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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Flagpole Sitting

11/20/2012 08:43 AM |

When it comes to jurisdiction over compounding pharmacies, Senator Lamar Alexander (R, TN), wants someone “on the flag pole” responsible for ensuring that non-traditional compounders meet set federal standards.

Alexander proposed allowing FDA to designate state boards of pharmacy as the primary regulator, if they are willing and able. If the state regulators proved unable or made mistakes, FDA would have the ability to take over regulation.

 FDA Commissioner Hamburg gave that idea a positive nod, saying it was one model that could be explored, but she still wants federal regulations to assure consistency.

“I think it should be made explicit,” she said. “I think for non-traditional compounding there should be federal standards that apply to certain types of practices that go to issues of quality and safety.”

FDA said those facilities should be required to submit to inspections, report adverse events and register with the agency.

On the House side, Tom Harkin (D, IA), suggested that compounding facilities shipping across state lines should come under FDA scrutiny. Other ideas included using a volume threshold and prescription records.

If the FDA gets more responsibility – which is looking increasingly likely -- they will also need more funding. Otherwise this is all just C-SPAN rhetoric.

Did somebody say “sequestration?”

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Malarkey Alert: IPAB Repeal

11/19/2012 07:24 AM |

The election may be over, but the malarkey continues.

The most recent Marlarkey Alert comes to us courtesy of the New York Times editorial page.

In defense of the IPAB, the Gray Lady editorializes:

The board, known as the Independent Payment Advisory Board, has been the subject of false attacks over the past few years by Republicans who claim that it will ration care, disrupt doctor-patient relationships, and tell patients what treatments they can receive. That is an outlandish way to describe a board that is prohibited by law from making any recommendations to ration care, raise premiums, increase cost-sharing, restrict benefits or limit eligibility.

That’s malarkey.

Medicare spending is expected to be $575.7 billion this year, jumping to over $1 trillion by 2022, as the country ages. Over the next 75 years, the program is projected to accumulate a $38 trillion budget shortfall.

Much of this enormous price tag goes towards financing Medicare Parts A (hospital insurance) and B (medical services -- including diagnostic tests and doctor visits). And yet, IPAB has no authority over either. And the board can’t make any substantive structural changes. Neither the fee-for-service structure nor enrollee premiums and fees can be altered.

The board’s only viable option is to further ratchet down reimbursement rates for Medicare providers, especially doctors, who are already losing money on Medicare patients. Indeed, the financial burden of too-low payments under Medicare has driven 17 percent of doctors and 31 percent of primary care physicians across the country out of the Medicare program altogether, according to a study from the American Medical Association.

If rates fall any lower, seniors will have an increasingly difficult time securing doctor appointments. Visits will be cut short to squeeze in patients and care compromised. 

Lawmakers must cast aside IPAB’s flawed approach (and the media malarkey surrounding it) and focus instead on innovative initiatives that address the program’s real cost-drivers while protecting seniors’ access to care.

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Greed and Gravity

11/16/2012 07:50 AM |

There is no greater disaster than greed.

 – Lao-tzu

The tragic deaths caused by the current drug compounding scandal could so easily have been avoided. The root cause wasn’t a drug shortage – it was greed.

A new effort by Representatives Ed Markey (D, MA), Henry Waxman (D, CA), John Dingell (D, MI), Frank Pallone, Jr. (D, NJ), Diana DeGette (D, CO), and Anna Eschoo (D, CA) is aimed at getting to the bottom of the problem by investigating the roles of the players in this horrible drama – specifically the actions of Group Purchasing Organizations (GPOs).

Mr. Markey: “As Congress fully investigates all the causes of the tragic meningitis outbreak in an effort to protect patients in the future, we need to look at the role GPOs play in the occurrence of drug shortages that could lead to increased reliance on compounding pharmacies.”

 “Through our investigation into drug shortages and the meningitis outbreak, we have discovered that medical professionals have increasingly been turning to compounding pharmacies for their prescription needs,” said Mr. Dingell. “It is important we study every potential cause of this outbreak in order to get to the bottom of this health crisis.  I look forward to working with my colleagues in the coming weeks to explore legislative approaches that will address this matter.”

Representative DeGette: “While we've begun to address drug shortages legislatively, it is clear we must aggressively investigate the factors that led to this deadly outbreak and determine possible remedies, so we can take steps to save patients lives.”

“If group purchasing organizations are taking advantage of their unique role in facilitating a safe and reliable drug supply, then we need to know about it,” said Representative Eshoo.

Mr. Waxman, “In light of the tragedy of the meningitis outbreak linked to contaminated compounded drugs, we need to look carefully at all factors that might be contributing to the increasing use of compounded drugs by hospitals and other health care providers.”

“This initiative is a courageous step forward in addressing the foundational reasons behind both the tragedies caused by drug compounding and the broader issue of preventable drug shortages,” said Peter J. Pitts, President, Center for Medicine in the Public Interest and a former FDA Associate Commissioner. “The anticompetitive manipulation of American healthcare by GPOs is an urgent topic for congressional action.”

The full Congressional release can be found here.

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Breakthroughs Anonymous

11/15/2012 08:28 AM |

BioCentury reports:

FDA issues first breakthrough designation

FDA has "recently" issued the first breakthrough drug designation, Janet Woodcock, director of the Center for Drug Evaluation and Research, said on Wednesday. She said the agency cannot disclose the sponsor or drug candidate. The breakthrough designation, created by the FDA Safety and Innovation Act, commits FDA to collaborate with a sponsor to accelerate product testing and development.

"Drug manufacturing could be the rate limiting step" for implementing the breakthrough drug pathway, Woodcock said at a meeting sponsored by Friends of Cancer Research and the Brookings Institution's Engelberg Center for Health Care Reform. FDA will work with sponsors to improve the chances that manufacturing is scaled-up and approved at the time when breakthrough drugs are approved, she added.

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Mirror, Mirror

11/14/2012 08:22 AM |

Mirror, mirror on the wall – whose delaying innovation most of all?

Should the new third leg of the FDA review process be … investment considerations?

Jonathan Leff, managing director of Warburg Pincus LLC, believes that the FDA should consider the cost of drug development when making its regulatory decisions.

At a conference on rare diseases conference sponsored by the Drug Information Association and National Organization for Rare Disorders, Leff said FDA should think about the effects of its demands on sponsors, such as the additional cost another efficacy trial would create should the agency demand it.

That’s a bad idea. While there are many things the FDA can and should do that would result in lower drug development costs – the issue has no place in the regulatory review process. It is as inappropriate a third leg as comparative effectiveness. The regulatory process must rest on the twin pillars of safety and efficacy.

Leff does not agree. “We must recognize that if a system is set up to ensure safety and efficacy and no mechanisms are designed into it to take the economic burden into account, we should expect to see as an unintended consequence, exactly what we have seen, which is relentlessly increasing time and cost.”

"Because of that time and cost, venture investors and pharma companies are now unable to make new investments to initiate development of many promising new therapies and as a result the next generation of potential breakthrough treatments … may never have a chance.”

Indeed, we are seeing an increase in time and cost – and these are serious roadblocks to continued investment in innovation. But whether this is entirely the fault of the FDA is not as simple as Leff makes it seem. He’s parroting the party line of those who choose to blame their developmental failures on the FDA. There is a medical device to address this myopia – it’s called a mirror.

Mr. Leff is only repeating the broader opinion of the investor community. The National Venture Capital Association's (NVCA) Medical Innovation & Competitiveness Coalition found that 39% of firms reduced investment in life sciences companies over the last three years. The same percentage expects to further decrease investment over the next three years. The venture capitalists surveyed largely blamed the Food and Drug Administration's restrictions and regulatory challenges for this trend.

Pharmaceutical companies must evaluate their level of responsibility for shrinking investment expenditures. If they want venture capitalists to continue investing in medical research and development, they must produce high-quality drugs worthy of FDA approval – and stop whining when their “miracle drugs” require more clinical evidence.

The solution isn’t for the FDA to weigh the financial investments of biopharmaceutical companies in its regulatory decision process. The solution is to make the regulatory process more predictable.

A quarter-century ago, the success rate for a new drug used was about 14 percent. Today, a new medicinal compound entering Phase 1 testing — often after more than a decade of preclinical screening and evaluation — is estimated to have only an 8 percent chance of reaching the market. For very innovative and unproven technologies, the probability of a product’s ability to make it to the market is even lower. We must work together to turn that around.

When Thomas Edison was asked why he was so successful, he responded, “Because I fail so much faster than everyone else.” Consider the implications if the FDA could help companies fail faster. Using the lower end of the Tufts University estimate of the average pre-tax cost of new drug development, $802 million:

• A 10 percent improvement in predicting failure before clinical trials could save $100 million in development costs.
• Shifting 5 percent of clinical failures to Phase 1, the earliest stage, from Phase 3, the latest stage, reduces out of pocket costs for developers by $15-$20 million.
• Shifting of failures to Phase 1 from Phase 2, the middle stage, would reduce their out of pocket costs by $12-$21 million.

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

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

 “I’ll tell you as a venture capitalist, I have been forced to not make investments in rare disease opportunities that we might have or almost certainly would have invested in 10 years ago because of a perception that if we do a clinical trial, and given all the unknowns in this area, miss slightly on the statistical goals, that would be game over for that development program,” he said during the conference.

That may be so. But drug reviews are not and should not be predicated on the calculations of venture capitalists.

But that doesn’t mean maintaining the status quo is acceptable.

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Any Questions?

11/13/2012 07:36 AM |

Matt Arnold at Medical Marketing & Media asks a few good questions about some tough post-election ACA issues.

How will IPAB cut spending, and who will sit on it?

More than nudging the country towards insurance universality, the guts of the ACA are comprised largely of deeply wonky schemes to try and “bend the curve” of healthcare spending. And then there's the Independent Payment Advisory Board, a scheme with teeth. Nobody's sure exactly how this broadly-drawn entity will operate, but the 15-member board -- for which members must be nominated by the President, in consultation with both parties' Congressional leaders, and approved by the Senate -- will have the power to impose spending cuts on Medicare when the program's spending outpaces projected growth and when Congress fails to pass cuts to offset those increases. Initial cuts will fall on doctors and pharmas, with hospitals and hospices coming in for cuts later on. Critics fear it will evolve into a federal formulary-setting body like the UK's NICE, restricting access to drugs deemed more costly than they're worth (fun fact: Sarah Palin called it “Death Panel-like). The legislation contains language specifically prohibiting the board from rationing care or limiting benefits, but opponents argue it will result in de facto rationing.  Republicans are fiercely opposed, along with enough Democrats that repeal is a real possibility, say some Congress-watchers. The board is scheduled to issue its first report in January, 2014, so nominations should be forthcoming soon.

How will CMS reset perverse incentives for provision of medical services?

One of the main goals of the ACA as to shift the US healthcare system away from an incentive structure that rewards quantity – of diagnostic tests and procedures and products, via reimbursement – and towards one that rewards quality, of health outcomes, patient feedback, etc. One way to do that could be through a UK-style comparative effectiveness regime of the sort that industry advocates feared PCORI (the Patient Centered Outcomes Research Institute) would become (early indications are that PCORI will be operating at a much more macro level than ‘Prescribe this, not this'). Another might be a risk-sharing scheme (AKA “Expanded Access”) in which pharmas and federal programs establish a measure of success for a therapy and companies reimburse the government when their products fail to meet that standard. In other words, will we measure for clinical effectiveness or for cost-effectiveness? 

What will the particulars of the Physician Payment Sunshine Act legislation that got rolled into the ACA look like?

The Centers for Medicare and Medicaid Services blew past its initial October 2011 deadline to issue guidelines on data collection, having bigger fish to fry – like setting up Obamacare's health exchanges. CMS then pushed back the start date for mandatory data collection to January, 2013. A final rule is expected by the end of the year, but nothing says CMS couldn't hit snooze again. The big question is preemption – will the ACA trump state laws, and if so, will it favor the more lax or the more draconian among them? Also, will the law require health insurers (including government) to report payments to physicians for things like academic detailing and switching patients to generics?

Will Democrats get anywhere in their efforts to limit biologics exclusivity to seven years?

The White House dearly wanted biologics exclusivity limited to seven years. The Administration got rolled by biopharmas, which succeeded in getting it set at 12 years, but the White House never stopped pushing to dial it back. With a fiscal reckoning fast approaching, everything is on the table, and the biopharma lobbies will be playing some serious defense.

How about that non-interference clause? 

Vice President Biden, in his debate with Rep. Paul Ryan, suggested he wouldn't mind another bite at a provision in the Medicare Part D prescription drug benefit law explicitly prohibiting the government from butting into negotiations between companies and private plans that administer the benefit. “If they allow Medicare to bargain for the cost of drugs like Medicaid can, that would save $156 billion right off the bat,” said Biden, whose party's left flank has tried and failed repeatedly to dislodge the clause. Given solid Republican control of the House, it seems unlikely that they might succeed now, but again, everything is on the table, and the politics of prescription drug prices are ever tricky.

Matt's complete article can be found here.
 

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Ova There

11/12/2012 08:41 AM |

December 7, 2011 is a day that will live in regulatory infamy. That’s when Secretary of Health and Human Services Kathleen Sebelius overruled the FDA decision on the over-the-counter status of emergency contraception.

By reversing an FDA decision, the Secretary set a dangerous precedent for all-comers to lobby Congress, the HHS and the White House on any and all FDA decisions—directly inserting politics into what must be a scientifically driven process.

On December 13, 2011, Senators Patty Murray and Maria Cantwell and a dozen other Senate Democrats asked the Obama administration to produce scientific justification for its decision to block girls 16 and younger from buying the emergency contraceptive Plan B over the counter.

In a letter to U. S. Department of Health and Human Services Secretary Kathleen Sebelius Tuesday, the 14 lawmakers said they wanted "medical and scientific evidence" behind Sebelius's unprecedented decision to overrule the Food and Drug Administration and block younger teenagers from buying the so-called "morning after pill" without a prescription.

Senator Murray also called for a Senate hearing on the topic. She asked the Secretary to testify in front of a Senate committee to explain her scientific views on the matter. Senator Murray stated, “I want to know what the scientific evidence is that the secretary made this decision on in overriding the FDA … Pharmaceutical companies here in this country make some very expensive decisions, and they need to know that the FDA is going to base a decision based on science.”

The hearing hasn’t happened yet.

Will it happen now that the election is over?

It should. And the hearing should broaden its scope to address the ability of the Secretary of Health and Human Services to reverse FDA decisions and whether the FDA Commissioner should serve a congressionally mandated six-year term in order to ensure the position is “above” the political fray – similar to that of the Director of the FBI—and then approved by the Senate.

Let the person chosen as FDA Commissioner serve as free of the political current as possible.

And a Senate hearing would be a good place to start.  

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When Puppies Fly

11/09/2012 08:16 AM |

John Boehner: “Obamacare is the law of the land.”

But what does that mean?

“For our district and for our country, the debate on Obamacare is over,” declared Bill Foster, a Democrat elected Tuesday to the House from a suburban Chicago district.

Not so fast.

As the New York Times reports, “Now comes another big hurdle: making it work.”

Jeff Goldsmith, a health industry analyst based in Virginia, offers a more pragmatic assessment, “If you actually are going to implement this law, people need to know what’s in it — not just the puppies-and-ice-cream parts.”

And, as any owner of a new puppy can attest -- it’s messier than it looks.

Aliter catuli longe olent, aliter sues.

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Show me -- or else.

11/08/2012 08:30 AM |

It seems that, in Missouri, the natural way to stop an ObamaCare-mandated state health exchange is to insist it be put to a vote. (Under the Patient Protection and Affordable Care Act, states have until 2014 to establish state-based insurance exchanges that may be run by the state, the federal government or through a partnership of both.)

On Election Day the Show Me State passed Proposition E, prohibiting the governor or any state agency from establishing or operating state-based health insurance exchanges unless and until they are authorized either by the legislature or by popular vote. Proposition E allows taxpayers to sue any Missouri state worker or agency involved with any part of the exchange process not required by federal law.

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Four More Years

11/07/2012 07:28 AM |

The next four years are going to be a battle over whether or not Uncle Sam continues to morph into Uncle Sam, MD.

Consider:

Uncle Sam as our nation’s largest payor.

Uncle Sam as funder of comparative effectiveness research.

Uncle Sam as “academic” detailer of comparative effectiveness research.

Uncle Sam as determiner of Essential Health Benefits.

Consider the continued disempowered physicians, patients -- and states (which are the true laboratories for healthcare delivery innovation).

Consider a battle royale over whether or not comparative effectiveness should become a third leg for FDA approvals (in addition to safety and efficacy).

Consider whether CMS reimbursement will move to a value-based reimbursement model, setting up the foundation for a US version of Britain's NICE. (Remember Don Berwick?)

Consider Medicare and Medicaid adopting the NHS model of "expanded access" (formerly known as "risk sharing") wherein reimbursement is granted -- but if predefined patient outcomes aren’t achieved, the pharmaceutical company reimburses CMS. Hm.

Consider FDA Commissioner Peggy Hamburg.  Will she stay or will she go? (I predict she will stay.)

I predict that (regardless of who gets elected) IPAB gets revoked on the Hill. IPAB is a slippery slope to broader government price controls, so it should be good riddance to dangerous rubbish – if the President doesn’t veto this first congressional rebuke to ObamaCare.

And what of the legitimacy of Billy Tauzin’s famous ACA “deal?” Will there be legislative action to revoke the Non-Interference Clause (as mentioned by Vice President Biden -- although not by name -- during the Vice Presidential debate)? Unlikely.

Will the President continue to push for 7 years of data exclusivity for biologics (as opposed to the 12 currently allowed under the ACA?) Will he have the chutzpah to claim he is pro-innovation if he does?

Will we have to endure another foolish waste-of-time debate over drug importation?

And did somebody say “Doc Fix?”

Ladies and Gentlemen, fasten your seatbelts and start your engines.

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The Ex(clusivity) Factor

11/06/2012 07:35 AM |

Those who believe that they are exclusively in the right are generally those who achieve something. -- Aldous Huxley

Rather than speculating as to where the FDA may go in a post-PDUFA V world, it's important to focus on where it has already gone. Without much attention or fanfare, the agency has established a new review board within the Center for Drug Evaluation and Research – the CDER Exclusivity Board. Its goal is to help the agency make consistent findings on whether products should be granted periods of marketing exclusivity.

The board began meeting in November 2011. It is comprised of agency employees from the Office of Regulatory Policy, the Office of Medical Policy, the Office of New Drugs, the Office of Pharmaceutical Sciences, the Office of Executive Programs in CDER and the Office of the Chief Counsel.

The focus is on five-year new chemical entity exclusivity, three-year new clinical trial exclusivity, and exclusivity for biological products, according to a brief description posted on the agency’s website.

“The board will not review or make recommendations with respect to all exclusivity determinations in these areas, but will assist the center in resolving certain matters, including issues that arise in the context of specific requests for exclusivity,” the web page states.

The board generally meets once or twice a month. Members review written submissions from sponsors and board members participate in meetings with sponsors regarding exclusivity determinations for their products.

Some exclusivity matters fall outside the board’s purview: The body generally will not make recommendations for 180-day generic drug exclusivity, seven-year orphan drug exclusivity, or six-month pediatric exclusivity. But it will work with other offices and groups within FDA that are responsible for dealing with these exclusivity determinations as needed.

Your PDUFA dollars at work.
 

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The FDA Agenda Setters

11/05/2012 08:12 AM |

Matt Herper of Forbes, in his article, Why Presidents Don't Shape The FDA, writes:

Generally speaking, Democrats like Obama want to ensure that medicines are safe and are less concerned with being friendly to the pharmaceutical industry, and Republicans like Romney believe in lowering regulatory barriers and getting medicines to market faster. But the reality is no president makes the FDA his top priority — which means that the agency is often shaped as much by the opposition as by the commander in chief.

In the bigger picture, this is an illustration that when it comes to reshaping federal agencies, Presidents are not as powerful as you might think — and a reminder that just because a politician campaigns for a change does not mean that he or she will be able to execute it.

The important truth to remember is that the FDA is an agency driven be career staff.  Of the 11,000 or so employees, under a dozen are political appointments (including the Commissioner). And all of those Schedule Cs reside within the Office of the Commissioner. That means 100% of employees in every FDA center are career government workers. Put another way – drugs are being reviewed exclusively by career employees.

To refer to the “Obama FDA” or the “Bush FDA” or a future "Romney FDA" is valid only insofar as the presidentially-appointed  Commissioner sets an agenda. And that is if the Commissioner has an agenda and (most importantly) can enlist senior career officials to buy into it.

During my tenure at the FDA, Commissioner Mark McClellan was able to convince the career leadership that the role of the agency was to be regulator and colleague to industry and, most importantly, change-agent. I believe we had many successes because of this agenda – and the public health was well served.

The most important tool any President has to impact the performance of the FDA is in his choice of an FDA Commissioner. And that appointment must be confirmed by the United States Senate.

Herper concludes:

From an FDA perspective here, the lesson may be that it’s best to insulate the agency from politics as much as possible. One way to do that, according to Peter Pitts of the Center for Medicine in the Public Interest, would be to put FDA commissioners on six-year terms, protecting them from political churn.

More to the point, a six-year term would allow a Commissioner to more fully pursue his or her agenda.

Neither President Obama or Governor Romney has demonstrated any interest in a fixed term for the Commissioner.  At least not yet.

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Power to the Patient

11/02/2012 08:20 AM |

While many on the East Coast are thinking about electrical power, a new global study by the IMS Institute for Healthcare Informatics shows that the use of healthcare IT to increase medication adherence could be a key factor in saving some $500 billion in healthcare spending worldwide -- and that a key factor is the power of information.

“Harnessing available information to set priorities, monitor progress and support behavior change among healthcare stakeholders – including policymakers, payers, clinicians, nurses, pharmacists and patients – is a vital first step,” he said.

Aitken said the increasing use of data in healthcare makes this a good time to apply the levers suggested by the study to lower healthcare costs, which are:

• Increase medicine adherence by addressing patient beliefs and behaviors at the point of prescription and during medicine intake.
• Ensure timely medicine use that prevents avoidable and costly consequences among patients with highly prevalent diseases that increase in severity if diagnosis and treatment are delayed.
• Optimize antibiotic use to turn the tide on rising antimicrobial resistance worldwide due to the misuse and overuse of antibiotics.
• Prevent medication errors throughout the medicine provision pathway, from prescription to administration.
• Use low-cost and safe generic drugs where available to leverage the under-exploited opportunity in post-patent expiry markets.
• Manage polypharmacy where the concurrent use of multiple medicines, particularly among the elderly, risks costly complications and adverse events.

“Not all of this is new. Adherence is not new,” he added. What is new, however, is the ability to use data and predictive modeling to find which patients best respond to what type of medication adherence reminders, he said. Some need a visit from a nurse, which is more costly than using a text or a tweet. Predictive modeling can help an organization use resources wisely to get the most adherence from patients.

The study also focuses on two key factors critical to driving improvement across the six levers: multi-stakeholder engagement and … the power of information.

Knowledge is Power.

The study can be found here.

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