Do you ever wonder who the champion advocate for the patient is in the healthcare reform debate? More importantly, is the focus on consumer choice taking a front seat while the issue of how consumers will ultimately respond to those choices is being ignored?

Many people, myself included, believe that in aggregate, individuals are best equipped to advocate for themselves. In fact, this hypothesis forms the foundation for a key component of Health 2.0, in which the consumer takes more responsibility for managing his or her healthcare and by default, the delivery of that care becomes more fluid and cost-effective.

The rub, however, is that data suggest that most consumers of healthcare rarely if ever speak up.

A fascinating report published in the September issue of Milbank Quarterly shows that fewer than 40% of 5,000 patients surveyed complained to healthcare insurance plans when a problem arose, even when it cost them upwards of $1,000 in out of pocket expenses or if care (or lack thereof) led to a more serious health condition. What’s more, less than 15% of patients took steps to opt out of their current plans and search for a more palatable option.

What these results demonstrate is that current response to the coverage being delivered neither safeguards the patient or serves to highlight the most significant problems among current plans in the marketplace.

Clearly, two major components are missing in the current debate: unique, consumer-driven advocacy and optimized patient responsiveness. Without these, even the most attractive new options on the table may ultimately fail to lead to better overall quality of care.

I’ve been tasked with presenting the genetic and rare disease perspective on comparative effectiveness.

I’ll dispense with ‘rare’ right away.  If by rare, we mean single gene disorders, then perhaps it is a useful designation.

If by rare, we just mean the equivalent of the US definition of orphan disease, i.e., less than 200,000 people in the US, then we should ask a few questions. In the old model of test and drug development, the ‘block buster, body count’, model, rare was a useful designation.  In a system built for BIG, then rare needed a boost.  In the new age of personalized medicine, all conditions are rare. In fact, they are usually an N of 1 after factoring in the myriad of genes involved, epigenetics, environment and so on.  As we enter new ways of dealing with common conditions, they too will be fragmented into dozens, sometimes hundreds, of rare conditions.  Thus rare and common conditions have similar challenges in that realm.  For these reasons, I recommend we lose the word ‘rare’.  I know it has a rich and abundant history in the Orphan Drug Act, but in addition to the aforementioned issues, I think siloing our thinking around disease into these social constructs of abundance of disease misses opportunities that would blossom were we to consider gene families, pathways and targets instead of incidence and prevalence.

Let’s move then to single gene disorders.  Comparative effectiveness would have to go on unemployment if it depended on single gene disorders for its first tasks.  Most single gene disorders do not have a treatment, let alone several.  And once one is developed, it is hard for a second one to be developed given the lack of financial incentives in the old paradigm.  Perhaps a first step in comparative effectiveness for single gene disorders is creation of ANY treatment for these conditions.

On to genetic conditions.  I think genetic conditions have already been spoken for in all of the preceding posts – because all disease is a mix of genes and environment, and so all of the conditions spoke about, either explicitly or implicitly, are genetic to some extent.  That said, I believe genetic diseases, and genomic signatures of attributes of disease, like tumors, offer ways to quantitatively measure expression thereby offering a new level of scientific scrutiny for disease.  While most genetically and genomically authored tests and treatments are nascent and have not yet been scrutinized, we are seeing some assessments of genetic tests, at least.  These are not complete comparative effectiveness studies, but they use methodology that might offer something to the field of comparative effectiveness.  If the field does use methodologies like those of EGAPP, then it will be important to do a broader assessment of the methodology before wide spread implementation.  From the website, “The project’s goal is to establish and evaluate a systematic, evidence-based process for assessing genetic tests and other applications of genomic technology in transition from research to clinical and public health practice.”  There are those who ask why almost all tests that EGAPP has assessed have failed to meet the requirements to pass into clinical practice, when some of these tests have done so in a variety of ways.  EGAPP is a good example of how hard it will be to do comparative effectiveness, since its assessment (far from comparative effectiveness) is so difficult, resource and time consuming.

However, I look forward to our foray into comparative effectiveness.  I believe it is time for the practice of medicine to move from being a cottage industry.  Comparative effectiveness, coming of age in the genomic/informatics era, while we move toward individualized medicine (commonly known as personalized medicine), will be an effective tool if, in the words of the post by Randel before me:

“A fair, open, cooperative public/private process, if designed properly will enhance the information needs of patients, physicians, and scientists and eliminate wasteful and ineffective medical technologies and procedures. Implemented without a fix of the payment system, however, the fear that this may stifle access and innovation will be realized.”  If we wish to move medicine out of the cottage industry realm, reimbursement has to come too.  There are exciting months ahead of us – let’s not call each other names like rare or common, genetic or infectious, popular or unpopular.  No disease, condition, or disorder should be left standing around the edges of the dance floor while the rest are dancing.

Stay tuned to the collaborative discussion about health reform resulting from the flash mob on June 17, 2009.

Inform Health Reform

http://fastercures.blogspot.com/2009/06/fastercures-joins-healthcare-advocates.html

I am not usually one to take on the Vatican. In fact, I toured its lovely treasure-filled buildings only three months ago and marveled at the wealth and power it denoted. However, the Pope’s recent pronouncements during his travels in Africa that condoms and abortions are morally wrong have filled me with righteous indignation. I too have spent time in Africa. But I wasn’t there to make pronouncements from on high. I was there to make a documentary about the increasing number of married women with AIDS in Kenya. I walked through Kibera slum and watched large families crammed into corrugated metal sheds without plumbing or heat.

Even so, I probably wouldn’t take on the Pope…except for an article in today’s Washington Post. Apparently, the Vatican’s top bioethics official said the two Brazilian doctors who performed an abortion on a nine-year-old rape victim “did not merit excommunication, because they acted to save her life.” HELLO! In my book, that’s called a pro-choice stand. Bravo for Archbishop Rino Fisichella, president of the Pontifical Academy for Life. Perhaps he should have a chat with his boss.

Abortion is not a black and white issue for me, despite having worked for the better part of a decade for Planned Parenthood Federation of America and NARAL on reproductive health issues. That’s why the pro-choice position has also seemed to me to be the reasoned one. It allows individuals to make decisions and encourages each of us to define for ourselves what is reasonable and acceptable. There is a trust factor in being pro-choice. For example, if there is the possibility that a nine-year-old may be raped by her stepfather, you want to believe that the pregnancy will be ended as swiftly and humanely as possible.

Good for Archbishop Fisichella for being able to see that issues related to reproductive health have gray areas! And that it is often possible to be both pro-life and pro-choice while having to accept difficult decisions. Is it too much to hope that Pope Benedict XVI might also see the light? If he truly listens to the people of Africa and other continents, and opens his eyes to their hopes for their own lives – I have faith that he may begin to understand the healing power of condoms and the life-affirming necessity for legal, safe abortions.

For several years, my family has held a grab bag holiday get-together. People are to bring inexpensive gifts, often intended more for laughs than for use. My extended family includes nieces and nephews ranging in age from 15 to 30, some with children and others who are sexually naïve, even if not virgins. Mine is not a family that discusses sex. My living siblings are reborn Christians, who deem appropriate only discussions of abstinence for those youth not yet married. However, as a nurse, I’ve made it clear that their auntie will not shy away from sexual discussions. I have been known to initiate such conversations with a niece or nephew and even their friends when I have some time with them.

“ONE PREMIUM LUBRICATED Lifestyles® LATEX CONDOM”

So this year, one of my grab bag gifts was condoms. My courage to make this a visible ‘gift’ at the holiday party arose from New York City’s model of making condoms available anywhere and everywhere. The city has rightfully been aggressive in distributing condoms since Thomas Frieden assumed the position of Commissioner of the Department of Health and Mental Health and saw that a large proportion of people in the city who are HIV-positive don’t know it and can be spreading it unintentionally by having unprotected sex. In 2007, the city launched a NYC-branded condom and has since given out over 70 million of them for free. (more…)

The New York Times recently reported on Health Reform Private House Parties. At this particular party, the participants easily sided with the angels: health care is a right, insurance should cover everything and coverage should be available from government and businesses. There, that was easy. Bet they had a nice time.

Partygoers were not asked for solutions and the devil is always in the details, so let’s use my favorite – flu shots.

First, the New York Times reported that party participants quickly agreed that “…health care was a right…” It makes me wonder; if health care is a “right,” what of “responsibility”?

- If I have a “right” to flu shots to help prevent flu disease, do I have a “responsibility” to get them to protect myself, reduce my medical care costs, improve economic productivity and protect others around me?
- If I don’t get the shot, infect others and cause them to be ill, am I responsible for the cost of their care?
- If others I infect miss work, am I responsible for the cost of temporary workers or losses incurred by their employer? Am I responsible to the government for the loss in taxable productivity?
- What if they die as a result of the flu I transmit to them? Am I culpable?

Second, participants also agreed “…that insurance should cover ‘everything,’ not just some services….”
- If a flu shot is covered and I choose not to get one, should I be required to pay for any doctor visits and medicines I need?
- Should my employer have the right to charge me for the temporary help that might need to be hired to replace me on the job?
- Should Uncle Sam have the right to tax me for earnings I would have made if I had been on the job?
- Should the cost of something so reasonably priced be covered for everyone? Really, I mean everyone? Even the wealthy who can well afford the cost?

Third, participants agreed that “…coverage should be readily available from the government, as well as from employers….” They agreed that individuals and businesses should have to pay a “small health care tax” to fund care.
- Who are they kidding, “small health care tax?” Have they looked at cost projections lately?
- And what about non-coverage barriers? Coverage is only one barrier to care. Ask those in rural areas, anyone not fluent in English or medically literate.

And yes, I’m still harping on flu shots. The season is not nearly over and there are plenty of doses left to protect the nation from the $87.1 billion – or more – that flu could cost us this winter.¹

1. Molinari NA, Ortega-Sanchez IR, Messonnier ML. The annual impact of seasonal influenza in the US: Measuring disease burden and costs. Vaccine 2007;25:5086-5087.

The following post appeared last week on the Get Better Health Blog:

President-elect Obama and Secretary of HHS designate, Tom Daschle, invited concerned Americans to discuss healthcare reform in community groups across the country. My husband and I hosted one such group at our home in DC yesterday. Although we had been instructed to compile a list of compelling stories about system failures – instead we decided to be rebellious and discuss “what’s right with the healthcare system” and compile a list of best practices to submit to the change.gov website.

The event was attended by a wide range of healthcare stakeholders, including a government relations expert, FDA manager, US Marine, patient advocate, health IT specialist, transportation lobbyist, real estate lobbyist, health technology innovator, Kaiser-trained family physician, medical blogger, and EMR consultant. Here is what they thought was “right” with the healthcare system: (more…)

Remember Harry and Louise? Sitting at their kitchen table, the nondescript couple burst onto the national stage in a 1993 television commercial that deftly attacked Bill Clinton’s proposed health care plan and made even those of us committed to universal health insurance afraid of its consequences.

I can’t recall the lines Harry and Louise used, but 15 years later I remember the message: America is built on choice, health reform will take away that choice, and if we pursue the proposed reforms we’ll deeply regret it. Then a medical student, I was convinced (albeit naively) that we should have a healthcare system like those of our European counterparts. I was seeing many, many patients who had waited too long to visit the emergency room because they didn’t have insurance – and the accompanying detritus of medical neglect. But I was moved by Harry and Louise. I am not a person who is easily swayed, and yet their message made sense to me. It put a chink in the ideological armor of the left that persists even now.

Today, as we think about how to pursue smart and sustainable healthcare reform, we would do well to remember that Americans – myself included – like to get something in return for their money. And we like to have meaningful choices. As we try to make the system better, let’s not sacrifice choice. In fact, expanding choice is actually the best rationale for reform. We like to think they have choice in our current system, but there are a myriad of choices that are made for us before we even walk into the doctor’s office or taken away from us after a decision is made.

Take advance directives. Americans cling to the false hope that we have the power to decide how we will die. That we can refuse unnecessary care, die with dignity, have the needs and the questions of our family members answered, and opt for extraordinary measures if we choose. This image of death on our own terms is a fallacy.

Last year, when my grandmother lay dying in a California hospital, I saw firsthand how little say a family has in the care a loved one receives at the end of life. Private insurance, a willingness to pay out of pocket, and two doctors in our family did nothing to give us choice. One morning, I came into her ICU room to discover that even though her care providers had observed an unexplained drop in the urine flow from her catheter, no one had checked it. No one had laid hands – or eyes – on the patient. The simple solution was the right one. Her catheter had slipped out of place. And this simple oversight meant she had been sitting in her own urine long enough to break down the skin and cause what would turn out to be a life threatening bed sore – if you can imagine that. As a family we chose to have her be turned regularly to heal the resulting bed sores. Yet despite dividing up the duties so there was a family member with her in the hospital 24 hours a day for months, we couldn’t always get hospital staff to change diapers or assist with turning her even though the evidence shows that these steps are essential.

Despite differences in opinion on her need for dialysis, we found we were at the mercy of the renal physician on duty that week. Just as we chose the follow the advice of one insistent physician to take the more aggressive step to undergo dialysis, that physician would go off service and we would be faced with a new doctor who adamantly refused that same course of action.

And even though she had proactively chosen her physician and her physician had attending privileges in the hospital in which she lay, we found this same doctor who had known her for decades had very little say in the care she received. Instead she was left at the mercy of a new hospitalist every week, each with no knowledge of her case and wildly varying approaches and expertise. I had no choice but to explain the nuances of her case to a new doctor each week. Years of working in medicine and policy were meaningless (in that way alone it was the ultimate democratizing experience). I was totally unable to protect the choices she had made for the end of her life. They willfully ignored her advance directives and the wishes of her family. And they blithely dismissed our choice to have accepted standards of care. What she did want she couldn’t have and what she didn’t want was forced upon her.

When the Aspen Institute polled Americans last summer, they overwhelmingly agreed that health span was more important than life span (91% to 9%). We want a system that helps us stay healthy, and living healthy years is more important to us than living more years. But this is not a choice we are given in our current system. We cannot choose our own definition of a healthy life in the current system.

So the next time you think you have a choice about the health care you receive – think twice. Do you have more reproductive choices than you did 15 years ago? Do you have more preventive health care choices within your insurance than you had 15 years ago? Do you have more choice to vote with your feet when you are treated with disrespect and callousness in a health care encounter than you did 15 years ago?

Harry and Louise were right. We do need choice. But Harry and Louise were also wrong. We will only protect and expand choice through change. And if you watch the latest (2008) installment of the Harry and Louise ads you will see that they too would take their words back.