A genomic revolution is underway in the discovery and development of more effective, less toxic targeted therapies for patients with cancer, and in particular, for patients with often rare blood-related cancers. In 2014, the FDA reported that 75% of NDAs were approved in first cycle reviews with 57% granted priority review. Almost half were for orphan indications and first in class agents. A good number of these approvals were classified as breakthrough therapies.

While their ultimate benefit to patients is an improvement in overall survival, many of these new agents, which lack the debilitating side effects of more traditional cytotoxic chemotherapeutic agents, are providing additional benefits by improving patient outcomes—particularly with respect to reduction in, and control of disease-related symptoms, improving patients’ ability to re-engage in normal day-to-day activities and contribute to the economy and their communities and decrease healthcare utilization costs.

High Costs Are Questioned

It is clear that the innovation, nimbleness and the more patient-centric focus of small to mid-sized biotech companies is transforming the management and lives of patients with rare and blood-related cancers. What is less clear is the justification for the cost of such therapies. In the emerging and evolving genomic-targeted therapy era, we no longer randomly screen molecular entities looking for minor differences between normal and cancerous cell activity, but rather rationally design compounds to interrupt or inhibit the activation of molecular targets validated as causative or seminal to the disease processes we desire to regulate.

As a result, the time to discover new molecular entities is dramatically reduced and their probability of success in clinical studies is similarly dramatically improved, thus reducing the time, cost and risk of drug development. So why then charge $300,000 annually for a treatment for hemophilia or paroxysmal hemolytic anemia if the time, risk and cost horizons are markedly reduced for developing these new breakthrough therapies. Even if companies provide co-pay assistance, can the national healthcare budget continue to absorb such costs—or should it?

Patients Face Financial Burden

In the UK, the National Institute for Health and Clinical Excellence requires companies to justify the incremental cost effectiveness (ICER) for new therapies based on the impact on quality of adjusted life years (QUALY) the agent provides to patients. This methodology incorporates a non-subjective, quantitative evaluation of the impact of new agents on healthcare utilization costs to determine the price and access for new therapies.

Such an objective methodology does not currently exist in the U.S. Because many of these new agents are targeting orphan diseases, they often “fly” below the radar screen of healthcare payers, but not below the radar screen of the patients and their families who have to bear the burden of their high price.

For the first time in more than 25 years, we are seeing “practice-changing” new therapies for patients suffering from a spectrum of blood-related cancers ranging from myelofibrosis and chronic lymphocytic leukemia to multiple myeloma and lymphoma. The challenge going forward for the biotech industry is to find the middle ground that sustains innovation while providing socially responsible access to these new breakthrough treatments.

  • Dr. James Bianco

    Dr. James Bianco is Founder, President, CEO of CTI Biopharma. Dr. Bianco founded this Seattle-based biopharmaceutical company in 1992 with a focus on bringing less toxic, more effective therapies to treat and cure cancer, creating three novel targeted therapies for patients with blood-related cancers.


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