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This week, scientists will gather in Washington, D.C., for an annual meeting devoted to gene therapy—a long-struggling field that has clawed its way back to respectability with a string of promising results in small clinical trials. Now, many believe the powerful new gene-editing technology known as CRISPR will add to gene therapy’s newfound momentum. But is CRISPR really ready for prime time? Science explores the promise—and peril—of the new technology.

• How does CRISPR work?
• What has CRISPR accomplished so far?
• So why isn’t CRISPR ready for prime time?
• With these caveats, do you even need CRISPR?
• CRISPR also has other issues
• And CRISPR still has big safety risks
• So what’s the bottom line?

Sanofi said it could raise its proposed $9.3 billion deal to buy Medivation if the U.S. cancer drugmaker engaged in talks, threatening to go directly to shareholders to oust the board if not...Sanofi Chief Executive Olivier Brandicourt wrote in a letter to the board of Medivation published on Thursday that the transaction was a priority for the French drugmaker and it was committed to seeing it through...Medivation said...it had received a letter from Sanofi that "simply restates an inadequate proposal that...substantially undervalues the Company, its leading oncology franchise, and innovative late-stage pipeline"...Medivation's first-quarter earnings missed most analysts' forecasts...company reported non-GAAP net income of 11 cents per diluted share, up from 8 cents a year earlier, but much less than the average 23 cents expected by analysts...Sanofi is keen to boost its presence in cancer treatments...Medivation, known for its Xtandi prostate cancer treatment...

Valeant Pharmaceuticals International Inc, which has acknowledged mistakes in its drug pricing practices amid U.S. congressional probes, said on Thursday it has formed a new committee to oversee pricing of the company's drugs...The Patient Access and Pricing Committee will initially be chaired by Joseph Papa, Valeant's new chairman and chief executive officer... the committee will review the pricing of Nitropress, Isuprel, Cuprimine and Syprine...Valeant raised the price of Isuprel by about 720 percent and Nitropress by 310 percent...Prices of the other two drugs, used to treat a genetic disorder that causes copper to build up in the body's organs, were raised by 5,878 percent and 3,162 percent, respectively...This new committee will take a disciplined approach to reviewing the company's pricing of drugs, and will consider the impact on patients, doctors, and our health care industry partners...

Biotech stocks dragged on the market Friday, and some "Fast Money" traders contended that the pain may not be done...Gilead Sciences shares sank 9 percent on the day, after the pharmaceutical company posted earnings and revenue Thursday that fell short of expectations. The iShares Nasdaq Biotechnology ETF (Exchange Traded Funds), meanwhile, dipped 2.7 percent on Friday...that investors need to be "careful" choosing biotech stocks amid headwinds like the ongoing criticism around drug pricing...Trader Guy Adami was also cautious on the sector, saying he would not buy any of its stocks until the biotech ETF cracks $285 per share. It closed at about $268 on Friday...

In the more than thirty years I have practiced pharmacy, I have witnessed a tremendous evolution in the profession. The clinical foundation and training of a pharmacist graduating today is leaps and bounds above where I started my practice. However, one thing has not changed: a pharmacists’ role in patient care goes well beyond dispensing medications...Today, as before, many pharmacists provide patient-centered services...We are the front line of the health care team and often see patients more than any other provider. Pharmacists have become the most over-trained and under-utilized professionals in America...While doctors should remain the quarterbacks, pharmacists must be given "provider status" so the profession is able to be the integral part of the health care team we are trained to be...With provider status, pharmacists would be added to the list of Medicare providers which would not only allow for the best possible care for patients but would also ensure it is done in the most cost-effective manner...In my short time in Washington I have come to realize just how hard it can be to advance commonsense reform...a bipartisan majority of 272 members have cosponsored the Pharmacy and Medically Underserved Areas Enhancement Act introduced by Representative Brett Guthrie. This legislation simply gives pharmacists provider status...let’s move forward...to improve access to quality and affordable health care for all Americans.

A row has broken out between Merck and New Zealand over a pricey new cancer drug in the latest example of how the cost of medicines is a flashpoint between drug makers and governments...At issue is Merck’s Keytruda (pembrolizumab), one of the new oncology treatments that harness the power of the body’s immune system to battle tumors. The medicine was approved in the United States two years ago to combat melanoma and, more recently, to tackle the most common form of lung cancer. Although priced at a hefty $150,000 a year, Keytruda is largely covered by public and private payers in the US...In New Zealand, the drug was approved last fall to treat melanoma. But since then, Pharmac, the government agency that decides whether coverage will be funded, has so far refused to endorse Keytruda. The agency contends evidence is lacking to verify whether the drug helps melanoma patients live longer compared with other new melanoma treatments or standard chemotherapy.

Two rival cholesterol-lowering injections from Sanofi and Amgen have been recommended by Britain's healthcare cost watchdog after the manufacturers offered special discounts to the country's state-run health service...Sanofi's Praluent (alirocumab)...and Amgen's Repatha (evolocumab) are both so-called PCSK9 medicines...The National Institute for Health and Care Excellence said in draft guidance on...that both drugs could be considered for use by people whose cholesterol is still not under control despite trying other treatments...It stressed that the medicines would only be cost-effective with the promised discounts...Both drugs cost more than 4,000 pounds ($5,780) per patient a year in Britain. That is already a lot less than their U.S. list price of around $14,000, but Sanofi and Amgen have committed to discount the British price by a further undisclosed amount for the UK National Health Service

Mike Glaicar, Business Development: Pharmacy Times...(PTNN) This weekly video program provides our readers with an in-depth review of the latest news, product approvals, FDA rulings and more.

Go to the doctor or hospital when you're sick in the hopes of getting better, and you might end up dead, instead...A new study estimates that medical errors are actually the third-leading cause of death in the United States, responsible for a whopping 251,454 fatalities in 2013...Only heart disease and cancer, which respectively killed 611,000 people and 585,000 people that year, outpaced medical errors...death from medical error has been understated, in large part, because such error by health providers is not included on death certificates...There is no official disease code for medical error, and death certificates rely on such codes...As a result, causes of death not associated with an ICD code, such as human and system factors, are not captured..."human error is inevitable" and "we cannot eliminate human error, we can better measure the problem to design safer systems mitigating its frequency, visibility and consequences."

The Colombian health ministry plans to override a patent that Novartis holds on its widely used Gleevec cancer treatment, the latest clash between the pharmaceutical industry and some governments over intellectual property and access to medicines...The move...could prompt the government patent office to grant a so-called compulsory license to allow a generic company to make a lower-cost version of the drug...Compulsory licenses allow generic drug makers to make low-cost versions of brand-name medicines without the consent of the company holding a patent...a Colombian government committee explained that issuing licenses to other companies would be in the public interest by widening access and saving health care dollars. Issuing a license would "restore competition for this product in the Colombian market,"..."A Declaration of Public Interest leading to a compulsory license should never be used as a mechanism to force price negotiations,"..."This runs counter to the spirit and intent of a compulsory license and its legal framework, and would create a damaging precedent that could apply to all patent-covered innovations — pharmaceutical or otherwise."