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  1. Mishra S, Bhat D, Venkatesh MP
    Orphanet J Rare Dis, 2024 Nov 29;19(1):446.
    PMID: 39614301 DOI: 10.1186/s13023-024-03377-6
    BACKGROUND: Rare disease (RD) management and orphan drug development in India face various hurdles regarding the implementation and adoption of comprehensive policies, lack of dedicated regulatory frameworks, and absence of epidemiological data. Current rare disease policy focuses more on strengthening the diagnostics and lacks a proper comprehensive treatment framework to ensure favorable clinical outcomes. Indian patients are largely excluded from global orphan drug clinical trials. This further alienates patients from access to rare disease treatment and available treatments come at high cost. This review-based study assesses the landscape of health policies and programs in India through a review of literature and guidelines, to identify strategic opportunities and recommendations for enhancing the overall care and support for the Rare Disease (RD) patient population and improving the orphan drug research ecosystem in India.

    DISCUSSION: The absence of specific regulations, shortage of healthcare resources, budget constraints, competing health priorities, lack of patient data, and insufficient research incentives discourage orphan drug development and global clinical trial inclusion, resulting in treatment inaccessibility and high costs. The Indian Government introduced the National Policy for Treatment of Rare Diseases (NPRD) to address these challenges. Several initiatives have been introduced to attract stakeholders with government-funded research, grants, incentives, and accelerated regulatory approvals of novel therapies that can ensure timely prevention and treatment of rare diseases. The National RD Registry by the Indian Council of Medical Research (ICMR) aims to provide prevalence data. Innovative approaches are required to improve rare disease management and promote orphan drug research. This will ensure the accessibility and affordability of life-saving therapeutics for India's rare disease patients.

    CONCLUSION: An integrated RD management and orphan drug research framework focusing on robust data management, patient-oriented policies to improve the treatment landscape, flexible regulations, strengthening rare disease registry with clinical and diagnostic data, and a favorable research ecosystem to promote indigenous research catering to the Indian population, will improve the treatment landscape and orphan drug research and development in India. This will ensure timely availability of therapeutics at affordable prices.

  2. Hutchinson PJ, Kolias AG, Tajsic T, Adeleye A, Aklilu AT, Apriawan T, et al.
    Acta Neurochir (Wien), 2019 Jul;161(7):1261-1274.
    PMID: 31134383 DOI: 10.1007/s00701-019-03936-y
    BACKGROUND: Two randomised trials assessing the effectiveness of decompressive craniectomy (DC) following traumatic brain injury (TBI) were published in recent years: DECRA in 2011 and RESCUEicp in 2016. As the results have generated debate amongst clinicians and researchers working in the field of TBI worldwide, it was felt necessary to provide general guidance on the use of DC following TBI and identify areas of ongoing uncertainty via a consensus-based approach.

    METHODS: The International Consensus Meeting on the Role of Decompressive Craniectomy in the Management of Traumatic Brain Injury took place in Cambridge, UK, on the 28th and 29th September 2017. The meeting was jointly organised by the World Federation of Neurosurgical Societies (WFNS), AO/Global Neuro and the NIHR Global Health Research Group on Neurotrauma. Discussions and voting were organised around six pre-specified themes: (1) primary DC for mass lesions, (2) secondary DC for intracranial hypertension, (3) peri-operative care, (4) surgical technique, (5) cranial reconstruction and (6) DC in low- and middle-income countries.

    RESULTS: The invited participants discussed existing published evidence and proposed consensus statements. Statements required an agreement threshold of more than 70% by blinded voting for approval.

    CONCLUSIONS: In this manuscript, we present the final consensus-based recommendations. We have also identified areas of uncertainty, where further research is required, including the role of primary DC, the role of hinge craniotomy and the optimal timing and material for skull reconstruction.

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