The anatomy of a good doctor
I met Dr. Stulbarg when I was twenty-two, and had just moved in with my boyfriend Stephen. Stephen had cystic fibrosis, but he’d been unusually healthy until now, when his lung collapsed on the way to a party. Then, in the hospital, his lung collapsed a second time. We sat on his bed together, talking with Dr. Stulbarg about what would happen next. Continue reading ... Your patients are rating you online: How to respond. Manage your online reputation: A social media guide. Find out how. (Source: Kevin, M.D. - Medical Weblog)
Source: Kevin, M.D. - Medical Weblog - October 25, 2013 Category: Family Physicians Tags: Patient Patients Source Type: blogs

Breathe Deeply: Did Insmed Release Dodgy Data For Its Drug?
Deciphering trial data can be a fascinating pastime, especially when the findings indicate a medication is truly capable of making a difference for patients. Other times, however, this activity can cause consternation if the results suggest something may be awry. Such may be the case with Phase III data released the other day by Insmed for its Arikace antibiotic for cystic fibrosis sufferers. The drugmaker released a poster presentation showing that its experimental medication demonstrated statistical non-inferiority compared with an existing Novartis (NVS) treatment, which is another way of saying that Arikace was equally...
Source: Pharmalot - October 22, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs

Mom: "Down Syndrome Research, Hope for My Daughter"
This year scientists announced a major breakthrough and a possible gene therapy for Down Syndrome. In cells taken from a person with Downs, they were able to silence the extra 21st chromosome. This may mean a targeted therapy to help fix the health and cognitive problems caused by have that extra bit of genetic material. Reactions were mixed. Some thought that people with Down Syndrome are perfect as God made them and we should do nothing to change them. I understand this reaction very well. We live in a society that kills 90% of people with Down Syndrome before they make it out of the womb. It is entirely natural to want ...
Source: Mary Meets Dolly - October 14, 2013 Category: Geneticists and Genetics Commentators Tags: Genetic Engineering Source Type: blogs

“You’re not as invincible as you think…”
For most of my young adult life, I lived in extremes—I was either overcommitted in school and extracurricular activities, or I was bedbound and hospitalized. I spent so much time trying to prove that illness didn’t define me that the quest for invincibility itself began to overwhelm my identity. I am a lifelong patient with multiple chronic illnesses, including primary ciliary dyskinesia (PCD), a rare genetic lung disease, so I’ve had to co-exist with symptoms and setbacks for as long as I could remember. As a college writing instructor, I interact with young adults every day, and I see firsthand the sens...
Source: Disruptive Women in Health Care - October 8, 2013 Category: Health Medicine and Bioethics Commentators Authors: dw at disruptivewomen.net Tags: Advocacy Choice Cost Coverage Policy Patients Politics Publc Health Young Adults Source Type: blogs

Forcing the decision of putting a value on a patients life
Pharmaceutical companies are forcing the decision of what is the value of a patient's life by putting the huge prices on new medications. There is a new cystic fibrosis drug out, ivacaftor, from Vertex Pharmaceuticals. It costs a paltry $294,000 per year. Doctors are even pushing back to the company about the high cost.If you are one of the patients with CF who would benefit from this new medication it probably is a life saver. But then you look at the price tag it puts on your life and wonder how you can afford it.My question to Vertex is how do you justify this cost. I am sure their reply will be somewhere along the line...
Source: Caroline's Breast Cancer Blog - October 8, 2013 Category: Cancer Tags: medication costs Source Type: blogs

FDA: The Growing Use of Freedom of Information Act Requests for FDA Data by Life Science Stock Traders
The Wall Street Journal reported on the use of FOIA (Freedom of Information Act) requests by investment firms with the intent to learn more about companies regulated by FDA. The story covers a wide scope of information requests with a focus on investment firms attempting to learn about possible adverse event reports and issues arising from FDA inspections. Using FOIA requests When SAC Capital Advisors LP was weighing an investment in Vertex Pharmaceuticals Inc., the hedge-fund firm contacted a source it knew would provide nonpublic information without blinking: the federal government. An investment manager for ...
Source: Policy and Medicine - October 3, 2013 Category: Health Medicine and Bioethics Commentators Authors: Thomas Sullivan Source Type: blogs

The Arguing Over PTC124 and Duchenne Muscular Dystrophy
This article at Nature Biotechnology does an excellent job explaining the details. Premature "stop" codons in the DNA of DMD patients, particularly in the dystrophin gene, are widely thought to be one of the underlying problems in the disease. (The same mechanism is believed to operate in many other genetic-mutation-driven conditions as well. Ataluren is supposed to promote "read-through" of these to allow the needed protein to be produced anyway. That's not a crazy idea at all - there's been a lot of thought about ways to do that, and several aminoglycoside antibiotics have been shown to work through ...
Source: In the Pipeline - September 18, 2013 Category: Chemists Tags: Business and Markets Source Type: blogs

23andMe Genetic Testing: What's the Deal?
Curious about what the future might hold for your health? After reading a great post on DNA testing by Charlotte at The Great Fitness Experiment, I became curious about 23andMe, a direct-to-consumer genetic testing outfit that's been getting quite a bit of buzz. As a former hypochondriac, and as someone with a family medical history that doesn't inspire much optimism for a long and healthy life, I knew this was a bit of a psychological gamble. Did I really want to know how screwed I might be genetically? Mightn't I be better off thinking happy thoughts, trying to be reasonably conscientious about diet and exercise, and h...
Source: Cranky Fitness - September 16, 2013 Category: Consumer Health News Authors: Crabby McSlacker Source Type: blogs

The Beautiful Disruption: How Open Health Care Data Puts Patients in Charge
Health care is on the verge of a beautiful disruption. Technology is driving a liberation of data that will fundamentally change our relationship with our patients. In this brief blog, we’ll: look at technology’s effects on grocery stores (and why we should care), highlight some innovative companies that are disrupting health care, and put it all together to recognize how patients will command more control over health care decisions and spending. But first—grocery stores. Grocery Store Technology Once upon a time, grocery store employees spent an evening each month counting the cereal boxes left upon ...
Source: richard[WINTERS]md - August 11, 2013 Category: Universities & Medical Training Authors: Richard Winters MD Tags: Uncategorized Source Type: blogs

The Beautiful Disruption: How Open Health Care Data Puts Patients in Charge
Health care is on the verge of a beautiful disruption. Technology is driving a liberation of data that will fundamentally change our relationship with our patients. In this brief blog, we will: look at technology’s effects on grocery stores (and why we should care), highlight some innovative companies that are disrupting health care, and put it all together to recognize how patients will command more control over health care decisions and spending. But first—grocery stores. Grocery Store Technology Once upon a time, grocery store employees spent an evening each month counting the cereal boxes left upon the s...
Source: richard[WINTERS]md - August 11, 2013 Category: Universities & Medical Training Authors: Richard Winters MD Tags: Uncategorized Source Type: blogs

The effect of Facebook on organ donor registration
Recently, there has been a great deal in the press surrounding organ transplantation.  Sarah Murnaghan, the courageous little girl with end stage cystic fibrosis, captured the nation’s attention as she waited for a life saving lung transplant.  The organ transplantation and procurement system in the US today is not without flaws.  Organs are at a premium and finding the best way to allocate them to matched patients who need them the most is a daunting task. Continue reading ... Your patients are rating you online: How to respond. Manage your online reputation: A social media guide. Find out how. (Sour...
Source: Kevin, M.D. - Medical Weblog - July 31, 2013 Category: Family Physicians Tags: Social media Facebook Nephrology Source Type: blogs

Extra Chromosome Silenced in Down Syndrome Cells
Amazing news was announced in the field of gene therapy this week. Scientists in Massachusetts have taken the cells of a person with Down Syndrome and have silenced the extra 21st chromosome in those cells. The Guardian has the story:Scientists have corrected the genetic fault that causes Down's syndrome – albeit in isolated cells – raising the prospect of a radical therapy for the disorder.In an elegant series of experiments, US researchers took cells from people with DS and silenced the extra chromosome that causes the condition. A treatment based on the work remains a distant hope, but scientists in the field said the...
Source: Mary Meets Dolly - July 18, 2013 Category: Geneticists and Genetics Commentators Tags: Genetic Engineering Source Type: blogs

Is the Cystic Fibrosis Foundation a Charity or a Venture Capital Firm?
This article deserves considerably more attention than it apparently initially received.Background The background was,What happens when a disease-fighting charity dives into venture capitalism?In the first case of its kind, the results include one of the planet's most expensive pills, huge sales projections for a drug company and windfalls for executives who sold stock in the glow of enthusiastic news releases about the drug.Kalydeco is a breakthrough drug designed from knowledge of the genetic roots of cystic fibrosis, a lung disease that kills most victims before they reach middle age. Developed by Vertex Pharmaceuticals...
Source: Health Care Renewal - June 10, 2013 Category: Health Medicine and Bioethics Commentators Tags: Vertex Pharmaceutical executive compensation disease-specific organizations manipulating clinical research mission-hostile management guidelines Cystic Fibrosis Foundation Source Type: blogs

GIGO: Will the Benefits of EHRs Outweigh the Trash They (Might) Create?
GIGO. I don’t hear that very often these days. It was a common refrain, though, when computer-enabled calculations became accessible to the “average” academic researcher for the first time. I was there. We were near giddy with enthusiasm. With more power to crunch more numbers came a sense of security – and even arrogance – that we could solve more problems with less effort, faster. At last, we thought, we’d find truth! No doubt, sometimes we did. But critics warned us: GIGO – Garbage In Garbage Out. If the data we used were garbage, so were our conclusions. I’ve already blog...
Source: Disruptive Women in Health Care - June 3, 2013 Category: Health Medicine and Bioethics Commentators Authors: dw at disruptivewomen.net Tags: HIT/Health Gaming Innovation Policy Technology EHR Electronic health record GIGO Source Type: blogs

Top stories in health and medicine, June 3, 2013
Brought to you by MedPage Today. 1. Online Program Boosts Mobility for MS Patients. A simple behavioral intervention delivered over the Internet helped multiple sclerosis (MS) patients increase their daily walking. 2. Vinegar Test May Reduce Cervical Ca Deaths. Vinegar has been used for everything from cleaning refrigerators to taming food odors, and now this common kitchen staple may reduce cervical cancer deaths. 3. Fosamax Benefits Bones in Young CF Patients. Young patients with cystic fibrosis and low bone mineral density (BMD) saw significant improvements in bone quality following a year on alendronate (Fosamax). Your...
Source: Kevin, M.D. - Medical Weblog - June 3, 2013 Category: Family Physicians Tags: News Cancer Endocrinology Neurology Source Type: blogs

Novel Nebulizer Technology Brings Seawater Therapy to Cystic Fibrosis Patients
Some years ago someone noticed that surfers with cystic fibrosis (CF) have improved lung function over their skateboarding peers, or really anyone else with CF that doesn’t get sprayed with saltwater on a regular basis. Turns out that while nature has created a good way to deliver hypertonic saline to the lungs, carrying an ocean around is not a practical way to receive therapy. Parion Sciences, a development-stage company working on commercializing these findings into a practical inhaler, has partnered with Cambridge Consultants to create technology that can deliver tiny droplets of saline deep into the lu...
Source: Medgadget - May 22, 2013 Category: Technology Consultants Authors: Editors Tags: Medicine Pediatrics Source Type: blogs

PhRMA Report: Over 5400 Medicines in Development and 70% are First in Class
According to report released by PhRMA, companies have more than 5,400 medicines in development globally, and more than 70% of therapies in the pipeline are potentially first-in-class and could offer patients new treatment options, and a notable number of potential therapies target diseases with limited treatment options such as ALS and rare diseases.  A breakdown of their report offers insight into the various medicines in development for different diseases and populations.    Older Americans  America’s biopharmaceutical research companies are developing 465 new medicines that target the 10...
Source: Policy and Medicine - April 24, 2013 Category: Health Medicine and Bioethics Commentators Authors: Thomas Sullivan Source Type: blogs

Partners for Healthy Dialogues: Offers Prescription for Healthy Physician-Industry Interactions
When it comes to our health, “collaboration between physicians and biopharmaceutical professionals is essential to improving the care we receive and developing new medicines.  Whether it is collaborating on research for new medicines or learning about the latest treatment options, interactions between physicians and biopharmaceutical professionals lead to better health and better medicine.”  This simple motto underlies a critical new initiative launched by a wide range of organizations, known as the “Partners for Healthy Dialogues.”  The partnership is a collaboration betwe...
Source: Policy and Medicine - April 9, 2013 Category: Health Medicine and Bioethics Commentators Authors: Thomas Sullivan Source Type: blogs

I need to widen my interests
Yesterday I went to a luncheon sponsored by the New England Coalition for Cancer Survivorship with a friend. She has had breast cancer and remarked when we arrived that she wasn't used to seeing men at cancer events. Breast cancer events are mostly filled with pink clad women.The lunch was wonderful with recognition of many people who are helping people with cancer cope with life after treatment - a worthy cause. I knew several people there and made some new friends and connections.However I started thinking (a dangerous undertaking to say the least) and decided I need to get more involved with the worlds of fibromyalgia a...
Source: Caroline's Breast Cancer Blog - March 24, 2013 Category: Cancer Tags: support fibromyalgia pain cancer rheumatoid arthritis Source Type: blogs

#ePharma Day 2 : Mobile Healthcare
The second half of the ePharma morning session started with a panel on serving Mobile Healthcare Customers lead by Joe Shields. Panelists included: Chris Crichton, Vice President, 5th Finger, A Merkle Company Sharon DeBacco, senior Director, Customer Communications & Operations, Ironwood Pharmaceuticals Scott Wolf, Chief sales Officer, Everyday Health. The panel covered topics such as – Is the mobile patient a moving target? What are the needs of the patient? Have these devices changed the pathway of a patient? The panel also covered the importance of educating the regulatory professionals, people who work on t...
Source: ePharma Summit - March 5, 2013 Category: Medical Marketing and PR Tags: 2013 epharma summit Digital Marketing for Pharma digital healthcare Source Type: blogs

Pharmalot… Pharmalittle… Good Morning
Good morning, folks, and how are you today? A spot of rain is falling on the Pharmalot corporate campus, where we are doing our best to energize the short people and tend to the usual early-in-the-day to-do list. Of course, the middle of the week only intensifies the need to move quickly. We trust you relate. So join us as we grab another cup of stimulation and dig in for another busy day. As always, here are some items of interest to help you along. Hope your day is smashing and do stay in touch… FDA OKs Pill For Post-Menopausal Sex Problems (Los Angeles Times) Allergan Settles Oklahoma Botox Case During Trial (Blo...
Source: Pharmalot - February 27, 2013 Category: Pharma Commentators Authors: Ed Silverman Tags: Uncategorized Allergan AstraZeneca Biosimilars Botox Cystic Fibrosis Elan FDA Flu GlaxoSmithKline Hospira Influenza Menopause Merck Narcolepsy Osphena Pandemrix Royalty Pharma SFDA Shionogi Tredaptive Vertex Pharm Source Type: blogs

Reason for Hope? - Novartis Rescinds Vasella's Golden Parachute
Enormous compensation of hired health care executives, out of all proportion, if related at all to whether their work had any positive effect on patients' or the public's health, has long been a concern on Health Care Renewal.  For example, back in 2006, we posted repeatedly (look here for links) about the billion dollar plus fortune amassed by the then CEO of UnitedHealthcare which vividly contrasted with the company's avowal to "make health care more affordable."We have posted many such stories.  Yet maybe there is a whiff of change in the air.  For the first time that I can recall, a gigantic pa...
Source: Health Care Renewal - February 25, 2013 Category: Health Medicine and Bioethics Commentators Tags: executive compensation perverse incentives golden parachutes Switzerland Novartis Source Type: blogs

White House PCAST: Rival Countries Gaining on US Medical Research Spending
The Nation once led the world in investments in research and development (R&D) as a share of gross domestic product (GDP), but more recently, the United States has been investing less in R&D than other leading and emerging nations invest.  Moreover, U.S. industry has been shifting its investments toward applied R&D, narrowing the support for basic and early-stage applied research, which is crucial to transforming innovation.   Without adequate support for such research, the United States risks losing its leadership in invention and discovery—the driving force behind the new industries and ...
Source: Policy and Medicine - February 14, 2013 Category: Health Medicine and Bioethics Commentators Authors: Thomas Sullivan Source Type: blogs

2012: Banner Year for New Drugs
Fueled by new cancer therapeutics, last year the annual new molecular and biological entity approval count from the U.S. Food and Drug Administration (FDA) saw its highest year since 1997. One-third of the novel products approved by the FDA’s Center for Drug Evaluation and Research (CDER) are used to treat cancers of the blood, breast, colon, prostate, skin and thyroid. As part of the U.S. Food and Drug Administration (FDA), the Center for Drug Evaluation and Research (CDER) promotes and protects the health of Americans by assuring that all prescription and over-the-counter drugs are safe and effecti...
Source: Highlight HEALTH - February 13, 2013 Category: Medical Scientists Authors: Walter Jessen, Ph.D. Source Type: blogs

Do We Really Know the Cause for Over 4500 Diseases?
Since I mentioned the NIH in the context of the Molecular Libraries business, I wanted to bring up something else that a reader sent along to me. There's a persistent figure that's floated whenever the agency talks about translational medicine: 4500 diseases. Here's an example: Therapeutic development is a costly, complex and time-consuming process. In recent years, researchers have succeeded in identifying the causes of more than 4,500 diseases. But it has proven difficult to turn such knowledge into new therapies; effective treatments exist for only about 250 of these conditions. It shows up again in this paper, just o...
Source: In the Pipeline - February 12, 2013 Category: Chemists Tags: Biological News Source Type: blogs

FDA: Enrichment Strategies to Improve Efficiency of Drug Development
Conclusion  Temple noted that there are some issues with predictive enrichment.  One is that you always believe the characteristic you use to enrich predicts the good responders; it may not do this as well as you hope.  So it’s very important to characterize the test that leads you to select those patients; then see whether it’s true that patients with the characteristic always (or most of the time) respond, and that patients without the characteristic don’t respond very much.   An issue to consider in any enrichment design is how much you need to study the people who don’...
Source: Policy and Medicine - February 12, 2013 Category: Health Medicine and Bioethics Commentators Authors: Thomas Sullivan Source Type: blogs

USMLE Questions – Characteristic Disease Findings
The United States Medical Licensing Examination (USMLE) is designed to emphasize knowledge of clinical scenarios and clinical pearls, even on Step I. Listed below are some commonly encountered disease findings and characteristics. Feature Disease 45, X chromosome Turner’s syndrome 5-HIAA increased in urine Carcinoid syndrome Aganglionic rectum Hirschsrpung’s disease Apple-core sign on barium enema Colon cancer Arched back (opisthotonos) Tetanus Argyll-Robertson pupil Syphilis Ash leaf on forehead Tuberous sclerosis Auer rods  Acute myelogenous leukemia Austin Flint murmur Aortic r...
Source: Inside Surgery - January 18, 2013 Category: Surgeons Authors: Editor Tags: Surgpedia USMLE diseases findings VMA water hammer pulse Source Type: blogs

Pipeline 2012
39 drugs approved in the USA in 2012 US Drug approvals in 2012 have reached a 15-year high with regulators giving the thumbs up to 39 new drugs. The figures from the US Food and Drug Administration show approved drugs were up on 2011, when 30 new medicines were given marketing authorisations. Of the 39 approved in 2012, 11 were for cancer treatments and almost 20 were designated orphan drug status. Some of the drugs allowed on the US market in 2012 included: Pfizer’s leukaemia drug Bosulif (bosutinib); Ariad Pharmaceutical’s leukaemia drug Iclusig (ponatinib); Johnson & Johnson’s multi-drug resi...
Source: PharmaGossip - January 6, 2013 Category: Pharma Commentators Authors: insider Source Type: blogs

Cystic Fibrosis and the Cost of Medicine
It’s difficult not to get excited about the prospect of genetic medicine, but as the results of research into genetics and personalized medicine continue to soar, so do the prices of disease-treating drugs. A recent story on NPR detailed the history behind...(read more) (Source: ADVANCE Discourse: Lab)
Source: ADVANCE Discourse: Lab - January 4, 2013 Category: Pathologists Authors: Michael Jones Tags: Current Events Molecular Diagnostics Pathology Research Source Type: blogs

Hello My Virtual Friend
Today's guest post comes from Emily Schaller, Founder/CEO of Rock CF Foundation. Follow her on twitter @RockCFem Technology is both a scary and amazing thing. Humans around the globe are plugged in and online all day and all night waiting for work emails, social media updates, breaking world news Tweets, and so much more. I must admit that I am guilty of all of these things. Every day you can turn on the news and see something wonderful or something tragic that has come from a social media. Digital communication has been a huge benefit for myself and patients with cystic fibrosis and other illnesses around the world. Bec...
Source: ePharma Summit - January 4, 2013 Category: Medical Marketing and PR Tags: social media and healthcare ePharma Source Type: blogs

Express Scripts CEO To Kids With Cystic Fibrosis: Find Another Way To Stay Alive
(Source: drugwonks.com Blog)
Source: drugwonks.com Blog - September 6, 2007 Category: Pharmaceuticals Source Type: blogs