Sickle Cell Society overturn NICE rejection of sickle cell drug

The National Institute for Health and Care Excellence (NICE) have been forced to review a new sickle cell drug, after their decision to reject it was challenged and overturned by the Sickle Cell Society and Pfizer.

Following an appeal for NICE to review the flaws in their decision to not to recommend the drug; Voxelotor, for the treatment of sickle cell anaemia, we are delighted that the appeal panel has overturned that decision and forced a review of the drug, which has been helping people with the condition to achieve a better quality of life.

Sickle Cell Disorder is the fastest growing condition in the UK, but is extremely underserved by treatment options. In the last two decades a new sickle cell drug has been approved for use in the NHS only once.

In November we heard that a groundbreaking gene editing therapy had been approved for the treatment of sickle cell disorder, but it has yet to undergo the scrutiny of NICE, who have the power to approve treatments for the NHS to prescribe, with costs taken into consideration.

Voxelator was initially rejected by NICE in July 2023, and we appealed that decision, along with Pfizer. The appeal panel recognised that NICE had breached its duties under the Equality Act 2010 by failing to recognise barriers to access and/or take into account health inequalities for patients with sickle cell disorder.

John James OBE, Chief Executive at the Sickle Cell Society said :

“We are encouraged by the success of our challenge to NICE, and await their more positive review of the drug, which takes full account of the health inequalities experienced by the sickle cell community.

“The systemic deficiencies in the treatment and care of those affected by this condition include access to new safe and effective disease modifying treatments.

“People are living with this condition, watching the decades slide by, and are seeing absolutely no change in the range of treatments they can access. It’s frustrating that not only are we still fighting for the basics, but also that institutions in control of NHS treatments are missing steps when it comes to enabling people to get the care and treatment they need and deserve.

“Voxelotor was approved safe and effective for use in the USA and Europe, so we ask, why not the UK? We hope that following this successful appeal, and after further consideration by NICE, the drug will become available across the NHS, and have transformative impacts on countless lives.”

Voxelotor offers hope for individuals living with sickle cell disorder. It has made a huge difference for people in the sickle cell community who were involved in the clinical trials.

Kalpna Sokhal lives with sickle cell disorder and had this to say:

“Since I started the Voxelotor trial about 5 years ago I have been so grateful because sickle cell crises no longer come with excruciating pain – instead it’s just slight pain so I’m able to manage with pain killers and avoid emergency hospitalisation.

“It has meant I can still see my husband and son every day without a hospital stay and they don’t have to watch me cry in excruciating pain for hours and days until the pain decides to go away.

“It meant the family summer holiday wasn’t over when I woke up with pains, and I was able to enjoy the sunshine and my son splashing in the pool, avoiding an overseas hospital stay, and an inevitable ruined holiday for us all.

“This medication has given me a new lease on life and a good quality of life. I am still able to be a mum, a wife, and work full time with my sickle cell condition no longer affecting my relationships and employment record. Now that I experience less painful sickle cell crises, my stress and anxiety levels have dramatically reduced.”