Informing Care Decisions: Emerging Technologies, Scientific Evidence, and Communication

Slide presentation from the AHRQ 2009 conference.

On September 14, 2009, Elise Berliner, PhD made this presentation at the 2009 Annual Conference. Select to access the PowerPoint® presentation (2.5 MB) (Plugin Software Help).


Slide 1

Slide 1. Informing Care Decisions: Emerging Technologies, Scientific Evidence, and Communication

Informing Care Decisions: Emerging Technologies, Scientific Evidence, and Communication

Elise Berliner, PhD
Director, Technology Assessment Program
Center for Outcomes and Evidence 

Slide 2

Slide 2. A child's toy version of Dr. McCoy's Medical Kit from Star Trek

Image: Star Trek toy doctor kit from the 1960's.

Slide 3

Slide 3. A screen capture of a blog post discussing a Star-Trek style medical analysis Tool  
Trekkie Medicine Cures With a Flick of the Wrist

Image: The beginning of an article from the blogs.amctv.com Website.

http://blogs.amctv.com/scifi-scanner/2009/01/star-trek-ultrasonic-medical-device.php
 

Slide 4

Slide 4. A screen capture of a blog post discussing a Star-Trek style medical analysis Tool (continued)

Image: Continuation of the article from the blogs.amctv.com Website.

Slide 5

Slide 5. Historical Example: Autologous Bone Marrow Transplantation (ABMT)

Historical Example: Autologous Bone Marrow Transplantation (ABMT)

  • Rescue from high dose chemotherapy with autologous bone marrow transplantation in patients with breast cancer
  • Reviews of uncontrolled trials in 1988-1989 found response rates (tumor shrinkage) of 58-80%
  • 1990: preliminary results of Phase II study showed 40% improvement in three year survival rates compared with historical controls treated with standard-dose chemotherapy

Sources: Welch and Mogielnicki 2002 BMJ 324:1088-1092; Mello and Brennan 2001 Health Affairs20:101-117

Slide 6

Slide 6. ABMT History Continued

ABMT History Continued

  • Methodological problems with studies
    • Uncontrolled
    • Selection bias: selection criteria for ABMT stricter than historical studies of standard dose chemotherapy
      • ABMT trial participants had demonstrated an objective response to previously administered chemotherapy
    • Short follow up time
    • Small sample size
  • Potential adverse effects

Source: Mello and Brennan 2001 Health Affairs 20:101-117 

Slide 7

Slide 7. ABMT History Continued

ABMT History Continued

  • Many insurance companies defined ABMT as "experimental"
    • No clear and consistent definition of "experimental"
  • Lawsuits for coverage
    • Massachusetts federal judge (1990): "To require that the plaintiff or other plan members wait until somebody chooses to present statistical proof ... that would satisfy all the experts means that plan members would be doomed to receive medical procedures that are not state of the art".

Reference: Welch and Mogielnicki 2002 BMJ 324:1088-1092 

Slide 8

Slide 8. ABMT History Continued

ABMT History Continued

  • 1996 GAO report: "Coverage of Autologous Bone Marrow Transplantation for Breast Cancer
    • "Although it is widely considered an experimental therapy, many health insurers are covering ABMT following high-dose chemotherapy for breast cancer"
    • Coverage policies influenced by fear of litigation and adverse public relations
    • Use increased rapidly from estimated 522 patients in 1989 to an estimated 4,000 in 1994
  • Four controlled clinical trials funded by NCI
    • Enrollment slower than expected
    • "We worry that women are not enrolling in these clinical trials because they mistakenly assume that HDC-ABMT is already a proven treatment and because they can receive it outside of studies" Dr. Jeffrey Abrams, NCI
    • Slow enrollment leads to delay in research results

Sources: GAO/HEHS-96-83 and press release at http://www.hhs.gov/news/press/1996pres/960528.html

Slide 9

Slide 9. ABMT History Continued

ABMT History Continued

  • Four RCTs presented at 1999 meeting of the American Society of Clinical Oncology meeting did not support use of ABMT
  • NEJM editorial in 2000: "To a reasonable degree of probability AMBT for metastatic breast cancer has been proved to be ineffective and should be abandoned"
  • Effect on patients
    • Estimated 42,680 ABMT procedures between 1990-1999
    • Acute toxicities: sepsis, pulmonary failure, veno-occlusive disease, cardiac failure, nephrotoxicity, hemorrhagic cystitis and cardiac toxicity
    • Chronic toxicities: acute myelogenous leukemia or myelodysplastic syndrome, bone marrow insufficiency, heightened vulnerability to opportunistic infections in the first year
    • Treatment related mortality rates up to 7% in controlled trials.

Sources: Welch and Mogielnicki 2002 BMJ 324:1088-1092; Mello and Brennan 2001 Health Affairs 20:101-117 

Slide 10

Slide 10. Particle Beam Therapy for Cancer

Particle Beam Therapy for Cancer

Redrawn schematic of a proton therapy center.

Adapted from a schematic of the Rinecker Proton Therapy Center, RPTC, Munich, Germany, under construction by ACCEL Instruments (http://www.proton-therapy.com; last accessed 06/16/2008).

The six images above compare the dose distribution of X-ray beams with proton beams.. Red is the maximum dose, followed by orange, yellow, green, blue, and purple.

From http://www.pi.hitachi.co.jp/rd-eng/product/industrial-sys/accelerator-sys/proton-therapy-sys/proton-beam-therapy/index.html 

Slide 11

Slide 11. Particle Beam Therapy For Cancer

Particle Beam Therapy For Cancer

Cancer typeSingle armRCTsNonrandomized comparativeTotal
Ocular804791
Head/neck532156
Spine9009
GI181221
Prostate143219
Bladder3003
Uterus4015
Bone/soft tissue6006
Lung170017
Breast2002
Miscellaneous140014

"When I was doing semiconductor device research, it was expected that I would compare my results with other people's previously published results and that I would comment on any differences. But it seemed to be different in medicine. Medical practitioners primarily tended to publish their own data; they often didn't compare their data with the data of other practitioners, even in their own field, let alone with the results of other types of treatments for the same condition. So I kept on doing cross-comparisons as best I could. "

Andy Grove, cofounder of Intel and prostate cancer patient, Fortune Magazine, May 13, 1996.

Slide 12

Slide 12. Particle Beam Therapy For Cancer

Particle Beam Therapy For Cancer

Table A. Comparators assessed in the randomized controlled trials

Cancer type and centerComparisonNSurvival (overall/specific)
Ocular (uveal melanoma)
MGH (US)Higher vs. lower dose proton RT188No/No
UCSF (US)Helium RT vs. I-125 brachytherapy136; 184Yes/Yes
CPO (France)Proton RT vs. proton RT + laser TTT151Yes/Yes
Head/neck (skull base chordoma/chondrosarcoma)
MGH (US)Higher vs. lower dose proton RT96Yes/No
Head/neck (brain glioblastoma)
UCSF (US)Helium RT vs. photo RT15Yes/Yes
GI (pancreatic cancer)
MGH and LLU (US)Photo RT + standard-dose proton vs. photon RT + high-dose proton393Yes/Yes
MGH (US)Photon RT + local photon boost vs. photon RT + local proton boost202; 191Yes/Yes

Abbreviations: CPO=Centre de protonthérapie d'Orsay; GI=gastrointestinal; LLU=Loma Linda University; MGH=Massachusetts General Hospital; N=number of enrolled patients; RT=radiotherapy; TTT=transpupillary thermotherapy UCSF=University of California San Francisco.

Slide 13

Slide 13. Particle Beam Therapy for Cancer

Particle Beam Therapy for Cancer

  • "In an ideal world, some oncologists say, most cancer patients would get this rare type of treatment, in which doctors use nuclear technology and magnets to fire protons into tumors at about two thirds the speed of light" (US News and World Report, April 2008)
  • "It all comes down to the physics," said Dr. Jerry D. Slater, the head of radiation medicine at Loma Linda University Medical Center in Southern California. "Every X-ray beam I use puts most of the dose where I don't want it." By contrast, he said, proton beams put most of the dose in the tumor" (NYTimes, December 26, 2007)
  • "The scientific debate could be resolved with a large trial in which patients were randomly assigned to X-rays or protons and tracked for years. Proton proponents say that would be a waste of time. "The laws of physics prove beyond a shadow of a doubt that proton radiation" is better, insists prostate cancer survivor Robert Marckini of Mattapoisett, Mass." (Forbes, March 16, 2009). 

Slide 14

Slide 14. Radiofrequency Catheter Ablation for Atrial Fibrillation

Radiofrequency Catheter Ablation for Atrial Fibrillation

Images of the inside of the heart where they do the catheter ablation.

http://www.healthline.com/sw/hr-nl-atrial-fibrillation-beyond-drug-therapies  

Slide 15

Slide 15. Radiofrequency Catheter Ablation for Atrial Fibrillation

Radiofrequency Catheter Ablation for Atrial Fibrillation

  • Many different types of catheters
    • First two catheters specifically approved for atrial fibrillation by the FDA in Feb. 2009
    • Many other catheters used "off-label" by physicians
  • Many different variations of the procedure
    • Different areas of the heart ablated
    • Different imaging techniques to guide the procedure (flouroscopy, MRI, CT, electroanatomic navigation)
  • Evidence is lacking to determine which of these variations would work best in different patients 

Slide 16

Slide 16. Radiofrequency Catheter Ablation for Atrial Fibrillation

Radiofrequency Catheter Ablation for Atrial Fibrillation

  • Reduces recurrence of atrial fibrillation when used as 2nd line therapy but studies only had short follow-up (=12 mo)
    • No long term evidence on mortality etc.
  • Low level of evidence on quality of life (methodological deficiencies in primary studies)
  • Insufficient data on use as 1st line therapy
  • Major clinical complications <5%, but quality of data is poor
    • Non-uniform definitions and assessments
  • Need more data on the elderly, patients with multiple co-morbidities, long-term (years) rates of AF recurrence, effects from radiation exposure, QOL, and mortality 

Slide 17

Slide 17. Radiofrequency Catheter Ablation for Atrial Fibrillation

Radiofrequency Catheter Ablation for Atrial Fibrillation

  • "Success rates are good, particularly for patients with intermittent atrial fibrillation, but some patients require repeat procedures and some still need to take medications afterward. Patients with persistent atrial fibrillation have lower success rates. Some serious complications can occur with this procedure, so the risks and potential benefits must be carefully weighed."
    --Tampa Tribune 2008
  • "I think they're successful maybe in the 80 percent range, but not like we'd like - which would be up in the high 90s," [Dr. R Dent Underwood] said. "But if you look back 10 years ago, it wasn't even an option."
    St. Paul Pioneer Press February 2008

Slide 18

Slide 18. Conclusions

Conclusions

  • Premature adoption of new technologies outside of clinical trials might lead to harm
  • The portrayal of the state of scientific evidence surrounding new medical technologies is often exaggerated by the physicians, patients and journalists.
    • Some technologies (such as catheter ablation) have less hype than others (such as proton beam)

Slide 19

Slide 19. Contact

Contact

Current as of December 2009
Internet Citation: Informing Care Decisions: Emerging Technologies, Scientific Evidence, and Communication. December 2009. Agency for Healthcare Research and Quality, Rockville, MD. http://www.ahrq.gov/news/events/conference/2009/berliner/index.html