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Precision Medicine: The Need for Expanded Compassionate Use Programs

Oct 16, 2020

ORION by VieCure

Volume 1, Issue 7

Devan Birch, BA and Fredrick Ashbury, PhD

The Burden of Cancer

Healthcare spending on cancer treatment is escalating at an alarming rate. In 2014, cancer treatment spending reached $88 billion. Between 2009 and 2014, a single new cancer drug launched for about $100,000 for one year of treatment.

Oncology’s paradigm shift away from the “one-size-fits-all” treatment philosophy to a precision oncology focus has yielded more therapeutic options to improve outcomes. Yet, these novel agents are very expensive. Launch prices for immunotherapies and targeted agents have climbed to as much as $400,000 for a year of treatment.

If a decision is made to pursue the genomic-based therapeutic options, the patient is placed in a decision crisis: the cost burden of treatment versus her/his ability to pay. For many patients who cannot afford these drugs or who lack coverage, access to these potentially lifesaving/life-prolonging options will be impossible”. Therefore, we need alternatives to cover the cost of such therapies. Greater access to compassionate use programs is one such alternative.

Our Study: Cost & Benefits of Precision Medicine

As part of a recent study with a client partner to compare the costs and benefits of precision medicine to standard of care in oncology (see ORION Volume 1, Issue 5), we explored compassionate use access among patients prescribed genomic-guided therapies. Based on the amount of precision medicine drug acquired through these programs, we were able to estimate the potential cost of the drugs if they were to be acquired at AWP-40%(average wholesale price – 40% discount).

What did we learn?

  • Using AWP-40%, over 4.3 million dollars over 3 years, of drug were acquired through compassionate use programs for patients we studied, of which some 3.5 million dollars of drug were acquired from only 3 companies (see figure below).

  • Moreover, of the 12 possible pharmaceutical suppliers that had drugs associated with the patients’ gene aberrations in our study, we found that over 75% of the drugs acquired through compassionate use programs came primarily from 3 companies.

Concluding Remarks

  • Our data reveal that some companies appear to be a preferred choice from which to order drugs. Perhaps the pattern of ordering drugs from some pharmaceutical companies is associated with the practice’s experience that compassionate drug approval is more efficient and effective in these companies compared to the normal 3-4 week cumbersome process of others? If so, the pharma industry should consider a shift in their business model to allow more rapid approval of compassionate use to increase adoption of novel therapies and to gather important real-world data (RWD), such as survival, ePROS, and cost-benefit data to accelerate approval for new indications.

  • Improved access to compassionate use could also build goodwill with physicians and patients as a direct result of faster approvals for superior personalized treatment options.

  • We can mitigate the financial toxicity of multiple failed lines of therapy, which result in countless lives lost prematurely and the waste of billions of dollars using ineffective treatments.

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