Myeloma Experts, Not Bureaucrats, Should Create Treatment Recommendations for Patients

Week in Review
Dr. Durie


In my previous blog, I wrote about the myeloma drug cost assessment report drafted by the Institute for Clinical and Economic Review (ICER). The potential contents of the ICER report continue to raise eyebrows—and considerable ire—among patients, who worry the report’s findings will limit treatment access. The American Society of Hematology (ASH) expressed its concern here. As the May 26th hearing on the draft ICER report draws near, the question being debated by patients and doctors, who are making their voices heard online, is:

Given legitimate concerns about the rise in overall healthcare costs, can any effort to assess value of drug treatment NOT threaten access to those treatments?

In simple terms, it is clear that since drug costs are high, there are only two choices to achieve financial feasibility: reduce costs or reduce access. To limit access based primarily upon costs is not valid or fair. A system is needed whereby available therapies are assessed by an expert panel—aware of costs—and recommendations made for realistic drug use.

Informed recommendations

The IMF formed the International Myeloma Working Group (IMWG)—an international body of more than 200 myeloma experts—precisely to address issues such as this. For the last two years, the IMWG has discussed myeloma therapies at the annual IMWG Summit Meetings in the context of access and costs. At this year’s meeting, on June 8th, attendees will meet in a special session to develop guidelines for therapy throughout the course of the disease.

The goal of this special session is to develop primary recommendations (those to be used a majority of the time) and secondary recommendations that allow for individualized choices based upon unique features of the disease, patient and/or physician preference and/or local/regional access issues.

Both efficacy and costs will be considered at this IMWG Summit session. The notion that 4 drugs can be better than 3 drugs has raised the specter of truly insurmountable costs for myeloma treatment. It is a moment to stop and consider carefully. Does an expensive new drug add value in a 3- or 4-drug combination? How does aggressive disease impact the use of 2- versus 3- or 4-drug combos? How manageable are patient costs for one regimen versus another? Many issues to consider.

In patients’ best interests

Since full outcomes data are NOT available for most new therapies or combinations, these guidelines can be based upon expert consensus opinion, including published data but supplemented by knowledge from ongoing clinical trials and clinical practice as best possible. If equivalent outcomes are predicted, the cheaper and/or more accessible option will be the primary recommendation. There are some choices for which more detailed recommendations will be required.

So, as we await the outcomes of the May 26th ICER hearing, I believe that recommendations from experts can provide a rational alternative and may be a better framework for decision making moving forward. Precise outcomes data will always lag behind the need to make recommendations. Given the current landscape, the IMWG can guide the way forward with the best interests of myeloma patients at heart. 

Image of Dr. Brian G.M. DurieDr. Brian G.M. Durie serves as Chairman of the International Myeloma Foundation and serves on its Scientific Advisory Board. Additionally, he is Chairman of the IMF's International Myeloma Working Group, a consortium of nearly 200 myeloma experts from around the world. Dr. Durie also leads the IMF’s Black Swan Research Initiative®.

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