Over the years, solutions to this problem were placed in the hands of law enforcement, which, among other measures, monitored physicians to assure they were not enabling existing addicts or creating new ones. Later, pharmacists were monitored as well and soon became engaged in measures of their own to prevent abuse. Recently, under the guise of patient safety, FDA joined in.

Intrusions led physicians to fear prescribing pain medicines for legitimate medical purposes and warned pharmacists away from essential community-based pain management. Law enforcement and regulatory barriers made it more difficult—even personally threatening and certainly more costly—for them to do so.

Isn’t it ironic that the law and the healing arts should clash? Early civilizations considered both to have been gifts from God. Both were intended to serve man, not to victimize the most vulnerable. Yet, today’s clashes have done precisely that, and jeopardize those in pain.

Those near death for whom society should have the most compassionate response have been harmed as a result. They have immediate needs that, in some cases, only powerful medicines can help.

Unrelieved pain takes a terrible toll on patients and their loved ones. Unable to get help from physicians and incapable of negotiating the maze of intrusive legal restrictions on their care, at one time families turned to politicians for intervention. They asked Congress to legalize heroin as a pain treatment. Thankfully, that era’s street drug-of-choice was not needed – we had better drugs than heroin. Congress did not grant their wish. But the families were right about one thing: those medicines were not being used. I know their frustration and anger. I was witness to the devastation they felt when loved ones had died, in pain unnecessarily.

Families took matters into their own hands in other ways. Some sued physicians for their failure to provide adequate pain relief. Imagine the physician’s dilemma: law enforcement sanctions for prescribing vs. malpractice suits for not.

Some aspects of pain management have improved since then and quality care standards monitor pain relief in institutions. Whatever improvements we achieve, however, are at risk if hysteria about abuse of legitimate medicines resurges. Relatively recent news reports about the illicit use of important medicines are only the latest—and certainly not the last—example. Press coverage about OxyContin abuse vilified—and victimized—the medicine, those who make it, and those who need it.

When law enforcement and medicine are at odds, dying people suffer all the more and my primary concern is for them. When some of us abuse medicines, those of us who use those same therapies for legitimate medical reasons become suspect.

Can’t we find a better way? Can’t we reduce suspicions about the valid use of pain medicines and correct the tragedy of under-prescribing and under-administering these critical solutions?

We can and we must. There are ways we can start:

• End the witch hunts against clinicians and protect them from harassment as they provide patients with medicines while we sort out our differences about legitimate prescribing and drug-abuse prosecution. Doing this will help patients now.
• Provide clinicians with immunity from malpractice litigation for failure to provide pain medications while we sort out the law enforcement issues. Doing this will help patients now.
• Radically accelerate improved clinical practices of pain management. Some efforts are underway nationally, but they’re insufficient and patients with only weeks or months to live cannot wait the average 17 years for even simple changes in clinical practice to change. 
• Require any individual who legislates, regulates, enforces or reports in the media on matters related to the abuse of pain medications work in hospice care with patients experiencing pain for a least one week. Doing this will help patients in the future, bringing better balance to future policy making, enforcement and reporting activity.
• All of us should resist the temptation to allow those of us who abuse pain-relieving medicines to numb our compassion for those who need them.

We must balance healing and the law better. Let’s not forget the compassion we should have for those in pain, as they live and especially as they die.

Can it be done? I believe so, and draw optimism for the future from my brother, Jim, and his wife Nancy, who for more than three decades volunteered their time to hospice. Jim wants to write a book. He’d call it, “You Live, Until You Die.” It comes from his experience of seeing the old and the sick die well, living—and often laughing—right up to the last moments. It would be a book of warm, tender, and funny stories. His stories echo the words of one cancer patient who helped us understand the importance of pain management back in my government days. She said, “I found that when I didn’t have the pain, I could forget I had cancer.”

How can we not embark on these changes?

To do less is to diminish the living of everyone, the dying as they leave us and the ones they leave behind. 

Cook Medical’s Zilver PTX is likely to become the first peripheral drug-eluting stent (DES) to be approved in the U.S., after an FDA advisory panel voted unanimously in favor of the device on Oct. 13. Approval would give the sponsor, Cook Medical (Bloomington, Indiana) access to a peripheral arterial disease (PAD) market valued at $1 billion, depending on whose figures one relies.

I attended the Oct. 13 advisory committee hearing for the device and observed the panel members comment that this was among the best submissions they had seen in some time. Cook presented a clear study that met all primary endpoints and showed improvement over percutaneous transluminal angioplasty, the current standard of care. To non-FDA experts like myself, it seemed as though Cook was recognized for setting a new bar for conducting clinical trials and collaborating with the FDA.

I imagine that Cook Medical’s leadership was able to make all the right clinical trial investments necessary for the long-term viability of the product’s market value, not just the ones that were on display at the advisory panel meeting.

Simply put, such a prudent investment for the long term is equally, if not more, essential when planning for the reimbursement success of any new medical device. Medicare already pays for peripheral endovascular interventions, both in the out-patient and the hospital in-patient settings. So as any “new” interventions receive FDA approval, such as a Zilver PTX, Medicare likely would pay for these within the existing payment levels. After all, these are existing technologies, using existing drugs, being utilized in a new application, so Medicare payment would default to those existing payment classifications.

How then can makers of novel devices – with clinical improvements over standard-of-care – seek premium pricing? Device developers must calculate the impact of a higher product price on customers’ (hospitals) before commercializing at a premium price. With the same Medicare reimbursement for the procedure regardless of stent cost, hospitals will think carefully about paying a premium to use a new device, such as the Zilver PTX. This makes premium pricing very difficult without some justifiable improvement elsewhere in the disease treatment continuum.

The Medicare reimbursement bar has been raised. To seek any additional product reimbursement for the hospitals, the device must prove it delivers “significant clinical improvement” over the standard of care currently reimbursed by Medicare. “Significant clinical improvement,” is a loosely defined standard, however, and is applied subjectively for each new technology. For example, does a new neurovascular implant have to show evidence of superiority over “standard of care” in a pivotal trial to justify a higher price? Is proof of non-inferiority of a new continuous glucose monitor sufficient? Is a separate study necessary? Should we invest scarce funding to support studies when non-inferiority is the goal?

I think the key to demonstrating substantial clinical improvement lies in demonstrating patients’ functional improvement – this could be walking, resuming activities of daily living, returning to work or reducing days away from work, and other patient-reported outcomes measures – in combination to show that the new treatment has improved a patient’s life. Medicare wants to see the measurable “So what?” quantified and compared to the “So what?” of existing treatment options. I am certain that Medicare will no longer be satisfied by clinical results that are considered equal to existing care. The mandated Medicare evidence bar – “significant clinical improvement” – is the key to product success. After all, “significant clinical improvement” is necessary to trigger a new technology add-on payment, reimbursable to hospitals in addition to existing payment.

Preparing to demonstrate significant clinical improvement for reimbursement purposes is an essential part of clinical trials strategy and execution – it must be built into planning and costs of clinical trials management. Doing so as part of clinical trials will accelerate time to market. Conversely, not having these data for Medicare may delay sales and revenues indefinitely.

Adi Renbaum, senior VP of health policy and reimbursement for the Neocure Group (Washington DC), joined the Neocure Group shortly after it was founded in 2006, and today heads its Washington DC office, where she leads the health policy and reimbursement practice. She has more than 17 years’ experience in the field of regulatory and clinical development strategies, and has negotiated directly with CMS and commercial payers to expand coverage and secure reimbursement for clients’ technologies. She also works closely with medical and specialty societies to build consensus about innovative products and for support with payers.

The history: In 2008, Avastin was given preliminary approval by the FDA for the treatment of breast cancer on the condition that the company would do more studies to demonstrate its effectiveness. Many women have been successfully treated with Avastin — a billion dollar drug for Roche.

But when Roche submitted the required follow-up studies in 2010, the data showed that there was no benefit from the drug for treating breast cancer.  Studies did not show significant impact on mortality or  improvement in quality of life.  In addition, the drug was associated with some significant side effects such as high blood pressure and blood clots.

In December, 2010, an FDA panel voted to withdraw the drug’s approval as a treatment for breast cancer.  Roche appealed the decision, and earlier this week, an FDA panel hearing the appeal unanimously decided, in a 6-0 vote, to withdraw the drug’s indication for breast cancer.  The final decision on whether or not Avastin loses the indication is ultimately up to the FDA Commissioner, Dr. Margaret Hamburg.

If Hamburg supports of the panel’s recommendation is critical. At the same time, this will fuel even more emotion and protests among patients. Here’s why:

• From the FDA’s side: The FDA issued a provisional approval with the explicit understanding that the final decision would be based on more conclusive studies. If the FDA maintains the drug’s approval despite the lack of scientific data, this would likely impact all of their future decisions to offer provisional approvals for potentially life-saving drugs.
• From the patients’ side: Women who have benefited from the medication have clearly voiced their opinion that the doctors and patients, not regulators, should decide whether or not to get potentially life-saving treatment.

An important fact to keep in mind is that Avastin will remain on the market because it is FDA-approved for certain types of lung, colon, kidney and brain cancers. Therefore, Avastin will still be available “off label” for treating breast cancer when patients and their doctors believe that it is the best option.  In those situations, the only real challenge will be a barrier to access is a financial one since expensive drugs used “off-label” are frequently not a covered benefit and very few women can afford to $80,000 per year out of pocket for Avastin.

Roche is understandably passionate about making this drug available to women, and they will be pursuing additional studies to prove its effectiveness.  But, in the mean time, there is a solution that’s not been discussed and doesn’t involve beating up on insurance companies: For women who decide (despite the data) that they want or need Avastin, Roche could simply decrease the cost.

What do you think?

Originially posted on Archelle on Health on Friday, July 1st.

Over the next year, 10 million people will likely be scored (without their knowing it, by the way) within a rage of 0-500. Those over 400 would be deemed ‘likely’ to use medication appropriately; those with lower scores would receive calls, cards, letters or some other intervention to boost their rates of adherence.

Halfway into the article, my HIPAA-involved policy-wonk self went bonkers. True, they don’t use medical records information but the fact that without consumers knowing it FICO can amass and organize other public information such as home and automobile ownership, job status, marital status, age and gender, then show payers how the weights of those factors vary by diagnosis enlightened me about the power of data mash-ups. Truth be told, it worried me as well. Who knew?

Wondering if the premise was even valid and also about a surprising finding from FICO’s work that women adhere to medication regimens less often than men, I called my guru on ‘all things adherence,’ Diana Long, to inquire.

She said FICO might, in fact, be ‘on to something’ saying that we in health care have been myopic about patients’ behavior. We think it’s ‘all about us’ and don’t consider that not taking meds or following doctors’ advice might be a reflection of factors other than how sick we felt, what side-effects we feared or what costs we bore. She pointed out, for example, that just because a child knows how to use an inhaler does not mean they have mastered the strategic thinking/planning required to remember that tonight is the night for soccer practice, they should ‘plan ahead’ and take the inhaler to school that morning. The factors we develop as we grow up  – including skill and motivation – to get up for a job every day may be the same factors that determine whether we’ll take our meds, eat right or exercise.

But what about women’s lower rates of adherence? Here is where ‘too much of a good thing,’ can backfire, in this case for the woman. Diana reminded me of a time she preceded me in a speech. She arrived on stage with a basket and proceeded to unpack it, demonstrated what she – as Dr. Mom – managed vis a vis medications for her:

• Two young sons,
• Dog,
• Husband,
• Husband’s parents, and
• Husband’s Grandfather.

She managed medications for herself and also for six other people – and four generations – of the family…oh, and the dog.

She reminded me of the data…most women also manage the household. And they also have jobs. Eventually, that gets to be ‘too much’ and the consequence is often her failure to take care of herself.

This is a holiday weekend and it’s too late now, but for tomorrow and the future, I’m hoping all the Dr. Mom’s reading this will remember to move themselves further up on the ‘to do’ list.

If for no other reason, do it so that you won’t have the extra hassle of fending off a set of dunning reminders to do so which may soon come our way.

There’s evidence that by spending a bit more on medication and bolstering prescription drug adherence among patients, total health spending can be lowered for vascular medical conditions. The study and data which leads to this conclusion is published in Medication Adherence Leads to Lower Health Care Use And Costs Despite Increased Drug Spending appears in the January 2011 issue of Health Affairs.

The study cites the World Health Organization’s report from 2003 that stated medication compliance rates globally averaged about 50%. This number may be high compared with other newer studies on adherence, such as reports from

Peoples’ lack of adherence to medication treatment regimes costs: diabetes patients who do not take their medications have a 58% increased risk for hospitalization and an 81% increased risk of mortality. The New England Healthcare Institute (NEHI) calculated that more than half of all Americans currently suffer from at least one chronic disease such as diabetes, heart disease and asthma at a cost to the economy of $1 trillion annually.

The Health Affairs study, sponsored by CVS (the retail pharmacy chain), established a causal link between medication adherence and outcomes: specifically, hospitalizations and total health costs. The researchers examined four conditions under the umbrella of vascular disease: congestive heart failure (CHF), hypertension, diabetes, and dyslipidemia (high cholesterol). Among these four conditions, bolstering adherence had positive impact (i.e., lowered) health costs across all categories, most markedly for CHF, as shown in the chart.

The average benefit-cost ratios from adherence for the four conditions were 8:4:1 for CHF, 10:1:1 for hypertension, 6:7:1 for diabetes, and 3:1:1 for dyslipidemia.

Health Populi’s Hot Points:  Focusing just on CHF, the average cost-savings per patient was $7,893. About 5.7 million Americans have CHF, and there are 400,000 new cases of CHF in the U.S. each year, according to the National Heart, Lung and Blood Institute (NHLBI). Do the math: bolstering medication adherence among CHF patients could save $billions to the U.S., which would positively and directly impact Medicare — the nation’s largest threat to financial security in the not-so-long-term.

Furthermore, improving medication adherence as described in this study would avert hospital admissions for patients with vascular conditions, which would enhance millions of Americans’ quality of life and productivity. This is one example of how to bend the cost curve in health, and it doesn’t require a lot of new technology – just strong doses of sound communication between patients and doctors, access to prescription drugs and medication adherence programs, and a culture of participatory health where patients feel vested in their own care in partnering with their physicians.

Posted originally on Health Populi by Jane Sarasohn-Kahn on January 10th.

Logically, if the weight of public and policymaker opinion continues in this direction, that innovation undermines the greater national goal of affordability, then this will inevitably lead to policy actions that reflect this line of thinking.  We’re already seeing this to some degree with the weighty pharmaceutical and device taxes that are part of the new health reform law and the creation of an independent board with the power to slash Medicare reimbursements.  While the intention makes sense, it is not clear this path will yield the intended, and worse, may contribute to unintended and undesirable consequences.

There’s too much at stake, both economically and in terms of societal well-being, to travel far down this path without having an absolute certainty that innovation does, in fact, make health care less affordable.  Do we just take it on faith that innovation drives costs skyward, or is there empirical evidence that says otherwise? 

To be sure, the literature includes a wide range of research on the relationship between innovation, costs, prices, and value.  Like almost every aspect of health care policy and research, this one is layered in complexity.  Sound bites can be misleading, definitions matter, and important findings can be misconstrued for political purposes and philosophical positioning.

So much has been written about the relationship between prescription drugs, costs, and prices. What does the literature say about the drug prices as they relate to health care cost growth?

Last month, Dr. Ernst Berndt of the Massachusetts Institute of Technology and Murray Aitken of IMS Health published the working draft of a paper that makes an important contribution to this debate.  Berndt and Aitken take on one of the widely accepted statements about the relationship between innovation and affordability – that prescription drugs are getting more and more expensive – and ask whether this thesis is supported by available pricing data.

The authors took a look at the Hatch-Waxman Act, the legislation crafted by Congress in 1984 that granted drugmakers a period of market exclusivity on their new products and then promoted consumer access to generic versions of those drugs once that period ended.  Some have suggested that the effects of Hatch-Waxman are tilted too far toward the interests of pharmaceutical companies, as exemplified by the regular reports issued by AARP monitoring drug price increases.   In fact, AARP said the prices for the top 217 branded drugs went up 9.3 percent in 2009 even though the consumer price index declined.

But AARP and other drug price critics are failing to take into account the impact of generic drugs and how patterns of drug use change over time.  That’s what Berndt and Aitken examined – not the price of name-brand pharmaceuticals in a vacuum, but rather the real-world purchasing of consumers, health insurance companies and pharmaceutical benefit managers.  And when viewed through that prism, the average price per prescription, between 2006 and 2009, actually declined by 21.3 percent.    Bottom Line: any brand price increases were more than offset by lower costs for generics.  

Real drug prices have also been falling in the Medicare Part D prescription drug program.  In the first two years of the program, cumulative spending was $69 billion, significantly lower than the $105 billion projected by government budget authorities. 

If, in fact, pharmaceutical price escalation was causing hardship for consumers, then policymakers would feel compelled to take strong, if not extraordinary measures, such as pricing caps or some other form of real or de facto price controls.

But, as the evidence makes clear, that is simply not the case.  Consumers are paying less, not more, for the medicines they need.

What is clear is that, 25 years after its enactment, the Hatch-Waxman measure is still having its desired effect.  A period of market exclusivity and patent protection is serving as an incentive for innovative pharmaceutical companies to develop new medications to meet patient needs.  Yet, there is still ample space for generics to yield affordability and accessibility.

With our society’s health compromised by a variety of threats, from the rapid rise in chronic disease to the escalating number of antibiotic-resistant superviruses, this is not a time to curtail medical innovation.  And as the Berndt-Aitken analysis shows,  it’s not clear there’s a compelling reason to do so. 

While Berndt and Aitken look specifically at drugs, one can make a similar case for many surgical procedures that use a non-invasive approach taking longer to perform and costing a little more in some cases, but reducing the overall cost of the episode of care and improving patient recovery.  As new approaches in homecare monitoring develop, we will see the same phenomena illustrated. Innovation increases cost at first and then lowers costs as competition creates cheaper but better technologies. 

While advances in medical science enable us to diagnose and treat a far greater number of illnesses, it is duplication, waste, errors, and overuse that are our enemy. They are fostered by a care delivery system based on fee for service payments and penalizing coordinated and integrated of care. Only innovation and better coordinated care can improve quality, reduce costs and save our healthcare system.

* This post is part of the Disruptive Women series on innovation.

Orphan Diseases – Bellwether of Health Care

“The only people interested in rare diseases are those who have them, and that’s not a lot,” an editor once told me when rejecting my proposal for a book on orphan diseases.

In reality, nearly thirty million Americans – one in ten – suffer from some 7,000 rare diseases, making them collectively very common indeed.  Though they differ wildly in their causes and manifestations, they share many characteristics in terms of the uncertainty, isolation, and costs imposed upon sufferers and their families, the challenges they pose for our health care system and sense of social responsibility, and their potential value to medical research.

In the United States, orphan diseases were defined by the Orphan Drug Act of 1983 as those that affect fewer than 200,000 people domestically.  This landmark legislation offered incentives – tax credits, a period of market exclusivity independent of patent protection, research grants – for manufacturers to develop clinically valuable treatments that would not be economically viable in the normal pharmaceutical market.  Previously there had been tragic instances of known, potentially useful therapeutics never taken to market, or others of already proven effectiveness that were withdrawn, because they did not meet a drug company’s threshold of profitability.  The definition encompasses some familiar disorders, such as cystic fibrosis, muscular dystrophy, hemophilia, Huntingdon’s Disease, and Crohn’s disease, along with thousands of much rarer conditions, including fibrodysplasia ossificans progressive (FOP), Jumping Frenchmen of Maine, and Sakati Syndrome, or acrocephalopolysyndactyly type III, which for many years was represented by a single patient in Saudi Arabia.

In less than thirty years since passage of the Act, more than 300 orphan drugs have been approved for market.  Some are produced by large pharmaceutical companies.  The biotechnology industry also benefited tremendously from the orphan drug market and its incentives.  In addition, as one pharmaceutical executive told me, big drug companies may need billion dollar drugs to support not only R&D but also massive corporate infrastructures and the sales and marketing machinery, but a small company aiming at a well-defined population, with a small number of specialized physicians and facilities, patient advocacy organizations, and unusually motivated patients, can get by quite nicely on mere tens of millions of dollars in revenues.

Research on orphan diseases is often unusually productive.  William Harvey, who discovered the circulation of blood, wrote in 1657 that nowhere does nature more openly “display her secret mysteries than in cases where she shows traces of her workings apart from the beaten path; nor is there any better way to advance the proper practice of medicine than to give our minds to the discovery of the usual law of Nature by careful investigation of cases of rare forms of disease.”  Sigmund Freud more generally stated in the New Introductory Lectures on Psychoanalysis that studying abnormal behavior was valuable because the broken structures of mental patients’ minds were as revealing as the fracture planes of a shattered crystal, which yielded clues to otherwise invisible molecular structures.  Rare diseases, often resulting from a single gene mutation or flaw in one step of a metabolic pathway or developmental process, may reveal stark outlines of underlying mechanisms difficult to tease out of complex, multifactorial diseases like hypertension, diabetes, obesity, or cancer.

Clues provided by rare diseases like Fragile X syndrome, Gaucher’s disease, and tuberous sclerosis complex provide insights into the mechanisms of more complex neurological disorders; WAGR and Chanarin-Dorfman syndromes give clues on obesity and fat metabolism; Schnyder crystalline corneal dystrophy helps elucidate cholesterol metabolism defects that may be implicated in heart attack and stroke; alpha-1-antitrypsin deficiency causes emphysema at an early age, allowing the lung condition to be studied separately from the effects of smoking and other factors in later life; and the study of progeria, a tragic premature aging disease with only a few dozen patients living truncated lives worldwide, sheds light on the normal process of aging.

Research on other rare disorders has already yielded important clinical and pharmaceutical discoveries.  The study of kuru, a rare neurodegenerative disorder found among members of a New Guinea tribe that engaged in ritualistic cannibalism, led Stanley Prusiner to his Nobel Prize-winning identification of prions.  Studies of the rare condition homozygous familiar hypercholesterolemia led to another Nobel Prize-winning discovery and the development of the important statin family of drugs.  A few hundred people suffer from the adenosine deaminase deficiency form of severe combined immunodeficiency disease – a variant of “bubble boy disease” – but the treatment strategy developed for it, of decorating therapeutic enzymes with protective polyethylene glycol (PEG) polymer chains, inaugurated a whole family of PEGylated drugs now used for the effective treatment of many more common diseases.

One unfortunate omission in the Orphan Drug Act was the field of medical devices, which were not given the same incentives as pharmaceuticals.  FDA’s device regulations do provide for what are called “custom devices” – variants of a commercially marketed device that are prescribed for an individual patient, like prosthetics or dental implants.  In the early 1980s, Cook Medical, an Indiana device company that manufactured a bladder stent for ureteral obstructions, custom produced a diminutive version for use in fetuses.  Cook sold this device at cost, as it was an effective way to treat an otherwise fatal condition affecting 200 fetuses each year.  When the number reached 680, FDA decided that the stents were no longer custom devices, but manufactured products, whether sold at a profit or not.  Cook successfully lobbied for new legislation, and the Safe Medical Devices Act of 1990 included provisions for a Humanitarian Device Exemption.  Alas, Cook won the battle but lost the war, because the exemption applies only for conditions affecting fewer than 4,000 Americans per year, explicitly bars manufacturers from making any profit, and provides incentives too paltry to encourage and facilitate the sort of robust R&D projects seen in the orphan drug world.

In addition to the research and product development benefits, orphan diseases present models of health care collaboration and service delivery that, though perhaps not fully scalable to more common diseases, inspire one to think of more efficient and effective approaches, particularly in an era of increasing specialization and of finer diagnostic differentiation among subtypes of medical conditions.  My own first exposure to an orphan disease, more than twenty years ago, came through a chance meeting with Dr. Frederick Kaplan of the University of Pennsylvania, an expert on FOP, a rare genetic bone disease in which muscles, tendons, and ligaments are progressively transformed into bone.  What struck me most – after the tragic oddity of the disease – was the extraordinarily close and productive collaboration between FOP clinicians, basic researchers (including Drosophila geneticists studying homologous gene defects in a boneless creature), and the patient population, all working together with a sense of respect and combined purpose that I had never seen before.  These three distinct populations directly informed and powerfully inspired one another with their respective understanding of the disease, and all of them benefited from the experience.

Because of the level of communication within rare disease communities, and with companies developing diagnostics or therapeutics for diseases where such candidates exist – there was tremendous efficiency and economy in the sharing and evaluation of knowledge.  And if, in the end, a treatment was developed, the companies’ ability to market products directly to a well-defined physician and patient population enabled everyone to benefit, eliminating the massive waste of buckshot marketing.

Perhaps nowhere are some of the most critical issues in health care and its costs thrown into harsher relief than with orphan diseases.  One might justifiably say that our approach to rare diseases is a bellwether of how our society will meet health challenges and deal with vulnerable populations generally.  A utilitarian or egalitarian approach recommends policies that yield the greatest good to the greatest number, not the disproportionate allocation of more money to patients whose conditions are rare or severe.  At some point benevolence towards heart-rendingly tragic cases may compromise the well-being of many.  On the other hand, a belief in equal rights suggests that we should, to the extent possible, help all people to become whole, even the blameless victims of random genetic mutations, and provide medical care adequate and appropriate to their conditions whether they are commonplace or not.

In a time of health care reform, we all are conscious that medical care is expensive, that we need to control costs, reduce errors and inefficiencies, and foster a system based on evidence rather than on marketing.  But “expensive” has meaning only in the context of what we are trying to do.  Inevitably we will develop better methods of screening for potential problems, targeting drugs at those who respond best to them, customizing therapies, and monitoring responses.  This costs.  But if greater up-front costs in approaches to smaller and smaller, more orphan-like populations lead to longer term savings or improved patient outcomes, the equation shifts.  What are we trying to do?  How important is health?  And what is our moral responsibility as a society, as humans, to care for the orphans or for others with more common serious ailments and infirmities who are also, ultimately, alone and distressed in their suffering?

For further information on orphan diseases and drugs, see:

• Thomas Maeder “A Few Hundred People Turned to Bone,” The Atlantic Monthly

• FDA’s Office of Orphan Product Development

• Humanitarian Device Exemptions

• The NIH Office of Rare Diseases Research

• The National Organization for Rare Disorders

Our panel this morning discussed the issues surrounding how the WHI results were interpreted and communicated to women and their health care providers. We recognize that hormones are not appropriate for all women, and look forward to hosting a future panel that highlights alternatives.

The speakers have a variety of backgrounds and experiences (and genders), and we aim to promote diversity of voices.

This was not normal breakfast conversation.

Today was a jolting – and disruptive – talk about what happens to women’s bodies when they age. (Who knew that if you’re menopausal and you don’t take your hormones, your vagina can literally dry up and shrink?)

The second in Disruptive Women’s 2010 breakfast series, today’s talk was titled, “News (Hot) Flash: Sex, Drugs & Menopause.” The breakfast at Johnny’s Half Shell, was sponsored by Medco – and we’re happy to say there were two men in the audience this month – double last time.

The breakfast started with a screening of trailer for the upcoming movie Hot Flash Havoc. (Think: Michael Moore tackles menopause.)

“It’s not available in theaters yet – but it will be,” said Disruptive Women’s Robin Strongin, introducing the film.

I’m genuinely sad that the documentary isn’t being released until October-ish. Normally, I don’t want to watch anything at 7:30 in the morning, but the little bit that was shown was so funny I can’t wait to see the whole thing. (Seriously, put it on your Netflix queue now.)

Introducing the speakers, Strongin briefly summed up a woman’s life cycle. “You start out life in this estrogen gel – like a gefilte fish,” she said. “Then you hit puberty, you’re either fertile or you’re not, then you’re pre-menopausal, then menopausal, then post-menopausal. Then you die.”

The talk today focused on the menopausal portion of the life cycle. Phyllis Greenberger, President and CEO of the Society for Women’s Health Research, started off speaking about the Women’s Health Initiative. “There was a lot of misinterpretation, some of the results reported were incorrect,” she said.

She quickly explained what they did, what was wrong, and what’s true today. The Women’s Health Initiative was a giant study of postmenopausal women, testing whether hormone replacement therapy could help prevent cardiovascular disease, cancer, and osteoporosis. The results were different for different age groups – women starting hormone therapy in their 70s had generally bad outcomes (increased risk of heart attack, breast cancer, stroke, etc.), while women starting in their 50s had generally good outcomes. But the results widely reported were the negative ones from older participants – so many women never heard about the rest of the research, or anything we’ve learned since!

The next speaker was Dr. James Simon, a clinical professor of obstetrics and gynecology at the George Washington School of Medicine – and a menopause researcher. (But he will forever be remembered to me as the man who scared the crap out of me about the future health and wellness of my vagina. I may not sleep tonight.)

The Women’s Health Initiative’s results scared a lot of menopausal women into quitting their hormones cold turkey. That, is a very bad idea, he said. Going off hormones makes women unhappy and unpleasant, but more disturbing, he said, “when women go off their hormones their vaginas dry up and get smaller.”

(!)

Which makes sex painful – so women stop having it. And, he says, marriages today have enough problems without eliminating sex (or arguing about it).

“No one wants to have sex when it hurts…. You can’t have good sex with a dried-up vagina. That’s a fact,” he said. “I can give you a two-hour lecture on why the parts don’t work.”

Uhm, great. Go on.

Instead, he told a horrifying story about one of his 55-year-old patients – a prominent writer for the Washington Post, who came to his office for her annual healthy woman exam. He asked her how she was feeling, how were things with her husband, how’s their sex life? Good, good, good, she said. Everything was fine.

Then she put her feet in the stirrups.

“I couldn’t even put the speculum in because it’s too shrunk and dry and small,” he said. “I could barely fit a pencil.”

(I have heard stories about women “drying up” and that if you don’t use it you lose it – but I thought that was just, well, talk. I didn’t think it was true.)

He asked her if she was having sex.

She was silent.

Then she started crying. “She cried and cried,” he said.

Painful dried up vaginas aren’t something a lot of women talk about. “It’s grin and bear it, tough it out, or give it up,” he says.

But, being an honorary Disruptive Woman – he laid it on the table.

And Susan Wysocki, president and CEO of the National Association of Nurse Practitioners in Women’s Health pointed out more menopause-related things a lot of young women don’t know – or talk about. Like, who knew some menopausal women off their hormones are in so much pain they can’t ride an exercise bike, or even comfortably sit down.

“Here we are,” she said, “saving at least one vagina at a time.”

She also discussed the fact that some women worry about taking hormones because they don’t want to get breast cancer. She says that sometimes a woman may have a teeny tiny potential tumor that could go un-noticed for years. But, sometimes the hormones can make it grow big enough to show up on a mammogram. And that way a woman can get treatment faster.

“That can be a good thing,” she said.

For more about menopause, hormone replacement, and the WHI study, you can read:

• Women’s Health Initiative site, National Institutes of Health
• “A Fresh Look at Post-Menopausal Hormone Therapy: Benefits and Risks,” Society for Women’s Health Research, January 2010 (updated April 28, 2010)
• “The Estrogen Dilemma,” New York Times Magazine, April 12, 2010
• Hot Flash Havoc movie site

Don’t miss the next Disruptive Women in Healthcare breakfast, “Childhood Obesity: A Big Fat National Challenge.” May 27 at Johnny’s Half Shell. Reserve your spot now!

The reasons for this are complex and varied.  This is a particularly vexing challenge for young, chronically ill patients, for people with mental health diagnoses and for the elderly who may suffer from memory impairment.  Anyone on a complicated drug regimen knows how committed one must be to remain adherent.

For some, cost is an issue while for others side effects can be unpleasant, travelling can compromise the best of intentions as can the need for refrigeration when none is available.  Some patients must take some drugs on an empty stomach and others on a full stomach.  Some patients are simply not ready to accept they have a serious, or lifelong illness. It is complicated.

Because the implications, both clinical as well as financial, are significant, we have invited a number of our Disruptive Women bloggers, as well as some other experts in the field, to join us in a series of policy posts on this critically important issue.

Beginning next week, on October 19th, we will launch our Drug Adherence series which will analyze this challenge from a number of perspectives:  patients, providers, researchers.  In addition, we will also offer innovative solutions.

At the completion of this series, we will compile all the posts into an e-book, just as we did when we tackled the issue of Comparative Effectiveness Research and created our Comparative Effectiveness Research e-book.

If  you or someone you help care for has experiences you would like to share, or you have research, solutions and other thoughts on this topic, I hope you will share them with us.

A common reaction around the Web has been “Finally!” – with remarks like “This is NOT a Hoax!” and “Just in time for Web 3.0,” the FDA has set a date to start figuring out “how to deal with Web 2.0.” (NPR Health Blog).

But after the initial shock and sarcasm subsides, the potential significance of the FDA’s (albeit long overdue) move forward this week starts to sink in – this could result in the most significant set of regulations since the FDA’s guidelines for broadcast direct-to-consumer (DTC) advertising in the late 1990s. We’re talking industry-changing stuff here – or rather, industries-changing, because you can be sure that pharmaceutical companies, physicians, consumers, Internet and social media companies, the advertising and public relations industries, and everyone whose revenue includes online advertising are all major stakeholders in this public policy debate.

So what has the FDA highlighted as the key elements for discussion of this issue? (List below drawn from the 9/21/2009 FR notice)

1. For what online communications are manufacturers, packers, or distributors accountable?
   • (paraphrased) What communications and discussions should be considered “by, or on behalf of” versus independent of influence from these companies – and when and how should companies “disclose their involvementor influence,” particularly “on third-party sites”? Should different types of online media platforms and different intended audiences of these platforms be considered differently when addressing these questions – if so, how?
2. How can manufacturers, packers, or distributors fulfill regulatory requirements… in their Internet and social media promotion, particularly when using tools that are associated with space limitations and tools that allow for real-time communications (e.g., microblogs, mobile technology)?
   • (paraphrased) How should product information be presented on these platforms so that users have appropriate access to both risks and benefits?
3. What parameters should apply to the posting of corrective information on Web sites controlled by third parties?
4. When is the use of links appropriate?
   • (paraphrased) Should there be rules about the use of “links to and from Web sites,” including links to or from unbranded websites to or from clearly branded company websites? And what research and data exists about the click-rates in different contexts of users seeking information about medical products?
5. Questions specific to Internet adverse event reporting
   • (paraphrased) How are companies that are obliged to report adverse effects of products using online media tools, if at all, to monitor information about adverse effects of their products? Should these companies be obliged to monitor and/or report information from online communications concerning adverse effects of their products?

We – and the FDA – want to know what you think. What are your gut reactions to all of this – do you find anything particularly worrying, are there any potential outcomes you’re especially hoping for?

If you submit any comments to the FDA – and we hope you will – come by and tell us about it in our comment section here (and we promise to do the same). More information about the public hearing and submitting comments is available here.

There are many reasons (cost, inconvenience, forgetfulness, unpleasant side effects) why patients don’t take their medicine. Medication adherence has become an issue of great concern within the health community, especially as we get older as a nation. So in this spirit, Verizon recently launched what it refers to as the Pill Phone — a new technology that allows people to make sure they keep to their medication regimens and help family members keep to theirs.

At its most basic, the Pill Phone is a medication information and reminder service.

Based on the best selling, medication reference guide (The Pill Book), the Pill Phone is a mobile, digital version of the book providing access to critical information on more than 1,800 medications including dosing, side effects, contraindications, photos and more. Currently, it is the only wireless application to have FDA approval (received in early 2006) for medication management.

An excellent example of the way in which patients can leverage technology to optimize their health care, the Pill Phone offers the following advantages:

- Provides automatic dosing reminders through visual and audio alerts.
- Provides detailed drug information, including the drug name, its picture, its class, warnings, and a side effect profile.
- Tracks and stores pill-taking records.
- Confirms when a dose was taken.

Women are the primary care givers to millions of elderly parents and relatives. In fact, nearly seventy percent of home health care givers are women. This is a tough job but very important, not only to their families but to society as a whole. New communications tools are now available to help make this difficult job easier.

The Pill Phone is just one example of the way in which cutting edge technology can help change how health care has traditionally been delivered. More and more, we are identifying the intersections between health and broadband technology. As broadband technology adoption increases, it offers consumers faster and better access to health information and their health care records—while also making a tremendous difference in medication adherence.

We are only at the beginning stages of what broadband networks can do to improve health care, increase access to health professionals and resources, and reduce health care costs. While a relatively simple application, the Pill Phone demonstrates the power broadband networks coupled with good applications and capable devices can bring to consumers and our healthcare system.