Health Insurance Carriers Can Restrict Recruitment on a Randomized Clinical Trial of Stem Cell Transplant for Autoimmune Disease

Transcription

1 Health Insurance Carriers Can Restrict Recruitment on a Randomized Clinical Trial of Stem Cell Transplant for Autoimmune Disease Keith M Sullivan, M.D. Duke University Medical Center For the SCOT (Scleroderma: Cyclophosphamide or Transplantation) Trial Investigators

2 Background Do Insurer Denials Restrict Clinical Trials? Peters NEJM 1994: 23% of requests for coverage for ABMT for breast cancer were denied by insurers. Light NEJM 1994 (accompanying editorial): More fairness, rationality and public accountability to insurance reimbursement for clinical trials is needed. GAO report 1999: No evidence of widespread limitations on patient access to NIH clinical trials by insurers. AHRQ Technology Assessment 2009: Although published data are near non-existent to quantify the magnitude of the effect of third party denials on recruitment into clinical trials, insurance policies do restrict recruitment onto NIH supported clinical studies.

3 Scleroderma (systemic sclerosis) ( the rheumatologists most ominous disorder ) At Diagnosis Rodnan Skin Score > 15 5 YR Survival 50% DLCO<60% 30%

4 Sponsored by: National Institutes of Health (NIH) through its Division of Allergy, Immunology and Transplantation (DAIT) in the National Institute of Allergy and Infectious Diseases (NIAID)

5 NORTHWEST FHCRC & Virginia Mason & U. of WA SCOT Study Sites NORTH CENTRAL Med. Coll. of Wisconsin U. of Michigan NORTHEAST Boston U. & Mass Gen. Hosp. Ctr for Rheum. UCLA & City of Hope SOUTHWEST California Pacific MC Mayo Clinic U. of WI Thom Jeff U. Hosp. for Special Surgery U. Chicago Mercy Arthritis UMDNJ North Shore Long Island Med. Univ. of Ohio Columbia University Georgetown U. U. of Cincinnati U. of Kentucky WUSTL SOUTHEAST Duke University U. of Tenn. Memphis Med. U. of South Carolina U. of Texas Southwestern U. of AL Birmingham Confirmed Transplant Centers Confirmed Rheumatology Centers U. of TX Houston & MD Anderson Cancer Center SOUTH CENTRAL October 26, 2005

6 SCOT Key Eligibility Criteria Age years Diffuse SSc with poor prognosis Extensive skin involvement Disease duration < 5 years Early internal organ involvement with either: Pulmonary disease ( DLCO or FVC <70%) Prior scleroderma renal crisis

7 Select Subjects by Screening Randomization Stem cell transplant Arm Cyclophosphamide (CTX) Arm Stem Cell Mobilization 2.5x10 6 CD34 High-dose immunosuppressive therapy: TBI 800 cgy + CTX 120mg/kg +ATGAM 90mg/kg followed by Auto-CD34+HPC transplantation Initial Pulse CTX of 500 mg/m 2 11 additional treatments of IV CTX 750 mg/m 2 at day intervals.

8 SCOT Eligibility Assessment Steps Prescreened Referrals to SCOT sites Enroll and Consent for Screen #1 If Eligible Eligibility Review Committee If Approved Application to Insurer for Coverage If Approved Screen #2 and Consent for Treatment If Eligible Randomization and Treatment Screen #1 History & Physical Exam by SCOT Rheumatologist and Transplanter, Clinical Labs, Rodnan Skin Score, Pulmonary Function Tests and DLCO, Electrocardiogram, Echocadiogram, Gastroduodenoscopy (to rule out GAVE) Screen #2 Repeat History & Physical Exam, Clinical Labs, Pulmonary Function Tests and DCLO, Bronchoalveolar lavage with cell count/cultures, High Resolution Chest Tomography on a dedicated machine, Research Labs and Specimen Storage, Health Insurer Supports: All Clinical Care Costs NIH Supports: All Research Costs including: Data Collection, Patient Travel for Follow-up visits X 7 yr, Repository Collections, Immune Function Studies, Mechanistic Studies, Monitoring of study sites and stem cell labs, Data Management and Analysis

9 Insurance Process: Initial Application (packets sent to insurers for SCOT treatment approval) o o A cover letter request from Transplant Center PI SCOT Rheumatology and Transplant physician consult notes o Results of Screen 1 o o Full Treatment Protocol and Consents Reprints of supporting prior publications Cy treatment for SSc (SLS study): NEJM 2006 SCT for SSc (FHCRC pilot): Blood 2007

10 Insurance Process: Denials Patient files appeal if denied. PI discusses disapproval directly with medical director of plan Reviews prior approvals by other Plans and the CMC Requests outside review by experts in scleroderma and transplant Submits additional published materials

11 95 pts submitted for insurance review SCOT Insurance Reviews (104 submissions for 95 patients) Approved on Initial submission 44(46%) Denied on Initial submission 51(54%) Approved on first appeal 13(14% of total; 36% of 1 st appeal) Denied on first appeal 23(24% of total; 64% of 1 st appeal) Did not Appeal 15(16% of total); Approved on 2 nd appeal 7(7% of total; 54% of 2 nd appeal) Denied on 2 nd appeal 6(6% of total; 46% of 2 nd appeal) Did not appeal 10(11% of total) Final Approval:67%; Never appealed the initial denial: 29%

12 Health Insurers Approving SCOT (104 submissions) First Submission No Appeals of Denial Initial+Appeal Approve Deny Appeal Approve Deny Approvals Non-Blue Commercial (29 submissions) (34%) (72%) Blue/Anthem Commercial (57 submissions) (65%) (68%) Public/Government Insurers (18 submissions) 6 12 (67%) (50%)

13 Reasons For Insurer Denials 26 Experimental or Investigational* 5 No clinical trial benefits 4 Diagnosis not covered 24 Unknown/unstated * Reprints of published promising results of treatment of SSc with Cy (NEJM 2006) and SCT (Blood 2007) were submitted with all insurance applications

14 Insurer Decisions and Study Randomization (97 patients) Randomized on SCOT trial Decisions by Insurers Yes No Approved on initial submission (44) A Approved on Appeal (25) 19 6 B Denied, No Appeal (14) 1 C 13 Denied on Appeal (14) 1 C 13 Totals 55 (57%) 42 A. Pt declined (3), Pt ineligible(3), Disease progressed (2), Co-Pay too high(1), Coverage Ran Out(1) B. Disease progressed (4), Pt ineligible(1), Pt declined(1) C. Self pay

15 Conclusions Health insurer payment decisions can be restrict entry onto a pivotal trial in autoimmune disease Data show some insurers delay and deny patient access on an NIH sponsored randomized clinical trial while others are inconsistent in approvals These restrictions do impede clinical research and the evidence needed to advance health care in America

16 The relevant matter in examining any form of treatment is not the reputation of its proponent, the persuasiveness of his theory, the eminence of its lay supports, the testimony of patients, or the existence of public controversy, but simply does the treatment work? - Dr. David Karnofsky

17 Acknowledgments Over 50 basic and clinical investigators at 33 universities in North America who are involved in the SCOT studies