Open research questions in Pharmaceutical studies and practices
80 unresolved questions extracted from the limitations and future-work sections of 532 Pharmaceutical studies and practices papers in our library. Each links back to the study that raised it.
What the literature leaves open
Long-term safety data on tirzepatide for weight management are still being collected, - Further studies on the safety profile of tirzepatide are needed
Real-World Safety Concerns of Tirzepatide: A Retrospective Analysis of FAERS Data (2022–2025) · 2025 · DOILimited real-world safety data on tirzepatide. Lack of long-term safety data on tirzepatide for weight management. Need for enhanced provider and patient education, clearer dosing guidelines, and proactive monitoring.
Real-World Safety Concerns of Tirzepatide: A Retrospective Analysis of FAERS Data (2022–2025) · 2025 · DOIFurther prospective, multicenter cohort studies are warranted to validate these findings and improve DDI management strategies in neonatal populations.
Evaluation of potential drug-drug interactions in the neonatal intensive care unit with two different decision support software: a retrospective study · 2025 · DOIBackground/aims: Due to a lack of standard pediatric prescribing information, medicines are often used in a dosage form or for an indication that has not been investigated in children.
Partnership of I-ACT for children (US) and European pediatric clinical trial networks to facilitate pediatric clinical trials · 2024 · DOIFurther studies should investigate the effects of tablet splitting on patient outcomes. Further studies should evaluate the quality control parameters of tablets from different manufacturers.
There is a lack of research on the effects of tablet splitting on the quality control parameters of alprazolam tablets. There is a need for regulatory guidelines and patient education to ensure safe and effective tablet splitting practices.
The lack of characterization of piperacillin pharmacokinetics in children after liver transplantation. The need for optimized dosing regimens for this population.
Piperacillin Population Pharmacokinetics in Plasma and Peritoneal Fluid and Dosing Optimization in Children Undergoing Liver Transplant: An Optimome Study · 2026 · DOIThere is a need to assess the bioequivalence of different formulations of amoxicillin and clavulanic acid. There is a lack of information on the pharmacokinetic parameters of amoxicillin and clavulanic acid in healthy Vietnamese subjects.
An IN VIVO bioequivalence study of two film-coated tablet formulations containing amoxicillin (875 mg) and clavulanic acid (125 mg) · 2026 · DOIHigh rates of treatment dropout among child and adolescent psychiatric patients. Limited understanding of the factors influencing treatment dropout. Difficulty in identifying high-risk family constellations.
Factors Influencing Treatment Dropout Among Child and Adolescent Psychiatric Patients: A Survival Analysis · 2026 · DOIThe need to identify factors influencing treatment dropout in child and adolescent psychiatric patients. The importance of understanding the impact of family structure and initial treatment approach on treatment adherence.
Factors Influencing Treatment Dropout Among Child and Adolescent Psychiatric Patients: A Survival Analysis · 2026 · DOIThe need for an update on the guidelines for therapeutic drug monitoring in neuropsychopharmacology. The need for a fundamental methodological revision on how to determine therapeutic reference ranges and dose-related reference ranges.
Consensus Guidelines for Therapeutic Drug Monitoring in Neuropsychopharmacology: Update 2026 – Pharmacokinetic, pharmacogenetic and clinical aspects · 2026 · DOIPharmacopsychiatry molar units to relate concentration to dose. Laboratories vary in the presentation of their results. They should always add the reference for the reference range to the result. The clinician should take note of the units (i. e., ng/mL, μg/L, μmol/L, or nmol/L) in which the results of the analysis are expressed. This is especially recommended for comparisons of values obtained from different laboratories or with those in the literature. To transform molar units into mass units and vice versa, conversion factors are given in Table 5. When drug concentrations are below the lower limit of quantification, which refers to the lowest concentration of the standard curve that can be measured with at least 80–120 % accuracy and 20 % precision, this limit should be indicated.1561 The results should be available for decision making within a clinically meaningful time. A 24 hours TDM service is desirable; however, a 48 hours turnaround time is sufficient in most cases. In the case of suspected intoxications, a few hours service is necessary.1391 To assist rapid intervention in patients at risk for toxicity or loss of tolerability, prompt information of the treating physician (i. e., a phone call) is required when the laboratory measures drug concentrations above the laboratory alert level (Table 5). We highly recommend that interpretation and pharmacological or pharmaceutical advice are provided with every assessment of a drug concentration, not only for those outside the therapeutic range. Expert interpretation and the adequate use of the information are essential to ensure the full clinical benefit of the TDM report. Reporting of results with inclusion of dose recommendations and other comments must be guided by the best available evidence. As shown in Fig. 4, it is important to check what the indication for TDM was, if the steady state was reached, if the through concentration was measured and what the level of recommendation was to draw correct clinical conclusions from the results. Expert knowledge may be necessary to calculate dose corrections or analyze drug–drug interactions. It is advantageous for the clinician to choose a laboratory that offers this service. Otherwise, the treating physician, a clinical pharmacologist or a trained expert of the clinic has to interpret the results and check for drug interactions that might explain the results. Access to specialist advice is also necessary if TDM results suggest that genotyping may be advisable, as shown in Fig. 5. It may even be legally required to include collaboration with a clinical pharmacologist. In Switzerland, a psychiatrist or a pharmacist may prescribe CYP genotyping, but it will only be reimbursed by insurances, when the test is prescribed by a physician specialized in clinical pharmacology.
Consensus Guidelines for Therapeutic Drug Monitoring in Neuropsychopharmacology: Update 2026 – Pharmacokinetic, pharmacogenetic and clinical aspects · 2026 · DOIThe study identifies a gap in the enforcement of regulations concerning pharmaceutical advertising in children's programs in Poland. The study highlights the need for effective regulation and oversight to protect minors from exposure to pharmaceutical marketing.
Pharmaceutical Advertising Targeting Children: A Comparative Perspective From Poland and Selected European Countries · 2026 · DOIThere is a lack of knowledge on the impact of patient characteristics on the enantiomeric distribution of citalopram. The study investigates the factors affecting the enantiomeric distribution of citalopram.
Distribution of citalopram enantiomers following medication with racemic citalopram - a naturalistic therapeutic drug monitoring study · 2026 · DOIIn infancy, medication costs typically accounted for less than 15% of total healthcare expenditure in studies reporting both components; childhood evidence was limited and suggested a smaller share of total costs.
The total number of delayed or otherwise incomplete pediatric PMRs is not publicly reported, - Limited data on the effectiveness of NC letters, - Study relied on publicly available FDA resources
Policy Without Penalty: A Review of Pediatric Research Equity Act Noncompliance Letters · 2026 · DOINearly half of PMRs remained delayed even after NC letter issuance, indicating that the FDA’s current enforcement power is insufficient to ensure the timely completion of mandated pediatric studies.
Policy Without Penalty: A Review of Pediatric Research Equity Act Noncompliance Letters · 2026 · DOIOne challenge identified is the lack of controlled studies in youth, making it difficult to determine the effectiveness and safety of off-label antipsychotics and psychiatric polypharmacy. Another challenge is the need to develop effective strategies for deprescribing psychiatric medications in youth, given the complexity of their medication regimens and the potential for negative reactions. The study also highlights the challenge of engaging caregivers and clinicians in the deprescribing process and ensuring that they have the necessary knowledge and support to make informed decisions.
Small sample size, - No statistical tests of significance were conducted, - The study was limited to two urban outpatient community mental health centers in New York
The lack of a streamlined application process for off-label therapies in paediatric oncology. The need for a centralised database of medicines information to support paediatric oncologists. The need for a collaborative approach to patient care in paediatric oncology.
Co-design of a paediatric oncology medicines database (ProCure) to support complex care provision for children with a hard-to-treat cancer · 2024 · DOIPrevious studies did not use a placebo with identical smell, taste, color, and texture to dicyclomine. There is a need to identify factors that would predict whether treatment would be successful.
There is a lack of studies on the effects of written information on patients in the UK. There is a need for more research on the effectiveness of different types of leaflets.
There is a lack of information about children's knowledge and orientations toward medicines and abusable substances. Prior studies have focused on adults' perceptions of children's medicine use.
Self-medication with OTC preparations can be inappropriate. Health professionals must balance encouraging self-care with preventing inappropriate self-medication. There is a need to develop effective methods for evaluating the appropriateness of use of non-prescription and home remedies.
The study had a response rate of 58.5%. The sample was limited to a student population.
Most-cited papers in Pharmaceutical studies and practices
- Developmental Pharmacology — Drug Disposition, Action, and Therapy in Infants and Children · New England Journal of Medicine · 2003 · 2,079 citations
- Medication Errors and Adverse Drug Events in Pediatric Inpatients · JAMA · 2001 · 1,417 citations
- A Teacher Rating Scale for Use in Drug Studies with Children · American Journal of Psychiatry · 1969 · 1,306 citations
- THE BEHAVIOR OF CHILDREN RECEIVING BENZEDRINE · American Journal of Psychiatry · 1937 · 807 citations
- Practice Parameter for the Use of Stimulant Medications in the Treatment of Children, Adolescents, and Adults · Journal of the American Academy of Child & Adolescent Psychiatry · 2002 · 537 citations
- The Transfer of Drugs and Therapeutics Into Human Breast Milk: An Update on Selected Topics · PEDIATRICS · 2013 · 437 citations
- A DIFFERENCE IN MORTALITY RATE AND INCIDENCE OF KERNICTERUS AMONG PREMATURE INFANTS ALLOTTED TO TWO PROPHYLACTIC ANTIBACTERIAL REGIMENS · PEDIATRICS · 1956 · 420 citations
- Evaluation and Management of Well-Appearing Febrile Infants 8 to 60 Days Old · PEDIATRICS · 2021 · 410 citations
- Reported Medication Use in the Neonatal Intensive Care Unit: Data From a Large National Data Set · PEDIATRICS · 2006 · 345 citations
- Off-Label Use of Drugs in Children · PEDIATRICS · 2014 · 336 citations
Most recent work
- Consensus Guidelines for Therapeutic Drug Monitoring in Neuropsychopharmacology: Update 2026 – Pharmacokinetic, pharmacogenetic and clinical aspects · Pharmacopsychiatry · 2026
- Artificial intelligence and precision medicine: a pilot study predicting optimal ceftaroline dosage for pediatric patients · Frontiers in Artificial Intelligence · 2026
- Evaluation of drugs and risk factors requiring therapeutic drug monitoring in the Neonatal Intensive Care Unit: a retrospective study · BMC Pediatrics · 2026
- Experimental evaluation of guideline-recommended pharmaceutical manipulations for hyperbolic tapering of psychiatric drugs · The British Journal of Psychiatry · 2026
- The Case for Prescribing Metformin Off‐Label for a Child in Remission from Ependymoma · The Hastings Center Report · 2026
- Exploring the Practice and Implications of Tablet Splitting in Pharmaceutical Care · Journal of Drug Delivery and Therapeutics · 2026
- Closing the Evidence Gap for Drugs in Children — Measures to Strengthen the Pediatric Research Equity Act · New England Journal of Medicine · 2026
- Double Dose of Prescription Medicines, Overuse or Un necessary use of OTC Medicines, Supplements & its Role of Social Medias in the Gulf & Middle East Countries · PriMera Scientific Medicine and Public Health · 2026
- Post‐Approval Pediatric Use of Drugs Granted Waivers from Pediatric Testing · Clinical Pharmacology & Therapeutics · 2026
- Simulated Y‐Site Compatibility of Calcium Gluconate and Benzylpenicillin With Maintenance Fluid Used in Neonatal Intensive Care Unit · Clinical and Translational Science · 2026
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