Enhancing access to treatment for Gaucher disease in India: The need for indigenous manufacturing.

Gaucher disease (GD) is a prevalent lysosomal storage disorder (LSD) that significantly impacts individuals' lives. However, the exorbitant prices of GD medications pose a major hurdle in ensuring widespread availability and affordability of treatment in India. The country heavily relies on imported medications, leading to high costs and limited access for many patients.

Lessons from the Rare Diseases Registry and Analytics Platform framework for development of a national rare diseases registry for India.

Rare diseases (RD) pose significant challenges for healthcare systems globally, necessitating the establishment of disease registries to facilitate research, diagnosis, and treatment. This article explores the development of a comprehensive national RD registry for India, informed by insights gained through interactions with experts from India and the Asia-Pacific Economic Cooperation (APEC) region. The social and technological challenges involved in creating and maintaining a national RDs registry are highlighted.

Assessment of the availability of repurposed orphan drugs in India.

India has a massive burden of rare diseases (RDs), with an estimated 96 million people living with RDs but limited options for treatment. Repurposing drugs used for other common conditions are essential alternative for RDs due to their cost-effectiveness and reduced timeline. India's patent regime prevents the evergreening of drugs, and a large generic manufacturing industry provides ample opportunity to explore the potential of repurposed drugs for treating RDs, known as repurposed orphan drugs (ROD).

Expansion of India's national child healthcare programme, Rashtriya Bal Swasthya Karyakram (RBSK), for rare disease management : a health policy perspective.

Rare diseases (RD) are severe and debilitating conditions. They are one of the leading causes of childhood mortality globally. In India, RDs have not been considered in most healthcare programs which usually cater to more common diseases.

Rare disease patients in India are rarely involved in international orphan drug trials.

We wished to determine whether rare diseases patients from India had been enrolled in international trials to develop novel orphan drugs. There are two reasons to be interested in this. (a) Different ethnic or racial groups may respond differently to a particular drug.

Discrepancies between FDA documents and ClinicalTrials.gov for Orphan Drug-related clinical trial data.

Clinical trial registries such as ClinicalTrials.gov (CTG) hold large amounts of data regarding trials. Drugs for rare diseases are known as orphan drugs (ODs), and it is particularly important that trials for ODs are registered, and the data in the trial record are accurate.

Integrating rare disease management in public health programs in India: exploring the potential of National Health Mission.

Rare diseases (RD) are conditions that affect a small number of people and hence do not get the focus on government health priorities in a resource-constrained setting such as India. Therefore, it is essential to focus on strengthening and utilizing the existing public health framework for the optimal usage of healthcare resources. In this regard, National Health Mission (NHM) is one of the crucial programs initiated by the government of India to address the health needs of the under-served.

The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study.

Background: Orphan medicinal products (OMPs) are intended for the diagnosis, prevention, management or treatment of rare diseases (RDs). Each RD affects only a small fraction of the population, and therefore, historically, industry hesitated to undertake relevant research and development (R&D). In response, the governments of many countries came up with orphan drug policies and RD policies which were hugely successful in incentivizing companies to do so.

The role of patient organizations in the rare disease ecosystem in India: an interview based study.

Background: Rare diseases (RDs) affect a small percentage of the population but can be severely debilitating and life-threatening. Historically, patient groups (PGs) have been the prime movers in raising awareness about these diseases and advocating for national supportive policies. They have also driven relevant research programs.