Puente: I've heard several times today that the NCCN Guidelines and the collaborative Value Pathways with NCCN will never put cost
over efficacy. That's a guarantee certain to be tested over time, as we all know that for oncology drugs it's a steep hurdle
to gauge overall survival benefits. Efficacy alone can be an ambiguous metric. A drug might post a spectacular improvement
against existing therapy in a small number of patients, but because of crossover effects performance in a larger trial set
is muted. This is evident in the international setting, where there is even less consensus on how a benefit is defined and
measured. Drug costs will also continue to rise, particularly in specialty areas like cancer, because of the complexity of
the follow up—not only in how drugs are administered, as part of the continuum of increasingly personalized care, but in the
post-marketing studies and trials that industry must commit to in responding to regulator concerns about safety.
Fitzgerald: There is a discrepancy too in the fact that diagnostics have a lower burden of proof in making the case for regulatory approval.
Yet all our guidelines panels want the same level of evidence, whether it is a drug, a device or a diagnostic. Industry must
develop evidence recognizing that stakeholders may require a different value proposition: what the FDA wants is one thing;
what the physicians or the payers want may be another.
Robert Martell, Tufts University Medical Center
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Gary Geipel, Eli Lilly & Co.: Pressures on innovators are coming from all points. Data that we generate to obtain a market license is not always going
to be sufficient to get a product supported on a pathway, while the very existence of a pathway can hinder the generation
of the real-world evidence that might lead to additional understanding of effectiveness. It's important to remember that cancer
progress almost always has occurred in a step wise incremental manner—often based on insights in the real world of practice.
Dr. Robert Martell, Tufts University Medical Center: It follows that, as personalized medicine takes root, indications for oncology drugs are becoming smaller. This puts significant
strain on the process and cost of obtaining regulatory approval. You can build down from an approved indication for ALK positive
lung cancer because the eligible patient population is large. But when you have a relatively smaller indication and then seek
approval for a still smaller sub-set of that, how do you do it at a cost commensurate to the potential size of the market?
You can't be funding 600 trials in such a situation. The system has to adjust: you might have a lower hurdle in winning initial
approval, followed by a structured, step-by-step process to better understand how an additional indication might add to the
disease fighting armamentarium, perhaps in combination with other therapies. Over 80 percent of oncology drugs are initially
approved as single agents, yet clinical exposure tells us that their best use comes as combinations. In fact, the NCCN Guidelines
for the top 10 malignancies recommend combinations as primary treatment in most situations.
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