How One Company Decides When to Pay for Experimental Therapies

Payment for bone marrow transplant (BMT) therapy for breast cancerfrom a managed care perspective will be influenced by clinicalarguments put forth by research and clinical communities, outcomesmeasures over time, and the subscription agreement itself, C.Michael Blackwood said at a symposium sponsored by the WesternPennsylvania Hospital.

As complete freedom of choice in health care is giving way tosome mechanisms of control, managed care is taking on a new role,he said. Now, health-care providers must make recommendationsto the insurance carrier, who applies internal clinical reviewprocesses before making payment recommendations to the health-careplan. "The dicey area, of course, is dealing with experimentaltherapies," said Mr. Blackwood, president and CEO of HealthAmericain Pittsburgh.

"The subscription agreement is a legal contract between theemployer, the employee, and the plan, and is very important forthe patient and us. Interpreting it is often a real challengebecause there are always shades of gray, no matter how hard youtry to make it straightforward. But that is the driving document,"Mr. Blackwood said.

Reviewing Experimental Therapies

To standardize the review process for all experimental procedurepayment requests, HealthAmerica developed a detailed experimentalprocedures policy, which they submitted to Pennsylvania's Departmentof Health. The policy defines the clinical review protocols thatmust be followed when making payment recommendations to the plan.

The review is performed by the company's utilization managementcommittee, composed of voting and nonvoting administrative membersand including two medical directors, senior internists, and familypractitioners, pediatricians, ob/gyns, and cardiologists. A medicaloncologist will soon be added because of the number of oncologycases being reviewed. Membership is rotated every year, and Mr.Blackwood's role as a nonvoting member is to ensure compliancewith the review protocol.

To aid the review process, the committee gathers information fromseveral sources: extensive literature searches of multiple databases;ECRI, a private Philadelphia company that researches various medicalinvestigative protocols and approaches; a group of HMOs who routinelycollect, update, and share information; and specialists who arefrequently requested to provide recommendations based on the diagnosisand clinical status of individual patients.

"We search for the most reliable information and best clinicalminds to give recommendations to that committee," he said.The group meets once every 2 or 3 weeks and often reviews fouror five transplant recommendations on a given day. "Thisplaces incredible pressure on the group for an accurate and timelyreview on behalf of patients and providers."

Committee decisions can be reviewed internally upon request, andthe patient or family has the right to appeal as high as the PennsylvaniaDepartment of Health. "There's a written protocol for that,and we follow it religiously," Mr. Blackwood said.

Difficult Decisions

In the last 2 years, HealthAmerica received nine referrals forBMT for breast cancer and denied two. "So you might ask---where'sthe problems in making a decision? Well, if you're sitting onthis committee, here are some of them," he said.

Members are required--and have an obligation to the patient--todetermine the safety and efficacy of every recommendation forevery patient. They must stay abreast of clinical indications,which are continually changing; define intraoperative and postproceduralrisks of the procedure itself, particularly for young patients;and evaluate the prognosis for each of the many treatment alternatives.

Mr. Blackwood perceives a trend toward bypassing conventionaltherapies and moving directly to newer therapies, like BMT. "Sodo we make recommendations with arguably insufficient literaturein support of a given approach for a given outcome, which mayrequire large expenditures and perhaps put the patient at risk?"

He also noted that there seems to be a treatment migration fromstage IV toward stage III disease. "We must determine whetherthat is appropriate. Obviously, we need to track outcomes overtime to see retrospectively if the treatment seems to work,"he said.

Struggle to 'Do the Right Thing'

He stressed that each decision made by the committee, becauseof its clinical impact on the patient and financial impact oneveryone involved, must be philosophically and morally sound."We struggle to do the right thing," he said, "butbecause predicting outcomes is difficult and because we oftenreceive conflicting recommendations from many well-respected cliniciansand researchers, it's often not clear from our chair what theright thing is."

The increasing numbers of BMTs being performed and approved everyday, coupled with increasing pressure from outside influences,will result in a dramatic increase in this mode of treatment."For us, however," he said, "the standard remainsthe same--we must be convinced the procedure is both safe andeffective within a reasonable degree of predictability for eachindividual or we won't make the recommendation. We feel a greatresponsibility not to put our patients at unnecessary risk, regardlessof pressure to do so,and we will stand fast on that.