Abstract
BackgroundIdentification of the determinants of access to investigational drugs is important to promote equity and scientific validity in clinical research. We aimed to analyze factors associated with the use of experimental antiretrovirals in Italy.MethodsWe studied participants in the Italian Cohort of Antiretroviral-Naive Patients (ICoNA). All patients 18 years or older who had started cART (≥ 3 drugs including at least two NRTI) after their enrolment and during 1997-2007 were included in this analysis. We performed a random effect logistic regression analysis to take into account clustering observations within clinical units. The outcome variable was the use of an experimental antiretroviral, defined as an antiretroviral started before commercial availability, in any episode of therapy initiation/change. Use of an experimental antiretroviral obtained through a clinical trial or an expanded access program (EAP) was also analyzed separately.ResultsA total of 9,441 episodes of therapy initiation/change were analyzed in 3,752 patients. 392 episodes (360 patients) involved an experimental antiretroviral. In multivariable analysis, factors associated with the overall use of experimental antiretrovirals were: number of experienced drugs (≥ 8 drugs versus "naive": adjusted odds ratio [AOR] = 3.71) or failed antiretrovirals(3-4 drugs and ≥ 5 drugs versus 0-2 drugs: AOR = 1.42 and 2.38 respectively); calendar year (AOR = 0.80 per year) and plasma HIV-RNA copies/ml at therapy change (≥ 4 log versus < 2 log: AOR = 1.55). The probability of taking an experimental antiretroviral through a trial was significantly lower for patients suffering from liver co-morbidity(AOR = 0.65) and for those who experienced 3-4 drugs (vs naive) (AOR = 0.55), while it increased for multi-treated patients(AOR = 2.60). The probability to start an experimental antiretroviral trough an EAP progressively increased with the increasing number of experienced and of failed drugs and also increased for patients with liver co-morbidity (AOR = 1.44; p = 0.053). and for male homosexuals (vs heterosexuals: AOR = 1.67). Variability of the random effect associated to clinical units was statistically significant (p < 0.001) although no association was found with specific characteristics of clinical unit examined.ConclusionsAmong patients with HIV infection in Italy, access to experimental antiretrovirals seems to be influenced mainly by exhaustion of treatment options and not by socio-demographic factors.
Highlights
Identification of the determinants of access to investigational drugs is important to promote equity and scientific validity in clinical research
When the first antiretrovirals entered clinical trials, in response to the need of patients for whom no effective treatment was available, the US issued a new regulation that allowed experimental drugs to be distributed outside clinical trials in the context of so-called Expanded access programs (EAP) [4]
Defined AIDS was present at enrolment in 16.9% of patients, while liver comorbitidy was present in 40.8%. 45.6% of patients were cared for in a teaching hospitals
Summary
Identification of the determinants of access to investigational drugs is important to promote equity and scientific validity in clinical research. Removing the barriers to accessing experimental drugs is an important goal from both a scientific and ethical point of view. For patients with serious or life-threatening diseases who cannot be treated effectively with approved drugs, access to experimental drugs may represent the only possible way of receiving effective treatment [3]. The issue of accessing experimental drugs has been debated for persons with HIV infection since the first decade of the epidemic. When the first antiretrovirals entered clinical trials, in response to the need of patients for whom no effective treatment was available, the US issued a new regulation that allowed experimental drugs to be distributed outside clinical trials in the context of so-called Expanded access programs (EAP) [4]. Similar regulations were issued in other countries [5]
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