Can we continue making progress in the fight against cancer, while at the same time meeting the challenge of demonstrating value for innovative therapies?

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I think we can.

A paper released today by Boston Healthcare Associates describes some of the important considerations in meeting these goals. The paper, Recognizing Value in Oncology Innovation, highlights the growing role of personalized medicine in cancer care. This trend towards focusing on cancer treatments that are targeted to patients with particular genetic variations or other biomarkers illustrates how innovation can drive better patient care and improved health care value.

The paper also describes the broader dynamics of progress in cancer care, in which new drugs and biologics receive approval from the FDA based on initial clinical trial results, and then accrue additional evidence on their role and value over a considerable period of time (in some cases decades).  Boston Healthcare identified five mechanisms through which this occurs:

  • development of evidence of improved outcomes in the initial FDA-approved indication;
  • use earlier in treatment line and in earlier disease stage;
  • use in different disease indications;
  • use in combination with other agents; and
  • use in combination with specific biomarkers.

We examined numerous existing therapies to characterize and illustrate these different mechanisms.  In the report, we highlight four specific therapies that illustrate these points.  For example, imatinib (Gleevec®) was first approved in 2001 for treatment of advanced stages of chronic myeloid leukemia and second-line treatment for the earlier, chronic phase of the disease.  Approval was based on surrogate endpoints showing patient response to treatment at the cellular level.  By 2007, the clinical benefit was dramatically demonstrated through research showing an 88% survival rate for patients after six years of treatment, compared to an average five year survival of 48% prior to imatinib. Similarly, the evaluation of docetaxel (Taxotere®) based on early results would have substantially underestimated the drug’s impact on survival for patients with squamous cell carcinoma of the head and neck by more than 4.5 years.

In the paper, we also examine cancer therapies for which the optimal use is guided by genetic markers [trastuzumab (Herceptin®) and cetuximab (Erbitux®)]. With cetuximab, for example, subsequent studies identified a subgroup of patients with a genetic mutation who were less likely to respond to the drug. Patients without the mutation showed an overall survival of 15.6 months, compared to 5.6 months for patients with the mutation.

The Personalized Medicine Coalition addresses this important issue in its payer principles, noting that evidence on the full value of new personalized medicine technologies emerges over time after initial introduction. “As with most new technologies, the evidence available early in their life cycle might not be of the highest level or as conclusive as that available for other diagnostics and therapeutics,” PMC says.

If therapies such as those described in the Boston Healthcare paper had been subject to strict coverage policies that imposed rigid, up-front assessment of value, they might never have reached many patients. Indeed, in countries that make use of these types of assessments, cancer patients have faced potential barriers to treatments like imatinib and trastuzumab. As we seek policies to support continued progress against cancer and addressing rising health care costs, it is important for policy-makers to understand this aspect of medical progress, and ensure that emerging personalized medicine technologies continue to be developed and made available to patients.

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