How One Company Decides When to Pay for Experimental Therapies

Payment for bone marrow transplant (BMT) therapy for breast cancer from a managed care perspective will be influenced by clinical arguments put forth by research and clinical communities, outcomes measures over time, and the subscription agreement itself, C. Michael Blackwood said at a symposium sponsored by the Western Pennsylvania Hospital.

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

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

Reviewing Experimental Therapies

To standardize the review process for all experimental procedure payment requests, HealthAmerica developed a detailed experimental procedures policy, which they submitted to Pennsylvania's Department of Health. The policy defines the clinical review protocols that must be followed when making payment recommendations to the plan.

The review is performed by the company's utilization management committee, composed of voting and nonvoting administrative members and including two medical directors, senior internists, and family practitioners, pediatricians, ob/gyns, and cardiologists. A medical oncologist will soon be added because of the number of oncology cases being reviewed. Membership is rotated every year, and Mr. Blackwood's role as a nonvoting member is to ensure compliance with the review protocol.

To aid the review process, the committee gathers information from several sources: extensive literature searches of multiple databases; ECRI, a private Philadelphia company that researches various medical investigative protocols and approaches; a group of HMOs who routinely collect, update, and share information; and specialists who are frequently requested to provide recommendations based on the diagnosis and clinical status of individual patients.

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

Committee decisions can be reviewed internally upon request, and the patient or family has the right to appeal as high as the Pennsylvania Department 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 for BMT for breast cancer and denied two. "So you might ask---where's the problems in making a decision? Well, if you're sitting on this committee, here are some of them," he said.

Members are required--and have an obligation to the patient--to determine the safety and efficacy of every recommendation for every patient. They must stay abreast of clinical indications, which are continually changing; define intraoperative and postprocedural risks 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 conventional therapies and moving directly to newer therapies, like BMT. "So do we make recommendations with arguably insufficient literature in support of a given approach for a given outcome, which may require large expenditures and perhaps put the patient at risk?"

He also noted that there seems to be a treatment migration from stage IV toward stage III disease. "We must determine whether that is appropriate. Obviously, we need to track outcomes over time 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, because of its clinical impact on the patient and financial impact on everyone involved, must be philosophically and morally sound. "We struggle to do the right thing," he said, "but because predicting outcomes is difficult and because we often receive conflicting recommendations from many well-respected clinicians and researchers, it's often not clear from our chair what the right thing is."

The increasing numbers of BMTs being performed and approved every day, 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 remains the same--we must be convinced the procedure is both safe and effective within a reasonable degree of predictability for each individual or we won't make the recommendation. We feel a great responsibility not to put our patients at unnecessary risk, regardless of pressure to do so,and we will stand fast on that.