HTA Organizations Identified and Described
On the basis of a literature review and input from experts, we identified seven examples of HTA organizations that might inform private payer coverage decisions related to personalized medicine:
- Blue Cross Blue Shield Technology Evaluation Center (BCBS TEC)16
- Emergency Care Research Institute (ECRI)17
- Evaluation of Genomic Applications in Practice and Prevention (EGAPP)18
- Institute for Clinical and Economic Research (ICER)20
- United States Preventive Services Task Force (USPSTF)21
Six HTA organizations (BCBS TEC, ECRI, EGAPP, Hayes, USPSTF, and UpToDate) had developed genomic technology reviews by the time of our study, and the seventh (ICER) was planning to conduct a genomic technology assessment.
The seven HTA organizations included two private companies, two independent panels developed by the government, one academic center, and two nonprofit organizations (). Four organizations made HTA reviews publically available; others charged a fee. The HTA organizations ranged in years of existence (from the 40-year-old ECRI to the 3-year-old ICER) and in the number of genetic reviews they produced (fewer than 15 by BCBS TEC, EGAPP, and USPSTF; more than 15 by ECRI; and more than 100 by Hayes and UpToDate).
Description of the HTA Organizations and Their Respective Reviews
HTA organizations varied in focus. Only one of them focused solely on genomics (EGAPP), and another (USPSTF) included only genomic technologies related to preventive services. Other organizations focused on assessing procedures by using novel technologies, imaging tests, and drugs and biologics. At least three HTA organizations also assessed laboratory tests and behavioral services (ECRI, Hayes, and UpToDate).
All seven HTA organizations conducted rigorous evidence assessment and contained description of the systematic evidence review. However, the HTAs answered differently formulated overarching research questions. USPSTF and EGAPP answered whether a technology should be used in clinical practice; ECRI and UpToDate provided a comprehensive topic review; BCBS TEC and Hayes evaluated evidence on the basis of their respective predefined criteria; ICER was concerned with comparative value of a technology. The majority (n = 5) provided evidence ranking, but only three (EGAPP, UpToDate, and USPSTF) provided recommendations for clinical use.
Payers' Perceptions of the Strengths and Shortcomings of HTA Reviews
All interviewed payers reported conducting internal technology assessment and using external HTA in their coverage decision making for personalized medicine. Payers valued the following strengths of the HTA reviews as related to decisions regarding personalized medicine (): expertise and credibility of reviewers (100%); rigor of scientific evidence evaluation (82%); whether HTA methodology was specific to genomics (73%); independence from external influences (73%). Payers valued evaluation rigor as highly as the HTA reviewer expertise. The majority (64%) considered genomic-specific methodology less important than other HTA strengths.
Strengths and Shortcomings of HTA Reviews
The reported shortcomings of external HTAs were related to review availability (73%) and to the inclusion of nonclinical factors (91%; ). Availability shortcomings included the small number of genetic reviews (64%), a lack of timeliness relative to payer coverage decisions (55%), and the increasing costs of fee-for-service reviews (45%). Fifty-five percent of payers claimed a heavier reliance on Hayes and/or ECRI, because they issued a higher number of reviews, albeit for a fee. The cost concerns were reported by both payers who used the fee-for-service HTAs and who stated that the fees were becoming prohibitive, and by payers who considered using them.
Our study explored how private payers use health technology assessment (HTA) in coverage decisions related to personalized medicine, including oncology.
- Both large and smaller payers used HTA more extensively for personalized medicine than for other technologies and preferred using multiple HTAs.
- A lack of HTA availability and timelines and insufficient inclusion of nonclinical factors limit their relevance in coverage decisions.
Our findings may inform:
- efforts to improve HTA relevance to private payers, particularly for oncology.
- the national HTA agenda, which may benefit from considering private payers' needs related to emerging technologies and how they intersect with patient and provider issues.
Ten of the eleven payers noted that, although all HTA reviews included evaluation of clinical evidence, few of them incorporated other factors that might be important in coverage decisions. Payers listed a spectrum of these factors: cost-effectiveness (82%), current level of adoption in clinical care (45%), incorporation of expert opinion (36%), barriers to adoption in care delivery such as a lack of infrastructure or logistical challenges (27%), and local regulations (eg, state coverage mandates; 27%). Interviewees noted that these factors played a role in their coverage decisions, especially regarding technologies with limited evidence and including many personalized medicine tests. All payers stated that cost-effectiveness was currently not a factor in their coverage decisions; however, at least seven of them believed that it would be a factor in the future as health care reform unfolds.
How HTA Organizations Are Used by Private Payers in Decisions Related to Personalized Medicine
All interviewed payers reported using at least one external HTA organization in their coverage decision making related to personalized medicine (). The number of HTA organizations used by individual payers ranged from one (n = 1) to all seven (n = 1); the majority of payers (n = 8) used three or more different HTA organizations. Large payers reported using more HTA organizations than smaller payers used. HTA reviews from BCBS TEC and USPSTF were reported as being used by most payers (91% and 82%, respectively), whereas ECRI and UpToDate were used by the least number of payers (36% and 45%, respectively). Fifty-five percent of payers stated that using multiple HTAs and comparing them helped them construct a complete evidence profile for a technology, because they had found no one source that was able to provide complete evaluation for genomics. They experienced a higher need for using reviews from multiple HTA organizations for genomics than for other technologies. The majority of payers (91%) found HTA reviews beneficial to their coverage decision making related to personalized medicine and used them for one or more of the following purposes:
- to help internal reviewers with question formulation and methodology (91%);
- to validate internal evidence analyses (36%); and/or
- to demonstrate credibility of decisions to providers and patients (36%).
At least five payers reported that they would like to use HTA reviews for question formulation more often, but because HTAs were not available in a timely manner, payers' internal evaluations were often completed before an external HTA was issued.
All payers found systematic evidence analyses by external HTAs useful, but their opinions differed on the usefulness of other HTA review components. Many payers found evidence ranking (45%) and HTA recommendations (55%) helpful. However, others found ranking confusing, preferred to use their own ranking methods, or deemed external recommendations not relevant to their decisions.