The myeloma community is at a critical moment in its history. While we are pleased that four new myeloma drugs (Darzalex®, Empliciti®, Ninlaro®, and Farydak®) were approved in 2015 by the US Food and Drug Administration, the annual price tag for some treatment combinations can climb well above six figures. Clearly, this is not a sustainable situation.

Recognizing our responsibility to lead the way in addressing costs effectively, members of the International Myeloma Working Group (IMWG), the research arm of the IMF, launched a plan of action at a special session of the June 2016 annual meeting held in Copenhagen this year.

“The discussion was very lively,” reported Prof. Jean-Luc Harousseau from France, who led the well-attended session. “Many are interested in this question. Are we [IMWG] able to have a significant impact on drug prices? Some were pessimistic. But others said we are a well-respected international group whose guidelines are followed around the world.”

An urgent need

Given the current budget-conscious economic climate, the IMWG recognizes there is an urgent need for clear myeloma treatment guidelines which consider cost, but which also protect patients from processes that may limit or distort access.

In the past two years, overly complicated and imperfect treatment value assessment tools have been offered by, among others, the American Society for Clinical Oncology, Memorial Sloan Kettering Cancer Center, and the National Comprehensive Cancer Network. But none drew the outcry that erupted over the myeloma treatment value assessment effort published in April 2016 by the Boston-based Institute for Clinical Economic Review (ICER).

Critics of the draft report were extremely worried that the Centers for Medicare and Medicaid Services (CMS) would turn to ICER for guidance on its proposed overhaul of Medicare Part B, as CMS had done previously in examining costs in other diseases. IMF Senior Director of Support Groups Robin Tuohy provided compelling testimony at a May 26th public hearing on the report, explaining why restricting access to myeloma drugs would have threatened the life of her husband Michael Tuohy, a 16-year survivor.

After an onslaught of criticism from patients, doctors, drug makers, and patient advocates, ICER issued a final report in June which admitted that the institute lacked the expertise to adequately assess the complexities of new myeloma therapies. It conceded that a “Fail First” policy is a mistake for myeloma patients. The report’s authors also indicated that they have come to understand that each myeloma patient is unique and that all therapies will be required during the course of
multiple relapses.

ICER, in essence, deferred to the expertise of doctors who work with myeloma therapies every day and to the experience of patients who have expressed both their needs and their views as impassioned advocates for all patients.

Time to act

But such efforts to shave costs off lifesaving treatments cannot and will not go unchallenged by myeloma experts. And so, the stage is set. It is time for the IMWG to act on behalf of patients.

In the next two months, IMWG members will:

• Prepare access or cost-stratified guidelines for use of therapies throughout the course of the disease, from frontline to relapse. The need for such guidelines was noted in a letter written by Dr. Philippe Moreau and Dr. S. Vincent Rajkumar, and published in The Lancet in July. In the face of rapid therapeutic gains in myeloma, they write, patients may not profit due to an “absence of a coherent strategy to tackle the heterogeneity of the disease, paucity of strategic trials and high cost of treatment.” To address the latter, the authors call for “the development of clear guidelines and treatment pathways that take efficacy, safety, and cost into account.” Such guidelines, they write, “should not be an extensive list of all possible options but rather a limited menu of one or two options that are judged to be preferred standards of care.”

• Initiate meetings of all stakeholders, including, of course, patients, pharmaceutical companies, insurers, central pharmacies, hospitals and clinics, health economists, and regulators, who will ultimately need to change the cost and reimbursement landscape.

IMWG consensus statements and guidelines routinely seek the maximum input and feedback to incorporate all opinions and suggestions. We anticipate that useful guidelines developed by the IMWG will also spur desperately needed changes and will transform the cost/payer landscape.

Give Where Most Needed

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