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Friday, September 16, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, your daily source for all things biotech. Check out the STAT Pharmalot newsletter for more on the drug industry, and follow us on Twitter for more: @damiangarde@megkesh, and @statnews.

Straight talk from the FDA's clinical trials guru

Robert Moscicki, formerly the chief medical officer of biopharma giant Genzyme, has settled into a comfortable regulatory role: He's now the FDA's deputy center director for science operations and spends much of his time overseeing clinical research. He sat down with STAT this week in Washington, D.C., for a quick chat. 

Can we talk about compassionate use programs, when very sick patients petition for access to experimental drugs? What impact does that have on companies?
FDA has been very supportive for expanded access. We get about 1,000 applications for that each year, and approve about 99 percent.

I think the biggest burden of expanded access is on the [drug maker] — they have the toughest decision to make, on whether they will give access. For many, it’s a matter of cost — particularly small companies, who often don’t have enough money to manufacture enough drugs for clinical trials. It’s a difficult decision for them.

Companies also worry about the fact that if a patient can have a drug through expanded access or compassionate use, maybe they’ll choose not to be in a clinical trial — or drop out if they know they got the placebo. And therefore, the trial will be busted.

And then some companies fear that adverse events may occur, in patients that are already extremely sick. So if something happens, that might derail their perception of the product. Of course, we did a little analysis of that, and over a 10 year period of all INDs related to expanded access, we found only two instances where the FDA put a clinical hold [due to adverse side effects].

We're seeing more companies getting FDA breakthrough designation for their drugs. What's driving that trend? 
I think in the biopharmaceutical world, there’s been a big change — partly driven by payers — because I think the days when you could create the fifth “me too” drug is gone. For a long time, for Big Pharma, there was much less risk when you’d create a once-a-day drug, when previously the standard was a twice-a-day drug. There’s not as much value there for payers anymore.

So I think pharma companies have bought into that idea that they need to create breakthroughs — tackling unmet needs, instead. They’re trying to create drugs that make not just a marginal difference, but a large one that will show value to the world.

How do small, orphan clinical trials differ from larger ones, in terms of FDA oversight?
Some orphan diseases are big enough that the standards are very applicable. Cystic fibrosis, for example, has 30,000 patients in the US. But there are many ultra orphans, where the sample size for a clinical trial is very limited.

So what the FDA has done, in many circumstances, is carefully applied flexibility into our expectations . We still want substantial evidence, of course. That’s the law. But we’ll consider a surrogate endpoint to rare disease.

23andMe is very good at marketing

This time last year, 23andMe was still in trouble with federal regulators for marketing consumer genetic tests detailing disease and drug sensitivity risks without the proper approval.

These days, though, 23andMe seems to be everywhere — thanks to a lot of savvy marketing.

23andMe now has its tests on the shelves at CVS and Target. It's promoting stories of people who have used the service to find their long-lost relatives. And yesterday, it signed up the governor of Nevada, who's sending away for a 23andMe spit kit as part of a population health study that aims to analyze the genetic data of 5,000 Nevada residents.

23andMe's competitors have also pulled out a few marketing tricks. Last year, for instance, Ancestry.com, made a splash with its unexpected contribution to presidential history — when it helped reveal that Warren Harding had a love child.

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Another biotech unicorn in no rush to go public

Intarcia, the Boston-based maker of an implantable diabetes treatment, just raised another $215 million in venture capital with no near-term plans for an IPO.

Intarcia CEO Kurt Graves told Fortune that his company expects to raise even more cash before the end of the year, which would bring its latest round to as much as $600 million and give it nearly $1.8 billion in cash. With all that money, Intarcia expects to win FDA approval for its implant, market the product, and start booking revenue long before it goes public. It's penciling in an IPO for late 2018.

Meanwhile, for biotechs that don’t have more than $1 billion in the bank, the IPO market remains lukewarm.

How do you get scientists to play nice?

Drug companies are constantly pairing up with academic institutions, promising “a fruitful collaboration” through which “basic research and drug discovery scientists may leverage each other's strengths in the fight against disease."

But what we on the outside often forget is that the motivations of an ivory tower-dwelling academic and drug company scientist are often quite different.

Pfizer Senior Vice President Morris Birnbaum wrote an editorial in Cell about the disconnect, and Derek Lowe picked up on it in Science Translational Medicine. Each highlights a key potential stumbling block: Academics are great at coming up with cool stuff, but drug companies are chiefly interested in cool stuff that leads to new drugs. And the difference can lead to friction.

So how do you bring the parties together? Drug companies would be foolish to write blank checks in support of basic research, however fascinating, that never results in an actionable drug candidate. And academics likely have no interest in becoming service providers on the hook for meeting monthly quotas. Instead, Lowe and Birnbaum advise the semi-strange bedfellows to chart a middle course. Everyone needs to understand everyone else, they write, if they want to walk away satisfied.

More reads

  • A bipartisan group of lawmakers introduced a bill that would require drug companies to justify price hikes of greater than 10 percent. (STAT)
  • Novavax's in-development vaccine for respiratory syncytial virus failed in a late-stage trial, sending the company's share price down more than 80 percent. (Press release)
  • Turmoil at the federal Office of Research Integrity could be bad news for biomedical research. (Washington Post)
  • Japanese drug maker Eisai is opening a genetics-focused research center in Massachusetts, hoping to develop targeted treatments for dementia, cancer, and autoimmune disease. (Press release)

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Enjoy the weekend. We'll be back with more news on Monday.

Damian & Meghana

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