Estimated impact for the NHS

Other treatment

There is currently no established treatment for mitochondrial disorders and clinical management is largely supportive. Interventions used to modify the disease process include dietary modification, pharmacological agents and a variety of nutritional supplements such as co-enzyme Q10, thiamine, vitamin C and vitamin E and exercise therapy (Pfeffer et al. 2012).

Costs of other treatment

Co-enzyme Q10 is available from 'special-order' manufacturers or specialist importing companies. Costs will vary depending on the brand or manufacturer.

Idebenone is a synthetic analogue of co-enzyme Q10. It is licensed for the treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (summary of product characteristics [SPC]: Raxone). Idebenone costs £6,364.00 for 180×150 mg tablets (MIMs, March 2017). This is £6,364.00 for 30 days' treatment at the SPC recommended dose of 300 mg three times a day.

Current or estimated usage

It is not possible to provide estimated usage based on the available data.

Likely place in therapy

There is currently no established treatment for mitochondrial disorders; clinical management, which may include dietary modification and nutritional supplements, is largely supportive.

There was very limited evidence found on the use of co-enzyme Q10 for mitochondrial disorders in children. The case reports included in this evidence summary have limited applicability to clinical practice.

Overall, the studies on the use of co-enzyme Q10 for mitochondrial disorders in adults and young people showed no advantage over placebo for the majority of outcomes assessed. However, these studies also had a number of limitations which restrict their applicability to clinical practice. The studies included in this evidence summary provide no information on the safety and tolerability of co-enzyme Q10 for the treatment of mitochondrial disorders.