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New BIO chair hopes for greater policy focus on rare diseases


The newly elected chair of the the largest industry organization and lobbying group runs a company focused on drugs for rare diseases, and he has an eye on that area of drug development for his tenure.

The Biotechnology Innovation Organization said Tuesday that it had elected as chair of the board for the 2019-2020 term Jeremy Levin. Levin is CEO of New York-based Ovid Therapeutics and former CEO of Teva Pharmaceutical Industries, a position in which he served from February 2012 to November 2013. He replaces outgoing Chair John Maraganore, CEO of Alnylam Pharmaceuticals.

In an interview at the BIO conference in Philadelphia Wednesday, Levin said rare diseases will be a particular area of interest for him at the organization. Ovid is also focused on development of rare neurological diseases, and its most advanced drug candidate is OV101, for Angelman syndrome – a nervous system disease that causes severe developmental problems – in adolescents and adults.

“One of the things I would hope is that in the immediate future we start to dive into and work on policies appropriate to rare disorders,” Levin said. For example, a drug for a rare disease may enroll small handful of patients in each country, such as three to five in the Netherlands or Germany. As such, with so much in the way of cross-border trade, there needs to be an understanding of how therapies will be brought to those patients and a greater policy focus on rare disorders, he said.

An estimated 30 million Americans live with any of more than 7,000 rare diseases, with 90 percent of those lacking a Food and Drug Administration-approved treatment, according to the National Organization for Rare Disorders.

Meanwhile, a negative challenge the industry faces comes in the form of legislation at the state and federal levels regarding drug pricing.

Levin said the industry should not be shy about taking on companies and individuals who take advantage of the healthcare system to increases prices of old drugs without adding any value to them. In particular, he referred to cases of old drugs that increase in price year over year, as well as actors like pharmacy benefit managers and untoward price increases in hospitals. But he distinguished that from cases of high-priced drugs that have received much of the attention in the news media, such as Gilead Sciences’ Sovaldi (sofosbuvir), which carries a list price of more than $80,000 but is a one-time treatment. Whereas the proceeds from drugs like Sovaldi can help fuel future innovation, he said, the routine price increases on old drugs do nothing to encourage innovation.

He pushed back against the idea of there being “massive price increases” on drugs across the board and said the misperception that has created remains the organization’s primary negative challenge. “The accumulated deficit that we have developed over the last 10-15 years in public trust is important, and that has led to frustration and anger in the public and given fruit and opportunity for people to make fallacious claims about what companies do,” Levin said.

It’s undeniable that certain trends have fueled the perception of such a large-scale, across-the-board increase in prices. For example, last month, the Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec-xioi), a Novartis gene therapy for spinal muscular atrophy that is the world’s most expensive drug, with a $2.1 million list price. However, the Institute for Clinical and Economic Review deemed the high-priced therapy cost effective given that if it’s fully curative, the alternative – such as treatment with Biogen’s Spinraza (nusinirsen) – would be more costly in the long run. Spinraza costs $750,000 in the first year and $375,000 per year afterward, but it must be taken over a lifetime. Generic drugs have seen significant price increases as well. A lawsuit filed by several states alleges that several generic drug companies, including Teva, conspired to increase prices on generic drugs. Then, of course, there have been cases of companies like Valeant Pharmaceuticals and Turing Pharmaceuticals, which became infamous for increasing the prices of very old drugs for rare disorders on which they had monopolies. Turing’s former CEO, Martin Shkreli, was reported in March to be illegally running a drug company from prison using a contraband mobile phone.

“The consequence of that negative challenge is that investors, the capital markets and entrepreneurs will be faced with an onslaught of negative legislation detrimental to innovation,” Levin said.

Photo: Alaric DeArment, MedCity News

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