Perioperative off-label use of gabapentinoids: evidence-based medicine validated or not?

BackgroundOff-label use (OLU) of a drug reflects a perceived unmet medical need, which is common in oncology. Cancer drugs are often highly expensive and their reimbursement is a challenge for many healthcare systems. OLU is frequently regulated by reimbursement restrictions. For evidence-based healthcare, treatment ought to be reimbursed if there is sufficient clinical evidence for treatment benefit independently of patient factors not related to the treatment indication. However, little is known about the reality of OLU reimbursement and its association with the underlying clinical evidence. Here, we aim to investigate the relationship of reimbursement decisions with the underlying clinical evidence.Methods/ designWe will extract patient characteristics and details on treatment and reimbursement of cancer drugs from over 3000 patients treated in three Swiss hospitals. We will systematically search for clinical trial evidence on benefits associated with OLU in the most common indications. We will describe the prevalence of OLU in Switzerland and its reimbursement in cancer care, and use multivariable logistic regression techniques to investigate the association of approval/rejection of a reimbursement requests to the evidence on treatment effects and to further factors, including type of drug, molecular predictive markers and the health insurer.DiscussionOur study will provide a systematic overview and assessment of OLU and its reimbursement reality in Switzerland. We may provide a better understanding of the access to cancer care that is regulated by health insurers and we hope to identify factors that determine the level of evidence-based cancer care in a highly diverse western healthcare system.

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COVID-19: Off-label therapies based on mechanism of action while waiting for evidence-based medicine recommendations

World Journal of Meta-Analysis ◽

10.13105/wjma.v8.i3.173 ◽

2020 ◽

Vol 8 (3) ◽

pp. 173-177

Author(s):

Matteo Scotto Di Vetta ◽

Marco Morrone ◽

Serafino Fazio

Keyword(s):

Mechanism Of Action ◽

Evidence Based Medicine ◽

Evidence Based ◽

Off Label ◽

Based Medicine

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Off-label drug use in home palliative care: A non-evidence-based established practice.

Journal of Clinical Oncology ◽

10.1200/jco.2017.35.31_suppl.225 ◽

2017 ◽

Vol 35 (31_suppl) ◽

pp. 225-225

Author(s):

Laura Velutti ◽

Carlotta Pavesi ◽

Diego Lopane ◽

Concetta Arcanà ◽

Annalisa Saetta ◽

...

Keyword(s):

Palliative Care ◽

Retrospective Analysis ◽

Evidence Based ◽

Care Pathways ◽

Administration Route ◽

Off Label Use ◽

Off Label ◽

Treatment Interruptions ◽

Infusion Duration ◽

Label Use

225 Background: Advanced cancer patients (pts) often present with multiple concomitant symptoms and off-label use of drugs (indications doses formulations administration route) is a common practice. Drugs mixture (DM) solutions by continuous administration are frequently used. However, evidence-based data in this setting is scarce. Methods: We performed a retrospective analysis of 576 consecutive care pathways provided by our Home Palliative Care (HC) Service from Jul 2010 to Jun 2017. Infusions were administered subcutaneously (sc) or by a venous (iv) access, by elastomeric pumps (EP) (5 days - 2 mL/h) with drugs diluted in 0.9% NaCl with no light protection. We analyzed: treated symptoms, drugs, doses/concentrations, solution transparency, administration route, infusion duration, clinical response, adverse events (AEs), compliance/treatment acceptability, device proper functioning, and treatment interruptions. Results: We analyzed 266 pts out of 576 care pathways treated with DM infusions, M/F 143/123, median age 70 yrs (21-92); treated symptoms: pain (50%), nausea/vomiting (20%), dyspnea/agitation (20%), dysphagia/mucositis (18%); infused drugs: morphine, midazolam, dexamethasone, ranitidine, metoclopramide, scopolamine, alizapride, haloperidol, furosemide, and ketorolac. Median number of drugs in the DM was 3 (1-6). Administration route was sc in 214 pts/80%, iv in 52 pts/20%. Median infusion duration was 5 days (0-64). Symptoms control was achieved within 72 h in 69% of pts. No AEs related to the infusion devices were observed. Compliance was good in 92% of pts, poor/inadequate in 8%. 5 EP malfunctions were observed: 1 human error, 1 device obstruction due to furosemide flocculation, 3 unexplained. Also, 3 pts required sc infusion interruption due to fluid accumulation near the injection site. Conclusions: This retrospective analysis on a large number of pts shows efficacy and tolerability of DM administration by EP in the HC setting. Treatment compliance was high, no AEs were observed, EP malfunction and therapy discontinuation were rare. However, there are still many open questions about the off-label use of drugs in this setting, and prospective trials are strongly suggested.

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Licensed medicines, off-label use or evidence-based. Which is most important?

Archives of Disease in Childhood ◽

10.1136/archdischild-2016-311527 ◽

2016 ◽

Vol 102 (1) ◽

pp. 53-54 ◽

Cited By ~ 12

Author(s):

Maurizio Bonati ◽

Evelyne Jacqz-Aigrain ◽

Imti Choonara

Keyword(s):

Evidence Based ◽

Off Label Use ◽

Off Label ◽

Label Use

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Off-label use of ketamine for treatment-resistant depression in clinical practice: European perspective

The British Journal of Psychiatry ◽

10.1192/bjp.2019.102 ◽

2019 ◽

Vol 215 (2) ◽

pp. 447-448 ◽

Cited By ~ 2

Author(s):

Álvaro López-Díaz ◽

Manuel Murillo-Izquierdo ◽

Elisa Moreno-Mellado

Keyword(s):

Clinical Practice ◽

Public Healthcare ◽

Evidence Based ◽

European Perspective ◽

Treatment Resistant Depression ◽

Off Label Use ◽

Treatment Resistant ◽

Off Label ◽

Resistant Depression ◽

Label Use

SummaryKetamine therapy for treatment-resistant depression in European national health systems may only be considered after attempting all evidence-based antidepressant strategies outlined in clinical guidelines. This paper seeks to explain the ethical, regulatory and procedural framework for the off-label use of ketamine for treatment-resistant depression within a public healthcare system.Declaration of interestNone.

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Medication Repurposing in Pediatric Patients: Teaching Old Drugs New Tricks

The Journal of Pediatric Pharmacology and Therapeutics ◽

10.5863/1551-6776-21.1.36 ◽

2016 ◽

Vol 21 (1) ◽

pp. 36-53 ◽

Cited By ~ 2

Author(s):

Martha M. Rumore

Keyword(s):

New York ◽

Pediatric Patients ◽

Drug Repurposing ◽

Evidence Based ◽

Off Label Use ◽

Level Of Evidence ◽

Off Label ◽

Pediatric Age Group ◽

Evidence Quality ◽

Label Use

OBJECTIVES: Gaps in pediatric therapeutics often result in off-label use and specifically, novel uses for existing medications, termed “drug repurposing.” Drug Information (DI) queries to a Pediatric Medication Resource Center of a large metropolitan pediatric hospital in New York and inherent difficulties in retrieving evidence-based information prompted a review of current medication repurposing for pediatric patients. The objective included characterization of innovative off-label use of medications Food and Drug Administration (FDA)-approved for 1 or more indications to treat a totally different disorder or indication in pediatric patients. METHODS: A systematic literature review was conducted to retrieve publications describing repurposed medications in pediatric patients. Excluded was FDA-approved indications used off-label in pediatric patients (e.g., different dose), preclinical data, adult use only, and experimental use. Evidence quality was classified using a modified American Academy of Neurology Level of Evidence. Results were analyzed using χ2 at p < 0.05. RESULTS: Over 2000 references were retrieved and reviewed. A total of 101 medications repurposed for novel off-label uses for pediatric patients were identified: 38 for neonates, 74 for children, and 52 for adolescents. Neonates and infants were least likely to receive a medication for a repurposed use. Strong or intermediate evidence existed in 80.2% of cases. The evidence was weak in 19.8%. No significant relationship was observed between the pediatric age group and strength of the literature. Most repurposed uses pertained to generic or widely used medications. Less than 5% of medications were first marketed after 2011. CONCLUSIONS: While not exhaustive, the present study represents the most comprehensive listing of novel uses exclusive to pediatric patients. Further research is needed to identify the frequency of repurposed uses. The valuable DI role of pharmacists in assessing repurposed uses is of expanding and increasing importance to ensure such uses are evidence-based.

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