Posts Tagged ‘Payment policy’

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

March 15, 2010

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.

Personalized Medicine and Health Care Reform: Looking Back, Looking Ahead

December 18, 2009

Will health care reform support personalized medicine?  In my mind, that depends on two important factors: 1) the extent to which health care reform is truly patient-centered (does it make room for patient differences, room for patient voices, and time for patient care?) and, 2) the extent to which it is innovation-friendly.

I’m focusing on the first topic in this post.  Earlier posts have rightly focused on comparative effectiveness research as one key provision.  If CER is structured correctly, it can help inform patients about optimal medical and health care options based on our differing needs.  These differences come from a number of factors, including different clinical conditions we may have, differences in our preferences and the way we view risk/benefit trade-offs, cultural differences, and certainly molecular differences.  CER structured to recognize, and respect, these differences can only accelerate the move to personalized care.  Yet it remains unclear if this is the kind of CER we’ll get.  I think only the Senate bill’s CER language gets us close to this goal, by fully including patients and providers in the process, fully embracing patient differences, and focusing the program on results communications and not national policy recommendations. 

CER is one of several aspects of health care reform that will have an impact on patient-centeredness and personalized medicine.  Just as important are provisions that will apply the scientific evidence to policy decisions.  This includes proposals to establish an independent Medicare advisory board, define physician “best practices” and performance standards, and establish standards for use of health information technology.   

These types of policies, when deployed as cost-control levers, could be one of the single biggest factors influencing the future of personalized medicine.  Payment policy measures designed to control costs and expand access—but that fail to encourage continued development and adoption of personalized medicine—could substantially delay or diminish opportunities for meaningful, measurable improvements in health care value and quality.

That’s because cost containment proposals that impose access restrictions based on average, population-wide study results risk ignoring the different needs of individual patients and discouraging adoption of personalized tests and therapies based on these differences.  For example, “pay for performance” programs focused on short-term provider efficiency could discourage physicians from using gene-based tests and targeted therapies to optimize care for the individual.  As a patient with epilepsy myself, I take a very personal interest in how these policies get developed and applied.  

This nexus of CER and policy levers was highlighted earlier this year in an NPR commentary from Anne Brewster, an internist and instructor at Harvard Medical School.  “Physicians may agree with the end goal, but many of us worry about the methods and unintended consequences.  Comparative effectiveness research sounds sensible.  Of course we need more studies to define best practices.  But I find myself afraid that the results will be used by policy makers, hospital administrators, and lawyers to further limit my autonomy by setting hard and fast rules about what is “right”.  Clinical situations are always nuanced, never black and white.  Perhaps it is semantic, but I want to feel that CER will empower rather than constrain me.” 

Enacting, and implementing, health care policies that support patient-centered care and the science of personalized medicine won’t be easy; but it is absolutely essential.  Let’s work together to make it happen.

By Tony Coelho, Chairperson, Partnership for Improved Patient Care

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