Estimating Population Health Benefits Associated with Specialty and Traditional Drugs in the Year Following Product Approval.

OBJECTIVE: Compared to traditional drugs, specialty drugs tend to be indicated for lower prevalence diseases. Our objective was to compare the potential population health benefits associated with specialty and traditional drugs in the year following product approval. METHODS: First, we created a dataset of estimates of incremental quality-adjusted life-year (QALY) gains and incremental life-year (LY) gains for US FDA-approved drugs (1999-2011) compared to standard of care at the time of approval identified from a literature search. Second, we categorized each drug as specialty or traditional. Third, for each drug we identified estimates of US disease prevalence for each pertinent indication. Fourth, in order to conservatively estimate the potential population health gains associated with each new drug in the year following its approval we multiplied the health gain estimate by 10% of the identified prevalence. Fifth, we used Mann-Whitney U tests to compare the population health gains for specialty and traditional drugs. RESULTS: We identified QALY gain estimates for 101 drugs, including 56 specialty drugs, and LY gain estimates for 50 drugs, including 34 specialty drugs. The median estimated population QALY gain in the year following approval for specialty drugs was 4200 (IQR = 27,000) and for traditional drugs was 694 (IQR = 24,400) (p = 0.245). The median estimated population LY gain in the year following approval for specialty drugs was 7250 (IQR = 39,200) and for traditional drugs was 2500 (IQR = 58,200) (p = 0.752). CONCLUSIONS: Despite often being indicated for diseases of lower prevalence, we found a trend towards specialty drugs offering larger potential population health gains than traditional drugs, particularly when measured in terms of QALYs.

The Decline in Vitamin Research Funding: A Missed Opportunity?

Background: The National Nutrition Research Roadmap has called for support of greater collaborative, interdisciplinary research for multiple areas of nutrition research. However, a substantial reduction in federal funding makes responding to these calls challenging. Objectives: The objectives of this study were to examine temporal trends in research funding and to discuss the potential consequences of these trends. Methods: We searched the NIH RePORTER database to identify NIH research grants and USASpending to identify National Science Foundation and USDA research grants awarded from 1992 to 2015. We focused on those that pertained to vitamin research. For the years 2000 to 2015, we examined funding trends for different vitamins, including vitamins A, B (one-carbon B-vitamins were considered separately from other B-vitamins), C, D, E, and K. Results: From 1992 to 2015, total federal research spending increased from ∼$14 to $45 billion (2016 US dollars). Although vitamin research spending increased from ∼$89 to $95 million, the proportion of grants awarded for vitamin research declined by more than two-thirds, from 0.65% in 1992 to 0.2% in 2015. Federal agencies awarded 6035 vitamin research grants over the time period, with vitamin A associated with the most research projects per year on average (n = 115) and vitamin K the fewest (n = 8). Vitamin D research projects were associated with the greatest average yearly project value ($34.8 million). Conclusions: Vitamin research has faced a disproportionate decline in research funding from 1992 to 2015. Insufficient federal research funding streams risk stalling progress in vitamin research and leaving important advancements unrealized.

Despite high costs, specialty drugs may offer value for money comparable to that of traditional drugs.

Specialty drugs are often many times more expensive than traditional drugs, which raises questions of affordability and value. We compared the value of specialty and traditional drugs approved by the Food and Drug Administration (FDA) in the period 1999-2011. To do this, we identified published estimates of additional health gains (measured in quality-adjusted life-years, or QALYs) and increased costs of drug and health care resource use that were associated with fifty-eight specialty drugs and forty-four traditional drugs, compared to preexisting care. We found that specialty drugs offered greater QALY gains (0.183 versus 0.002 QALYs) but were associated with greater additional costs ($12,238 versus $784), compared to traditional drugs. The two types of drugs had comparable cost-effectiveness. However, the distributions across the two types differed, with 26 percent of specialty drugs--but only 9 percent of traditional drugs--associated with incremental cost-effectiveness ratios of greater than $150,000 per QALY. Our study suggests that although specialty drugs often have higher costs than traditional drugs, they also tend to confer greater benefits and hence may still offer reasonable value for money.

Cost-effectiveness in the contemporary management of critical limb ischemia with tissue loss.

BACKGROUND: The care of patients with critical limb ischemia (CLI) and tissue loss is notoriously challenging and expensive. We evaluated the cost-effectiveness of various management strategies to identify those that would optimize value to patients. METHODS: A probabilistic Markov model was used to create a detailed simulation of patient-oriented outcomes, including clinical events, wound healing, functional outcomes, and quality-adjusted life-years (QALYs) after various management strategies in a CLI patient cohort during a 10-year period. Direct and indirect cost estimates for these strategies were obtained using transition cost-accounting methodology. Incremental cost-effectiveness ratios (ICERs), in 2009 U.S. dollars per QALYs, were calculated compared with the most conservative management strategy of local wound care with amputation as needed. RESULTS: With an ICER of $47,735/QALY, an initial surgical bypass with subsequent endovascular revision(s) as needed was the most cost-effective alternative to local wound care alone. Endovascular-first management strategies achieved comparable clinical outcomes but at higher cost (ICERs ≥$101,702/QALY); however, endovascular management did become cost-effective when the initial foot wound closure rate was >37% or when procedural costs were decreased by >42%. Primary amputation was dominated (less effectiveness and more costly than wound care alone). CONCLUSIONS: Contemporary clinical effectiveness and cost estimates show an initial surgical bypass is the most cost-effective alternative to local wound care alone for CLI with tissue loss and can be supported even in a cost-averse health care environment.