- Express Scripts: Specialty meds driving up US drug spend (biopharmadive.com)
An estimated three out of every 1,000 Americans ring up a bill of more than $50,000 in annual prescription drug costs last year, according to a new analysis of health plan members published...by Express Scripts... The number of people who meet this threshold for high pharmaceutical expenditures has risen sharply, by 35%, since the PBM's last report on the trend in 2014. While numerically small, this group of patients accounted for more than 20% of total U.S. spending on prescription drugs in 2016...higher utilization of pricey specialty medicines for cancer, multiple sclerosis and rare diseases was the primary driver of spending among patients with annual pharmacy costs over $50,000. Yet that was a change from 2014, when new hepatitis C drugs and compounded medicines goosed spending...Nearly 96% of the plan members who cleared the $50,000 in annual spending threshold used specialty medicines, most notably cancer therapeutics. While the average annual cost of oncology drugs was lower than some other specialty categories, a quarter of all spending by this group was on cancer treatments...
- Biotech Stock Mailbag: Sarepta, Biomarin, Spark, Raptor (thestreet.com)
…I appreciate your prediction of the approval for the DMD drugs but I think you're too optimistic about Sarepta Therapeutics. I believe FDA approves Biomarin but tells Sarepta that another trial must be run…why Biomarin over Sarepta? Because what Biomarin is seeking to do -- make a convincing argument for drisapersen's approval based on messy clinical data…It's something that the FDA has done before with drugs for rare diseases… Sarepta's eteplirsen, by contrast, requires the FDA to set a regulatory precedent which the agency might regret later.
- Drugmakers ‘hijacked’ the FDA’s orphan system to score premium pricing on mass-market meds: report
There’s no denying that financial incentives for orphan drug development spawned meds that have saved hundreds of thousands of lives. But they’ve also helped mass-market drugmakers rack up millions in incentives, tax breaks and patent-protected profits—in some cases through monopoly pricing...About one-third of the orphan drug approvals the FDA doled out since the program began more than 30 years ago have been for repurposed, large-market products or drugs with multiple orphan green lights...Best-sellers such as Crestor…,Abilify…,Herceptin…,and Humira...fall into the category of big sellers whose makers snagged millions in government incentives—not to mention seven years of exclusive rights on the market—when they resubmitted their therapies as treatments for smaller populations...What we are seeing is a system that was created with good intent being hijacked…Repurposing a drug isn’t necessarily a bad thing, of course, if it can help get a treatment to additional patients...But when the orphan incentives allow competition-free drugmakers to charge whatever prices they want for their meds?...Now...it seems like...this practice may be driving up prices...Industry lobby groups...are unsurprisingly in favor of maintaining the status quo. With rare diseases “tragically killing and brutalizing mostly children,” incentives for orphan drugmakers should be kept in place...the risk of losing incentives in the system far outweighs the benefit of trying to save a few pennies on the health care dollar...
- India a potential center for orphan drug development and demand (fiercepharmaasia.com)
India may be sitting on a pharmaceutical gold mine. It has more than 7 times the number of people affected by rare diseases than the United States, a less expensive workforce for making drugs and an often-criticized price-control system that, by definition, does not affect orphan drugs…One India-based company sees a profitable outcome for its orphan drug, a sign the nation could become a major source of the often-overlooked treatments as other drugmakers watch to see if the optimism is justified and lasts… India offers the advantage of cheaper drug development, orphan drugs often have already produced or at least studied relatives, and many of them…are curative, thus reducing the burden on healthcare systems. And, it has a lot more potential patients.