Journal of Pharma and Drug Regulatory Affairs (e-ISSN:2582-3043)

Orphan Drugs: A Thematic View

Fri, 03 Nov 2023 00:00:00 +0530

Orphan diseases are all medical conditions that impact 0.65 to 1 out of every 1000 people, according to the World Health Organization. Since these are not financially stable and feasible, they are typically not explored for their pathophysiology or the emerging therapy alternatives. On January 28, 1983, in the USA a law known as the Orphan Drug Act was formed to promote the study, creation, and approval of medications for rare diseases. There are currently as many medications for other infectious diseases as there are for tropical infectious diseases, with 11 pharmaceuticals (4.87%) recognized as orphan drugs. Several medications having orphan status are used to treat illnesses that no longer meet the requirements for orphan designation. Some businesses also manufacture and produce orphan medications. Nuclear cloning, rational drug design, and high throughput screening are all methods used in drug discovery programs that could lead to the development of new medicines. This article discusses orphan drugs, medical orphans, rare diseases, the orphan drug act, orphan drug design, the global market scenario of orphan drugs and various databases that contain information about orphan drugs. Patients who have been ignored by for-profit medication discovery efforts have hope for the future.

Anti-Obesity Medications: Emerging Pharmacological Advances and Drug Discovery Perspectives

Leena Yadav, Dipesh Singh Rajput, Aashutosh Shinha — Thu, 20 Jul 2023 00:00:00 +0530

The treatment of disorders including hypertension, adult-onset diabetes, and high cholesterol that are intimately linked to obesity has come a long way in the previous 50 years. However, therapy for obesity has proved difficult, with anti-obesity drugs (AOMs) generally failing to deliver on their promises and raising concerns about their safety. In this article, we present a brief review of AOM evolution, highlighting key milestones and discussing the challenges that still need to be overcome. Recent developments, such as a deeper appreciation for the molecular gut-brain interface, have sparked the search for next-generation AOMs that show promise for causing substantial and long-lasting weight reduction without compromising patient safety. In the United States alone, the prevalence of obesity has increased by a factor of four since 1975, with more than a third of adults and twenty per cent of teenagers being classified as overweight or obese. Obesity raises the risk of several chronic diseases, including type-2 diabetes, cardiovascular disease, and cancers of the colon, oesophagus, liver, rectum, pancreas, gallbladder, and kidney. Several major therapeutic targets have caught the interest of the scientific community despite several failures. They are indicative of the current state of the art in identifying and progressing new medication candidates to human testing. With obesity as the major treatment goal, research into four target areas (leptin, ghrelin, mitochondrial uncouplers, and growth differentiation factor 15 [GDF15]) was launched and progressed.

Medical Devices with Cyber Security for Managing the Life Cycle of Health Technology

Teja Velpula, Koushik Yetukuri — Mon, 30 Oct 2023 00:00:00 +0530

Safeguarding virtual medical devices from emerging cyber-related dangers needs a continuous lifecycle approach, where cyber security is networked into the life cycle and both integrates and reinforces the security risk management processes. A vital process is the efficient management of medical devices across the device life cycle. To maintain the safety and security of devices and data, effective security by design relies on the capacity to comprehend and keep up with cybersecurity challenges. All areas of medicine surgery and community health employ devices extensively nowadays. Since ancient times, medical devices have been employed to treat and identify disease. Cybersecurity is the Safeguarding against malware of internet-connected systems, including data, software and hardware. Lifecycle management is essential to ensuring that medical device is available and in excellent condition when patient care is offered, as well as to maximize their intended lifespan. Wearable technology is currently employed in healthcare settings, although there are a variety of barriers that prohibit this. With the advancement of sensor and computing technologies, devices will become more capable and a necessary part of our instruments for practicing health. The standard lays forth precise technical specifications for producers and developers to follow in order to guarantee the security of their goods through their lifecycles. The focus of cyber security issues when it comes to medical devices exploited in domestic and global networks is emerging. Every citizen in society needs access to health technology, which is a global requirement.

Applications of Proteomics in Human Medicine

Vivek Kumar Tiwari, Bairi Harika — Fri, 13 Oct 2023 00:00:00 +0530

Technology is used in proteomics to count and estimate how many proteins are present in each cell, tissue, and organism. It aids in the identification of an organism's proteins and the comprehension of the structure and functions of a particular protein, complementing other "omics" technologies like genomics and transcriptomics. Proteomics is fundamentally complicated because it entails categorizing and examining all of the protein signatures in a genome. Mass spectrometry is the foundation of contemporary proteomics, with LC-MS-MS and MALDI-TOF/TOF being widely utilized devices. However, finding biomarkers is still a challenge in proteomics due to their entanglement and dynamic nature. Consequently, using a proteomics technique in conjunction with genomics and bioinformatics will help to interpret the data about biological systems and how illness alters them. However, most studies have only examined a small portion of the blood proteins. This review emphasizes the several types of proteomics, the available approaches, and their applications in numerous research fields.

Artificial Intelligence in Regulatory Affairs for Growing Pharmaceutical Companies

V. S. R. L. Sushma Kondaveti, Sreenu Thalla, Koushik Yetukuri — Fri, 20 Oct 2023 00:00:00 +0530

Healthcare product development is about to undergo a drastic change because of artificial intelligence (AI), which has the potential to greatly benefit patients as well as other stakeholders like regulators and industry. One of the most significant factors contributing to this development is the capacity of AI applications to handle routine logical problems and procedural difficulties faced by compliance officers. AI can quickly and accurately manage enormous amounts of information, which may theoretically enhance regulatory compliance. It is safe to claim that technology will soon enable manufacturers to understand compliance requirements quickly and take the necessary actions. Finally, the sustained application of AI in the regulatory and legal sectors has the potential to drastically reduce the number of individuals required to execute the operations. The numerous AI applications are growing rapidly in how organizations are promoting innovation and developing their business strategies. AI has streamlined and had numerous positive effects on the pharmaceutical industry throughout the years, from developing new and improved medications to treating rare disorders. In this article, examples of companies using AI technology are mentioned.