Ace inhibitor use in patients with Duchenne and Becker muscular dystrophy slows progression of myocardial fibrosis
(Source: Notes from Dr. RW)
Source: Notes from Dr. RW - September 6, 2017 Category: Internal Medicine Tags: cardiovascular neurology Source Type: blogs

You ’ve Got to Be Carefully Taught: How to Explain Hatred to Your Children
With world events occurring at lightning/frightening speed, adults who may be bewildered themselves, may feel at a loss to answer the questions their young ones may have about topics they see broadcast on television or hear about on the school bus. In the wake of the virulent rally in Charlottesville and those that have followed since, it is an even more important topic for parents to address. Children will ask questions and it is crucial for answers to be available and not brushed under the rug, as it might seem easier to do. One such parent is Stefanie Nicolosi, a Philadelphia area photographer. In an article for Newswor...
Source: World of Psychology - August 29, 2017 Category: Psychiatry & Psychology Authors: Edie Weinstein, MSW, LSW Tags: Bullying Children and Teens General Minding the Media Parenting Psychology Bigotry Charlottesville Hatred Nazis Prejudice Racism sexism teaching moment white nationalism Source Type: blogs

CRISPR and Identity
Dr. Joel Reynolds, a postdoctoral fellow at The Hastings Center recently wrote a very poignant essay in Time magazine arguing that our increasing ability to edit our own genetic code risks eventually eliminating the very genetic code that results in people like his younger brother Jason, who was born with muscle-eye-brain disease, resulting in muscular dystrophy, hydrocephalus, cerebral palsy, severe nearsightedness and intellectual disability. In... // Read More » (Source: blog.bioethics.net)
Source: blog.bioethics.net - August 15, 2017 Category: Medical Ethics Authors: Mark McQuain Tags: Genetics Health Care bioethics biotechnology enhancement Health Care Practice human dignity Human Identity syndicated Source Type: blogs

Mallinckrodt Settles Anti-Competitive Practices Suit
Mallinckrodt and the Federal Trade Commission (FTC) recently agreed to settle allegations of raising drug prices and engaging in anti-competitive practices by paying a $100 million fine and allowing a competitor to produce a similar medication. The drug, H.P. Acthar Gel, is used to treat infantile spasms and multiple sclerosis. Mallinckrodt allegedly raised the price of the prescription from $40 per vial to over $34,000 per vial. According to New York Attorney General Eric Schneiderman’s office, Mallinckrodt’s United States subsidiary (Questcor) purchased the drug in 2001 and raised the price an estimated 85,000% over...
Source: Policy and Medicine - August 1, 2017 Category: American Health Authors: Thomas Sullivan - Policy & Medicine Writing Staff Source Type: blogs

Orphan Diseases Or Population Health? Policy Choices Drive Venture Capital Investments
The US exhibits a remarkable pipeline of biopharmaceutical innovation, with 170 new drugs and biologics launched into the market between 2011 and 2015 and another 22 drugs approved in 2016. A striking feature of the pharmaceutical pipeline is the large percentage launched for the treatment of small “orphan” indications, defined by the Food and Drug Administration (FDA) as including fewer than, often many fewer than, 200,000 patients in the United States. Almost half (74) of the products approved by the FDA between 2011 and 2015 were for orphan indications, twice the number (36) approved during the same period by the Eu...
Source: Health Affairs Blog - July 21, 2017 Category: Health Management Authors: Dayton Misfeldt and James C. Robinson Tags: Costs and Spending Drugs and Medical Innovation Orphan Drug Act orphan drugs venture capitalism Source Type: blogs

Top Companies in Genomics
From portable genome sequencers until genetic tests revealing distant relations with Thomas Jefferson, genomics represents a fascinatingly innovative area of healthcare. As the price of genome sequencing has been in free fall for years, the start-up scene is bursting from transformative power. Let’s look at some of the most amazing ventures in genomics! The amazing journey of genome sequencing Genome sequencing has been on an amazing scientific as well as economic journey for the last three decades. The Human Genome Project began in 1990 with the aim of mapping the whole structure of the human genome and sequencing it. ...
Source: The Medical Futurist - May 30, 2017 Category: Information Technology Authors: nora Tags: Genomics Personalized Medicine AI artificial intelligence bioinformatics cancer DNA dna testing DTC gc3 genetic disorders genetics genome sequencing personal genomics precision medicine Source Type: blogs

How long can high drug prices last?
“Pigs get fat, but hogs get slaughtered,” the saying goes. And so may it prove to be true for the pharmaceutical industry. Three articles, all published May 3, illustrate the greed and egregious pricing by certain drug companies that are gaining public recognition and scrutiny. As an example, Marathon invested $370,000 to obtain the license for the data on “deflazacort,” a steroid available for about $1,200 a year in the United Kingdom. This medication is prescribed to treat muscular dystrophy, a condition that predominantly affects young boys. The company then secured FDA approval, renamed the drug “Emflaza,” ...
Source: Kevin, M.D. - Medical Weblog - May 20, 2017 Category: General Medicine Authors: < a href="http://www.kevinmd.com/blog/post-author/robert-pearl" rel="tag" > Robert Pearl, MD < /a > Tags: Meds Medications Source Type: blogs

The Little Louisiana Purchase
By ROBERT PEARL, MD “Pigs get fat, but hogs get slaughtered,” the saying goes. And so may it prove to be true for the pharmaceutical industry. Three articles, all published recently, illustrate the greed and egregious pricing by certain drug companies that are gaining public recognition and scrutiny. Marathon Pharmaceuticals LLC serves as a case in point. Over the last 15 years, its chairman and CEO Jeffrey Aronin generated a billion-dollar valuation for the company. As reported in a Wall Street Journal article, “Drug Price Revolt Prods a Pioneer to Cash Out,” he achieved this milestone not by inventing ne...
Source: The Health Care Blog - May 12, 2017 Category: Consumer Health News Authors: John Irvine Tags: Uncategorized Source Type: blogs

New Checks and Balances For Big Pharma
By ROBERT PEARL, MD “Pigs get fat, but hogs get slaughtered,” the saying goes. And so may it prove to be true for the pharmaceutical industry. Three articles, all published recently, illustrate the greed and egregious pricing by certain drug companies that are gaining public recognition and scrutiny. Marathon Pharmaceuticals LLC serves as a case in point. Over the last 15 years, its chairman and CEO Jeffrey Aronin generated a billion-dollar valuation for the company. As reported in a Wall Street Journal article, “Drug Price Revolt Prods a Pioneer to Cash Out,” he achieved this milestone not by inventing ne...
Source: The Health Care Blog - May 12, 2017 Category: Consumer Health News Authors: John Irvine Tags: Uncategorized Source Type: blogs

The Louisiana Purchase
By ROBERT PEARL, MD “Pigs get fat, but hogs get slaughtered,” the saying goes. And so may it prove to be true for the pharmaceutical industry. Three articles, all published recently, illustrate the greed and egregious pricing by certain drug companies that are gaining public recognition and scrutiny. Marathon Pharmaceuticals LLC serves as a case in point. Over the last 15 years, its chairman and CEO Jeffrey Aronin generated a billion-dollar valuation for the company. As reported in a Wall Street Journal article, “Drug Price Revolt Prods a Pioneer to Cash Out,” he achieved this milestone not by inventing ne...
Source: The Health Care Blog - May 12, 2017 Category: Consumer Health News Authors: John Irvine Tags: Uncategorized Source Type: blogs

Orphan Drugs: Pursuing Value And Avoiding Unintended Effects Of Regulations
Amid ongoing scrutiny of high pharmaceutical drug prices, debate about the value of “orphan drugs”—those designed to treat rare conditions that affect fewer than 200,000 individuals—continues to capture public and policy maker attention. The latest examples involve Emflaza and Spinraza, drugs used to treat the rare genetic childhood disorders of Duchenne muscular dystrophy and spinal muscular atrophy, respectively. Both have been approved by the Food and Drug Administration (FDA) and provide meaningful efficacy and value to patients affected by these conditions. However, outcry has developed in response to ...
Source: Health Affairs Blog - May 4, 2017 Category: Health Management Authors: Joshua Liao and Mark Pauly Tags: Costs and Spending Drugs and Medical Innovation Insurance and Coverage Quality Duchenne Muscular Dystrophy government regulation Orphan Drug Act orphan drugs value-based pricing Source Type: blogs

Value Frameworks For Rare Diseases: Should They Be Different?
The US health care system is increasingly focusing on value as a basis for reimbursement of pharmacotherapies and devices, and as a result the use of “value frameworks” for measuring and comparing treatment value has grown in recent years. However, the therapies assessed by most frameworks frequently apply to modest-to-large disease populations, rather than the smaller populations affected by rare diseases, where the factors driving value may differ. Rare diseases are different from diseases affecting larger populations in several fundamental ways. In the United States, a rare disease is defined as one that affects few...
Source: Health Affairs Blog - April 12, 2017 Category: Health Management Authors: Anupam Jena and Darius Lakdawalla Tags: Costs and Spending Drugs and Medical Innovation Quality Orphan Drug Act Precision Medicine rare disease treatment treatment value value frameworks Source Type: blogs

Senators Ask GAO to Investigate Potential Orphan Drug Act Abuse
In a March 3, 2017, letter to the United States Government Accountability Office, Senators Orrin Hatch, Chuck Grassley, and Tom Cotton raised the idea that regulatory or legislative changes might be needed to “preserve the intent of this vital law” that gives drug makers lucrative incentives to develop drugs for rare diseases. The letter notes that, “[w]hile few will argue against the importance of the development of these drugs, several recent press reports suggest that some pharmaceutical manufacturers might be taking advantage of the multiple designation allowance in the orphan drug approval process.” The sena...
Source: Policy and Medicine - April 6, 2017 Category: American Health Authors: Thomas Sullivan - Policy & Medicine Writing Staff Source Type: blogs

A Better Balance Between Accelerated Access And High-Priced New Drugs: A New Conditional Approval Option
There are two different storms brewing in the pharmaceutical world. On the one hand there is increasing opposition to the very high prices of drugs. On the other hand there is ever more pressure to accelerate access to drugs for seriously ill patients. President Trump expressed both of these very different concerns in his January 31st meeting with pharmaceutical executives, during which he called drug prices “astronomical” while also vowing to “streamline” the process of drug approval. It is vital that the cost of new drugs not overwhelm patients and the health care system. It is also important to get drugs to desp...
Source: Health Affairs Blog - March 20, 2017 Category: Health Management Authors: Robert Bohrer Tags: Costs and Spending Drugs and Medical Innovation Drugs and Medical Technology accelerated approval conditional approval drug pricing Duchenne Muscular Dystrophy early access parallel track Source Type: blogs

What Experts in Law and Medicine Have to Say About the Cost of Drugs
By ANDY ORAM Pharmaceutical drug costs impinge heavily on consumers’ consciousness, often on a monthly basis, and have become such a stress on the public that they came up repeatedly among both major parties during the U.S. presidential campaign–and remain a bipartisan rallying cry. A good deal of the recent conference named Health Law Year in P/Review, at the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School, covered issues with a bearing on drug costs. It’s interesting to take the academic expertise from that conference–and combine it with a bit of commo...
Source: The Health Care Blog - February 2, 2017 Category: Consumer Health News Authors: John Irvine Tags: Uncategorized Andy Oram Drug Pricing Pharma Source Type: blogs