Rare Disease Policy

HC asks Centre, Delhi govt for report on stocks of rare disorder injection

The Delhi High Court has directed the Centre and Delhi government to file their reports on the status of stocks of Antihemophilic Factor injection, which is administered to persons suffering from a rare genetic blood disorder. The court asked the governments to also give their status reports on the existing supply chain system for all the hospitals under their administration and listed the matter on December 12. "The respondents are directed to file status reports with respect to the stock of the AHF injections, and the existing supply chain system for all the hospitals under their administration. The same be done within ten days," Justice Sanjeev Narula said in an order passed on November 28. The court was hearing a petition by several persons suffering from haemophilia disease, which is a rare genetic blood disorder. The petitioners submitted that the treatment for this disease is expensive and requires injections of Antihemophilic Factor (AHF). The said while the treatment for

Need to create awareness about rare diseases in society: CJI Chandrachud

Chief Justice of India DY Chandrachud on Saturday emphasised on the need to create awareness about rare diseases among the society, on being empathetic and supportive to such parents and their families regardless of cultural, religious or traditional barriers. He said we can no longer afford to be unaware of genetic diseases. Citing the National Policy of Rare Diseases launched by the Ministry of Health and Family Welfare in March 2021, he said that further research is necessary to define the definition of rare diseases and stressed on the need to ensure equitable access to advanced medical therapy like gene therapy in a diverse nation like India. Speaking at the conference organised by Narayana Nethralaya Foundation on Gene therapy and precision medicine, Justice Chandrachud noted, "In a country like India, home to the largest population in the world with over 4,600 distinct population groups, many of which are endogamous, we face a heightened burden of rare diseases. Unfortunately

Mpox deaths rise by 107 in one week, Africa CDC calls toll unacceptable

African countries recorded more than 100 mpox-related deaths in the past week, the continental health body said Thursday as it described the rising toll as not acceptable. Dr Jean Kaseya, director-general of the Africa Centre for Disease Control and Prevention, said 107 new deaths and 3,160 new cases had been recorded in the past week, just a week after his agency and the UN World Health Organisation launched a continental response plan. In the one week, we lost 107 (people). It's too much. It's not acceptable, Kaseya said as he emphasised the need for stronger cross-border surveillance. Mpox belongs to the same family of viruses as smallpox but causes milder symptoms like fever, chills and body aches. People with more serious cases can develop lesions on the face, hands, chest and genitals. In testing for the disease, men recorded the highest positivity rate at 63%, while children under the age of 15 were at 41%. Kaseya said there was need for increased testing and resources to .

Rare disease patients suffer as CoEs under-utilise funds, shows data

5 of 12 CoEs formed to treat rare diseases have spent less than 35% funds allotted

Rare disease patients ask for sustained funding from health ministry

LSDs are primarily treated with Enzyme Replacement Therapy (ERT), which is a lifelong treatment and can cost anywhere between Rs 17 lakh to Rs 50 lakh depending on the weight of the patient

Rare flesh-eating bacteria that can kill in two days spreads in Japan

At the current rate of infections, the number of cases in Japan could reach 2,500 this year, with a 'terrifying' mortality rate of 30%

Rare Disease Day 2024: All you need to know about rare disease communities

Rare Disease Day 2024 is vital to spreading awareness about rare diseases. This extraordinary day is observed on the last day of February and it presents a unique opportunity

Medicines for four rare diseases become cheaper due to domestic production

Medicines for four rare diseases have become available at drastically cheaper rates as Indian pharma companies are now producing them reducing the reliance on expensive imported formulations, Union Health Ministry officials said on Friday. The slashing of prices comes as the ministry has prioritised action related to 13 rare diseases along with sickle cell anaemia. Drugs for four of these diseases -Tyrosinemia Type 1, Gaucher's Disease, Wilson's Disease and Dravet-Lennox Gastaut Syndrome - along with sickle cell Anemia have been approved and are being manufactured indigenously. Four more drugs for three diseases - Tablet Sapropterin for Phenylketonuria, tab Sodium Phenyl Butyrate and tablet Carglumic Acid for Hyperammonemia and Capsule Miglustat for Gaucher's disease - are under process for approval and are likely to be available by April 2024, official sources said. With these drugs being manufactured indigenously, the annual cost of Nitisinone capsules, used in the treatment of .

Delhi HC constitutes committee to implement 2021 Rare Disease Policy

The Delhi High Court has constituted a five-member committee for the implementation of the National Rare Disease Policy-2021, including procurement and "indigenisation" of therapies and drugs for patients who are mostly children. The members of the National Rare Diseases Committee would include secretary, Union health ministry, or one of his nominee, director general of the Indian Council for Medical Research (ICMR), the Drug Controller General of India, and Dr Madhulika Kabra and Dr Nikhil Tandon from AIIMS-Delhi, the court ordered. It was dealing with a batch of petitions concerning the treatment of several children suffering from rare diseases. Justice Prathiba M Singh said urgent steps in close coordination among the medical community, therapy providers and government agencies were needed as under the present framework, Centres for Excellence were not centrally coordinated, leading to lack of timely availability and adequate therapies for patients with rare diseases. The court

Kids suffering from rare diseases: HC seeks presence of Health Secretary

Stating it cannot turn a blind eye to the medical condition of 40 children suffering from rare diseases, the Delhi High Court has sought the presence of the secretary of the Union Health Ministry before it following the non-compliance of its order directing release of Rs 5 crore for their treatment. Justice Prathiba M Singh, who was hearing a batch of petitions relating to their treatment, said the effectiveness of the medicines already administered to such children would be completely obliterated if further doses are not continued and asked the official to be physically present in court on May 10 when she would consider whether notice of contempt ought to be issued. "The Secretary, Ministry of Health & Family Welfare shall physically remain present in Court on the next date of hearing. On the next date, the Court shall consider whether notice of contempt ought to be issued in these matters. List on 10th May, 2023," ordered the court. The petitioners before court are children ...

Imported drugs for rare diseases to be exempt from customs duty

Normally, medicines attract a basic customs duty of 10%, while some life-saving drugs or vaccines attract a concessional rate of 5%, or are completely exempt from duties

Govt exempts import duty on drugs for personal use to treat rare disease

The government has exempted basic customs duty on all drugs and food for special medical purposes imported for personal use for treatment of rare diseases. The import duty waiver will come into effect from April 1. Also, the government has exempted Pembrolizumab (Keytruda), used in treatment of various cancers, from basic customs duty. Drugs/Medicines generally attract basic customs duty of 10 per cent, while some categories of life saving drugs/vaccines attract a concessional rate of 5 per cent or Nil. "The Central Government has given full exemption from basic customs duty on all drugs and Food for Special Medical Purposes imported for personal use for treatment of all Rare Diseases listed under the National Policy for Rare Diseases 2021," a finance ministry statement said. Food for Special Medical Purposes is a food formulation intended to provide nutritional support to persons who suffer from a specific disease, disorder or a medical condition, as a part of their dietary ...

Ensure sustainable funding for Lysosomal Storage Disorders, says MPs

Patients diagnosed with ultra-rare genetic conditions, including Lysosomal Storage Disorders, require sustainable funding support, 24 Parliamentarians from across party lines wrote to Union Health Minister Mansukh Mandaviya, seeking his intervention in the matter. Under the aegis of the Parliamentary Support Group for Rare Diseases, the MPs from both Lok Sabha and Rajya Sabha said this in a memorandum to the health ministry on Rare Disease Day, stressing the need for a sustainable fund to manage certain diseases under the National Policy for Rare Diseases, 2021. Rare Disease Day is observed every year on the last day of February. "Classified as Group 3a conditions in the National Policy for Rare Diseases, 2021, we would like to draw your attention and seek measures in putting up a mechanism to ensure continuity to all these patients, subject to a review of the progress of these patients by competent members of the Central Technical Committee on rare diseases set up by Ministry of ..

Centre releases Rs 22.20 cr for treatment of patients with rare diseases

The Union Health ministry has released Rs 22.20 crore for 134 patients undergoing treatment for rare diseases at eight designated hospitals since the guidelines for providing financial aid to such patients were issued last August. On May 19, the ministry increased the financial support from Rs 20 lakh to Rs 50 lakh for patients belonging to all categories of rare diseases, and on August 11 issued guidelines to grant financial aid to such patients under the National Policy for Rare Diseases, 2021. According to officials, the number of Centres of Excellence (CoEs) designated for treating those with rare diseases like Gaucher Disease, Tyrosinemia, Severe Combined Immunodeficiency (SCID) and others have also been increased from eight to eleven. The 11 CoEs are AIIMS, New Delhi, Maulana Azad Medical College, New Delhi, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, Post Graduate Institute of Medical Education and Research, Chandigarh, Centre for DNA Fingerprinting an

Govt releases Rare Disease Policy draft, proposes to give Rs 15 lakh help

The Union Health Ministry intends to notify certain medical institutes as Centers of Excellence for Rare Diseases

After new rare diseases policy, govt incentives needed for finding cures

Medical experts say India also needs an orphan drug policy to provide incentives to pharmaceutical companies to innovate and manufacture drugs for rare diseases