Expanded Distribution of an Investigational Drug in Parallel with Ongoing Controlled Clinical Trials: The Didanosine Model

Pike, Isadore M.; Daniels, Michelle; Wirtz, Carol; Stout, J. J.; Quigley, James J.

doi:10.1093/clinids/19.6.1071

Cited by 4 publications

(4 citation statements)

References 2 publications

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“…[15][16][17][18] Although such comparisons are attractively simple, they are problematic, because data from most privately funded trials, unpublished studies, and expanded-access programs are unavailable; because the local or national surveillance data used for comparison may not represent all the patients for whom investigational therapies would be appropriate; and because the characteristics of persons who do not participate in trials are not accounted for. [19][20][21] To address these issues, we used nationally representative data from the HIV Cost and Services Utilization Study (HCSUS) to determine the characteristics of the participants in trials of medications for HIV and whether or not patients with HIV had access to research trials and experimental treatments.…”

Section: Resultsmentioning

confidence: 99%

“…11,20,21,36 Non-Hispanic white patients, men who had sex with men, patients with higher incomes, a higher level of education, or private health insurance, and those who received care closer to a clinical-trial center were all more likely to have received experimental medications at some time while receiving HIV care (Table 1).…”

Section: Access To Experimental Medicationsmentioning

confidence: 99%

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Participation in Research and Access to Experimental Treatments by HIV-Infected Patients

et al. 2002

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Section: Resultsmentioning

confidence: 99%

Section: Access To Experimental Medicationsmentioning

confidence: 99%

Participation in Research and Access to Experimental Treatments by HIV-Infected Patients

et al. 2002

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“…The design of the abacavir EAP shared many of the features of EAPs for other antiretroviral agents reported elsewhere [18][19][20][21][22][23]. Consistent with the definition of an EAP, this program included patients with a life-threatening disease (HIV infection) and a paucity of therapeutic alternatives.…”

Section: Discussionmentioning

confidence: 99%

Abacavir Expanded Access Program for Adult Patients Infected with Human Immunodeficiency Virus Type 1

Kessler

Johnson

Follansbee³

et al. 2002

Clinical Infectious Diseases

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Expanded access programs (EAPs) provide medication to patients with life-threatening, treatment-refractory illnesses before regulatory approval and allow the acquisition of safety information. A 2-part, multisite EAP to evaluate abacavir, a carbocyclic nucleoside reverse-transcriptase inhibitor for use in combination antiretroviral therapy, was conducted. The EAP involved >13,000 adults infected with human immunodeficiency virus type 1 (HIV-1) who no longer responded to commercially available treatment regimens. Part A (open-label trials) examined the efficacy, safety, and tolerance of abacavir, and part B (provision of abacavir through expanded access) assessed only the occurrence of serious adverse events. By month 2 of abacavir-containing treatment, plasma HIV-1 RNA levels decreased by > or =0.5 log(10) in 31.4% of patients, and 5.6% of the patients had HIV-1 RNA levels decrease to <400 copies/mL. Drug-related serious adverse events were reported by 7.7% of patients, the most common of which were nausea, skin rash, diarrhea, malaise or fatigue, and fever. Approximately 4.6% of patients experienced a hypersensitivity reaction that was possibly drug related. Overall, the types and incidences of adverse events reported in the abacavir EAP were similar to those reported in phase 2 and 3 clinical trials evaluating abacavir.

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“…These figures are generally higher than what is reported in our study. However, these studies were conducted mostly during the 1990s when a great number of new drugs were in registration studies [24] and when the patients had limited treatment opportunities and large expanded access programs were set up [25]. …”

Section: Discussionmentioning

confidence: 99%

Determinants of access to experimental antiretroviral drugs in an Italian cohort of patients with HIV: a multilevel analysis

Girardi

Scognamiglio

Angeletti

et al. 2012

BMC Health Serv Res

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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.

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Expanded Distribution of an Investigational Drug in Parallel with Ongoing Controlled Clinical Trials: The Didanosine Model

Cited by 4 publications

References 2 publications

Participation in Research and Access to Experimental Treatments by HIV-Infected Patients

Participation in Research and Access to Experimental Treatments by HIV-Infected Patients

Abacavir Expanded Access Program for Adult Patients Infected with Human Immunodeficiency Virus Type 1

Determinants of access to experimental antiretroviral drugs in an Italian cohort of patients with HIV: a multilevel analysis

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