Clinical Research and Experimental Treatment:

Who Participates, Who Pays?

A meeting in NYC, November 3, 1997

Health Care Choices, Inc., a nonprofit health care educational organization put this meeting together on November 3, 1997 in New York City.

Keynote address by Lewis Morris, Esq. Assistant Inspector General-This office regulates Medicare issues and under Medicare no experimental treatments are allowed. There would be money to cover such costs but it is being "wasted" by non-rendered services and over-testing. Morris mentioned a settlement made by testing labs, Smithkline Beecham, as one example, found guilty of billing medically unnecessary tests at $300 million. Double billing by both a teaching doc and his intern is another example. Possible total abuse of $23 billion per year. If this could be cleared up, Medicare could afford to change its coverage.

The morning panel's topic was: Should Health Insurance Cover Experimental Treatment? moderated by James R. Tallon, President of The United Hospital Fund. Cliff Hudis, MD, Assistant Attending Physician, Sloan-Kettering spoke about the nature of trials, from lab experiments to Phase I looking for maximal safe dose, must end up getting the active dose. Then Phase II which checks the activity of the dose, the safety and is done with a small amount of patients (25-40). Phase III looks at how it compares to existing protocols. Translational is when the product is used by patients and their physicians. A newer approach is clinical observation, Phase II as a confirmatory observation and Phase III still how it compares. He mentioned that these methods allow us to "inch" ahead most of the time.

Michael Stocker, MD, President of Empire Blue Cross/Blue Shield said that Empire is the largest payer of experimental treatment in New York State. The decisions are made on an individual basis due to the overall confusion in this area. Empire discovered it was better to pay for bone marrow transplant(for example) than argue the case due to the bad publicity and high legal costs. When denials have been upheld, its because there has been no "proof" for the treatment Somewhat more than half the denials were upheld by the review company.

They use an outside review process run by Medical Care Management Corporation. It is believed that physicians submit better and more detailed information when it is known that the information will go to an outside agency. There are usually less requests from MD's since they know it will be refused if experimental! Law suits in this area have greatly reduced down from 20 in 1996 to 0 in 1997. Empire really does not want to pay for treatment that is unproven or experimental.

Dr. Stocker suggested that since we have data, we establish data bases so that this information can be used to allow trials to investigate new treatments in all arms. This would allow faster patient accrual and faster results. Elliot Shaw,Director of Government Affairs, The Business Council of New York State Inc. suggested that large business in New York State get directly involved in medical issues. He named Kodak, Xerox, IBM, Bell Atlantic and stated that they have large enough medical departments to help make decisions. Larry Kramer, Co-Founder of Gay Men's Health Crisis and Act-UP, suggested that employers and drug companies should be in the audience. He personally sees no need for clinical trials since the outcome is almost always known and they do not work well. He believes most clinical trials are mediocre. Consensus committees choose only the most basic trials and peer review is a joke! When the product comes to the marketplace, it works for some and not others. He explained that companies like Merck and Johnson and Johnson worked directly with AIDS activists and others like Hoffman-LaRoche would not. He suggested that the relationship between breast cancer activists and drug companies is too remote. (I suggested to him that he pay more attention to the complementary/alternative world of treatment). He then said that no cure for anything ever came out of NIH.

He believes that the faster the drugs get into patients, the less it will cost. Drug companies should pay the most since they benefit most financially. He suggested that the audience would be fighting to defend a system that doesn't work. He further suggested a database on the internet to which patients could input information. Work has begun on this idea and he would like to see it work for other disease areas. He mentioned that he had met with Dr. Stocker and found areas of agreement.

The afternoon's panel was Clinical Research and Development of Experimental Trials moderated by Charles Aswad, MD, Executive Vice President of the Medical Society of the State of New York

Charles Vogel, MD, Florida based clinician who works with many clinical trials, discussed the success of chemotherapy by pointing to Acute Mytoblastic Leukemia. He said that 70% of young men with advanced testicular cancer are "cured" and above 60% of children with cancer are "cured". He said a large number of children had been enrolled in trials but only 3% of adults are. Why are there so few? He suggested that the trials are large and complex with a big infrastructure at the institutions in order to participate. Possibly the 5-6 pages of informed consent with a listing of each and every possibility, in lay terms, was a discouragement. He said that it was more lucrative for physicians to give standard therapies. Dr. Vogel mentioned that most of the time, costs are covered by the agency sponsoring the trial. Peter Goldschmidt, MD, DrPH, DMS, President of Medical Care Management Corporation an external review company discussed some aspects of his five year old company. They advise patients at no charge as to best treatments, and review doctors advice. They advise on issues of high technology, high risk, rare treatments, quality management and workers' compensation. There are over 500 credentialed clinical reviewers at over 100 medical centers, reviewers are matched to each case avoiding conflicts of interest with 3 members per panel. All questions relate solely to the individual patient. They check benefits and risk of treatment to patient. In reviewing over 6000 cases in the last five years for 150 clients, they find that about 40% of the patients are not eligible for the proposed protocols. Litigation has been threatened in 0.05% of the cases.

Finally, some health plans share the reviews with the patients and clinicians and new information on options are provided to the patient. Arthur Levin, VP, Technology and Clinical Practice Assessment, Prudential HealthCare stated that it is his group that decides what treatments have scientific merit and what will be covered. They are currently researching what makes women get mammograms and why. He said some companies will call on leading experts in a field for their opinions or to elicit the prevailing opinions. Prudential uses formal technology assessment.

There are certain generic problems in clinical trials: a) Failure to study important questions b) Duplication of trials c) Poor trial design-hard to know what is being looked for and when it is found. d) obsolete trials And patient-specific problems include: a) Ineligible patients b) "Off trial" mechanisms c) Inadequate informed consent

Other issues include: a)Utilization-how many people are actually involved? b) Cost/cost shifting-What does it cost? Who is paying now and who wants insurers to cover the costs? No firm answers. c) Trial eligibility-what constitutes an eligible trial? d) Future trials-universe expansion

Factors influencing costs include: 1)Intra-trial(i.e. more tests, imposed by design of trial) 2) Institutional factors-larger centers, travel and follow-up involved. 3) Global negotiated rates 4) In network equivalent care(designated places payors do business with.

There are a variety of state proposed mandates concerning off label drugs, specialized networks, new cancer therapies, basic benefit design and rider benefit design. Mary McCabe, RN Director, Office of Clinical Research Promotion, NCI discussed their sponsorship of treatment studies including: reviewing trials, approval by cancer therapy evaluation program, cooperative group studies-Phase III and Clinical-Phases I and II as well as studies under innovative grants.

There are 6600 investigators, 1500 institutions, 16,000 patients, 340 therapeutic studies in follow-up including prevention trials. Medical practice can be changed as a result of this work. She specifically mentioned several drugs such as ATA, fludarabine, zinecard, and pentostatin. She also pointed out that a multi-clinic study had detected the leukemia many breast patients were presenting with - AML (acute myoletic leukemia). This was not found in any single viewing but as an observation among the many locations.

This program is undergoing a huge reassessment on efficiency, duplicate studies, the difficulty for doctors to participate and other issues.

Paula Waterman, MS, Executive Director, Institutional Review Board, Food and Drug Administration-stated that her office does the interface to protect human rights in the clinical section of FDA. There are many written regulations in this area, and they mostly regulate where a drug is involved but will advise on other issues. In private research, which is not FDA regulated, there is no protection. There is a regulatory right to have information presented in language that can be easily understood and a patient cannot be asked to waive away rights or release anyone from responsibility. It must be stated that the process is investigative and experimental and include the following: Duration, procedures to be followed, experimental procedures, description of risks, description of benefits if any, disclosure of alternative treatments, confidentiality issues(FDA can inspect), explanation as to complications, medical coverage if needed and who to contact for answers. A statement that participation is voluntary is needed and the subject can cease participation at any time. Will the results be made available if significant new findings arise? The subject can be told how many other people are involved.

Conclusions: Insurance companies can refuse to pay for even standard therapies if they are part of a clinical trial. This is true for devices also. Just signing a consent form should not remove a patient from possible reimbursement. Right now the questions have not been resolved, individual solutions by 3rd party payors is still the current system. These are sometimes looked at on a case by case basis. Sometimes it is a marketing decision to promote the company. Sometimes there is a rider than the employee or group can purchase. Their needs to be involvement by the patient community for this to change. Dr. Vogel stated again that he believes there are advances to be made in a clinical trial setting. "We don't yet have the data everyone wants".

A most interesting and informative event.

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