... Gottlieb believes that drugs “are priced to some measure of the cost of the capital -- including the investment capital -- that’s required to discover and develop them. And the risk and time and cost of the regulatory process are a big part of that equation.” The Commissioner’s plan will include a “broad range of steps we’ll take to make sure that our own regulatory tools and policies are modern and risk based -- and designed to facilitate the development of potentially breakthrough new treatments.”
... “This new policy will address the issue of targeted drugs, and how we simplify the development of drugs targeted to rare disorders that are driven by genetic variations, and where diseases all have a similar genetic fingerprint, even if they have a slightly different clinical expressions.” The guidance will clarify circumstances in which FDA may approve a cancer drug based on its molecular mechanism of action rather than the specific tissue or organ where tumors occur. It will also help sponsors develop drugs for rare subsets grouped by laboratory testing, so they can be studied in a single clinical trial.
... Gottlieb also understands that regulatory transparency cannot be a “for thee but not for me” proposition. Per the Commissioner, “We should be making sure that we try to provide as much information back into the market of ideas as possible. There are places across this agency where we bottle up too much information.” He singles out complete response letters as a “place where we should ask hard questions because there’s some very important information in those communications.”
... FDA is holding a public meeting on July 18 to solicit ideas about ways to administer the Hatch-Waxman Act “to help ensure the intended balance between encouraging innovation in drug development and accelerating the availability to the public of lower cost alternatives to innovator drugs is maintained.” Gottlieb has said he hopes to hear ideas at the meeting about ways to prevent manufacturers of branded drugs from blocking competition.
The full article is well worth the price of admission.