Gene Therapy Update Part 2

Last week we wrote about recent news in the field of CRISPR/Cas9 research. But CRISPR is not the only approach to gene editing under development, nor the most advanced.   First Human Gene Editing Data — But Not from CRISPR… Read »

Gene Therapy Update Part 1: Big Advances and Big Questions for CRISPR

The gene therapy field has seen several developments in recent months, especially around CRISPR, with both significant achievements and further controversies about the safety of the gene editing approach.   CRISPR Cure for Duchenne’s Muscular Dystrophy (DMD) in Dogs  … Read »

Seminal Immuno-oncology Discoveries Honored by 2018 Nobel Prize

Seemingly spontaneous remissions from terminal cancers are known to occur on rare occasions.  For over 100 years, this observation has spurred scientists to attempt to engage the immune system to fight cancer, with little success. But on October 1, the… Read »

The Growing Influence of ICER

We have previously written about the Institute for Clinical and Economic Review (ICER), the independent watchdog whose mission is to offer an objective evaluation of the clinical and economic value of prescription drugs, medical tests, and other healthcare innovations.  … Read »

U.S. Drug Spending: Where the Money Goes

Two recently published analyses are providing a more in-depth picture of prescription drug spending in the United States. Such evaluations illustrate the complexities facing policy makers and regulators seeking to help reduce patient and payer drug costs. In addition, they… Read »

FDA Issues Set of Guidelines on Gene Therapy

Back in December, the U.S. Food and Drug Administration (FDA) granted its first approval for a gene therapy product, Spark Therapeutics’ Luxturna. According to the Alliance for Regenerative Medicine, as many as 50 more such treatments — addressing a diverse… Read »

As Brexit Approaches, Concerns about Impacts Abound

On March 29, 2019, the United Kingdom is scheduled to leave the European Union (EU). Since the UK passed the referendum in 2016 to leave the economic bloc, there have been plenty of uncertainties on how this will affect not… Read »

The Drug Pricing Blueprint — and Audience Reactions

In May of this year, the Trump White House issued its “Blueprint to Lower Drug Prices” in response to the Administration’s views that excessively high drug prices “burdening the American people” were in part caused by “foreign freeloading” and a… Read »

Steps Toward the Use of Real World Evidence

“Real-world evidence” (RWE) is data on the use and accrued long-term benefits/risks of a drug or device that is gathered outside of a clinical trial — generally after the product has been approved. Clinical trials don’t often reflect the breadth… Read »

Drug Pricing and Pharma

In September 2015, Martin Shkreli, then CEO of Turing Pharmaceuticals, sparked widespread outrage in the United States when the company raised prices on an off-patent treatment for toxoplasmosis, Daraprim, by over 5,000%, just one month after Turing acquired the drug…. Read »

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