The Lancet July 25, 2020

Emtricitabine and tenofovir alafenamide vs emtricitabine and tenofovir disoproxil fumarate for HIV pre-exposure prophylaxis (DISCOVER): primary results from a randomised, double-blind, multicentre, active-controlled, phase 3, non-inferiority trial

Mayer KH, Molina JM, Thompson MA, et al.

Bottom Line

In the DISCOVER trial, F/TAF was shown to be non-inferior to F/TDF for HIV pre-exposure prophylaxis in MSM and transgender women, while demonstrating a more favorable bone and renal safety profile.

Key Findings

1. Emtricitabine/tenofovir alafenamide (F/TAF) was non-inferior to emtricitabine/tenofovir disoproxil fumarate (F/TDF) for HIV prevention, with an incidence rate ratio (IRR) of 0.47 (95% CI 0.19–1.15); the upper bound of 1.15 was well below the prespecified non-inferiority margin of 1.62.
2. After 8,756 person-years of follow-up, there were 22 incident HIV infections: 7 in the F/TAF group (0.16 infections per 100 person-years [95% CI 0.06–0.33]) and 15 in the F/TDF group (0.34 infections per 100 person-years [95% CI 0.19–0.56]).
3. F/TAF was statistically superior to F/TDF across all six prespecified bone mineral density (BMD) and renal biomarker safety endpoints, demonstrating less decline in spine and hip BMD and a more favorable renal profile.
4. Adverse events leading to study drug discontinuation were rare in both study arms (1% for F/TAF vs. 2% for F/TDF).
5. Participants taking F/TAF experienced mild increases in weight and blood lipid concentrations compared to those on F/TDF, consistent with the lack of TDF's known weight-suppressive and lipid-lowering effects.

Study Design

Design
RCT
Double-Blind
Sample
5,387
Patients
Duration
48-96 wk
Median
Setting
Europe and North America
Population HIV-negative adult cisgender men who have sex with men (MSM) and transgender women who have sex with men, at high risk for HIV acquisition
Intervention Emtricitabine 200 mg and tenofovir alafenamide 25 mg (F/TAF) once daily
Comparator Emtricitabine 200 mg and tenofovir disoproxil fumarate 300 mg (F/TDF) once daily
Outcome Incident HIV infection

Study Limitations

Cisgender women and people who inject drugs were excluded from the trial, meaning the findings could not immediately be generalized to individuals exposed to HIV through receptive vaginal sex or intravenous drug use.
The absolute number of incident HIV infections was very low (n=22), which limited the statistical power to formally prove superiority of F/TAF in preventing HIV or to conduct extensive efficacy subgroup analyses.
Participants demonstrated remarkably high adherence compared to typical real-world populations, which may obscure differences in forgiveness for missed doses between the two regimens.
Black and Hispanic/Latino individuals, who are disproportionately affected by the HIV epidemic in the United States, were underrepresented in the study population.

Clinical Significance

The DISCOVER trial established F/TAF (Descovy) as a highly effective, FDA-approved alternative to F/TDF (Truvada) for HIV PrEP in men who have sex with men and transgender women. Because F/TAF limits systemic tenofovir exposure while maintaining high intracellular active metabolite levels, it mitigates the bone loss and renal tubular toxicity associated with TDF. It is now widely preferred for individuals with baseline osteopenia/osteoporosis or renal impairment, though clinicians must weigh this against the potential for mild weight gain and lipid increases.

Historical Context

Since the landmark iPrEx trial and the 2012 FDA approval of Truvada (F/TDF), daily oral F/TDF remained the sole medication approved for HIV PrEP. However, TDF's established association with declines in bone mineral density and renal function raised concerns for lifelong prophylactic use in healthy populations. Tenofovir alafenamide (TAF), a targeted prodrug, had already replaced TDF in many HIV treatment regimens due to enhanced safety. DISCOVER was the pivotal phase 3 trial that demonstrated TAF's efficacy in the prevention setting, leading to the 2019 FDA approval of Descovy for PrEP (excluding those at risk from receptive vaginal sex).

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the pharmacokinetic profile of tenofovir alafenamide (TAF) differ from tenofovir disoproxil fumarate (TDF), and how does this mechanism explain the improved bone and renal safety outcomes observed in the DISCOVER trial?

Key Response

TDF is converted to active tenofovir in the plasma, leading to higher circulating levels that can cause proximal tubule toxicity and bone mineral density loss. TAF is a prodrug primarily cleaved intracellularly (by Cathepsin A in lymphoid cells), resulting in a 90% lower plasma tenofovir concentration. This targeted delivery reduces off-target renal and bone toxicity while maintaining high intracellular concentrations of the active drug.

Resident
Resident

Given the non-inferiority of F/TAF to F/TDF demonstrated in DISCOVER, in which specific patient populations would you definitively choose F/TAF over F/TDF for PrEP, and when might F/TDF still be preferred?

Key Response

F/TAF is preferred for patients with pre-existing renal dysfunction (eGFR between 30-60 mL/min) or those with known osteoporosis/osteopenia. F/TDF is still preferred (or required) for populations not studied in DISCOVER, such as cisgender women at risk via vaginal sex, patients with significant dyslipidemia or weight gain concerns (as TDF has a lipid-lowering and weight-suppressive effect compared to TAF), or when cost/generic availability is the primary barrier.

Fellow
Fellow

The DISCOVER trial highlighted improvements in bone and renal parameters for F/TAF, but how should we weigh the metabolic differences—such as weight gain and lipid profile changes—observed with TAF when discussing long-term cardiovascular risk in an aging PrEP population?

Key Response

While F/TAF avoids the well-documented renal and bone toxicity of F/TDF, patients transitioning to or initiating TAF often see a relative increase in weight and atherogenic lipids (partially due to the loss of TDF's statin-like effect). A nuanced fellow-level approach requires balancing these competing toxicities: mitigating osteo/renal issues versus potentially exacerbating metabolic syndrome and cardiovascular risk, necessitating individualized cardiovascular risk stratification for patients on lifelong PrEP.

Attending
Attending

When educating junior clinicians on the DISCOVER trial, how do you contextualize the clinical significance of the statistically significant but absolute small changes in eGFR and bone mineral density between F/TAF and F/TDF, particularly in light of healthcare economics and generic availability?

Key Response

An attending must bridge clinical trial statistics with real-world practice. While DISCOVER showed statistically significant bone/renal benefits for F/TAF, the absolute event rates for fractures or clinical renal failure in young, healthy MSM on F/TDF are exceptionally low. Teaching points should focus on high-value care: avoiding routine switching to expensive branded F/TAF for healthy, asymptomatic patients while appropriately reserving it for those with genuine risk factors, thus stewarding healthcare resources.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The DISCOVER trial utilized a non-inferiority margin based on retaining a specific percentage of the historical efficacy of F/TDF compared to placebo. How does the unexpectedly low overall incidence rate of HIV in both study arms challenge the statistical power, margin selection, and interpretation of non-inferiority in modern prevention trials?

Key Response

In highly effective prevention trials, event rates (HIV infections) are extremely low. A PhD critique examines how the prespecified non-inferiority margin behaves when baseline incidence is lower than anticipated. Extremely low event rates make it difficult to rule out a clinically meaningful relative loss of efficacy without massive sample sizes, highlighting the fragility of confidence intervals and the challenge of proving non-inferiority when the denominator of events approaches zero.

Journal Editor
Journal Editor

As a peer reviewer evaluating the DISCOVER trial, what major concerns would you raise regarding the study's generalizability due to its exclusion criteria, and how does this limit the sweeping label indications of F/TAF for universal PrEP?

Key Response

A critical reviewer would flag the homogeneity of the trial population, which exclusively studied MSM and transgender women. Because tenofovir concentrations differ drastically across tissue types (e.g., cervicovaginal vs. rectal tissues), establishing non-inferiority in rectal exposure does not guarantee efficacy for vaginal exposure. Reviewers must demand strict caveats in the manuscript preventing off-label extrapolation to cisgender women or persons injecting drugs.

Guideline Committee
Guideline Committee

How does the evidence from the DISCOVER trial inform the CDC and WHO PrEP guidelines regarding the strength of recommendation for F/TAF, and what specific demographic and exposure-route limitations must the guidelines explicitly codify when outlining first-line regimens?

Key Response

Based on DISCOVER, current CDC guidelines recommend F/TAF as an FDA-approved PrEP option with a strong level of evidence (Level IA) but explicitly restrict this recommendation to MSM and transgender women. Guidelines must explicitly codify that F/TAF is NOT recommended for people at risk through receptive vaginal sex due to a lack of efficacy data in this compartment. The committee must position generic F/TDF as the universal first-line option across all demographics, with F/TAF as an alternative for specific populations.

Clinical Landscape

Noteworthy Related Trials

2010

iPrEx Trial

n = 2,499 · NEJM

Tested

Emtricitabine/tenofovir disoproxil fumarate (F/TDF) daily

Population

HIV-negative men or transgender women who have sex with men

Comparator

Placebo

Endpoint

Incident HIV infection

Key result: Daily oral F/TDF reduced the risk of HIV acquisition by 44% overall, and up to 92% in those with detectable drug levels.
2015

IPERGAY Trial

n = 400 · NEJM

Tested

On-demand (event-driven) Emtricitabine/tenofovir disoproxil fumarate (F/TDF)

Population

High-risk men who have sex with men

Comparator

Placebo

Endpoint

Incident HIV infection

Key result: Event-driven PrEP with F/TDF reduced the risk of HIV infection by 86% compared to placebo.
2021

HPTN 083 Trial

n = 4,566 · NEJM

Tested

Long-acting injectable cabotegravir (CAB-LA) every 8 weeks

Population

Cisgender men and transgender women who have sex with men

Comparator

Daily oral Emtricitabine/tenofovir disoproxil fumarate (F/TDF)

Endpoint

Incident HIV infection

Key result: CAB-LA was superior to daily oral F/TDF for HIV prevention, reducing the risk of HIV acquisition by 66% compared to F/TDF.

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