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Understanding the FDA – Biotech In the Money

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  • Chapters
  • 1. Why the Paradigm Shift at the FDA, over the Past 5 Years?
  • 2. Is Anything Binding With the FDA Anymore, or Are Companies Just Not Listening?
  • 3. Chronic Disease vs. Critical Needs: the FDA Fast Track, and Recourses for Companies
  • 4. Biosimilars are Without a Champion at the FDA
  • 5. FDA Very Actively Enforcing Compliance Regulation
  • 6. If You Had Unlimited Powers, How Would You Reform the FDA?
  • 7. Combination Therapy in the Eyes of the FDA. Can We Let FDA Off the Hook? The System Return to a More Rational Process

1. Why the Paradigm Shift at the FDA, over the Past 5 Years?


During past month, many important life science events were FDA driven : Dendreon (DNDN) with the Provenge approval, Intermune (INTM) with Pierfenedone’s rejection, the discontinuation of Merck’s biosimilar EPO program after the company received feedback from the agency, NicOx failed to gain FDA panel’s endorsement for Naproxcinod.. Hence this FDA SPECIAL of Biotech in the Money. Marina Bozilenko and Ed Allera (DC Attorney, FDA expert) discuss the tremendous change at the FDA in the past 5 years, as it underwent a complete philosophical change. Contributing factors are the Medicare Monetization Act (MMA, Medicare Part D), the change of control of Congress to the Democrats, the Food & Drug Amendments Act, the new (Obama) Administration and its Healthcare Reform. Ed argues that the perception of government was that “the pendulum had swing too far to the approval side.” Now, in the post-Vioxx-era, with Congressional oversight on the FDA, there is a much greater risk aversion.


2. Is Anything Binding With the FDA Anymore, or Are Companies Just Not Listening?


One of the biggest complaints companies have with FDA is that it changes regulations on a whim, which results in missed timelines and significant increases in clinical development spending. FDA’s primary complaint is that companies dont listen. Ed Allera explains FDA gives nuanced answers, and too often, companies hear what they want to hear. Breaking new ground (with FDA) in this day and age is very difficult. Is the FDA’s fault or the companies’ fault? Ed argues companies should learn to listen, and since FDA is “holding all the cards” it is healthy to get as many people as possible (Congress, key opinion leaders, media) looking at the trial to add more transparency and accountability to the FDA reviewal process. Ed also comments on the recent Dendreon Provenge approval.


3. Chronic Disease vs. Critical Needs: the FDA Fast Track, and Recourses for Companies


Is there still such a thing as “significant clinical need” or is everything a “chronic disease” now? While it used to be easier to develop drugs for cancer or infectious diseases, that doesn’t seem to be the case anymore. Are there any legal recourses to an “unfair” FDA decision? Can companies sue the FDA? How does sunlight bring sanity?


4. Biosimilars are Without a Champion at the FDA


Will all the talk about biosimilars finally result in concrete guidelines? Ed Allera explains that every initiative needs a champion, and that biosimilars have never had their champion inside FDA. Is there a chance they will?


5. FDA Very Actively Enforcing Compliance Regulation


The FDA has been very actively enforcing compliance. Curiously, FDA inspections are more stringent in the U.S. than abroad. Ed Allera talks about the FDA;s “vendetta” against pre-natal vitamins, and the failure of their transparency initiative.


6. If You Had Unlimited Powers, How Would You Reform the FDA?


If the FDA were to be reformed, Ed Allera would start by ensuring a more fair risk benefit calculation (current concerns being over rare events that are hard to detect), and re-initiating the dialogue on what is acceptable risk. As we move to more sophisticated technology platforms that can gather information on adverse events FDA should leverage this data. Additionally, Ed believes that the effort to control the message drug companies put out is ill considered given the amount of information that is available to patients online.


7. Combination Therapy in the Eyes of the FDA. Can We Let FDA Off the Hook? The System Return to a More Rational Process


Does FDA understand the critical need for combination therapies, and will they help the industry get some of these approved? Ed Allera believes FDA has an abstract understanding of combination therapy, and is being cautions because “they’ve already gotten beaten up on antibiotics.” Ed argues that given the amount of off label prescriptions and phase 4 breakthroughs, FDA could be held to a less stringent critique if something does go wrong. Ed also brings up the issues of demonstrating value add and comparative effectiveness. To the question “is there any good news?” Ed answers that the state of the current regulatory environment is just a phase, and that as more people raise issues, the system will return to a more rational process.


This interview was conducted at the NASDAQ Marketsite, on May 26th, 2010, in New York City.

Featuring:

  • Marina Bozilenko, Executive Producer, BioBusiness.TV
  • Edward John Allera, Chairman, FDA/Biotechnology Section, Buchanan Ingersoll & Rooney

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

Related Videos:

  1. IPO Review, OSIP, Medivation, Xenoport, Genzyme – Biotech in the Money
  2. Hepatitis C, Neurology, Healthcare Reform, Genetic Testing – Biotech in the Money
  3. Genzyme / Sanofi : Why The Price Isn’t Quite Right
  4. An Interview with Steve Burrill
« VICL – Vical CEO Vijay Samant interviewed by Stephen Willey
AMRS – Amyris CEO John G. Melo Interviewed by Boonsri Dickinson »

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  • About the Show


    A financial perspective on hot industry topics, the state of the markets, and the inner-workings of the biotech industry.

    Hosted by Marina Bozilenko, Head of Biotechnology & Pharma, William Blair & Company.

  • Related Videos

    1. IPO Review, OSIP, Medivation, Xenoport, Genzyme – Biotech in the Money
    2. Hepatitis C, Neurology, Healthcare Reform, Genetic Testing – Biotech in the Money
    3. Genzyme / Sanofi : Why The Price Isn’t Quite Right
    4. An Interview with Steve Burrill
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