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16 March 2020

List of Medicines to be Used with Caution in Primary Mitochondrial Disease

IMP is aware that the recently published updated List of Medicines to be Used with Caution in Primary Mitochondrial Disease has caused some confusion within the mitochondrial disease patient community. 

Elja van der Veer, Chair of IMP, stated, “We are grateful for the feedback we have received from patients around the world directly and via our member organisations. We updated the List to ensure that the latest insights and new clinical and scientific evidence was considered. There are some drugs that previously had a blanket restriction on them. This has resulted in some patients not gaining access to them, when in fact they would have a positive impact on their health. The evidence that was reviewed by the experts demonstrated that although some drugs cause an adverse reaction in some patients, they can be beneficial to others and should not be labelled harmful to all patients with primary mitochondrial disease. We recommend that patients speak to their treating specialist who will be able to advise them on their individual case.”

The List was compiled by 16 independent international experts in mitochondrial medicine, including clinicians, pharmacologists and scientists, who reviewed scientific data regarding 50 drugs/drug groups. The experts used a rigorous process combining a scientific method, evidence-based research and the latest peer-reviewed literature to reach a consensus on all the drugs on the original list, ensuring they were able to accurately identify drugs which had the potential to cause mitochondrial toxicity as opposed to those which had been previously listed based on patient case reports which could not be linked directly to their mitochondrial disease.

No pharmaceutical companies were involved at any stage (during the process or from a financial perspective).

It is crucial to remember that people can respond differently to drugs and side effects can occur in anyone and are not necessarily related to primary mitochondrial disease. 

The List has been produced as a guideline for patients, carers and non-specialist doctors. Patients should seek advice from their medical specialist, who will consider their personal medical history. 

 

Frequently Asked Questions

Why are some drugs that have previously been deemed ‘unsafe’, now reclassified as ‘safe’?

The most up-to-date evidence-based data indicates that some drugs, previously thought to be ‘unsafe’, actually benefit some patients. It is vital that these drugs are not dismissed or withheld from patients who need them.

Patients should seek guidance from their medical specialist, who will take their individual case into account before prescribing any drug. All drugs included in the List should be used with caution. 

Should I use a drug that has been categorised safe, if I have had an adverse reaction in the past?

Patients who have an adverse reaction to a medicine are advised to consult their clinician, even if the medicine has been categorised 'safe' for them on the list.

Can one drug have a different effect on people with the same type of mitochondrial disease?

Yes! Every patient reacts differently to medication – seek advice that is relevant to you and do not rely on anecdotal evidence, even if it's from other patients with the same disorder.

Should my medical specialist follow this List?

The List has been produced as a guideline for patients, carers and non-specialist doctors. Patients should seek advice from their medical specialist, who will consider their personal medical history before prescribing any drugs.

Were pharmaceutical companies involved in the new List? 

No pharmaceutical companies were involved at any stage (during the process or from a financial perspective).

Who were the experts that updated the List?

The following clinicians, pharmacologists and scientists from six different countries produced the list. They are experts in mitochondrial medicine and represent many of the world’s leading clinical and research institutes.

Maaike C. De Vries, Shamima Rahman, Laurence Bindoff, Michelangelo Mancuso, Gráinne S. Gorman, David A. Brown, Mitchell E. Allen, Amel Karaa, Nandaki Keshavan, Costanza Lamperti, Robert McFarland, Yi Shiau Ng, Mar O'Callaghan, Robert D. S. Pitceathly, Frans G. M. Russel, Kristin N. Varhaug and Tom J. J. Schirris.

See reference article for more background on the experts.

 

Access List of Medicines to be Used with Caution in Primary Mitochondrial Disease

© 2020 Int. Mito Patients