FDA

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When CMS approved Solvadi, Gilead’s $84,000 drug for hepatitis C, the stakes were raised in drug price wars.  Two opposing forces, one, a financial push toward lower costs came up against an opposing force of public sentiment. The FDA’s goal of getting 90% of patients moved from costly branded prescriptions to generics met with an an large outcry in social and traditional media for providing the best available care, rallying around the story of a patient.   The wave of sentiment seems to have won over CMS.

Granted, CMS was likely considering the reversal in its policy on Solvadi, but it was the May 12th coverage by the Kaiser Family Foundation and NPR of the patient who was denied treatment, and the amplification across social media that turned the tide toward coverage.

Solvadi had not been approved by the patient’s prescription drug carrier, so physicians lobbied CMS for coverage of Solvadi and the life of the patient. Solvadi appears to cure liver cancer in 90% of the patients who take it as recommended. CMS agreed. As a single payer, they have the incentive to balance drug costs and benefits with other costs and benefits, and new therapies often win the fight for coverage.

Getting Covered: The decision to pay for drug combinations is often quicker than FDA approval of the drug combinations

Objective health policy observers such as KFF note that in the early days of successful antiviral drug treatment for HIV, payers allowed doctors to “mix and match” medications in “off-label” or unapproved combinations as they thought best.  Medicare is often slow to approve the physician-driven cocktails, so getting CMS to adopt the strategy was a win for many very sick people in this country, as it sets a precedent for “exceptions.”

One doctor at Beth Israel Health System in Boston has a trial that has shown that combining Solvadi with another high-cost treatment, Olysio (by Janssen, cost $66,000 for a course of treatment) resulted in 90-100% cure rate.

The CMS statement in this case noted that that “the new policy will apply broadly to hepatitis C patients whose doctors prescribe the combined use of the two drugs because they meet certain criteria laid out in January by the Infectious Diseases Society of America and the American Association for the Study of Liver Diseases.” Those guidelines recommend the combined use of the two drugs in patients with advanced liver disease who have failed to be cured by earlier drug regimens – even though the FDA has not yet approved the combination—because Medicare guidelines say a patient must have access to a therapy if his or her condition warrants it.

Continue reading “What’s the Value of a Cancer Cure?”

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flying cadeuciiThe Food and Drug Administration has spent decades refining its processes for approving drugs and devices (and is still refining them), so what would happen if they extended their scope to the exploding health software industry?

The FDA, and its parent organization, the Department of Health and Human Services, are facing an unpleasant and politically difficult choice.

Sticking regulatory fences into the fertile plains of software development and low-cost devices will arouse its untamed denizens, who are already lobbying Congress to warn the FDA about overreaching. But to abandon the field is to leave patients and regular consumers unprotected. This is the context in which the Food and Drug Administration, the Office of National Coordinator, after consultation with outside stakeholders, released a recent report on Health IT.

I myself was encouraged by the report. It brings together a number of initiatives that have received little attention and, just by publicizing the issues, places us one step closer to a quality program. Particular aspects that pleased me are:

  • The suggestion that quality programs should start to look at electronic health records (p. 8). EHRs have been certified by various bodies, but usually just to check off boxes and declare that the systems comply with regulations–neither the quality of their user interfaces nor the quality of their implementations have been questioned. Reportedly, the FDA considered “safety and quality standards” for electronic health records in 2010 but couldn’t get them adopted. It also checks certain forms of clinical decision support, but only if they are built into a regulated device. The current HHS report refers back to aspirational documents such as a Health Information Technology Patient Safety Action & Surveillance Plan and a set of guidelines on the safety of EHRs.
  • A call for transparent reporting and sharing of errors, including the removal of “disincentives to transparent reporting”–i.e., legal threats by vendors (p. 25). Error reporting is clearly a part of the “environment of learning and continual improvement” I mentioned earlier. A regulation subgroup stated the need most starkly: “It is essential to improve adverse events reporting, and to enable timely and broader public access to safety and performance data.” Vague talk of a Health IT Safety Center (p. 4, pp. 14-15) unfortunately seems to stop with education, lacking enforcement. I distinctly disagree with the assessment of two commentators who compared the Health IT Safety Center to the National Transportation Safety Board and assigned it some potential power. However, I will ask ONC and FDA for clarification.
  • A recognition that software is part of a larger workflow and social system, that designing it to meet people’s needs is important, and that all stakeholders should have both a say in software development and a responsibility to use it properly.

Don’t imagine that the FDA is unused to regulating software. For quite some time they have instituted practices for the software used in some medical devices , and have tried to keep them up-to-date.

A waterfall-like process of risk assessment and testing called computer system validation has long been required for pharma and devices.

Continue reading “Health IT: The Coming Regulation”

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An advantage astrologers have over genetic testing is that the prediction of astrologers are personally verifiable. An astrologer once emphatically stated that I had no chance of a career in international cricket or Bollywood. So far both claims have turned out to be remarkably accurate.

How does one personally verify a “12.5 %” increased chance of lung cancer, the sort of number the vendor for genetic testing 23andMe produces? If one develops lung cancer how would one know that the chances were indeed 12.5 %, not 6.25 % or 25 %?

We die only once. Whether one ends up with lung cancer or doesn’t, the veracity of the claim can be made only empirically. Meaning we need to see how many develop lung cancer out of 10, 000 people just like us.

Yet there is an element of scientific precision in the number, augmented by the decimal point. And it is precisely because genetic testing tends towards science not metaphysics that it falls within the dominion of the Food and Drug Administration (FDA). FDA does not regulate palm readers.

FDA has asked 23andMe to stop sales of its genomic testing.

As a libertarian seeped in the Austrian school of Economics, I am generally disposed against regulations. I also share the sentiments of the monetarist Milton Friedman that the true costs of the FDA must also include the treatment opportunities foregone in their lengthy review process.

So it hurts me to be somewhat sympathetic of FDA’s stance on 23andMe, even as I think an outright ban was a tad harsh.

Continue reading “One Libertarian’s Dilemma: Genetic Testing or the FDA”

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It’s been clear for more than a decade that trans fat is a dangerous substance that increases the risk of heart disease.  Denmark banned its use in 2003.  Several American cities and states have followed suit, but the use of trans fat is still widespread despite the availability of suitable substitutes.

Over the past 10 years, trans fat consumption is thought to have contributed to an estimated 70,000 needless American deaths. Given  that universal, voluntary cooperation to eliminate trans fat hasn’t happened, the Food and Drug Administration (FDA) is justifiably seeking to designate trans fats as unsafe.

A nationwide ban on artery-clogging artificial trans fat is a long-overdue first step toward improving American diets, fighting obesity and limiting the risk of chronic disease. But it is just the first step in what should be a far broader campaign to help consumers make healthier choices at mealtime.

Public lack of awareness of the impact of prepared foods on individual health is not limited to trans fat.   When dining out, even in establishments that avoid trans fats in preparing food, Americans face a range of health risks often without realizing it. People are routinely served far more calories than they can burn.

They are routinely served too many low nutrient foods and insufficient quantities of fruits, vegetables, and whole grains.  What should become routine instead is the availability of menu options that put people’s health first.

Hopefully, the FDA’s trans fat initiative will succeed – previous city/state bans and labeling improvements have already managed to cut daily consumption by Americans from 4.6 grams in 2006 to 1 gram in 2012 – and pave the way for the creation of other standards and regulations regarding the quantity and quality of food that is offered to diners in restaurants.

The lack of such standards makes it difficult, if not impossible, for most people to recognize when they are being put at risk for a chronic disease.   If people are served too much of something (like calories), they would have to compensate by eating less later; conversely, if they are served too little of something (like vegetables), they would have to eat more later to neutralize the risk of chronic disease.

But most people lack the information they need to judge or track the quantity and quality of the nutrients they consume.

Continue reading “The FDA Ban on Trans Fat Should Be Just the Beginning”

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Emergency doses of a meningitis vaccine not approved for use in the U.S. are likely to be on the way to Princeton University to halt a meningitis outbreak that has already sickened seven students. There are approved meningitis vaccines available, but they do not protect against Meningitis B—a strain not covered by the shots given in the U.S. and not a strain prevalent here.

Government health officials said Friday they have agreed to import Bexsero, a vaccine licensed only in Europe and Australia that does protect against meningitis B.  And that decision seems entirely reasonable given the threat that this nasty strain of meningitis poses.

That said, the question arises — what should those getting the vaccine be told?  Are they being offered a proven vaccine, an experimental vaccine, a vaccine believed to be the best choice given the threat of an epidemic or something else?  Can a student, campus worker or faculty member refuse the vaccination and stay in school or in a dorm?  Should those who have visited the campus recently be tracked down and offered the vaccine?  If people do refuse should they identify themselves in anyway as unvaccinated and to whom?

In general when using a drug or device that has not been approved by the FDA or other federal advisory agencies those who are offered the vaccine should be treated more as research subjects than patients.  They should be told all the facts about the vaccine, why it has not been approved in the U.S. and about the all too real threat that meningitis poses.  They should be given the opportunity to ask questions.  There is a duty to try and monitor those who get the vaccine or a representative sample of such persons, to watch for both efficacy and safety.  Vaccine refusal might be accepted, herd immunity could help in this regard, but university officials will need a policy concerning refusers and where they ought to go to study, live and work until the potential epidemic subsides.

Using a vaccine very likely to be safe and effective to stave off an outbreak of a nasty disease makes good moral sense.  However, it is important to treat emergency use as such and to do what can be done to inform subjects, track the results in those who are vaccinated and to find a path for those who will not accept vaccination.

Arthur Caplan, Ph.D. is head of the Division of Bioethics at New York University Langone Medical Center. This post originally appeared in bioethics.net blog.

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Between October 1 and 17, the federal government ceased all nonessential operations because of a partisan stalemate over Obamacare. Although it is premature to declare this the greatest example of misgovernance in modern U.S. Congressional history, this impasse ranks highly.

One casualty of the showdown was any consideration of changes to lessen the impact of the across-the-board sequestration cuts that began on March 1. The cuts have caused economic and other distress across the nation, including serious impacts within the health care sector. Nearly eight months into sequestration, we can move beyond predictions and begin to quantify these effects.

Consider the following impacts of sequestration on Federal health agencies and activities:

NATIONAL INSTITUTES OF HEALTH

Cuts to the FY13 budget: $1.71 billion or 5.5%

This includes:

A 5.8% cut to the National Cancer Institute, including 6% to ongoing grants, 6.5% to cancer centers, and 8.5% to existing contracts

A 5.0% cut to National Institute of General Medical Sciences, and a 21.6% drop in new grant awards

Among the effects:

  • 703 fewer new and competing research projects
  • 1,357 fewer research grants in total
  • 750 or 7% fewer patients admitted to NIH Clinical Center
  • $3 billion in lost economic activity and 20,500 lost jobs
  • Estimated lost medical and scientific funding in California, Massachusetts, and New York alone of $180, $128, and $104 million respectively.

Dr. Randy Schekman, whose first major grant was from the National Institutes of Health in 1978, said winning this year’s Nobel Prize for Medicine made him reflect on how his original proposal might have fared in today’s depressed funding climate. “It would have been much, much more difficult to get support,” he said.  Congresswoman Zoe Lofgren (D-Calif.) noted the irony that because of sequester cuts, NIH funding was reduced for the research that resulted in Yale’s James Rothman sharing in the 2013 Nobel Prize for Medicine.

Continue reading “The Sequestration Cuts That Are Harming Health Care”

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The shutdown could not stop the rollout of the state and federal exchanges.

That’s because the Obama administration, sensing a political fight in the offing with Republicans, wisely prepaid the bill for the insurance exchanges and other key components of the rollout.

On the other hand, the fiscal standoff is having a very real impact on the infrastructure that supports healthcare across the United States.   Agencies from the Centers for Disease and Control to the National Institutes of Health have seen their money turned off. Others have seen their staffing levels sharply reduced with non-essential employees furloughed.

It doesn’t take a wild imagination to imagine potential deadly consequences if something goes wrong. If for example, flu season strikes early or a drug recall  is needed.  Much of the pain will be felt over time.  As the shutdown drags on, you can expect problems that are brewing under the surface to become much more visible …

Here’s a review of what’s happening:

Centers For Disease Control and Prevention
Funding for monitoring of disease outbreaks turned off. Lab operations sharply scaled back. 24/7 operations center to remain online.  With some scientists predicting a severe 2013-2014 flu season, this is cause for concern …

National Institutes For Health
Enrollment in new clinical trials suspended, impacting thousands of patients suffering from serious diseases. No action on grant proposals. Minimal support for ongoing protocols.

Food and Drug Administration
Food safety inspections sharply cut back. Monitoring of imports eliminated.  Oversight of production facilities curtailed, again potentially an issue with flu season on the way.The good news? Because drug approvals are funded by industry “user-fees” FDA approvals of new drugs will continue.

Centers For Medicare and Medicaid Services
Key ACA related operations intact.  The bad news for docs and patients – claims and payment processing expected to continue but with slower service than usual. With purse strings tight, this is likely to become more of a problem as shutdown drags on. In the unlikely event that a shutdown continues for more than a month, the impact on physician practices could be much more serious.

Continue reading “How the Federal Government Shutdown Is Hurting Healthcare: Agency by Agency”

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Health care providers and consumers are increasingly using mobile technology to exchange information. Many health IT providers readily acknowledge that some level of oversight is required to ensure patient safety and privacy protections, but many providers question whether the FDA is the right agency for the job and want to see the FDASIA recommendations.

Can the FDA, with its already limited resources and lengthy review cycles, regulate the fast-moving health IT industry? Should it? Health IT is fundamentally different from a medical device in many ways. For oversight purposes, the key differentiator between the two is the opportunity for clinical intervention in the use of health IT. Many medical devices interact directly with the patient (such as an infusion pump or pacemaker). Most health IT, on the other hand, merely provides information to clinicians, who ultimately make independent, experienced care decisions. Physicians are informed, but not controlled, by the information. This leads to a vast difference in the patient risk proposition and rigid regulatory oversight is not appropriate.

Advocates of a broad health IT oversight framework – which encompasses mobile health IT – are urging the FDA to delay release of its final guidance, particularly in light of a July 2012 Congressional mandate for the creation of a comprehensive oversight framework that avoids regulatory duplication.

But some mobile medical application developers are pressing the FDA to move forward immediately, believing its guidance will reduce the regulatory uncertainty that they believe is stifling innovation and investment in some aspects of mHealth.

Continue reading “Regulating Health IT: When, Who, and How?”

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Here’s an interesting clinical dilemma brought to my attention by another physician.

She was asked to refill a prescription for a drug called domperidone to help a patient with lactation. Domperidone is not FDA approved in the United States for any indication. However, in Europe and in Canada it is approved as a promotility agent for patients with a condition called gastroparesis, which causes the stomach to empty very slowly and results in chronic nausea and vomiting. As a side effect the drug is also known to increase the production of prolactin, a hormone that stimulates milk production. In the case of this physician’s patient, she had adopted a child and found that the medication had effectively enabled her to produce milk and nurse, with seemingly no untoward effects. It’s unclear who had initially prescribed the drug, but various online lactation support forums discuss it as an option  for women who have trouble with lactation.

The questions: Is it legal, ethical or good medical practice for a physician in the United States to write a prescription for domperidone for a patient who has been using it for lactation with good results? How about for gastroparesis? Where does one get the drug? Is it even legal to sell the drug in the United States?

I’ve cared for at least two patients who have used domperidone. In both instances it was ordered by prescription from an overseas source by a local gastroenterologist. In these two cases my patients had tried just about everything on the market in the United States for gastroparesis and were still struggling with debilitating symptoms. In one case, my patient had required hospitalizations and ultimately a feeding tube because of intractable vomiting. The drug was ineffective in both patients and it was eventually discontinued.

Continue reading “Should a Doctor Prescribe Drugs that are Unapproved by the FDA?”

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Over four years of Congresses, Sage Bionetworks has drawn together leading thinkers and doers throughout the fields of genetic research and drug development. For two days each year, the conference floor is colonized by clumps of eagerly networking PhDs from academic, pharma, government, non-profits, biotech firms, and patient advocacy groups–people who often glide from one domain to another within this tight-knit cohort.

A cohort, certainly, we can characterize this group of attendees, sharing as they do a mysterious language drawn from years of research most of us will never understand. But is it a community? That will be tested over the following year as Sage Bionetworks lets go of the Congress. Founder Stephen Friend says it is up to others to create the next Congress, and its success or failure will be a measurement of the sweat and passion that Friend and Sage have put into attempts to build a community.

Why should a reader look further at this struggle among a tiny elite, rather than clicking on the next article? Well, first, if you’re one of the 48% of Americans who took a prescription drug this month, you should be concerned about where new breakthrough drugs will emerge. If you visit this web site because you want a more responsive health care system that can match patients to treatments more quickly and cheaply, recognize that new methods are important nowhere as much as at the foundation of the system where new treatments are discovered. And if you are just curious about the potential for global cross-institutional teams and loose networks connecting experts with ordinary members of the public to find creative solutions to old problems, this article will provide insights.

Don’t get too close, you don’t know what I have

The premise on which Friend founded Sage is that research and drug development have stagnated and cannot progress without more collaboration and data sharing. Therefore, with all due regard for the presentations at the recent Sage Congress on cancer research projects and other individual experiments, the real theme of the conference is in the keynotes about open source, the use of social media, and crowdsourcing. The challenge of this community–if we find that it has indeed become a community–is to analyze and deal with the particular challenges that genetic research and drug development inject into trends toward open collaboration.

Continue reading “Open Research For Open Cures: A Report From Sage Congress”

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