Cancer R&D's tenuous value chain


The good news in oncology is that the "R" of R&D is alive and well - at least judging from from this week's AACR-NCI-EORTC conference, "Molecular Targets and Cancer Therapeutics". Although there haven't been many transformative, "aha" moments, there continues to be broad and deep science, innovation and passion in basic cancer research that lays a strong groundwork for discovering effective therapies. Further ahead in the value chain, however, the rest of the cancer drug development enterprise is in trouble. Worrying themes recurred in presentations and hallway chatter at the conference - and other big issues didn't come up, but are lurking in the background. Some big challenges to oncology drug development came to mind at "Molecular Targets" that need attention from senior R&D leaders so they don't block us from translating emerging science into effective medicines:

1. So many ideas, so little time: Early clinical development is the single biggest bottleneck in cancer R&D. We have plenty of great hypotheses of new combinations and dosing schedules to test, or niche populations that might benefit from a particular therapy, but conventional early trial designs are unwieldy, and force us to use weak preclinical models to pick only a handful of ideas to take into the clinic. In late development, we've gotten better at moving quickly from Phase 1b into Phase 3 and executing diagnostics-led trials, but our early-stage studies are still extremely conventional and not fit-for-purpose for the wide range of ideas we need to investigate. In order to capitalize on emerging preclinical science and identify the best ideas to advance into later studies, pharma R&D groups need to aggressively explore and vet new approaches, like alternate Phase 1 designs and adaptive Phase 1b and Phase 2 trials.

2. Acing the (diagnostic) test: How do we bridge the gap from research centers to widespread practice in cancer diagnostics? Everyone at "Molecular Targets" seems to agree we're headed toward multiplex testing at diagnosis and beyond to guide treatment choice, notwithstanding some scientific and clinical disagreements over platforms, analytes and technologies. But the elephant in the room is regulatory approval for tests in parallel with the associated drugs, which is hindering the path to the market. Pharma companies are keeping an eye on the evolving landscape, but there are worrying signs that FDA does not have a clear vision of how to move forward. Senior R&D executives need to more proactively engage government and the public on this key issue so we can remove the regulatory overhang and move cancer tests and drugs forward to the public.

3. The price is (not) right: Drug pricing isn't an appropriate topic for a cancer research conference, but it's the elephant in the room when it comes to pharma R&D decision-making. During the "Molecular Targets" conference, two new articles appeared in the lay press (here and here) that question the high prices of oncology drugs. Senior pharma executives are going to question the sustainability of cancer R&D, especially when they're asked to endorse proposals for combination therapies and drugs for niche populations. It's time for oncology R&D leaders to push their commercial colleagues to explore and implement novel pricing schemes, such as performance-based pricing, so this issue doesn't derail the entire cancer drug development enterprise.

It's clear from the "Molecular Targets" conference that this is an exciting time to be doing basic cancer research and developing new oncology drugs. But it's time for pharma R&D executives to focus on key issues related to clinical trials, diagnostics and pricing to get this great science to patients. Otherwise, the effort and money being invested in cancer R&D might be for naught.