Sickle cell disease is a disabling ailment that significantly impairs sufferers' quality of life. Nonetheless, treatment for sickle cell disease has been limited for years, and there has been a dearth of medicines available to people whose lives have been impacted by the affliction. Crizanlizumab is a ground-breaking medication that has the potential to transform hundreds of lives, and NICE has recommended it as the first novel treatment for sickle cell disease in two decades.

What exactly is sickle cell disease?

Treating sickle cell disease

The National Institute for Health and Care Excellence (NICE) has approved Crizanlizumab, a drug manufactured by Novartis Pharmaceuticals, as treatment for preventing recurring pain crisis in adults with sickle cell disease aged 16 or older this week.

According to NICE, the medication, which was licenced in the United States in 2019, is the first novel treatment for the disease in 20 years in the UK's National Health Service.

NICE, a government-funded body that provides national guidance on how to improve healthcare in England and Wales, is recommending that the medicine be used in the UK under a special arrangement under which more than 300 people will receive treatment in the first year and more than 450 in subsequent years.

Managing sickle cell disease

As there is much ambiguity about the treatment's long-term effectiveness and cost-efficiency, crizanlizumab cannot be recommended for routine usage on the NHS at this time. Rather than that, the committee advised therapy via the Managed Access Agreement (MAA) with the NHS, which will allow people to receive crizanlizumab until new data from clinical studies is gathered to resolve these issues.

This decision was made on the basis of crizanlizumab's cost-effectiveness under the conditions of the MAA. Additionally, the committee considered the substantial unmet demand for sickle cell disease treatments and NICE's goal of eliminating health inequities in its decision-making.

Clinical evidence indicates that those treated with crizanlizumab experience significantly fewer sickle cell crises per year than those treated with other standard therapy alternatives. In the English healthcare system, sickle cell pain crises are treated with regular blood transfusions and the generic drug hydroxycarbamide (known as hydroxyurea in the US.)

Sickle cell disease in India 

SCD disproportionately affects India's most vulnerable tribal communities. Despite several studies demonstrating the epidemiological burden of disease among tribal populations and the practicality of screening procedures in low-resource settings, knowledge and action gaps persist in implementing comprehensive programmes to care for impacted communities.

Due to the increased frequency of SCD among forest-dwelling tribal populations, there is an urgent need for equity-based intervention, considering the other factors that contribute to socioeconomic disparities among SCD-affected tribal communities.

Affordability of treatment options 

With over 300,000 newborns born each year with severe variants of sickle disease, it has been a "grossly ignored health problem," according to the World Health Organisation.

Until 2017, when the US Food and Drug Administration authorised L-glutamine, an oral powder manufactured by the US Emmaus Medical Pharmaceutical firm, Hydroxyurea, a medication approved in 1998 for adults in the US, was the only drug approved to treat sickle cell disease.

L-glutamine has not been approved by the European Medicines Agency, while another medication, Voxelotor, developed by Global Blood Therapeutics (GBT), a biopharmaceutical firm based in the United States and licenced in the United States in 2019, has not been approved outside the United States.

Disclaimer- The views and opinions expressed in this article are those of the author's and do not necessarily reflect the official policy or position of M3 India.

The author is a practising super specialist from New Delhi.