then1st amendment doesn't protect misleading or deceptive speech. Which means that the trial bar and AGs will still seek damages under The False Claims Act. What should companies do? Generate clear guidelines on the level of evidence that should be used when sharing off-label information. Should a letter to NEJM discussing one doctor's observations about the novel use of medicine be widely shared? Probably not. But should a company be able to share information based on observational or registry research, comparative trials or new mechanisms of action? Yes. Similarly, companies should come up with a clear set of guidelines for talking about and responding about off label uses in social media. And I think it will be critical to develop a legal strategy reflecting this important ruling. For too long critics of the biopharma industry have been able to flood the digital commons with misleading, deceptive and sensationalist claims based on junk science. The appellate court ruling gives companies a bit more freedom to participate in conversations previously dominated by critics and fearmongers. They should use it or they might lose it.   Read More & Comment...

Juice on Safe Use

12/05/2012 07:59 AM |

I was pleased to chair the Fifth Annual Risk Management and Drug Safety Summit.

REMS is a tactic. Safe Use is a strategy and Safe Use is the new normal.

Some take-aways:

According to Greg Fiore, MD (Chief Medical Officer and Acting Head of Global Pharmacovigilance at The Medicines Company), we are now in a “new world order” where pharmacovigilance teams finally have a seat at the table.  But, per Fiore, we are still spending too much time, talent, and treasure of process – and not nearly enough on insights development and use.

Good points. Could this be because ever-more process is a good excuse for actually doing something?  Such action would require not just more dollars and FTEs for PV teams – but would also mean changing the cognitive mapping of the way many companies do business. It might actually mean that patient safety takes priority over marketing and sales.

Fiore also mentioned the need to consider what benefits a more aggressive use of social media might mean for pharmacovigilance.

Is it time to “friend” Gerald Del Pan?

Josephine Torrente, JD (a Director at Hyman, Phelps & McNamara), made the point that REMS is labeling (a view held by most at the conference) and that the implications for shared REMS must be taken into account – especially when it comes to biosimilars. Josephine also addressed the issue of possible FDA waivers from shared REMS programs. She doesn’t see it as likely.

Is this another possible point of political intrusion? As with Plan B, will the HHS Secretary decide to grant such a waiver if the FDA declines to do so? Ms. Torrente feels petitioning the Humphrey Building for such divine intervention would have little chance of success. Glad to hear that.

The class-wide Opioid REMS was much on everyone’s mind, and Stuart Kim, JD (Senior Regulatory Counsel, Covidien), made a strong case for a more active role by state-level stakeholders. The context for this was the crucial need to differentiate the desire to address abuse versus misuse. The failure to recognize the different nature of these issues will lead to negative and unintended consequences. He also asked, per the CME requirements, why we are not looking to a more thoughtful level of measurement. Just “ticking the box” on CME won’t solve any problems. Rather, Stuart pointed to a goal of a Moore’s Scale Level 7 achievement of actually improving patient outcomes.

For more on Moore’s Scale, see here.

Addressing abuse is one thing – but smartly dealing with misuse (while not as sexy or politically potent) is at least of equal import.

After Stuart’s presentation, I offered the following prediction:

FDA will not approve generic opioids minus the innovator-developed abuse-resistant technologies – unless they are forced to. IP issues? You bet. Watch this space for more on this issue as it rises to the top of the FDA agenda. (And don’t be thrown off-guard by what’s going on north of the border.)

Eleanor Segal, MD (Consultant, Segal PV Systems), discussed pharmacovigilance trends in the EU, with specific focus on how the EU is actually becomes less harmonized both between it’s own members – and with the FDA. And the new EU directive on Phase IV studies didn’t give anyone a warm and fuzzy feeling.

James Frame, MD (Medical Director, David Lee Cancer Center, Chair of ASCO REMS Working Group, and President, West Virginia Oncology Society) spoke about the “onerous” burdens that many REMS plans are placing on physicians. Not news, but he made the good point that such burdens impact patient access issues such as time spent with physician, availability of support staff to address patient needs – both of which could actually lead to less optimal care. Unintended consequences – but certainly not unpredictable when both sponsor and agency fail to take the weight of “practice burden” into account.

It’s the risk of risk mitigation.

Maybe its time for the FDA (and sponsors) to engage a “physician representative” when it comes to REMS design.

Day One ended with a thoughtful presentation by former FDAer Lynn Mehler, JD (Partner, Hogan Lovells). Lynn predicted that the FDA would be looking forward to more class-wide REMS.

We shall see.

Richard Hermann, MD (Safety Science Physician, Patient Safety, Global Regulatory Affairs, AstraZeneca) brought the issue of risk/benefit analysis via a validated grid into the conversation. His baby, the BRAT, is only one methodology under discussion – but it is an important one in the “date versus information” debate. He urged the audience not be prisoners of process and to fight cultures that are resistant to change be they in industry or at the FDA.  He acknowledged that many firms are operating in a “change-weary environment.”

Well, if the Pope can start tweeting and off-label promotion is protected free speech, there is hope for us all.

What will tomorrow bring.

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Off-Label Chinsanity

12/04/2012 07:49 AM |

Fasten your seatbelts.

Court: off-label promotion protected

The U.S. Court of Appeals for the Second Circuit said in a 2-1 ruling in United States v. Caronia on Monday that the "government cannot prosecute pharmaceutical manufacturers and their representatives under the [Food, Drug and Cosmetic Act (FDCA)] for speech promoting the lawful, off-label use of an FDA-approved drug." In the ruling, Judge Denny Chin wrote that so long as the off-label use of the FDA-approved drug is legal, the government's interpretation of FDCA's misbranding provisions to prohibit and criminalize the promotion of off-label use "unconstitutionally restrict[s] speech." FDCA prohibits misbranding, but does not expressly prohibit the promotion or marketing of drugs for off-label use. Chin noted that off-label promotion that is false or misleading is not protected by the First Amendment. In a dissenting opinion,

Judge Debra Ann Livingston said the ruling "calls into question the very foundations of our century-old system of drug regulation," adding that if drug companies "were allowed to promote FDA-approved drugs for nonapproved uses, they would have little incentive to seek FDA approval for those uses."

The case concerns Alfred Caronia, a former specialty sales consultant at Orphan Medical, which was acquired by Jazz Pharmaceuticals plc. He was convicted in 2008 of conspiracy to introduce a misbranded drug into interstate commerce based on audio recordings in which he promoted the off-label use of narcolepsy drug Xyrem sodium oxybate. Caronia, who was sentenced to one-year probation and 100 hours of community service, appealed, arguing that his conviction was based solely on his speech and therefore violated his First Amendment rights.

The appeals court vacated and remanded to the lower court the decision convicting Caronia.

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CMPI Brings Sanity to the Big Apple's Soda Ban

12/03/2012 04:55 PM |

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CBO Scores One For Innovation

12/03/2012 04:48 PM |

A Congressional Budget Office memo about the offsetting costs of Medicare Part D concludes that a 1 percent increase in the number of prescriptions filled leads to 1 percent reduction in Medicare spending in the absence of prescription drugs.  

So I did some rough calculations:  According to CMS the percent increase in Rx filled per year in Part D from 2007 -2009  was 5.6 percent.   That lead to an increase in part D spending of about 5.7 billion.   Assuming a 5.6 percent decline in the rate of medicare spending I come up with a savings of $30 billion. (The additional spending that would have occured in the absence of drug utilization.)   That means every dollar spent on Part D saves about $6 in Medicare spending.   Lichtenberg estimates $1 dollar of  drug spending  on new drugs saves $7.   This is a bit below his estimate (few new drugs were introduced since 2006) but now CBO has essentially "blessed" the offset


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2013: A Healthcare Odyssey

12/03/2012 07:40 AM |

According to “Pay it Forward,” Medical Marketing & Media’s overview of 2013 …

After a tumultuous few years of healthcare policymaking, the recent election brought a big victory for Obamacare. It's now all systems go for the law, with sweeping change for the American healthcare system and tens of millions more insured. Expect this status quo to be anything but boring.

ACA implementation isn't the most immediate cause for anxiety among healthcare policy types. That honor goes to the “sequester,” a high-stakes game of budgetary chicken set to play out over the next month or so. Last year's standoff between the White House and Congressional Republicans over the “debt ceiling” was resolved, in classic Washington fashion, by kicking the can down the road a bit, but with a twist—if the two sides couldn't agree on painful cost savings by January 2, a legislative “trigger” would be tripped prompting brutally deep cuts (of $1.2 trillion over nine years) to defense and social spending. Medicare is largely exempt from the cuts—limited to 2% of its budget—and Medicaid and CHIP are off-limits. FDA, however, would face deep cuts—projected at $318 million—that would slow approvals and rules-making, according to the Office of Management and Budget, and effectively freeze PDUFA.

“Sequestration would be a complete disaster for all involved,” says Peter Pitts, former FDA associate commissioner. “The FDA simply doesn't have any slack in its budget and would not have the bodies to do things on time. This is not the status quo but a significant step backwards.”

The rest of the article can be found here.

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Obamacare's impact on small business owners

11/30/2012 11:29 PM |

From the Associated Press:

NEW YORK (AP) — Rose Wang looks at her staff of 70 employees and wonders if she'll have to lay off some of them to comply with the health care law.

The owner of Binary Group Inc., an information technology firm based in Alexandria, Va., is one of many small business owners who will be required to provide health insurance for her staffers under a provision of the law that goes into effect on Jan. 1, 2014. Wang already provides insurance, but she has struggled with premiums that have soared as much as 60 percent annually, so she requires employees to contribute to their coverage. She's worried because she doesn't know how much she'll have to pay under the Affordable Care Act.

Wang's worry is a gut-wrenching dilemma that many small business owners are concerned that they may face. Now that President Barack Obama has won re-election, the health care overhaul, which presidential candidate Mitt Romney promised to dismantle, is marching forward. Companies must decide before the start of 2014 what they'll do to comply with the law. Right now, no one knows how much the insurance will cost, and owners aren't sure if they'd be better off not buying it and paying a government a penalty of $2,000 per worker. Some owners are even threatening to defy the law. The big challenge for most small businesses is that they just don't have enough information to make concrete plans.

If Wang can't afford the insurance, she says that some of her staffers may have to go.

"I would have to say, 'look, guys, you're family to me in many respects, but this family also depends on having the kind of cash flow available to keep the lights on and keep employing most of you,'" Wang says. "It would have to come down to that."

Read the full piece here.


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More is not always better

11/30/2012 08:13 PM |

"Less is More," said Walter Gropius. But, according to Mies van der Rohe, "More tastes better."

Alas -- there's no accounting for taste.

From the pages of Medical Marketing & Media:

European regulators to publish clinical trials data

The head of the European Medicines Agency has said it's a matter of how, not if, the body will mandate publication of clinical trials data – and that has pharmas on both sides of the Atlantic sweating the potential implications for global competitiveness.

EMA executive director Guido Rasi said last week “The European Medicines Agency is committed to proactive publication of clinical trial data, once the marketing authorization process has ended. We are not here to decide if we publish clinical trial data, but how.”

His remarks came at the start of a daylong workshop in London on data transparency around clinical trials. The agency has convened several advisory groups, including some representation from industry, to grapple with thorny issues including patient confidentiality, data formats, rules of engagement, good analysis practice and legal aspects. They will start work in early 2013 and are expected to deliver their recommendations by the end of April, with mandatory publication of trials data taking effect on January 1, 2014.

“The implications are rather significant relative to the continued protection of intellectual property rights,” said Peter Pitts of the Center for Medicine in the Public Interest, “and continued incentives—or disincentives—to invest in innovation.”

The full article can be found here.

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Healthcare Innovation? "Aye" says Dubai

11/28/2012 10:36 PM |

Gulf News

Experts stress need for innovation in healthcare

Regulation will ensure adequate supplies of medicinal stocks, says ministry

By Carolina D’Souza, Staff Reporter

November 28, 2012

Dubai: The UAE is intensifying its efforts to increase availability of medical technologies and drugs. This key message was delivered by the UAE Ministry of Health (MoH) during the opening of a two-day conference on Wednesday.

The Ministry believes that the UAE is one of the leading countries in the region to provide innovative medicine.

The conference titled ‘Competitiveness Forum: Health Care and Access to Innovative Medicines in the UAE’ brought together senior officials from the MoH, healthcare authorities, Ministry of Economy and GCC regulatory bodies as well as representatives from the private pharmaceutical sector.

Speaking to Gulf News, professor Peter Pitts, president of the Centre for Medicine in the Public Interest (CMPI), said, “The UAE has smartly figured out that by partnering with the pharmaceutical industry, it can bring innovation to the country and make it a hub of innovation.”

He said that the UAE is recognised for its speed in marketing new medicines and providing patients with the newest medical technologies. Stressing on the need for innovation in medicine, he explained that incremental innovation is more common — than one-time innovation — through which companies enhance a product or a technology.

Extending lives

“Stakeholders, governments and regulators should support innovation in healthcare — it extends peoples’ lives,” said Pitts.

Dr Amin Hussain Al Amiri, assistant undersecretary for Medical Practices and Licensing at the MoH, said that innovative medicines are key drivers for growth in the healthcare sector. “The UAE is one of the leading countries, providing its residents with all kinds of medicine, including life-saving drugs. Through regulations, we will ensure that new medicines are introduced and adequate stocks are maintained for all healthcare needs.”

Dr Ola Al Ahdab, pharmaceutical consultant for registration and drug control at the MoH, told Gulf News that access to timely and advanced medicine is a top priority for the UAE government.

Chair of the conference organising committee Dr. Yaqoub Haddad added, through this conference, the pharmaceutical companies and stakeholders will be able to identify strengths and weaknesses in the healthcare sectors to continue to provide access to innovative medicine.

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Are We In A Post Wakefield Era For Immunization

11/28/2012 08:28 AM |

MMR vaccine rates return to pre-Wakefield levels UK NEWS | NOVEMBER 28, 2012 BEN ADAMS Immunisation levels for measles, mumps and rubella in England have reached their highest recorded coverage since the Wakefield scandal in the late 1990s. In the past year, 91.2% of children had received their first dose of the MMR vaccine by their second birthday - a rise from 89.1% in 2010-11, according to new data from the Health and Social Care Information Centre. This is the first time coverage has passed 90% since 1997-98 (when it was 90.8%) and is 0.6% points away from a coverage peak recorded in 1995-96 (91.8%). Related Links The Lancet retracts Wakefield’s flawed MMR paper England and Wales see surge in measles cases MMR link to autism based on falsified data, says BMJ study In 1998 the British doctor Andrew Wakefield published a study in The Lancet that suggested a strong link between the MMR vaccine and rates of autism and bowel disease in children. UK media coverage caused a storm of controversy over the issue, and researchers and the government could do little to persuade some parents that the MMR jab was safe. Media coverage of the issue hit a peak in 2001-2, and vaccination levels of the combined vaccine fell dramatically to less than 80% a year later. It is thought this then contributed to outbreaks of measles in some parts of the country. Since then, however, Dr Wakefield’s work has publicly been retracted by The Lancet, and he was struck off from the General Medical Council in 2010, which is believed to have helped return rates back to the levels before 1998. While rates are returning to pre-Wakefield levels, they are still some way off the 95% of children that the World Health Organization says should be vaccinated in order to create herd immunity against the three diseases. England’s rates of immunisation for MMR also remain lower than the rest of the UK, the HSCIC notes. HSCIC chief executive Tim Straughan said: “[The] report marks a significant point in the continued rise of MMR coverage since it hit a low in 2003-04 – as for the first time in 14 years, nine out of 10 children in England have had the MMR vaccine before they turn two. However, although MMR coverage at two years has risen in all regions of England, and overall the country's coverage has increased in recent years, the national figure remains below the World Health Organization target of at least 95%.”  Read More & Comment...

Will punishing hospitals mean better outcomes?

11/28/2012 04:19 AM |

Medicare’s tough love is not going over well everywhere. Academic medical centers are complaining that the penalties do not take into account the extra challenges posed by extremely sick and low-income patients. For these people, getting medicine and follow-up care can be a struggle.

At Barnes-Jewish Hospital, Dr. Lynch said physicians from all over the Midwest referred their sickest heart patients to his facility for transplants and other major interventions. But those patients can skew his hospital’s readmissions numbers, he said: “The weaker your heart, the more advanced your emphysema, the more likely you are to be readmitted to the hospital.”

Dr. Lynch said Barnes-Jewish set up follow-up appointments for patients who didn’t have their own doctors. But about half of the patients never showed up, he said, even after the hospital made reminder phone calls and arranged for free rides. Sending nurses to see patients at home did not significantly reduce readmission rates either, he said.

“Many of us have been working on this for other reasons than a penalty for many years, and we’ve found it’s very hard to move,” Dr. Lynch said. He said the penalties were unfair to hospitals with the double burden of caring for very sick and very poor patients.

“For us, it’s not a readmissions penalty,” he said. “It’s a mission penalty.”

Read the full New York Times piece here.


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Recognizing the Brakes on Breakthrough Therapies

11/26/2012 09:14 AM |

The first step towards solving a problem is to identify that a problem exists.

The breakthrough therapy provision in FDASIA is aimed at shortening the development time for drugs intended to treat serious or life-threatening conditions and for which preliminary clinical evidence suggests a substantial improvement over existing therapies. But the devil is in the developmental details.

But the promise of speeding innovative medicines to market could be blunted unless issues related to manufacturing, companion diagnostics and international harmonization are adequately addressed.

That was the message from a multi-stakeholder panel discussion at the November 14^th Conference on Clinical Cancer Research, co-sponsored by the Friends of Cancer Research and the Engelberg Center for Health Care Reform at Brookings.

Panelists and FDA representatives agreed that if a drug is ushered through the development process, hold-ups will arise from other areas. As a result, discussions surrounding chemistry, manufacturing and control issues for new drugs and biologics will need to take place much earlier in the clinical development process than currently happens to avoid a situation where a breakthrough therapy is approved but the manufacturing processes are not in place to support commercial marketing.

Discovery is one thing. Development and commercialization are another. Nobody said it was going to be easy.

Breakthrough treatments that require companion diagnostics also could be potentially problematic, as the development, validation and approval of the diagnostic may not be able to keep pace with the drug’s abbreviated clinical development program.

Under FDASIA, sponsors must request that their product be designated as a breakthrough therapy, and FDA must respond within 60 days. For drugs receiving the designation, the agency must work in a variety of ways to expedite the products’ development and review, including providing timely advice to the sponsor to ensure the development program is as efficient as possible and involving senior managers and experienced review staff in a collaborative cross-disciplinary review.

Under the law, FDA must issue draft guidance by January 2014 on the process and criteria for making designations and actions the agency will take to expedite development and review.

What about international harmonization? During the development of the FDASIA legislation, Woodcock cited the need for FDA and EMA to “be on the same page” about abbreviated development programs for breakthrough therapies. And, according to a conference issues brief, absent an equivalent development pathway in the EU, “drugs that receive breakthrough designation in the U.S. may still be required to go through the traditional drug development pathway for EMA approval. These differing requirements may make the breakthrough pathway an unattractive option for drug sponsors.’

Richard Pazdur raised the question as to what happens when subsequent studies of a breakthrough-designated product fail to confirm the encouraging efficacy signal seen early in development. Bob Temple suggested the agency’s guidance on breakthrough designations would account for such a scenario. “The concept clearly includes the possibility that it looked really good at first and then now it doesn’t and so it’s designation could go away,” he said. “I’m sure there will be a provision for that.”

That's one thing we can most certainly be sure of. Caveat emptor.
 

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Regulatory Shoals for Curosurf

11/26/2012 08:46 AM |

“PR” does not stand for “Post Regulatory” – nor should it.

Via an untitled letter concerning a pitch letter, the FDA sent out an important holiday reminder that press materials are, indeed, regulated speech.

The target of the October 31st letter was Cornerstone Therapeutics. Their press release for its Curosurf (indicated for rescue treatment of premature infants experiencing Respiratory Distress Syndrome) was, according to the FDA, false and misleading due to omission of risk info and presentation of unsubstantiated superiority claims. A pretty standard objection, and an obvious mistake that should have been avoided by the Cornerstone communications staff.

Communications departments and PR agencies beware – play by the rules or suffer the consequences.

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The Gray Lady on the Gray Market

11/23/2012 07:44 AM |

New York Times editorial from November 22nd:

The Problem of Fake and Useless Drugs

Global health authorities are making only fitful progress toward eliminating fake or substandard drugs that cause widespread suffering and death in the developing world. Delegates from 76 member countries of the World Health Organization met this week in Buenos Aires to lay the groundwork for more forceful action.

No one knows precisely how much fraudulent or substandard medicine is sold around the world, but the fragmentary data are alarming. In poor countries, half of the medicines used to treat some deadly diseases have been found to be fakes that had little or no active ingredient; worse yet, some contained toxic substances.

One estimate by experts is that at least 100,000 people die every year from substandard and fake medicines for cancer, heart disease, infectious diseases and other ailments. Fake malaria drugs pose a real risk of hampering the international effort to curb the disease. In wealthy nations, substandard or fraudulent drugs have caused thousands of adverse reactions and some deaths.

The United States, despite having a strong regulatory agency, is hardly immune to this problem. In recent years, fake doses of Avastin sold to physician groups for cancer treatment lacked the active ingredient; a weight-loss medicine contained undeclared ingredients that posed a health risk; and compounding pharmacies have produced contaminated drugs that caused meningitis and deaths.

An analysis by health experts published in the British Medical Journal last week lamented the lack of progress in addressing this scourge. The group called for a global treaty backed by governments, drug companies and nongovernmental groups to ensure a safe drug supply.

Crackdowns by international police and national regulatory agencies have made progress in shutting down dangerous online pharmacies. But, in poor countries, the greater problem is keeping bad products out of the supply chain that serves pharmacies and health care provider groups. In Kenya, even Doctors Without Borders, a smart operation, was duped into buying fake antiretroviral drugs to combat AIDS, which it gave to thousands of patients before the fraud was discovered.

There is an urgent need for a more vigorous international effort. That might include stronger surveillance and research to determine the extent of the problem, clearer definitions for bad drugs (substandard, counterfeit, falsely labeled, falsified or fraudulent drugs, for example) and new international laws to make it a crime to traffic in such medicines.   Read More & Comment...

Expanding Essentialism

11/21/2012 07:36 AM |

Avoiding the therapeutic cliff.

HHS has issues a proposed rule that would increase the number of drugs eligible for reimbursement by insurers under the Affordable Care Act's essential health benefits requirements. In a previous bulletin, HHS said it planned to require that essential health benefits cover only one drug in each therapeutic class included in benchmark plans put forward by each state. The new rule essentially changes the language from 'only one' to 'at least one' drug, requiring that insurers cover at least one drug per therapeutic area or the same number of drugs in each category and class as specified in the state benchmark plan, whichever is greater.

BioCentury reports that the proposed rule also states that a plan "must have procedures in place that allow an enrollee to request clinically appropriate drugs not covered by the health plan." Comments on the proposed rule are due Dec. 26. Previous comments on HHS' bulletin said a one-drug requirement would have provided insufficient access to medications for some conditions. Medicare Part D requires that drug plans provide access to "all or substantially all" medications in six therapeutic classes.

HHS also released a proposed rule that prohibits health insurance companies from discriminating because of a preexisting or chronic condition, as well as a proposed rule on implementing and expanding employment-based wellness programs.

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