More patients help drug firms pass 'valley of death'

August 11, 2014
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There’s no question the pharmaceutical industry has boatloads of cash.

Global sales of prescription drugs total nearly $1 trillion annually, according to IMS Health, a Connecticut-based market research firm.

Drug companies spend about $200 billion per year on researching and developing new drugs, according to life sciences investment firm Burrill & Co.

On top of that, venture capitalists last year plowed $4.5 billion into U.S. drug discovery firms, according to a MoneyTree report from Thomson Reuters and the National Venture Capital Association.

So when industry consultants and analysts a few years ago predicted patient advocacy groups would become critical sources of development funding, I was skeptical.

But patient groups—especially those formed around rare and untreatable diseases—are indeed playing a crucial role in getting experimental drugs across the proverbial “valley of death”—where none of those deep-pocketed players want to put their money.

That’s how it worked for Indianapolis-based Chondrial Therapeutics LLC, as I wrote about in this past weekend’s IBJ. Co-founders Dr. Mark Payne and Steve Plump, who used to be chief marketing officer at Indianapolis-based drug giant Eli Lilly and Co., had pitched their drug to large pharma and biotechnology firms.

Payne got two years of funding from Shire Pharmaceuticals, but when the company looked for another round to run a clinical trial, it came up empty handed.

So it turned to a patient organization, Friedreich’s Ataxia Research Alliance. One board member has now put $1.1 million into Chondrial to help it prepare for its first human trial in 2016.

Patients, along with their families and committed advocates, are turning out to be better sources of funding because they tolerate risk better than drug companies and investors. For patients—particularly those suffering from a disease with no treatment, as is the case with Friedreich’s ataxia—the risk of losing their money is, of course, a much smaller risk than losing their life.

Drug companies, while they may not be spending much money at these early stages, are more active than ever in talking to patient advocacy groups about developments in their disease.

Ron Bartek, who co-founded Friedreich’s Ataxia Research Alliance with his wife after their son was diagnosed with the rare genetic disorder at age 11, told me the interest of major drug companies has soared since the mid-2000s. He noted that Pfizer Inc. sent a team of employees to Bartek’s for an entire day to get the lay off the land on Friedreich’s ataxia.

The research alliance has built up a clinical database of more than 2,200 patients, which is critical for getting drug researchers started. It has also raised $30 million to fund research into cures for the disease.

"I know of no other more passionate pursuit than a parent’s pursuit of their child’s health," Bartek said.

That passion also translates into a willingness to pay high prices for drugs that give hope where none existed. Companies such as Novartis and Genzyme (now part of Sanofi) have gotten rich by launching disease-altering treatments for ailments that were once incurable. As patients live longer, the drugs can rack up huge sales.

“The research economics become very different if it truly has an impact on long life,” said Dr. Tim Franson, chief medical officer of Indianapolis-based life sciences consulting firm YourEncore, which maintains a network for 8,000 former pharmaceutical professionals.

Patients are also good sources of funding for a reason beyond money. Active patient groups like Friedreich’s Ataxia Research Alliance can help drug companies find the patients they need in a hurry.

Only one in every 50,000 people suffer from Friedreich’s ataxia, so finding a patient and then finding a doctor to conduct a clinical trial with him or her can be hugely expensive and time-consuming.

But patient groups have typically already done the hard work of attracting the relevant patients. When Bartek helped Edison Pharmaceuticals find patients for its latest Phase 2 trial of its drug for Friedreich’s ataxia, it took only two hours and 43 minutes.

Most drug companies take weeks, at least, to find the patients they need.

In addition, patient groups can actually help set the standard for success in a drug trial. The Wall Street Journal reported last week that a group for patients suffering from Duchenne muscular dystrophy—a rare form of an already rare disease—worked with the U.S. Food and Drug Administration to change the “primary endpoint” an experimental drug will be expected to meet to receive market approval.

Parent Project Muscular Dystrophy, a group focused on Duchenne muscular dystrophy, worked with 80 scientists, clinicians and drug professionals to write draft guidance for pharmaceutical companies trying to develop drugs to treat the fatal condition. The guidance calls for different measures of success than the typical six-minute walking test so that younger patients and those already seriously disabled by the disease could participate in the drug trials.

Franson was on the team that helped write that guidance for FDA, which still must approve it. But he’s encouraged by FDA’s willingness to be flexible.

“We’re certainly hopeful that this Duchenne example is replicated, in rare diseases but even outside of it,” Franson said. “I give them a lot of credit for stepping out and trying to establish new models for rare diseases. I’m very optimistic for how this will impact in other diseases.”

  • nice approach in developing new medicine
    It is a nice strategy to approach parents or patients families for research. I really hope there will be a good progress especially in duchenne muscular dystrophy research.

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