Clinical Trials Access and Reimbursement William B. Farrar, MD Clinical trials continue to play an extremely important role in clinical oncology, but of the nearly 1 million cancer patients diagnosed in the United States each year, only 2.5% (25 ,000) participate in such trials. Access to clinical trials has been enhanced by the adoption by the NCT of a promotional campaign that involves seminars, dissemination of information to national and local news media, and assisting in making information about clinical trials available to patients and physicians. Factors that hinder accrual of patients to clinical trials include: (1) physicians' concern about losing contact with patients, (2) physicians who feel that "experimental therapy" is not as good as "s tandard therapy," and (3) the time it takes to discuss and implement clinical trials. Reimbursement for clinical trials has also become a major concern. Who should pay for clinical research? Many insurance companies have been reluctant to pay for "experimental therapy" but will pay for "standard therapy." If this trend continues, the whole concept and organization of clinical trials , i.e. , Phase I, II, and III trials, will be greatly hindered. Solutions must be found, and third party payers need to realize that an investment in clinical trials today will decrease the long term costs of state-of-theart care. Cancer 67 :1779-1782, 1991.
T
I-I E D SIGN ADO DU T of clinical trial s continue
to play an extremely important role in clinical oncology. Over the past 5 years considerable progress has been made via the clinical trial process, i.e., conservative treatment of breast cancer (NSABP), interfero n for hairy cell leukemia, MV A (methotrexate, vinblastine, doxorubicin , cisplatin) for bladder ca ncer, and preoperative chemotherapy and limb salvage in osteosarcoma. Only by participation in well-designed prospective clinical trials could these advances in treatment have been accomplished . Anecdotal data and nonexperimental surveys conducted through hospital and regional tumor registries have no role in current clinical oncology. Many conclusions drawn from nonexperimental data have led to year of unnecessary costl y treatm ent and immeasurable suffering. We as oncologists and other related profes ionals need to continue to stri ve to take part in well-d signed
Presented at the American Cancer Society ational Conference on ancer and the hanging Healthcare ystem. an Franci co, alifornia, . May 3-5. 1990. From the Division of urgi al Oncology, DepaI1ment of Surgery, OhIO State University ollege of Medicine, olumbus, Ohio. Address for reprint : William B. Farrar. MD, 907 Doan Hall . 410 W. 10th Avenue, olumbus.01-l43210. Accepted for publication eptember 14, 1990.
clinical trials to answer specific scientific que lions. This conclusion beco mes even more important as reimbursement for clinical trials becomes increasingly difficult to obtain. Our objective is to explore access and reimbursement issues confronting both physicians and patients who are involved in clinical trials now and in the coming year. A full appreciation of the problems of access and reimbursement for patient care in clinical research requires an understanding of the process whereby promising new drugs and treatm ent approaches are e aluated. Potentially effective drugs and treatments are first evaluated in Pha e I studies, which are designed to determine the relationship between toxici ty and dose- chedule of treatment. The e studies usually involve t 5 to 25 patients and are conducted almost excl usively in specialized research centers, where coordinated clinical , pharmocologic, immunologic, and other studies are performed. The information obtained from Phase I studies is then used to evaluate re ponse in specific tumor types in Phase II trials. The e trials typically require 20 patients with a specific disease. Phase III trials are randomized clinical trials that compare a standard therapy with a new treatment. The e trial require large numbers of patients and are be t carried out in national cooperative groups. Both Phase II and Pba e III trials can
1779
1780
CANCER
March 15 Supplement 1991
be carried out by physicians in private practice and community clinics if access to the clinical trials is made possible. Of the nearly 1 milJion cancer patients diagnosed in the United States each year, only about 2.5% (25,000) participate in NCI-sponsored clinical trials. This low percentage of enrollment impedes completion of many welldesigned trials that ask relevant scientific questions. DeVita reported at a recent ASCO meeting that if 10% of patients with common tumors participated in clinical trials, most trials could be completed within I year instead of the current 3 to 5 years required for adequate accrual. Many more trials could be completed in a more timely fashion . Conclusions drawn from 5-year follow-up would consequently become evident that much sooner, thereby leading to better therapy.' What has been done by the NCl and the national cooperative groups to increase access to these clinical trials? What can we do as physicians and related professionals to increase access to these trials? Let us look at these questions in detail and see if there are any solutions. In 1988 the NCI called for patient accrual to be of the highest priority. According to Wittes, "The field of cancer research has never been so rich in opportunity to develop new cancer treatments as it is now, not only in the rare tumors but also in common epithelial tumors of adults. We are excited about the opportunity to make a real difference in outcome for cancers that affect large numbers of Americans.'" To assist in this effort, the NCI adopted a promotional campaign that involved conducting seminars, placing feature stories in national and local news media, and assisting in making information on clinical trials available to patients and physicians. Physician Data Query (PDQ) was also set up to assist physicians to learn what clinical studies were available for all different types of tumors. Considerable information has been made available for patients, such as a 23-page NCr booklet, "What Are Clinical Trials All About?" The goal of this effort was to double the number of patients entered in clinical trials by 1992 (25,000 to 50,000). The result of this effort has become evident in the increased accrual that has occurred in most of the cooperative groups. Progress toward this goal has been substantial, but considerably more work is needed. One can easily see that the NCI effort has been successful, but continued improvement is necessary. The backbone of access to clinical trials is twofold and includes both comprehensive cancer centers and national cooperative groups. The Phase I and many Phase II studies are completed at institutions that have comprehensive cancer centers. Recent restructuring of comprehensive cancer centers has placed new emphasis on outreach programs to improve accrual in clinical trials. National cooperative groups funded by the NCT continue to be the
Vol. 67
mainstay of Phase III and many Phase II trials. Access to clinical trials has been aided greatly by community clinical oncology programs (CCOP) and cooperative group outreach programs (CGOP). CCOP are undertaken in hospitals affiliated with a national cooperative group that has the ability to place patients into protocols. Each has its own principal investigator and also is responsible for a number of cancer control studies. The CGOP are implemented in community hospitals affiliated with member institutions for the purpose of conducting clinical cancer research. These programs have also aided in patient accrual. Many programs have been instituted to increase patient access to clinical trials. However, the most important factors to consider with regard to access are physician and patient education. All of these previously described programs make contributions; however, if the physician never discusses the trials with patients, accrual will not improve. Let us first address the question, why are physicians reluctant to place patients into clinical trials? The most important factors are (1) fear of losing contact with the patient, (2) physician's belief that answers to a specific trial are already known, and (3) the time required to discuss and implement a clinical trial. Why do physicians fear that they may lose control and follow-up of their patients? Unfortunately, that fear is often founded on past experience. We as leaders in oncology need to do everything possible to alleviate that fear. We need to communicate frequently with the referring physicians both by phone and letter. We need to let the referring physician know what side effects to expect and what to do when the patient returns home and develops fever and vomiting in the middle of the night. Communication is the name of the game-patient accrual will improve only as communication improves. Reluctance by many physicians, especially surgeons, to explain the options of therapy to their patients continues to be a problem. It is much easier to tell a patient that one option is the treatment of choice rather than to explain that the best treatment option is unknown and that participation in a clinical trial is as good an option for therapy as is currently available. A good example of this is the national melanoma trial, which is a randomized trial evaluating 2 versus 4 cm margins. A second randomization is an immediate or delayed lymph node dissection. Many surgeons believe they already know how to treat melanoma, although no randomized prospective data will support their view. If all surgeons had supported this ongoing study, it would have been successfu lly completed years ago. The only solution to this difficult problem is continuing physician education. Reluctance of physicians to enter patients into trials was further substantiated by the report of Hunter e/ a/. 2 This study looked at all patients that were entered in the
NO.6
CLIN ICAL TRIALS
CCOP physician patient log between August 1984 and November 1985. There were 44, 156 newly diagnosed cancer patients with 40% (17,773) avai lable for an NCIapproved protocol ; 56% of patients wit h a protocol avai lable were clinically eligible and on ly 19% were entered in the protocol. In the 37% who were eligible for a protocol but did not participate, the physician preference for a specific or alternati ve treatment was the predominant reason for not participating. One-third of the eligible patients refused to participate for a number of reasons. Experimentation, toxicity, and costs accounted for more than 50% of the specific reasons for patient refusal. Only through better physician education can we hope to improve these n umbers. The third factor is time. Without question, discussing and implementing a clinical trial is time-consuming. Even with nurse and data manager assistance, an average of 6 hours of the physician's time is required . A good exa mple is the ongoing trials for node-negative breast ca ncer. Discussi ng all of the options included in the current tri als requires an average of 4 to 6 hours. Many physicians would prefer to take the easier "standard therapy" route. Access to clinical trials is available in every section of the country because the national cooperative group, CCOP, and CGOP are so widespread. Success can be seen by the continuing increasing accrual of patients by many cooperative groups. The major problem con tinues to be physician and patient educatio n. We need to continue to support physicians through the cooperati ve groups and continue to educate physicians that protocol therapy in most instances is better than "standard therapy". As Frei, stated, "Clinical trials involve the very best treatment given by experts ver /IS the very best treatment with someth ing added to it which will not make it worse and might make it better. I do not know of any instance where the new therapy was worse than the standard therapy.") The oth er major topic to be considered is extremely timely and important: reimbursement for clinical trials. As health care cost continue to make headlines, funding of clinica l trials has become an increasing problem. Many insurance companies will not reimburse experimental trials. Until the la t few years, an informal but effective coalition to lower the costs of health care for clinical trials has existed. This coalition consisted of the federal govern ment (N T) to fund worthy groups, universities to fund research space and personnel, pharmaceutical industries to provide funds for testing new drugs, and third party payers to cover patient care co t . Thi coalition served as an excellent means to provide tate-of-the-art care through well-designed clinical trial .4 ach of the components of this coalition has been affected in some way by the increasing demand of the cost
Farrar
1781
of medical care, posing a difficult question. Who should fund clinical research as we enter thc I990s? The success of the design and conduct of clinical trials over the past 20 years is without question , but if experimental therapy (Phase I and II trials) are not supported, clinical research progress will grind to a halt. We have already addressed the difficulties encountered in placing patients on clinical trials by answering questions such as how this therapy will benefit the patient. What are the risk ? What are other options? Is this therapy as good as standard therapy? Now we also need to address the question of who is going to pay for this therapy, including cost of drugs, physician visits, laboratory procedures, radiographs, hospitalization, and complications from therapy. The patient and the physician need to know the answers to these questions before insti tuting the study. To address some of these questions, let us first look at the weaknesses in the current system and then see if solutions can be identified. One of our greatest problems is inconsistency in coverage among insurance companies. One company may approve a patient for a clinical trial and another carrier may not. Some pay for laboratory studies but not hospitalization. Some pay for outpatient chemotherapy but will not pay for hospitalization for complications from investigational therapy. As a result, some patients are afraid to accept experimental therapy. They cannot afford it if insurance refuses to pay. Do we have a system in which only the wealthy will benefit from new and innovative therapy? The current system also tends to hinder clinical research progress. Insurance companies will pay for standard therapy and labeled drugs; however, they will not pay for investigational therapy or unlabeled drug. If these policies continue, our entire system of clinical trial design will be jeopardized. In looking at the u e of drugs for unlabeled indicators, Antman el a/. 4 pointed out that both the FDA and the Medicare laims Manual clearly state that insurance carriers are to be re pon ible for reimbursement for appropriate unlabeled uses. This expectation is not borne out in actual practice. Upon review of a survey of 165 private practice oncologists with 3500 medical records, Antman el a/. 4 found that an average of46% of the chemotherapeutic agents were off-label. Off-label use is common; it is medically necessary, and it i supported by both FDA and HCFA policy. When third party carriers refuse to reimburse unlabeled use, they are exercising poor judgment. Their refusal will not lower medical costs but will actually raise long-term costS.4 Another concern with the current system is the apparent shift of decision-making power from the physician and patient to third party payers. Do the phy ician and the patient decide or is the treatment actually being dictated by the third party payers? If a phy ician and a patient both agree that the best treatment i an "unlabeled" drug
1782
CANCER March J5 Supplement 199 I
but the insurance company will not pay for it, the only option for treatment may be an "old" standard therapy, which has proved not to be as effective as the "unlabeled" drug. Who is actually making treatment decisions? The final weakness in the area of reimbursement is probably of greatest concern. That is the inability to carry out the conduct and design of our current clinical trial system. Phase [ and some Phase II studies are not reimbursed by third party payers. Even some Phase m trials are currently denied jf the carrier is aware that the treatment is part of a clinical trial. Tfthis process is not halted, we will have no way to formulate and conduct Phase TTl trials because no Phase [ or Phase II trials will be completed. We will be unable to test new technology to determine whether it is cost-effective. We will be unable to know if a cheaper new drug is as good as the more expensive "standard" drug. We may be able to cut costs for the present; however, progress will stop and treatment will be more costly in the future. If progress is to continue in our clinical trial process, solutions to all of these problems must be found. How should the costs of clinical research be borne? There is no easy answer to this question. The NCI and NIH must continue to renew efforts to support clinical trials. Cancer continues to be a leading cause of death in the United States and continued research is essential. Recently the entire comprehensive cancer center concept ·has undergone change. The number of CCC has decreased. What effect is this decreasing number going to have on Phase I and II trials? Where will those trials be done? As stated, the NO has outlined a program to increase the accrual of patients into clinical trials. Much success has been achieved; however, if more money is not funneled into
Vol. 67
the system to fund clinical trials, the process will be seriously jeopardized. Obviously, insurance companies and patients themselves will have to bear part of the burden. We need to make certain that we do not evolve into a system in which only the wealthy can afford " up-to-date" therapy or insurance companies dictate the type of therapy a patient can receive. Either of these options would signal disaster. What should be our immediate goals with regard to access and reimbursement for clinical trials? With regard to access, we have been successful in providing information and protocols to small communities. We need to strive to make it easier for the physician by finding ways to fund secretarial and nursing support. We also need to continue to strive to educate the physicians about the need for clinical trials. There is no easy solution to reimbursement issues. We must pool all resources, such as federal government, private industry, third party payers, universities, large community hospitals, and the patients themselves to come to a compromise that will ensure the survival of clinical trials research. The crisis is here, action is underway, and we as oncology specialists must assist in finding the answers. REFERENCES I. News. J NaIl Cancer Insl 1988; 71 :619-620. 2. Hunter el at. Selection factors in clinical trials: Results from the Community Clinical Oncology Program Physician's Log. Cancer Trea/ Rep 1987; 71 :559-565. 3. Merz B. NCI seeks to boost study participation: Medical NelliS and PerspeclivesJAMA 1988; 260:1337. 4. Antman KH, Aledort LM. Yarbro J el at. Cost-effectiveness and reimbursement in patient care. Semin !lema/ol 1989; (Suppl 3) 26:3245.
T
I-I E D SIGN ADO DU T of clinical trial s continue
to play an extremely important role in clinical oncology. Over the past 5 years considerable progress has been made via the clinical trial process, i.e., conservative treatment of breast cancer (NSABP), interfero n for hairy cell leukemia, MV A (methotrexate, vinblastine, doxorubicin , cisplatin) for bladder ca ncer, and preoperative chemotherapy and limb salvage in osteosarcoma. Only by participation in well-designed prospective clinical trials could these advances in treatment have been accomplished . Anecdotal data and nonexperimental surveys conducted through hospital and regional tumor registries have no role in current clinical oncology. Many conclusions drawn from nonexperimental data have led to year of unnecessary costl y treatm ent and immeasurable suffering. We as oncologists and other related profes ionals need to continue to stri ve to take part in well-d signed
Presented at the American Cancer Society ational Conference on ancer and the hanging Healthcare ystem. an Franci co, alifornia, . May 3-5. 1990. From the Division of urgi al Oncology, DepaI1ment of Surgery, OhIO State University ollege of Medicine, olumbus, Ohio. Address for reprint : William B. Farrar. MD, 907 Doan Hall . 410 W. 10th Avenue, olumbus.01-l43210. Accepted for publication eptember 14, 1990.
clinical trials to answer specific scientific que lions. This conclusion beco mes even more important as reimbursement for clinical trials becomes increasingly difficult to obtain. Our objective is to explore access and reimbursement issues confronting both physicians and patients who are involved in clinical trials now and in the coming year. A full appreciation of the problems of access and reimbursement for patient care in clinical research requires an understanding of the process whereby promising new drugs and treatm ent approaches are e aluated. Potentially effective drugs and treatments are first evaluated in Pha e I studies, which are designed to determine the relationship between toxici ty and dose- chedule of treatment. The e studies usually involve t 5 to 25 patients and are conducted almost excl usively in specialized research centers, where coordinated clinical , pharmocologic, immunologic, and other studies are performed. The information obtained from Phase I studies is then used to evaluate re ponse in specific tumor types in Phase II trials. The e trials typically require 20 patients with a specific disease. Phase III trials are randomized clinical trials that compare a standard therapy with a new treatment. The e trial require large numbers of patients and are be t carried out in national cooperative groups. Both Phase II and Pba e III trials can
1779
1780
CANCER
March 15 Supplement 1991
be carried out by physicians in private practice and community clinics if access to the clinical trials is made possible. Of the nearly 1 milJion cancer patients diagnosed in the United States each year, only about 2.5% (25,000) participate in NCI-sponsored clinical trials. This low percentage of enrollment impedes completion of many welldesigned trials that ask relevant scientific questions. DeVita reported at a recent ASCO meeting that if 10% of patients with common tumors participated in clinical trials, most trials could be completed within I year instead of the current 3 to 5 years required for adequate accrual. Many more trials could be completed in a more timely fashion . Conclusions drawn from 5-year follow-up would consequently become evident that much sooner, thereby leading to better therapy.' What has been done by the NCl and the national cooperative groups to increase access to these clinical trials? What can we do as physicians and related professionals to increase access to these trials? Let us look at these questions in detail and see if there are any solutions. In 1988 the NCI called for patient accrual to be of the highest priority. According to Wittes, "The field of cancer research has never been so rich in opportunity to develop new cancer treatments as it is now, not only in the rare tumors but also in common epithelial tumors of adults. We are excited about the opportunity to make a real difference in outcome for cancers that affect large numbers of Americans.'" To assist in this effort, the NCI adopted a promotional campaign that involved conducting seminars, placing feature stories in national and local news media, and assisting in making information on clinical trials available to patients and physicians. Physician Data Query (PDQ) was also set up to assist physicians to learn what clinical studies were available for all different types of tumors. Considerable information has been made available for patients, such as a 23-page NCr booklet, "What Are Clinical Trials All About?" The goal of this effort was to double the number of patients entered in clinical trials by 1992 (25,000 to 50,000). The result of this effort has become evident in the increased accrual that has occurred in most of the cooperative groups. Progress toward this goal has been substantial, but considerably more work is needed. One can easily see that the NCI effort has been successful, but continued improvement is necessary. The backbone of access to clinical trials is twofold and includes both comprehensive cancer centers and national cooperative groups. The Phase I and many Phase II studies are completed at institutions that have comprehensive cancer centers. Recent restructuring of comprehensive cancer centers has placed new emphasis on outreach programs to improve accrual in clinical trials. National cooperative groups funded by the NCT continue to be the
Vol. 67
mainstay of Phase III and many Phase II trials. Access to clinical trials has been aided greatly by community clinical oncology programs (CCOP) and cooperative group outreach programs (CGOP). CCOP are undertaken in hospitals affiliated with a national cooperative group that has the ability to place patients into protocols. Each has its own principal investigator and also is responsible for a number of cancer control studies. The CGOP are implemented in community hospitals affiliated with member institutions for the purpose of conducting clinical cancer research. These programs have also aided in patient accrual. Many programs have been instituted to increase patient access to clinical trials. However, the most important factors to consider with regard to access are physician and patient education. All of these previously described programs make contributions; however, if the physician never discusses the trials with patients, accrual will not improve. Let us first address the question, why are physicians reluctant to place patients into clinical trials? The most important factors are (1) fear of losing contact with the patient, (2) physician's belief that answers to a specific trial are already known, and (3) the time required to discuss and implement a clinical trial. Why do physicians fear that they may lose control and follow-up of their patients? Unfortunately, that fear is often founded on past experience. We as leaders in oncology need to do everything possible to alleviate that fear. We need to communicate frequently with the referring physicians both by phone and letter. We need to let the referring physician know what side effects to expect and what to do when the patient returns home and develops fever and vomiting in the middle of the night. Communication is the name of the game-patient accrual will improve only as communication improves. Reluctance by many physicians, especially surgeons, to explain the options of therapy to their patients continues to be a problem. It is much easier to tell a patient that one option is the treatment of choice rather than to explain that the best treatment option is unknown and that participation in a clinical trial is as good an option for therapy as is currently available. A good example of this is the national melanoma trial, which is a randomized trial evaluating 2 versus 4 cm margins. A second randomization is an immediate or delayed lymph node dissection. Many surgeons believe they already know how to treat melanoma, although no randomized prospective data will support their view. If all surgeons had supported this ongoing study, it would have been successfu lly completed years ago. The only solution to this difficult problem is continuing physician education. Reluctance of physicians to enter patients into trials was further substantiated by the report of Hunter e/ a/. 2 This study looked at all patients that were entered in the
NO.6
CLIN ICAL TRIALS
CCOP physician patient log between August 1984 and November 1985. There were 44, 156 newly diagnosed cancer patients with 40% (17,773) avai lable for an NCIapproved protocol ; 56% of patients wit h a protocol avai lable were clinically eligible and on ly 19% were entered in the protocol. In the 37% who were eligible for a protocol but did not participate, the physician preference for a specific or alternati ve treatment was the predominant reason for not participating. One-third of the eligible patients refused to participate for a number of reasons. Experimentation, toxicity, and costs accounted for more than 50% of the specific reasons for patient refusal. Only through better physician education can we hope to improve these n umbers. The third factor is time. Without question, discussing and implementing a clinical trial is time-consuming. Even with nurse and data manager assistance, an average of 6 hours of the physician's time is required . A good exa mple is the ongoing trials for node-negative breast ca ncer. Discussi ng all of the options included in the current tri als requires an average of 4 to 6 hours. Many physicians would prefer to take the easier "standard therapy" route. Access to clinical trials is available in every section of the country because the national cooperative group, CCOP, and CGOP are so widespread. Success can be seen by the continuing increasing accrual of patients by many cooperative groups. The major problem con tinues to be physician and patient educatio n. We need to continue to support physicians through the cooperati ve groups and continue to educate physicians that protocol therapy in most instances is better than "standard therapy". As Frei, stated, "Clinical trials involve the very best treatment given by experts ver /IS the very best treatment with someth ing added to it which will not make it worse and might make it better. I do not know of any instance where the new therapy was worse than the standard therapy.") The oth er major topic to be considered is extremely timely and important: reimbursement for clinical trials. As health care cost continue to make headlines, funding of clinica l trials has become an increasing problem. Many insurance companies will not reimburse experimental trials. Until the la t few years, an informal but effective coalition to lower the costs of health care for clinical trials has existed. This coalition consisted of the federal govern ment (N T) to fund worthy groups, universities to fund research space and personnel, pharmaceutical industries to provide funds for testing new drugs, and third party payers to cover patient care co t . Thi coalition served as an excellent means to provide tate-of-the-art care through well-designed clinical trial .4 ach of the components of this coalition has been affected in some way by the increasing demand of the cost
Farrar
1781
of medical care, posing a difficult question. Who should fund clinical research as we enter thc I990s? The success of the design and conduct of clinical trials over the past 20 years is without question , but if experimental therapy (Phase I and II trials) are not supported, clinical research progress will grind to a halt. We have already addressed the difficulties encountered in placing patients on clinical trials by answering questions such as how this therapy will benefit the patient. What are the risk ? What are other options? Is this therapy as good as standard therapy? Now we also need to address the question of who is going to pay for this therapy, including cost of drugs, physician visits, laboratory procedures, radiographs, hospitalization, and complications from therapy. The patient and the physician need to know the answers to these questions before insti tuting the study. To address some of these questions, let us first look at the weaknesses in the current system and then see if solutions can be identified. One of our greatest problems is inconsistency in coverage among insurance companies. One company may approve a patient for a clinical trial and another carrier may not. Some pay for laboratory studies but not hospitalization. Some pay for outpatient chemotherapy but will not pay for hospitalization for complications from investigational therapy. As a result, some patients are afraid to accept experimental therapy. They cannot afford it if insurance refuses to pay. Do we have a system in which only the wealthy will benefit from new and innovative therapy? The current system also tends to hinder clinical research progress. Insurance companies will pay for standard therapy and labeled drugs; however, they will not pay for investigational therapy or unlabeled drug. If these policies continue, our entire system of clinical trial design will be jeopardized. In looking at the u e of drugs for unlabeled indicators, Antman el a/. 4 pointed out that both the FDA and the Medicare laims Manual clearly state that insurance carriers are to be re pon ible for reimbursement for appropriate unlabeled uses. This expectation is not borne out in actual practice. Upon review of a survey of 165 private practice oncologists with 3500 medical records, Antman el a/. 4 found that an average of46% of the chemotherapeutic agents were off-label. Off-label use is common; it is medically necessary, and it i supported by both FDA and HCFA policy. When third party carriers refuse to reimburse unlabeled use, they are exercising poor judgment. Their refusal will not lower medical costs but will actually raise long-term costS.4 Another concern with the current system is the apparent shift of decision-making power from the physician and patient to third party payers. Do the phy ician and the patient decide or is the treatment actually being dictated by the third party payers? If a phy ician and a patient both agree that the best treatment i an "unlabeled" drug
1782
CANCER March J5 Supplement 199 I
but the insurance company will not pay for it, the only option for treatment may be an "old" standard therapy, which has proved not to be as effective as the "unlabeled" drug. Who is actually making treatment decisions? The final weakness in the area of reimbursement is probably of greatest concern. That is the inability to carry out the conduct and design of our current clinical trial system. Phase [ and some Phase II studies are not reimbursed by third party payers. Even some Phase m trials are currently denied jf the carrier is aware that the treatment is part of a clinical trial. Tfthis process is not halted, we will have no way to formulate and conduct Phase TTl trials because no Phase [ or Phase II trials will be completed. We will be unable to test new technology to determine whether it is cost-effective. We will be unable to know if a cheaper new drug is as good as the more expensive "standard" drug. We may be able to cut costs for the present; however, progress will stop and treatment will be more costly in the future. If progress is to continue in our clinical trial process, solutions to all of these problems must be found. How should the costs of clinical research be borne? There is no easy answer to this question. The NCI and NIH must continue to renew efforts to support clinical trials. Cancer continues to be a leading cause of death in the United States and continued research is essential. Recently the entire comprehensive cancer center concept ·has undergone change. The number of CCC has decreased. What effect is this decreasing number going to have on Phase I and II trials? Where will those trials be done? As stated, the NO has outlined a program to increase the accrual of patients into clinical trials. Much success has been achieved; however, if more money is not funneled into
Vol. 67
the system to fund clinical trials, the process will be seriously jeopardized. Obviously, insurance companies and patients themselves will have to bear part of the burden. We need to make certain that we do not evolve into a system in which only the wealthy can afford " up-to-date" therapy or insurance companies dictate the type of therapy a patient can receive. Either of these options would signal disaster. What should be our immediate goals with regard to access and reimbursement for clinical trials? With regard to access, we have been successful in providing information and protocols to small communities. We need to strive to make it easier for the physician by finding ways to fund secretarial and nursing support. We also need to continue to strive to educate the physicians about the need for clinical trials. There is no easy solution to reimbursement issues. We must pool all resources, such as federal government, private industry, third party payers, universities, large community hospitals, and the patients themselves to come to a compromise that will ensure the survival of clinical trials research. The crisis is here, action is underway, and we as oncology specialists must assist in finding the answers. REFERENCES I. News. J NaIl Cancer Insl 1988; 71 :619-620. 2. Hunter el at. Selection factors in clinical trials: Results from the Community Clinical Oncology Program Physician's Log. Cancer Trea/ Rep 1987; 71 :559-565. 3. Merz B. NCI seeks to boost study participation: Medical NelliS and PerspeclivesJAMA 1988; 260:1337. 4. Antman KH, Aledort LM. Yarbro J el at. Cost-effectiveness and reimbursement in patient care. Semin !lema/ol 1989; (Suppl 3) 26:3245.
