«AHRQ Healthcare Horizon Scanning System – Potential High-Impact Interventions Report Priority Area 08: Functional Limitations and Disability ...»
AHRQ Healthcare Horizon Scanning System – Potential
High-Impact Interventions Report
Priority Area 08: Functional Limitations and Disability
Agency for Healthcare Research and Quality
U.S. Department of Health and Human Services
540 Gaither Road
Rockville, MD 20850
Contract No. HHSA290-2010-00006-C
5200 Butler Pike
Plymouth Meeting, PA 19462
Statement of Funding and Purpose This report incorporates data collected during implementation of the Agency for Healthcare Research and Quality (AHRQ) Healthcare Horizon Scanning System by ECRI Institute under contract to AHRQ, Rockville, MD (Contract No. HHSA290-2010-00006-C). The findings and conclusions in this document are those of the authors, who are responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services.
This report’s content should not be construed as either endorsements or rejections of specific interventions. As topics are entered into the System, individual topic profiles are developed for technologies and programs that appear to be close to diffusion into practice in the United States.
Those reports are sent to various experts with clinical, health systems, health administration, and/or research backgrounds for comment and opinions about potential for impact. The comments and opinions received are then considered and synthesized by ECRI Institute to identify interventions that experts deemed, through the comment process, to have potential for high impact. Please see the methods section for more details about this process. This report is produced twice annually and topics included may change depending on expert comments received on interventions issued for comment during the preceding 6 months.
A representative from AHRQ served as a Contracting Officer’s Technical Representative and provided input during the implementation of the horizon scanning system. AHRQ did not directly participate in horizon scanning, assessing the leads for topics, or providing opinions regarding potential impact of interventions.
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Financial Disclosure Statement None of the individuals compiling this information has any affiliations or financial involvement that conflicts with the material presented in this report.
Public Domain Notice This document is in the public domain and may be used and reprinted without special permission.
Citation of the source is appreciated.
Suggested citation: ECRI Institute. AHRQ Healthcare Horizon Scanning System Potential HighImpact Interventions: Priority Area 08: Functional Limitations. (Prepared by ECRI Institute under Contract No. HHSA290-2010-00006-C.) Rockville, MD: Agency for Healthcare Research and Quality. December 2014. http://effectivehealthcare.ahrq.gov/index.cfm/ i Preface The purpose of the AHRQ Healthcare Horizon Scanning System is to conduct horizon scanning of emerging health care technologies and innovations to better inform patient-centered outcomes research investments at AHRQ through the Effective Health Care Program. The Healthcare Horizon Scanning System provides AHRQ a systematic process to identify and monitor emerging technologies and innovations in health care and to create an inventory of interventions that have the highest potential for impact on clinical care, the health care system, patient outcomes, and costs. It will also be a tool for the public to identify and find information on new health care technologies and interventions. Any investigator or funder of research will be able to use the AHRQ Healthcare Horizon Scanning System to select potential topics for research.
The health care technologies and innovations of interest for horizon scanning are those that have yet to diffuse into or become part of established health care practice. These health care interventions are still in the early stages of development or adoption, except in the case of new applications of already-diffused technologies. Consistent with the definitions of health care interventions provided by the Institute of Medicine and the Federal Coordinating Council for Comparative Effectiveness Research, AHRQ is interested in innovations in drugs and biologics, medical devices, screening and diagnostic tests, procedures, services and programs, and care delivery.
Horizon scanning involves two processes. The first is identifying and monitoring new and evolving health care interventions that are purported to or may hold potential to diagnose, treat, or otherwise manage a particular condition or to improve care delivery for a variety of conditions. The second is analyzing the relevant health care context in which these new and evolving interventions exist to understand their potential impact on clinical care, the health care system, patient outcomes, and costs. It is NOT the goal of the AHRQ Healthcare Horizon Scanning System to make predictions on the future use and costs of any health care technology. Rather, the reports will help to inform and guide the planning and prioritization of research resources.
We welcome comments on this Potential High-Impact Interventions report. Send comments by mail to the Task Order Officer named in this report to: Agency for Healthcare Research and Quality, 540 Gaither Road, Rockville, MD 20850, or by email to: email@example.com.
ii Contents Executive Summary
Central Nervous System Disorder Intervention
Dimethyl Fumarate (Tecfidera) for Treatment of Relapsing Forms of Multiple Sclerosis...........2 Genetic Disorder Interventions
Eliglustat Tartrate (Cerdelga) for Treatment of Gaucher’s Disease Type 1
Elosulfase Alfa (Vimizim) for Treatment of Morquio A Syndrome
Idebenone (Catena) for Treatment of Duchenne Muscular Dystrophy
Sensory Disorder Interventions
Corneal Collagen Cross-Linking (VibeX/KXL System) for Treatment of Progressive Keratoconus
Pediatric Vision Scanner Screening for Strabismus and Amblyopia
Retinal Prosthesis System (Argus II) for Treatment of Retinitis Pigmentosa
Tasimelteon (Hetlioz) for Treatment of Non–24-Hour Sleep-Wake Disorder
Prosthetic Arm with Body-Machine Interface (DEKA Arm System) to Restore Natural Arm Function After Amputation
Spinal Cord Injury Intervention
Intraoral Tongue-Drive Computerized System to Maneuver Electric Wheelchairs
Figures Figure 1. Overall high-impact potential: dimethyl fumarate (Tecfidera) for treatment of relapsing forms of multiple sclerosis
Figure 2. Overall high-impact potential: eliglustat tartrate (Cerdelga) for treatment of Gaucher’s disease type 1
Figure 3. Overall high-impact potential: elosulfase alfa (Vimizim) for treatment of Morquio A syndrome
Figure 4. Overall high-impact potential: idebenone (Catena) for treatment of Duchenne muscular dystrophy
Figure 5. Overall high-impact potential: corneal collagen cross-linking (VibeX/KXL System) for treatment of progressive keratoconus
iii Figure 6. Overall high-impact potential: Pediatric Vision Scanner screening for strabismus and amblyopia
Figure 7. Overall high-impact potential: retinal prosthesis system (Argus II) for treatment of retinitis pigmentosa
Figure 8. Overall high-impact potential: tasimelteon (Hetlioz) for treatment of non–24-hour sleep-wake disorder
Figure 9. Overall high-impact potential: prosthetic arm with body-machine interface (DEKA Arm System) to restore natural arm function after amputation
Figure 10. Overall high-impact potential: intraoral tongue-drive computerized system to maneuver electric wheelchairs
iv Executive Summary Background Horizon scanning is an activity undertaken to identify technological and system innovations that could have important impacts or bring about paradigm shifts. In the health care sector, horizon scanning pertains to identification of new (and new uses of existing) pharmaceuticals, medical devices, diagnostic tests and procedures, therapeutic interventions, rehabilitative interventions, behavioral health interventions, and public health and health promotion activities. In early 2010, the Agency for Healthcare Research and Quality (AHRQ) identified the need to establish a national Healthcare Horizon Scanning System to generate information to inform comparative-effectiveness research investments by AHRQ and other interested entities. AHRQ makes those investments in 14 priority areas. For purposes of horizon scanning, AHRQ’s interests are broad and encompass drugs, devices, procedures, treatments, screening and diagnostics, therapeutics, surgery, programs, and care delivery innovations that address unmet needs. Thus, we refer to topics identified and tracked in the AHRQ Healthcare Horizon Scanning System generically as “interventions.” The AHRQ Healthcare Horizon Scanning System implementation of a systematic horizon scanning protocol (developed between September 1 and November 30, 2010) began on December 1, 2010. The system is intended to identify interventions that purport to address an unmet need and are up to 3 years out on the horizon and then to follow them up to 2 years after initial entry into the health care system.
Since that implementation, review of more than 18,000 leads about potential topics has resulted in identification and tracking of about 2,000 topics across the 14 AHRQ priority areas and 1 crosscutting area; about 550 topics are being actively tracked in the system.
Methods As part of the Healthcare Horizon Scanning System activity, a report on interventions deemed as having potential for high impact on some aspect of health care or the health care system (e.g., patient outcomes, utilization, infrastructure, costs) is aggregated twice a year. Topics eligible for inclusion are those interventions expected to be within 0–3 years of potential diffusion (e.g., in phase III trials or for which some preliminary efficacy data in the target population are available) in the United States or that have just begun diffusing and that have completed an expert feedback loop.
The determination of impact is made using a systematic process that involves compiling information on topics and issuing topic drafts to a small group of various experts (selected topic by topic) to gather their opinions and impressions about potential impact. Those impressions are used to determine potential impact. Information is compiled for expert comment on topics at a granular level (i.e., similar drugs in the same class are read separately), and then topics in the same class of a device, drug, or biologic are aggregated for discussion and impact assessment at a class level for this report. The process uses a topic-specific structured form with text boxes for comments and a scoring system (1 minimal to 4 high) for potential impact in seven parameters. Participants are required to respond to all parameters.
The scores and opinions are then synthesized to discern those topics deemed by experts to have potential for high impact in one or more of the parameters. Experts are drawn from an expanding database ECRI Institute maintains of approximately 150 experts nationwide who were invited and agreed to participate. The experts comprise a range of generalists and specialists in the health care sector whose experience reflects clinical practice, clinical research, health care delivery, health business, health technology assessment, or health facility administration perspectives. Each expert uses the structured form to also disclose any potential intellectual or financial conflicts of interest ES-1 (COIs). Perspectives of an expert with a COI are balanced by perspectives of experts without COIs.
No more than two experts with a possible COI are considered out of a total of the five to eight experts who are sought to provide comment for each topic. Experts are identified in the system by the perspective they bring (e.g., clinical, research, health systems, health business, health administration, health policy).
The topics included in this report had scores and/or supporting rationales at or above the overall average for all topics in this priority area that received comments by experts. Of key importance is that topic scores alone are not the sole criterion for inclusion—experts’ rationales are the main drivers for the designation of potentially high impact. We then associated topics that emerged as having potentially high impact with a further subcategorization of “lower,” “moderate,” or “higher” within the high-impact-potential range. As the Healthcare Horizon Scanning System grows in number of topics on which expert opinions are received and as the development status of the interventions changes, the list of topics designated as having potentially high impact is expected to change over time. This report is being generated twice a year.
For additional details on methods, please refer to the full AHRQ Healthcare Horizon Scanning System Protocol and Operations Manual published on AHRQ’s Effective Health Care Web site.
Results The table below lists the 13 topics for which (1) preliminary phase III data for drugs were available; (2) information was compiled and sent for expert comment before November 4, 2014, in this priority area; and (3) we received five to seven sets of comments from experts between January 1, 2014, and November 13, 2014. (This priority area included 107 topics that were being tracked in the system as of November 4, 2014.) We present summaries on 10 topics (indicated below by an asterisk) that emerged as having high-impact potential on the basis of experts’ comments. The material in this Executive Summary and the report is organized alphabetically by disease state and then by intervention. Readers are encouraged to read the detailed information on each intervention that follows the Executive Summary.
Discussion The AHRQ priority area of functional limitations encompasses a wide range of disease states and conditions. For purposes of horizon scanning, AHRQ defines this area using the U.S.