 "blood test")
The Haemophilia and Health Collective of North (HHCN), a registered body of healthcare professionals working in the domain of haemophilia care, will engage in discussions with several state governments to adopt prophylaxis as the standard care practice for treating Haemophilia.
An official with the body said that HHCN is advocating the inclusion of prophylaxis as the standard of care for Haemophilia A patients in India.
"There will be discussions with various state governments to help in providing the treatment to patients," the official said.
Haemophilia is a rare genetic bleeding disorder in which the blood does not clot properly, leading to spontaneous bleeding as well as bleeding following injuries or surgery. The condition can lead to substantial morbidity, disability, and reduced life expectancy.
Experts note that India is home to the second-largest population of patients living with Haemophilia A (caused by a lack of protein called clotting factor VIII), with more than 136,000 people affected by the condition.
Speaking on the effects of such a large caseload on the country’s economy, Dr Nita Radhakrishnan, associate professor and head of department, paediatric haematology-oncology, Post Graduate Institute of Child Health, Noida, said that patients are forced to drop out of school or remain absent from work due to the regular frequency of bleeds. "This can have various direct and indirect effects on India’s gross domestic product (GDP), and also affect the standard of living for Haemophilia patients," she said.
According to HHCN data, a person with Haemophilia A, on average, experiences 30 to 35 bleeds a year, with each bleed reducing at least 15 days of life and possible complications leading to early mortality or morbidity.
Current treatment modalities for Haemophilia A in India primarily focus on on-demand therapy to address bleeding episodes as they occur. While on-demand therapy is used by around 95 per cent of all patients in India, medical experts say that it is difficult to sustain economically.
Highlighting the hardships faced by patients to avail on-demand therapy, Dr Radhakrishnan said that patients have to travel to the nearest government-approved haemophilia centre in pain. This problem is further compounded in states like Jammu and Kashmir and Uttarakhand, which have geographical disadvantages.
The medical body’s move to advocate for government support for prophylaxis comes after they proposed the first set of local directives for haemophilia treatment in collaboration with National Health Mission (NHM) representatives from 16 states last year. Prophylaxis in Haemophilia consists of regular administering of therapeutic products aimed at preventing bleeding, especially joint haemorrhages. This is either done with factor VIII concentrates or as non-factor therapy with drugs such as emicizumab.
According to a recent study, only 4 to 5 per cent of Haemophilia A patients are given prophylactic treatment in India.
Commenting on the need to adopt prophylactic treatment for haemophilia, Dr Naresh Gupta, chairman, HHCN, said that it is the recommended therapy route for patients with high bleed rates or those who live in remote or distant locations from treatment centres.
This is in line with World Haemophilia Federation’s guidelines, which also recommend the use of prophylaxis with non-factor therapy. "Globally, countries are moving towards advanced non-replacement therapy," he added.
Global data shows that prophylaxis can reduce the bleed rate from 35 to 40 events a year to 0 to 4 events a year.
Speaking on using prophylaxis, Dr Radhakrishnan added that in the case of factor VIII concentrates, the patient requires an intravenous (IV) infusion three times a week. "This is a very strenuous exercise for the patient, as they cannot come to the haemophilia centre thrice a week, every week for the rest of their lives.
"So patients and their carers are taught IV infusion. But even then, many states do not support prophylaxis and for those who do, providing medicines for home therapy may not be allowed," she said.
Some patients may also develop factor inhibitors, leading to the usage of expensive bypass agents. "With the emergence of newer drugs such as emicizumab in 2019, it is now easier to administer prophylactic treatment to patients in India," she added.
Emicizumab can be administered subcutaneously (under the skin) like insulin. "In the limited number of patients that emicizumab has been used, doctors have seen zero to no recurrent bleeding, thus improving their quality of life," Dr Gupta said.
"Since health is a state subject, there is an ongoing effort from individuals and groups to discuss giving a budget for prophylaxis with the state governments," Dr Radhakrishnan said.
Prophylaxis can be a heavy budgetary burden on states, with only 13 states in India procuring emicizumab as of now. The same number of states have included prophylaxis as a standard of care for Haemophilia A.
The body is now recommending a preliminary budgetary calculation for payers and policymakers to understand the budgetary requirements of each state and central reimbursement bodies.
