By ELAINE SCHATTNER, MD

Well, it’s time to resume our dis­cussion of Bending the Cost Curve in Cancer Care.

We’ve reached the end of the list, on ideas to reduce oncology costs put forth by Drs. Smith and Hillner in the May 25 issue of the NEJM. Really this 10^th and final point intended for oncol­o­gists is two-​​in-​​one: “The need for cost-​​effectiveness analysis and for some limits of care must be accepted,” they chart. So doctors should embrace studies of com­par­ative effec­tiveness and cost effectiveness.

Hard to argue with reason — they’re correct, of course. They write:

… The national imper­ative is to empower a trans­parent, acceptable, equi­table, polit­i­cally inde­pendent agency for guidance in making tough choices in the public interest so that doctors do not have to make them at the bedside.⁶⁰ Ulti­mately, we will have to make deci­sions based on some cri­teria, and comparative-​​effectiveness⁶¹ and cost-​​effectiveness⁶² analyses are good ways to align resource use with the greatest health benefit.

This sounds great, and is probably right, but I don’t think it’s realistic.

A more detailed con­sid­er­ation on the issue of cost-​​effectiveness, IMO, came out a few weeks later, also in NEJM: Com­par­ative Effec­tiveness Research and Patients with Mul­tiple Chronic Con­di­tions. This piece, by Drs. Mary Tinetti and Stephanie Stu­denski, con­siders the prob­lematic appli­cation of CER in the real world.

The problem with CER, these authors emphasize, is that most medical patients have more than one con­dition and many are elderly; clinical trials tend to include, exclu­sively, patients who don’t have more than one major illness are rel­a­tively young. This limits the physi­cians’ abil­ities to apply data to their patients.

What’s more, reported results tend to focus on central results, but most patients fall else­where on mea­sured curves:

The het­ero­geneity of treatment effects will further com­plicate CER. Although studies typ­i­cally report average effects, most par­tic­i­pants expe­rience more or less benefit and harm than average. Such het­ero­geneity results from vari­ability in patients’ initial level of risk for a given outcome, in their respon­siveness to treatment, and in their vul­ner­a­bility to adverse effects — issues with par­ticular rel­e­vance to patients receiving treatment for mul­tiple coex­isting conditions.

The authors, who rec­ognize the need for better research to support treatment deci­sions, write that “CER will probably accel­erate the movement toward outcome-​​driven decision making, reim­bursement, and quality assessment. As this shift occurs, we must move toward a focus on cross-​​disease, “uni­versal” out­comes in research and clinical care.” Their thesis gets more abstract (which I admire), but meets a wall or two: the lack of con­sensus on a set of uni­versal health out­comes, dif­ferent para­meters mea­sured by the likes of the VA admin­is­tration, CMS, the FDA, NIH and other huge agencies.

They make a prac­tical sug­gestion, about the need for head-​​to-​​head com­par­isons in CER:

… inter­ven­tions such as exercise that affect mul­tiple con­di­tions simul­ta­ne­ously should be a high priority…Studies should include assessment of the burden of treat­ments for patients and fam­ilies. Another CER pri­ority should be the exam­i­nation of treat­ments for common pairs of dis­eases in which treatment of one may exac­erbate the other. For example, when hyper­tension and osteo­porosis coexist, what treatment best min­i­mizes the risk of adverse car­dio­vas­cular out­comes without increasing the risk of falls and fractures?

All of this sounds rea­sonable to this patient-​​doctor, but it’ll take a lot of time and money to accom­plish effective CER that encom­passes the needs and con­di­tions of suf­fi­cient numbers of patients in disease and age com­bi­na­tions to power any mean­ingful studies. You have to wonder at some point, as I have been lately, is all this clinical research worth the effort?

That said, I respect this paper’s con­clu­sions on CER:

Researchers have largely shied away from the com­plexity of mul­tiple chronic con­di­tions — avoidance that results in expensive, poten­tially harmful care of unclear benefit. We cannot improve health care’s quality, effec­tiveness, and effi­ciency without addressing its greatest con­sumers. Devel­opment and testing of inno­v­ative approaches to care for patients with mul­tiple chronic con­di­tions could prove the most lasting legacy…

My bottom line: CER, and con­sid­er­ation of treatment costs, should underlie reduction of cancer care costs in the near and long-​​term future. As to how we accom­plish suf­fi­ciently careful research, and avoid inap­pro­priate cutting of helpful treat­ments — espe­cially those that prove ben­e­ficial for some younger and oth­erwise healthy cancer patients — is one of the two main chal­lenges ahead.

(The other big chal­lenge, mainly a moral one, is the subject of rationing, to which Smith and Hillner allude but don’t detail, and which subject I won’t address in this post.)

Mean­while: thorough, apo­litical, nuanced and trans­parent reporting of trial results would help doctors, patients and the general public under­stand what infor­mation is available.

Finally, in the next month or so I will look back over the full, provocative and gen­erallyexcellent list by Drs. Smith and Hillner, and see what holds hope for the future of cancer med­icine. What’s clear is that the path ahead man­dates clear thinking through some very tough clinical decisions.

Elaine Schattner MD is an  trained oncol­ogist, edu­cator and jour­nalist who writes about med­icine. She teaches at Weill Cornell Medical College.  You’ll find her blog, where this post first appeared, at Medical Lessons.