Thomas Maeder, our May 2010 Man of the Month, is the author or co-author of twelve books and numerous articles in national publications, and has long experience in the biomedical field as a writer, educator, and consultant. Below, he shares his thoughts on rare disorders and their place in the drug market.

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of known, potentially useful therapeutics never taken to market, or others of already proven effectiveness that were withdrawn, because they did not meet a drug company’s threshold of profitability.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.

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Many thanks to our speakers, Phyllis Greenberger, Dr. James Simon, and Susan Wysocki, and to Disruptive Women’s Wendy Grossman for the following summary post.

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!

It has been estimated that 3 out of 4 people report that they do not take their medications as directed, resulting in hundreds of billions of dollars annually in related medical costs and an enormous number of hospital admissions and readmissions.

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.

Big news: The FDA is holding a public hearing to discuss online promotion of FDA-regulated medical products – including prescription drugs, prescription biologics, and medical devices. The hearing will be November 12 and 13, 2009 in Washington, DC (registration closes October 9 – see also registration instructions from Eye on FDA), but public comments can be submitted in writing or electronically now through February 28, 2010. View the docket details and full Federal Register notice.

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.

“Drugs don’t work in patients who don’t take them.” This quote, by the former Surgeon General, C. Everett Koop, M.D, appeared in a New England Journal of Medicine article on drug therapy and adherence.

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.

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