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Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

ABSTRACT: This work aimed to establish whether paediatric needs in cardiovascular diseases have been met by paediatric investigation plans (PIPs) produced since the development of the European Union Paediatric Regulation in 2007. The European Medicines Agency repository was searched for patterns in the development of paediatric medicines in general. Next, positive PIPs related to cardiovascular diseases were scrutinized for outcomes and compared to specific paediatric cardiovascular needs. In total, 1866 PIPs were identified with 12% corresponding to decisions taken for cardiovascular medicines. However, despite this therapeutic area having the greatest number of overall PIPs, only 14% of established needs in paediatric cardiovascular diseases were addressed by PIPs with positive decisions. Further, 71.9% of PIPs with decisions in cardiovascular disease corresponded to full waivers, so the product would not be studied in paediatrics. Despite the progress found in overall numbers of PIPs published, cardiovascular products are still commonly used off-label in paediatrics. Particularly, there is a need to develop products to treat heart failure and hypertension, two areas with clear unmet clinical needs in paediatrics. A case study on valsartan showed that industry, regulators, health technology assessment bodies, and prescribers should work together to reduce off-label use of paediatric cardiovascular diseases (CVD).

SUBMITTER: Faulkner B

PROVIDER: S-EPMC7761547 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature

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Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

Faulkner Bethany B Delgado-Charro M Begoña MB

Pharmaceutics 20201202 12

This work aimed to establish whether paediatric needs in cardiovascular diseases have been met by paediatric investigation plans (PIPs) produced since the development of the European Union Paediatric Regulation in 2007. The European Medicines Agency repository was searched for patterns in the development of paediatric medicines in general. Next, positive PIPs related to cardiovascular diseases were scrutinized for outcomes and compared to specific paediatric cardiovascular needs. In total, 1866 ...[more]

PMID: 33276598

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Project description:IntroductionPharmaceutical industry is no longer allowed to develop new medicines for use in adults only, as the 2007 Paediatric Regulation requires children to be considered also. The plans for such paediatric development called Paediatric Investigation Plans (PIPs) are subject to agreement by the European Medicines Agency (EMA) and its Paediatric Committee (PDCO). The aim of this study was to evaluate the key characteristics of oral paediatric medicines in the PIPs and the changes implemented as a result of the EMA/PDCO review.MethodsAll PIPs agreed by 31 December 2011 were identified through a proprietary EMA-database. PIPs were included if they contained an agreed proposal to develop an oral medicine for children 0 to 11 years. Information on the therapeutic area (EMA classification system); target age range (as defined by industry) and pharmaceutical characteristics (active substance, dosage form(s) as listed in the PIP, strength of each dosage form, excipients in each strength of each dosage form) was extracted from the EMA website or the EMA/PDCO assessment reports.ResultsA hundred and fifty PIPs were included corresponding to 16 therapeutic areas and 220 oral dosage forms in 431 strengths/compositions. Eighty-two PIPs (37%) included tablets, 44 (20%) liquids and 35 (16%) dosage forms with a specific composition/strength that were stored as a solid but swallowed as a liquid e.g. dispersible tablets. The EMA/PDCO review resulted in an increase of 13 (207 to 220) oral paediatric dosage forms and 44 (387 to 431) dosage forms with a specific composition/strength. For many PIPs, the target age range was widened and the excipient composition and usability aspects modified.ConclusionThe EMA/PDCO review realized an increase in the number of requirements for the development of oral dosage forms and a larger increase in the number of dosage forms with a specific composition/strength, both targeting younger children. Changes to their pharmaceutical design were less profound.

Project description:BackgroundGlobally, cardiovascular disease (CVD) is a leading cause of death and disproportionately affects low- and middle-income countries (LMICs). The WHO Model List of Essential Medicines (WHO EML) is a tool for improving accessibility and availability of medicines. This study compared the 2021 WHO EML CVDs basket of medicines with latest available national essential medicines list (NEMLs) for South Africa and 15 Southern African Development Community (SADC) countries to assess consistency in CVDs medicine listing.MethodsThis descriptive, desktop review study compared SADC NEMLs. A comparator list was extracted by combining cardiovascular medicines listed in the 2021 WHO EML for adults and children. SADC country NEMLs were obtained from the WHO Essential Medicines and Health Products Information Portal. Consistency of NEMLs was calculated as a percentage coverage of CVD medicines listed in the 2021 WHO EML. SA hospital and primary health care (PHC) level NEMLs were included as separate formularies.ResultsThe SA hospital level NEML scored 70% consistency with the 2021 WHO EML. Tanzania (84%), Namibia (81%) and Angola (79%) scored the highest consistency. The mean consistency for SADC NEMLs was 66%. The lowest scoring country was Eswatini at 26%. The SA PHC NEML scored 35%. The least listed medicines were beta-blockers, angiotensin receptor blockers (ARBs), clopidogrel (43%) and paediatric formulations (furosemide (21%); digoxin (43%)). Individual antihypertensive medicines were most commonly listed. Botswana and Lesotho were the only countries to list a single pill combination (SPC) for the treatment of hypertension.ConclusionsThis comparison indicates that South Africa and most SADC countries are aligned with 2021 WHO EML recommendations. The inclusion of age-appropriate formulations for children as well as ARBs and SPC for the treatment of hypertension may improve patient adherence and cardiovascular outcomes in these countries. More frequent updates to NEMLs should improve consistency. NEMLs were not available for two countries, and these therefore did not form part of this study. Country health expenditure in ranking the consistency of NEMLs was not accounted for. LMICs adopting the essential medicine list strategy should consider imposing minimum consistency thresholds to the WHO EML to improve accessibility and availability of CVD medicines.Trial registrationNot applicable.

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Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

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Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

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