Evaluation of marketing authorization and labels of medicines in 2021 WHO Model List of Essential Medicines for Children in China, the Russian Federation and Brazil
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SUBMITTER: Li C
PROVIDER: S-EPMC10913573 | biostudies-literature | 2024 Jan
REPOSITORIES: biostudies-literature
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Project description:BackgroundTuberculosis (TB) remains a major cause of morbidity and mortality globally despite effective treatments. Along with high-quality health services, essential medicines are a key tool in curbing TB related mortality. Examining relationships between listing TB medicines on national essential medicines lists (NEMLs) and population health outcomes related to amenable mortality is one way to assess TB care.MethodsIn this cross-sectional study of 137 countries, we used linear regression to examine the relationship between the number of TB medicines listed on NEMLs and TB related mortality while controlling for country income, region and TB burden.ResultsMost countries listed essential TB medicines to treat latent, drug-sensitive and disseminated TB but few listed enough for multi-drug resistant TB (MDR-TB) therapy. The total number of TB medicines listed ranged from 1 to 29 (median: 19, interquartile range: 15 to 22). Over 75% of the variation in health outcomes were explained by the number of TB medicines listed, gross domestic product (GDP) per capita, region and high-burden MDR-TB status. The number of TB medicines listed was not associated with TB mortality.ConclusionMost countries list essential TB treatments and the variation in TB outcomes is explained by other factors such as GDP.
| S-EPMC8924688 | biostudies-literature
Project description:BackgroundSome evidence supports the use of a short list of essential medicines to improve prescribing. We aimed to create a preliminary essential medicines list for use in Canada.MethodsThe 2013 World Health Organization Model List of Essential Medicines was initially adapted by the research team. Fourteen Canadian clinicians gave suggestions for changes to the list. Literature relevant to each unique suggestion was gathered and presented to 3 clinician-scientists who used a modified nominal group technique to make recommendations on the suggested changes. Audits of prescriptions of 2 Toronto-based family health teams (an inner city clinic and a suburban site) between Aug. 1, 2013, and July 30, 2014, were performed to identify common prescriptions that were not on the draft list. Literature relevant to these additional medications was gathered and shared with the clinician-scientist review panel to determine whether each should be added to the list, and a list was developed. The audits were repeated based on the final list to provide a preliminary assessment of the coverage of the list.ResultsThe multistep process produced a list of 125 medications. The medications included on this list covered 90.8% and 92.6% of prescriptions at the inner city clinic and the suburban site, respectively. In total, 93% of the patients seen at the inner city clinic and 96% of the patients seen at the suburban clinic had all or all but 1 of their medications covered by the list.InterpretationA preliminary list of essential medicines was developed that covered most, but not all, prescriptions at 2 primary care sites. The list should be further refined based on wider input.
| S-EPMC5378503 | biostudies-literature
Project description:BackgroundThe popular use of herbal medicines necessitates national regulatory authorities to have efficient mechanisms for the control of these products including marketing authorization (MA) and safety follow-up. Herbal medicines like conventional medicines require assessment of efficacy, safety and quality information before MA can be granted. However, the complete proof of safety is mainly based on the history of the long-term traditional use. Herbal medicines can cause adverse reactions due to various factors and thus require clinical trials to ensure their safety. Herbal medicines treatment practices involve combinations of different plants to achieve the desired effect while multiple herbal components have been known to cause herbal-herbal toxicity and interactions due to variety of complex active ingredients in plants. Compliance with regulatory requirements on herbal medicines has been shown to be difficult for manufacturers since different countries have different regulatory requirements with wide variations which results in the MA of very few herbal medicines. Limited studies on dossiers of marketing authorization of herbal medicines have been performed in other countries, with no studies in African regulatory system settings. The aim of this study is to determine the type of safety documentation that is submitted on herbal medicines application dossiers to support MA in Tanzania.MethodsA cross-sectional retrospective study of herbal medicines dossiers submitted at the Tanzania Medicines and Medical Devices Authority from 2009 to 2020 was conducted.ResultsAs many as 75% of the herbal products applications were combination products made by more than one herbal substance or plant. Out of 84 dossiers subjected to analysis the majority did not provide evidence of preclinical (55%) and clinical safety data (68%). Evidence of safety data in humans was mostly from the literature (70%) and not manufacturers' clinical studies. Quality parameters with safety implications were not included in 48% and 23% of the active herbal substance and finished product specifications, respectively.ConclusionAnalysis of the herbal medicine dossiers submitted showed major deficiencies of safety data to support MA. Manufactures need to provide evidence to support the safety of their products for evidence-based regulatory decisions and to avoid multiple reviews of the applications.
| S-EPMC10658996 | biostudies-literature
Project description:BackgroundThe WHO Essential Medicine List for Children was released on the 30th anniversary of the general Essential Medicine List in 2007, to recognise special needs for medicines in children, and to promote the inclusion of paediatric medicines in national procurement programmes. This study aimed to investigate the alignment of the medicines included in the Albanian reimbursement medicines list of the Mandatory Healthcare Insurance Fund (AMHIF) and the Essential Medicine List for Children.MethodsA quantitative evaluation was performed to compare the paediatric medicines included in the 2022 list of the AMHIF and the 2021 WHO Essential Medicine List for Children. In addition, vaccines in the Albanian vaccination programmes for children were compared to the ones listed on the WHO Essential Medicine List for Children.ResultsBoth lists had a total of 284 active ingredients in common, whereas 14 of 24 vaccines were found to be in common in the Essential Medicine List for Children list and the Albanian vaccination programmes.ConclusionsThis is the first study in Albania to investigate the alignment of the WHO EMLc and AMHIF list. In case of the same active ingredient there were many deviations in terms of dosage form, strength and indication.
| S-EPMC10775712 | biostudies-literature
Project description:ObjectivesChallenges remain in ensuring universal access to affordable essential medicines. We previously estimated the expected generic prices based on cost of production for medicines in solid oral formulations (ie, capsules or tablets) on the WHO Model List of Essential Medicines (EML). The objectives of this analysis were to estimate cost-based prices for injectable medicines on the EML and to compare these to lowest current prices in England, South Africa, and India.DesignData on the cost of active pharmaceutical ingredients (APIs) exported from India were extracted from an online database of customs declarations (www.infodriveindia.com). A formula was designed to use API price data to estimate a cost-based price, by adding the costs of converting API to a finished pharmaceutical product, including the cost of formulation in vials or ampoules, transportation and an average profit margin.ResultsFor injectable formulations on the WHO EML, medicines had prices above the estimated cost-based price in 77% of comparisons in England (median ratio 2.54), and 62% in South Africa (median ratio 1.48), while 85% of medicines in India had prices below estimated cost-based price (median ratio 0.30). 19% of injectable medicines in England, 9% in South Africa, and 5% in India had prices more than 10 times the estimated cost-based price. Medicines that appeared in the top 20 by ratio of lowest current price to estimated cost-based price for more than one country included numerous oncology medicines-irinotecan, leuprorelin, ifosfamide, daunorubicin, filgrastim and mesna-as well as valproic acid and ciclosporin.ConclusionsEstimating manufacturing costs can identify cases in which profit margins for medicines may be set significantly higher than average.
| S-EPMC6773329 | biostudies-literature
Project description:This study aimed to determine to which extent data on potential pharmacogenetic-pharmacokinetic (PG-PK) interactions are provided to, and assessed by, the European Medicines Agency (EMA) in novel drug marketing authorization applications (MAAs), and whether regulatory assessment of PG-PK interactions is adequate or could be optimized. For this purpose, we retrospectively analyzed MAAs of small molecule drugs assessed by the EMA between January 2014 and December 2017. As per two key requirements in the EMA's guideline, we analyzed cases where (i) a single functionally polymorphic drug metabolizing enzyme (DME) metabolizes > 25% of the drug, or (ii) the drug's PK shows high interindividual variability not explained by other factors than PG. Results showed that, of 113 drugs analyzed, 53 (47%) had ≥ 1 functionally polymorphic DME accounting for > 25% of the drug's metabolism, yielding 55 gene-drug pairs. For 36 of 53 (68%) of the products, CYP3A4 was the major DME. Compliance with European Union (EU) guidance on PG-PK issues in drug development was notably different for CYP3A4 substrates vs. non-CYP3A4 substrates. Adequate PG-PK data were provided during registration in 89% (16/18) of cases concerning non-CYP3A4 substrates, compared with 32% (12/37) of cases concerning CYP3A4 substrates. Concluding, PG-PK interactions related to non-CYP3A4 substrate drugs were, in general, addressed adequately in EU MAAs. PG-PK information on CYP3A4 substrates was available less frequently, despite some available evidence on the functional relevance of CYP3A4 polymorphisms. A more harmonized approach toward assessment of PG-PK issues in EU MAAs seems warranted, and a discussion on the relevance of CYP3A4 polymorphisms, such as CYP3A4*22, is recommended.
| S-EPMC7484984 | biostudies-literature
Project description:With the implementation of the new EU regulation on in vitro diagnostics (IVDR) in May 2022, notified bodies will be required to assess Companion Diagnostics (CDx). The EMA and national medicines agencies will be consulted on the performance and safety of CDx. In this paper, we report on our systematic review on how the EMA has dealt with CDx in dossiers for marketing authorization procedures, in 2017-2019, and in scientific advice procedures in 2016-2020, prior to the implementation of the new IVDR. Out of 167 medicines approved or refused by the EMA, CDx played a role for 20 medicines during assessment. Both European public assessment reports (EPARs) and the internal day 80 and day 120 assessment reports (ARs) of the EMA centralized marketing authorization procedures for these 20 medicines were analyzed in detail to determine how CDx were assessed. Likewise, in 46 of 159 cases in which scientific advice was provided, CDx were mentioned in the question-and-answer section of the scientific advice, and these were analyzed in an analogous manner. Our analysis indicates that clinical performance and analytical performance of the CDx were the most-discussed topics, being discussed 11 and seven times in the 20 EPARs and 59 and 29 times in the ARs, respectively. For scientific advice, clinical and analytical performance was discussed 65 and 22 times in the 46 retrieved mentions of scientific advice. Other aspects in relation to CDx were discussed as well, although at a lower frequency, in assessment reports and scientific advice. Overall, our analysis demonstrates that, despite the absence of an obligation from a legal point of view, EMA has gained experience on the assessment of CDx, most notably regarding its analytical and clinical performance. This experience may be useful in situations in which the EMA and national agencies of EU member states will formally be consulted by notified bodies regarding the performance and safety of CDx. In addition, the issues raised in the EPARs, ARs and scientific advice reports provide insight for applicants on aspects of CDx that need careful consideration.
| S-EPMC9120537 | biostudies-literature
Project description:ObjectivesWe used the WHO draft nutrient profile model (NPM) to evaluate baby foods targeted at infants and young children (IYC) aged 6-36 months in the Russian Federation to assess their suitability for marketing.DesignA cross-sectional study in Moscow (Russian Federation).SettingNutrition information of baby food was primarily collected from retailer websites, with some complementary data from physical stores. Both specialist stores for IYC and general supermarkets were included.ParticipantsTwo hundred and thirty baby food products targeted to IYC were selected. Breastmilk substitutes and products targeted at children over 3 years old were excluded.Main outcome measuresPer cent of missing nutrition data, per cent of products with added sugar or sweetener and exceeded sodium or salts, per cent of products marketed as suitable for IYC under 6 months.ResultsMost products were 'ready-to-eat', including fruit (n=42, 18.5%) and vegetable (n=29, 12.8%) purees, meat, fish or cheese purees (n =26, 11.5%); 'dry or instant cereal/starchy foods' (n=27, 11.9%), including predominantly dry cereals, 'juices and other drinks' (n=26, 11.5%). 95% (n=219/230) of products were missing total sugar information, 78% (n=180/230) were missing either sodium or salt, and 25% (n=57/230) were missing total fat. Among products with sugar and sodium information, 41% (n=94/230) included added sugar or sweeteners, and 48% (n=24/50) exceeded the NPM sodium threshold. 40% of products (n=92/230) were marketed as suitable for IYC aged under 6 months.ConclusionBaby foods marketed for IYC showed a high per cent of missing nutrition information and disparity with WHO's guidelines for complementary feeding, age of introduction, sugar and salt content. Stronger regulation is needed in this area to minimise higher risk of non-communicable diseases (NCDs) in later life.
| S-EPMC9872470 | biostudies-literature
Project description:Background: Reference to so-called real-world data is more often made in marketing authorization applications for medicines intended to diagnose, prevent or treat rare diseases compared to more common diseases. We provide granularity on the type and aim of any external data on efficacy aspects from both real-world data sources and external trial data as discussed in regulatory submissions of orphan designated medicinal products in the EU. By quantifying the contribution of external data according to various regulatory characteristics, we aimed at identifying specific opportunities for external data in the field of orphan conditions. Methods: Information on external data in regulatory documents covering 72 orphan designations was extracted. Our sample comprised public assessment reports for approved, refused, or withdrawn applications concluded from 2019-2021 at the European Medicines Agency. Products with an active orphan designation at the time of submission were scrutinized regarding the role of external data on efficacy aspects in the context of marketing authorization applications, or on the criterion of "significant benefit" for the confirmation of the orphan designation at the time of licensing. The reports allowed a broad distinction between clinical development, regulatory decision making, and intended post-approval data collection. We defined three categories of external data, administrative data, structured clinical data, and external trial data (from clinical trials not sponsored by the applicant), and noted whether external data concerned the therapeutic context of the disease or the product under review. Results: While reference to external data with respect to efficacy aspects was included in 63% of the approved medicinal products in the field of rare diseases, 37% of marketing authorization applications were exclusively based on the dedicated clinical development plan for the product under review. Purely administrative data did not play any role in our sample of reports, but clinical data collected in a structured manner (from routine care or clinical research) were often used to inform on the trial design. Two additional recurrent themes for the use of external data were the contextualization of results, especially to confirm the orphan designation at the time of licensing, and reassurance of a large difference in treatment effect size or consistency of effects observed in clinical trials and practice. External data on the product under review were restricted to either active substances already belonging to the standard of care even before authorization or to compassionate use schemes. Furthermore, external data were considered pivotal for marketing authorization only exceptionally and only for active substances already in use within the specific therapeutic indication. Applications for the rarest conditions and those without authorized treatment alternatives were especially prominent with respect to the use of external data from real-world data sources both in the pre- and post-approval setting. Conclusion: Specific opportunities for external data in the setting of marketing authorizations in the field of rare diseases were identified. Ongoing initiatives of fostering systematic data collection are promising steps for a more efficient medicinal product development in the field of rare diseases.
| S-EPMC9413272 | biostudies-literature
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.
| S-EPMC9733348 | biostudies-literature