IOM Review of FDA-Approved Biologics Labeled or Studied for Pediatric Use

Field, Marilyn J.; Ellinger, Lara K.; Boat, Thomas F.

doi:10.1542/peds.2012-2412

Cited by 12 publications

(5 citation statements)

References 19 publications

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“…Extrapolation is the process of approving a BioS for all of the approved indications of the originator drug, even if the BioS has not been formally studied in all the indications or populations of the originator product (3)(4)(5). In clinical practice, extrapolation of molecules in the same class sharing the same mechanism of action from adult to pediatric (6,7) or across indications (8,9) is common in case there are not enough data available or when clinical trials are ongoing. Extrapolation allows keeping the cost of BioS competitive with subsequent greater market availability (4,10,11).…”

Section: Extrapolationmentioning

confidence: 99%

Use of Biosimilars in Pediatric Inflammatory Bowel Disease

Ridder

Assa

Bronský

et al. 2019

J. pediatr. gastroenterol. nutr.

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Biologic therapies have changed the outcome of both adult and paediatric patients with Inflammatory Bowel Disease (IBD). In September 2013, the first biosimilar of infliximab was introduced into the pharmaceutical market. In 2015, a first position paper on the use of biosimilars in paediatric IBD was published by the ESPGHAN IBD Porto group. Since then, more data have accumulated for both adults and children demonstrating biosimilars are an effective and safe alternative to the originator. In this updated position statement we summarize current evidence and provide joint consensus statements regarding the recommended practice of biosimilar use in children with IBD.

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Section: Extrapolationmentioning

confidence: 99%

Use of Biosimilars in Pediatric Inflammatory Bowel Disease

Ridder

Assa

Bronský

et al. 2019

J. pediatr. gastroenterol. nutr.

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“…However, appropriate labeling of clinical safety and efficacy data for children has improved in the United States over the last decade through a series of federal legislative actions, culminating in the permanent authorization of the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act (PREA) under theFDASafetyandInnovationActof 2012. [15][16][17][18][19] European pediatric legislation has also developed in parallel with that in the United States. These legislative developments are summarized in Fig 1. Under this legislation, a pediatric assessment is required for all drug or biological product applications or supplemental applications for a new active ingredient, new indication, new dosage form,newdosingregimen, ornewrouteof administration.…”

Section: Current State Of Safety Research In Pediatric Rheumatologymentioning

confidence: 99%

Using Registries to Identify Adverse Events in Rheumatic Diseases

et al. 2013

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The proven effectiveness of biologics and other immunomodulatory products in inflammatory rheumatic diseases has resulted in their widespread use as well as reports of potential short- and long-term complications such as infection and malignancy. These complications are especially worrisome in children who often have serial exposures to multiple immunomodulatory products. Post-marketing surveillance of immunomodulatory products in juvenile idiopathic arthritis (JIA) and pediatric systemic lupus erythematosus is currently based on product-specific registries and passive surveillance, which may not accurately reflect the safety risks for children owing to low numbers, poor long-term retention, and inadequate comparators. In collaboration with the US Food and Drug Administration (FDA), patient and family advocacy groups, biopharmaceutical industry representatives and other stakeholders, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) and the Duke Clinical Research Institute (DCRI) have developed a novel pharmacosurveillance model (CARRA Consolidated Safety Registry [CoRe]) based on a multicenter longitudinal pediatric rheumatic diseases registry with over 8000 participants. The existing CARRA infrastructure provides access to much larger numbers of subjects than is feasible in single-product registries. Enrollment regardless of medication exposure allows more accurate detection and evaluation of safety signals. Flexibility built into the model allows the addition of specific data elements and safety outcomes, and designation of appropriate disease comparator groups relevant to each product, fulfilling post-marketing requirements and commitments. The proposed model can be applied to other pediatric and adult diseases, potentially transforming the paradigm of pharmacosurveillance in response to the growing public mandate for rigorous post-marketing safety monitoring.

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“…These laws resulted in critical changes in drug labeling for pediatric patients because unique pediatric dosing is often necessary because of the growth and maturational stages of pediatric patients [31]. Furthermore, since the implementation of these incentive programs, a majority of biologics (vaccines, anti-toxins, and insulin) approved include pediatric information in their labeling [32].…”

Section: Addressing Disparities In Clinical Trial Accrualmentioning

confidence: 99%

Scientific and Ethical Considerations for Increasing Minority Participation in Clinical Trials

Wilder¹

2018

Clinical Trials in Vulnerable Populations

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Since its inception, a major weakness in clinical trial research has been an inability to recruit diverse populations into clinical trials. These under-represented populations are mostly comprised of the poor, the elderly, children, women, and racial/ethnic minorities (African Americans and Hispanics). This fundamental weakness is further exacerbated by the fact that these same groups are often disproportionately affected by the diseases being studied in clinical trials. There are various patient specific, provider specific, and policy related causes for the existence of these disparities. Regardless of the cause, the lack of participation of these groups in clinical trials raises important questions about the quality and ethics of clinical research. The goal of this document is to discuss the evidence and reasons behind disparities in clinical trial participation. We also provide a discourse on potential mechanisms to address disparities in clinical trial accrual including the ethical considerations of financial incentives, the impact of a more stringent policy and review process for product approval from the Food and Drug Administration (FDA) including a diversity mandate with an associated population black box warning.

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IOM Review of FDA-Approved Biologics Labeled or Studied for Pediatric Use

Abstract: A majority of biologics approved in the past 15 years include some pediatric information in their labeling, and pediatric trials have been registered for a substantial majority of these products.

Cited by 12 publications

References 19 publications

Use of Biosimilars in Pediatric Inflammatory Bowel Disease

Use of Biosimilars in Pediatric Inflammatory Bowel Disease

Using Registries to Identify Adverse Events in Rheumatic Diseases

Scientific and Ethical Considerations for Increasing Minority Participation in Clinical Trials

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