In a follow up to the last post, there are indications that regulators and developers are beginning to look at different models to better address health needs. According to a Market Watch interview with Margaret Hamburg, commissioner of the FDA, she indicated that the agency had to do a better job of evaluating and approving new treatments and to get them to patients more quickly.
"We're relying on 20th-century approaches for review of 21st-century therapies," Hamburg said. She said the science of regulation needs to adapt and become more innovative, just like the market it oversees. I think that is a refreshing statement. Hamburg further noted that lots of time and money are wasted on ineffective treatments because of the aging regulatory approach. Hamburg urged Congress to pass stalled legislation to provide budget to give the agency tools to update its approach.
Following the theme of adaptive trial design, Fierce Biotech reports UCSF is taking a leading role in the I-Spy 2 cancer trial. This trial will test up to 12 therapies in an attempt to identify the right population for the right therapy on a much smaller budget.
Initially five breast cancer drugs from three companies - Abbott, Amgen and Pfizer - will be tested for their efficacy. The study uses MRI to track tumor response to shorten time lines. The vision is a 300-patient phase III trial instead of a 3,000 patient trial. As one drugs fails in the process another can be sustituted in for testing. Thus, the trial adapts to results as it proceeds.
Finally, the Pharma Reform blog talks about changes that need to occur in R&D to adapt to the realities of the new environment. It points out five areas of traditional thinking to reconsider for a new approach:
- Kill early, kill fast. This would sem best suited to the "mass market" use of a drug. However, in the era of personalized medicine, drugs that may not have made the cut before might work well for patient subpopulations in a more targeted approach.
- 'What's the fastest, commercially viable indication that will get us to market". This may no longer be viable as FDA, Third Party payers, and others want to see data to support specific claims for use and reimbursement. We're entering an age of comparative effectiveness.
- "if we do that study or analyze that data, we might find something we don't want to know" Product liability and coverups continue to make headlines. companies are expected to do the science to understand their products and to be transparent about it.
- "our research is built around the biological target X program" Focusing on single targets is hit or miss. Companies will need to take a more comprehensive approach to understanding disease progression and will develop multiple ideas on how to stop disease progress across more than one perhaps interacting targets. This will cut dependency on a single target.
- "we can do it cheaper and better in-house" Innovation demands outside and inside collaboration. Many functions can be outsourced to groups with equivalent if not better expertise. This will provide more flexiblity around facilties, equipment and personnel and reduce overall costs.
New ways of thinking and doing are coming.
Posted by Bruce Lehr October 11th 2010.