The World Health Organization (WHO) is to launch a pilot project this year to evaluate inexpensive copies of costly drugs for biotechnology cancer to make these drugs more accessible in the poorest countries.
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The US House of Representatives, controlled by the Republicans, passed a new version of the American Health Care Act to repeal and replace Obamacare, by a vote of 217 to 213. The Republican leaders of the house have blurred to gain sufficient support for the vote after adding a series of changes since an earlier version was taken from a vote in Parliament at the last minute of March.
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Researchers have developed a custom algorithm that predicts the impact of certain foods on an individual's blood sugar, according to a new study published in PLOSComputational Biology.
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Roche Diagnostics UK and Ireland launches the next generation of INR patient self-monitoring meter for patients on warfarin anticoagulant therapy. The new CoaguChek INRange meter allows patients to send their results to their clinic via an App on their phone using wireless connectivity.
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Takeda Pharmaceutical Company Limited announced that ALUNBRIG™ (brigatinib) has received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for the treatment of patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib. This indication is approved under Accelerated Approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. ALUNBRIG, which previously received Breakthrough Therapy Designation from the FDA, is a once-daily oral therapy that may be taken with or without food.
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The Indian Council of Medical Research (ICMR) has launched "The Indian Rare Disease Registry" to meet the unmet needs of patients with rare diseases and to help the country develop data and information to support research and Development and improve innovation.
This India registry launched on 27th April 2017 during The National Initiative for Rare Diseases (NIRD), organized by ICMR, AIIMS, JNU and PRESIDE, seeks to take the first steps to identify patients.
The scope of this registry will evolve over time, maturing from an outreach/community-building effort or a means for a basic understanding of patient and disease characteristics, to a supportive mechanism for research funding and attracting health care providers. It intends to comprehensively cover the spectrum of rare and ultra-rare disorders prevalent in the country but initially it shall only gather data of conditions which have an established treatment available in India or globally. With time, many of the other diseases (some without treatment) would also be incorporated.
The registry is an effort to give Indians who suffer from any form of rare disease a chance to be visible and would initially be based on the hospital or doctor. The Registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves a predetermined scientific, clinical, or policy purposes.
The benefits of the Registry include monitoring prevalence, incidence & natural history of disease over a period of time towards guiding policy decisions; support research initiatives that aim to better understand the distribution and determinants of rare diseases; facilitating access to innovations in genetics, molecular and computational biology, and other technological advances for patients suffering with rare diseases; and bridge the lack of data on rare disorders in our population thus facilitating access to supportive care for countless individuals suffering from these disorders.
The registry will seek to benefit for the patients as they will be identified and therefore would have increased possibility of access to treatment depending upon the inclusion criteria. For the government, it will know exactly the number of patients and therefore provide resources to help address the patients’ needs. For research bodies, the registry will be a source of plethora of information which will be made available for research and development activities to flourish in India. For publications, the data would form the basis of several publications thereby strengthening the country’s stand as a global leader in healthcare and for clinical trials, the patients and local R&D would also benefit.
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A growing number of healthcare institutions and the growing demand for modernization of health care, according to experts, have made it even more important to set up a robust computer system by the Indian health accessible and affordable.
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The National Pharmaceutical Pricing Authority (NPPA) has set price caps for an additional 15 drugs, including those used for the treatment of heart disease, infections, cancer, hypertension and pain relief.
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ProQR Therapeutics N.V. announced that the Company can start the Phase 1/2 trial for QR-110, named PQ-110-001, in Leber’s congenital amaurosis Type 10 (LCA 10) patients. QR-110 is a novel investigational RNA therapeutic targeting LCA 10 due to the p.Cys998X mutation, a severe genetic rare disease that causes children to lose sight in the first years of life. LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available.
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Alder BioPharmaceuticals, Inc. a clinical-stage biopharmaceutical company developing monoclonal antibody therapeutics, today announced the presentation of positive data from its Phase 2b clinical trial evaluating eptinezumab (formerly ALD403) for the prevention of migraine.
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