2017 ACC/AAP/AHA Health Policy Statement on Opportunities and Challenges in Pediatric Drug Development: Learning From Sildenafil

Sable, Craig; Ivy, D. Dunbar; Beekman, Robert H.; Clayton-Jeter, Helene D.; Jenkins, Kathy J.; Mahle, William T.; Morrow, W. Robert; Murphy, M. Dianne; Nelson, Robert M.; Rosenthal, Geoffrey L.; Stockbridge, Norman; Wessel, David

doi:10.1016/j.jacc.2017.04.007

Cited by 3 publications

(2 citation statements)

References 25 publications

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“…In lung treatment, SLD nano-transformers were prepared using a modified lipid hydration technique 41 . The STARTS-1 and -2 trials and the 2012 US Food and Drug Administration (FDA) product labeling for the use of sildenafil in pediatric patients with pulmonary hypertension improved communication and mission alignment with regard to pediatric drug trials 41 . Administration of sildenafil shows beneficial effects and is likely to be used in chronic kidney disease.…”

Section: Teratogen Testmentioning

confidence: 99%

Review: The Discovery and Development of Sildenafil Citrate

Septifani¹,

Yetti²,

Asra³

2021

Asian J Pharm Res Dev

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Background: Griseofulvin is a fungistatic antifungal drug used to treat dermatophytosis. Dermatophytes that are often found include Trichophyton rubrum, Trichophyton mentagrophytes, Microsporum canis, Microsporum gypseum, Epidermophyton floccosum. Purpose: This review article aims to discuss the drug discovery review of griseofulvin. Data Source: The author created this review article using the literature study method relevant to the purpose of the review. Sources of information from national journals and international journals that are accessed through online sites such as Google Scholar, Research Gate, Science Direct, Springer Link, and NCBI. Key words were used to find the journals, namely griseofulvin, dermatophyte, toxicity. Conclusion: The conclusion of this article is that griseofulvin is used as an antifungal drug containing chlorine from Penicillium griseofulvum isolated against mycelium fungus. This drug is non-toxic, so it is effective and safe to fight various types of fungal infections of the skin such as tinea capitis and remains the drug of choice for dermatophytes.

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Section: Teratogen Testmentioning

confidence: 99%

Review: The Discovery and Development of Sildenafil Citrate

Septifani¹,

Yetti²,

Asra³

2021

Asian J Pharm Res Dev

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“…These include strict requirements from regulatory bodies; ethical restrictions; recruitment and retention of patients; creation of suitable formulations for use by children; and selection of suitable endpoints . Innovative trial designs, novel endpoints, and incorporation of technology are likely to be required for the full evaluation of drugs in pediatric populations …”

Section: Introductionmentioning

confidence: 99%

Treatment of pediatric pulmonary arterial hypertension: A focus on the NO‐sGC‐cGMP pathway

Beghetti¹,

Gorenflo

Ivy

et al. 2019

Pediatric Pulmonology

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Objective While pulmonary arterial hypertension (PAH) is rare in infants and children, it results in substantial morbidity and mortality. In recent years, prognosis has improved, coinciding with the introduction of new PAH‐targeted therapies, although much of their use in children is off‐label. Evidence to guide the treatment of children with PAH is less extensive than for adults. The goal of this review is to discuss the treatment recommendations for children with PAH, as well as the evidence supporting the use of prostanoids, endothelin receptor antagonists (ERAs), and phosphodiesterase type 5 inhibitors (PDE5i) in this setting. Data Sources Nonsystematic PubMed literature search and authors’ expertise. Study Selection Articles were selected concentrating on the nitric oxide (NO)‐soluble guanylate cyclase (sGC)‐cyclic guanosine monophosphate (cGMP) pathway in PAH. The methodology of an ongoing study evaluating the sGC stimulator riociguat in children with PAH is also described. Results Despite recent medical advances, improved therapeutic strategies for pediatric PAH are needed. The efficacy and tolerability of riociguat in adults with PAH have been well trialed. Conclusion The pooling of data across trials, supplemented by registry data, will help to confirm the safety and tolerability of prostanoids, ERAs, and PDE5i in children. Ongoing studies will clarify the place of sGC stimulators in the treatment strategy for pediatric PAH.

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Mandatory requirements for pediatric drug development in the EU and the US for novel drugs—A comparative study

2022

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Mandatory pediatric legislation has been implemented in the European Union (EU) and the United States (US) to increase research and the availability of drugs for the pediatric population. Differences in the legislative framework can cause different pediatric requirements for similar indications granted for similar drugs across jurisdictions. This cross-sectional study compares the pediatric requirements for therapeutic indications granted at the time of initial approval for novel drugs approved in the two regions from 2010 to 2018. We collected the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) decisions to grant a waiver and/or to agree on a pediatric development plan and deferrals hereof at marketing authorization (MA) from publicly available documents. An agreed pediatric development plan was required for 66% (N = 188/285) and 63% (N = 134/212) of the indications granted in the EU and the US at the time of approval, respectively. Almost all (EU; 98%, US; 89%) were deferred until after MA. Based on the broad scope of the EU Pediatric Regulation, an additional 36 PIPs originated from the indications granted at MA. In the subset of indications granted for drugs approved in both the EU and the US (N = 232), significantly more indications resulted in an agreed pediatric development plan for one or more subsets of the pediatric population in the EU (N = 185) as compared to the US (N = 82). This was based on the exemption of orphan designated drugs in the US and the broader scope of the EU Pediatric Regulation. However, indications subject to the mandatory pediatric legislation in both regions (N = 131) most often had similar regulatory requirements for the inclusion of the pediatric population from the EMA and the US FDA (83%, N = 109). In conclusion, when comparing mandatory pediatric requirements, more pediatric development plans were agreed upon in the EU than in the US, in line with the broader mandates of the EU Pediatric Regulation. However, authorities most often had similar regulatory requirements when an indication was subject to pediatric legislation in both regions.

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2017 ACC/AAP/AHA Health Policy Statement on Opportunities and Challenges in Pediatric Drug Development: Learning From Sildenafil

Cited by 3 publications

References 25 publications

Review: The Discovery and Development of Sildenafil Citrate

Review: The Discovery and Development of Sildenafil Citrate

Treatment of pediatric pulmonary arterial hypertension: A focus on the NO‐sGC‐cGMP pathway

Mandatory requirements for pediatric drug development in the EU and the US for novel drugs—A comparative study

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