3 More Stock-Moving Binary Events to Watch in 2012

• Report this Comment On December 31, 2011, at 3:45 PM, PhillyDan wrote:

  Brian - the FDA is turning over a new leaf, yes to be fair, it may or may not help Arena or Vivus but don't bet against it. My question to you is why you have not reported on this significant development being undertaken by the FDA?

  The "Obesity Project"

  This project is the start of the FDA to host a series of disease-specific meetings to identify key risk/benefit goals of patients. The goal is to obtain the patient's perspective on disease severity and unmet medical need.

  (JS comment: As stated, the intent is not help Arena, Vivus or Orex. But, in my opinion, the results of these meetings will be coming into the FDA in the first half of 2012. These results/feedback certainly can work to help change the attitude/environment of the FDA towards Obesity, we can only hope.)

  Focus on Identifying Patient Priorities—Not Salvaging Applications.

  The GW project won't directly help (implies indirect help) any of the pending sponsors. For one thing, Woodcock says it is just getting organized and should start in the fall or winter of 2011. (Actually is is underway.)

  More to the point, though, the goal of the effort isn't to salvage pending applications, but to set better standards for future drug development in the class. The weight loss drug experiences, in effect, have provided a case study that supports the agency's push to build in better regulatory standards up front in the regulatory process.

  GW will "serve as a neutral convener" to host "a series of meetings that include patients, patient societies, bariatricians, ethicists, regulated industry, different regulators and so forth." The plan will be to "talk about obesity and what are potential benefits you could get from reducing weight. What evidence is there about their benefit? What are the harms that we are considering in these obesity drugs and how do you balance those two?"

  The goal will be to drill down into explicit tradeoffs in risks and benefits for weight loss, to give the agency a deeper understanding of how to make risk/benefit judgments in the class—and to give sponsors better guidance on how to design trials (and choose interventions) to optimize the risk/benefit ratio.

  The incorporation of patient perspectives into FDA's benefit-risk decision-making is a major change to its approval calculus, being pursued on multiple fronts. (Brian: The leaf is already turning...)

  In addition to the weight loss project and the workshops described in the PDUFA discussion with industry, FDA has also recently contracted with Sonal Singh in the Department of Medicine at Johns Hopkins for further work on the effort to create a systematic benefit-risk assessment process.

  FDA describes the objective of the Hopkins project also with the patient's view of benefit-risk high in the priorities. The agency is looking to the Hopkins benefit-risk process to "allow for quantitative and qualitative input from various stakeholders." The objective is to find a way to "systematically address risks and benefits in a transparent patient-centered way."

  In addition, the agency is doing extensive work on developing patient-reported outcomes to gather more data about patient experiences in clinical trials themselves.

  A Better Way?

  For Woodcock, in other words, the GW project isn't just aimed at a better path forward in obesity, but a better model for drug development overall.

  "What happens now is that people bring us stuff, development programs that may have cost $500 million to complete, and they say: 'Is this good enough?'" Woodcock said. "We might take it to an advisory committee and then everybody scratches their head." (JS comment: Sounds familiar!)

  "What would really be preferable is to work with the patients and the treating physicians in a proactive way before the development programs are done" to define exactly how individual patients weigh the burden of their disease and the potential harm of therapies. She noted a recent survey of IBS patients conducted by the International Foundation for Functional Gastrointestinal Disorders which asked patients explicitly whether they would trade a 1 in 100 mortality risk for symptom relief. "That kind of input is very important."

  Rather than debate those issues when the development program is done, Woodcock says, it is far better to "lay that all out in advance and then have people do development programs against those standards that are basically consensus standards."

  The seeds of the GW project in obesity followed from the rejection of the three pending applications. In March, Woodcock met with medical and patient representatives from the Obesity Action Coalition, the Obesity Society, the American Dietician Association, and the American Society for Metabolic & Bariatric Surgery. The associations described the meeting as an opportunity to discuss their "concerns about innovation" in pharmacotherapy for the condition.

  CDER will be working with GW School of Public Health & Health Services Professor Christine Ferguson, who is also director of the STOP Obesity Alliance, whose steering committee includes the Obesity Society and the Obesity Action Coalition, as well as other prominent medical (AHA, ADA), health industry (AHIP), employer and labor organizations.

  Ferguson herself joined the GW faculty after serving as head of the Massachusetts Department of Health under then-Governor Mitt Romney (R.). She also spoke during the 2011 Pharmaceutical Research & Manufacturers of America annual meeting in a session focusing on the need to address the societal cost of diseases like obesity. (Doesn't sound like a lifestyle issue to me...)

  On FDA's side, the project will be managed directly out of Woodcock's office. That will further insulate the initiative from direct consideration of the near-term weight loss therapies.

• Report this Comment On January 02, 2012, at 12:00 PM, TechNicole wrote:

  You go Philly Dan!

  Maybe you need to take Brian's place.

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