How is the introduction of a new medicine regulated in the UK?

Transcription

1 Information provided to Duchenne muscular dystrophy patient organisations regarding Raxone (idebenone) and the Early Access to Medicines Scheme in the UK (EAMS 46555/0001) A medicine called Raxone, which is produced by Santhera Pharmaceuticals and is also known as idebenone, is to be made available in the UK through the Early Access to Medicines Scheme (EAMS). Raxone has been given a positive scientific opinion by the Medicines and Healthcare Regulatory Agency (MHRA) for slowing the decline of respiratory function in patients with Duchenne Muscular Dystrophy (DMD) from the age of 10 years who are currently not taking glucocorticoids. As a result, Raxone is now available in the UK before a European marketing authorisation is granted. The manufacturer has separately applied to the European Medicines Agency (EMA) for a marketing authorisation in Europe (also known as a licence ), including the UK, and this is going through the process of review at present. Before giving an EAMS positive opinion the MHRA reviewed information supplied by the manufacturer and determined that Raxone is considered overall to have an acceptable safety profile, based on the information provided, and was satisfied that the company is able to supply the medicine according to a consistent quality standard. In conclusion, considering the very high level of unmet need in this patient population, and that the benefits outweigh the risks, early access to Raxone for DMD patients as defined in the scope of the EAMS indication is considered to be justified. This medicine will not be suitable for everybody and people with DMD need to discuss their own condition with a specialist doctor who knows them and what has been happening to them. Details of the EAMS assessment are available in the Public Assessment Report at ne_final_public_assessment_report.pdf

2 FAQs How is the introduction of a new medicine regulated in the UK? A prescription medicine that is proposed to be used in a new medical condition must be licensed for that use in the countries of the European Union by the European Medicines Agency. The Committee on Human Medicinal Products (CHMP) makes a thorough review of detailed information provided by the manufacturer of the medicine and will recommend if a product should receive a Marketing Authorisation (also known as a product licence ) in the European Union. The information includes the results of clinical trials that have been completed using the medicine in people with the condition that is being studied. What is the Early Access to Medicines Scheme? The aim of the Early Access to Medicines Scheme (EAMS) is to provide earlier availability of promising medicines, before the normal European product licensing process is completed, to patients in the UK where there is high unmet clinical need. The medicinal products included in the scheme are those that are intended to treat, diagnose or prevent seriously debilitating or life threatening conditions where there are no adequate treatment options. The scheme is regulated by the Medicines and Healthcare Regulatory Agency (MHRA) in the UK. More information about the scheme can be found on its website here: dex.htm The MHRA scientific opinion provides benefit and risk information to doctors who may wish to prescribe the unlicensed medicine under their own responsibility. The EAMS programme is only open to include new people until a product gets a European product licence, then no new people can be added to the scheme. What is Raxone? Raxone is a medicine that contains a substance called idebenone in tablet form. Idebenone is a potent anti-oxidant and is believed to improve the functioning of mitochondria (important for the generation of cellular energy) inside cells. Who will be able to have this treatment in the scheme? Raxone will only be available with a prescription from a specialist doctor with experience in managing people with DMD. A specialist doctor can advise on whether the scheme might be suitable for an individual person. Raxone has received positive opinion for use within the EAMS programme to slow the rate

3 of decline of breathing ability in patients with Duchenne Muscular Dystrophy (DMD) from the age of 10 years who are not taking glucocorticoids (also known as steroids ). This is because clinical trials have only been completed with this medicine in patients of this age with DMD who were not taking steroids. Breathing ability is usually measured by lung function tests, and typically these show that function begins to decline in people with DMD around the time they lose ambulation and require a wheelchair most of the time. Lung function decline must be confirmed by spirometry (which measures the maximum flow and amount of air that can be exhaled in one breath) before Raxone is given. Raxone is given without glucocorticoids and can only be given to patients previously treated with glucocorticoids, such as prednisolone or deflazacort, or in patients who are not candidates for continuation or commencement of glucocorticoid treatment. Importantly, Raxone is not a replacement for steroids and people with DMD should not decide to stop steroids solely so that they can start Raxone. A specialist doctor with experience of managing people with DMD can advise on the benefits of continuing steroid therapy for an individual person. Is this medicine used to treat other conditions? Raxone already has a European product licence for treatment of an inherited mitochondrial eye disease, Leber s Hereditary Optic Neuropathy (LHON). The patient information leaflet for this condition can be found here: How does the EAMS work? The EAMS allows medicines to be prescribed by UK specialist doctors to treat people before it has a European product licence. This is not a clinical trial; it is more like how the medicine will be used in real life once it has a licence. However extra care will be taken as this is a new medicine in DMD. Treatment will only be provided in specialist centres who are experienced in managing DMD and there is the requirement for certain monitoring to ensure the safety of patients during the EAMS. People with DMD may discuss together with their doctor whether they are suitable for this treatment and a joint decision would be made whether they want to go ahead. They would be asked to sign a consent form once they have considered all the information. The company that makes Raxone will provide the medicine to the NHS free of charge during EAMS.

4 Where will the treatment be available? This scheme is only available to the National Health Service in England, Scotland, Ireland and Wales. Raxone will only be available through EAMS on prescription through specialist centres which are experienced in treating DMD. The MHRA scientific opinion provides benefit and risk information to doctors who may wish to prescribe the unlicensed medicine under their own responsibility. When will the treatment be available? The MHRA has given Raxone a positive scientific opinion allowing access to Raxone through EAMS and specialist centres in the NHS should be able to implement the scheme across the UK within a short time. Because of the ongoing European regulatory review the EAMS programme will likely be open to include new people for a few months, as once Raxone gets a European licence then no new people can be added to the EAMS scheme. Who can advise someone with DMD about eligibility for the scheme? The first step is for a person with DMD to discuss it with their specialist doctor. What does the treatment involve? Raxone comes as tablets which need to be taken three times a day with food. If someone has difficulty in swallowing, tablets can, if necessary, be crushed into semi-solid food, or into liquid feed for administration through a tube into the stomach. What are the possible side effects? Like all medicines, Raxone can cause side effects, although not everyone gets them. The MHRA and the Company will make available a leaflet written for people with DMD that explains more about Raxone and its effects. Specialist doctors will also be able to explain this further. A risk management plan has been developed to ensure that Raxone is used as safely as possible. Are there extra tests and hospital visits required? In most circumstances no extra tests are required for participants in the scheme beyond what is routine practice. Doctors will want to keep in touch regularly with their patients to know how they are finding the new treatment. In particular they will want to know about any side effects they experience.

5 Who pays for the treatment during EAMS? During EAMS the medicine is provided free-of-charge to the NHS by the company who makes it. The NHS pays for other aspects of clinical care, such as tests and clinic appointments, just as they do at present. The NHS will begin to pay for the medicine after EAMS has closed and once NHS funding has been agreed in each country of the UK. What happens if/when the medicine gets a licence in Europe? The scientific opinion is based on the information supplied to the MHRA on the benefits and risks of a promising new medicine. As such this is a scientific opinion and should not be regarded as a medicine licensed by the MHRA or a future commitment by the MHRA to licence such a medicine. An EAMS scheme has to close to new patients if/when the medicine receives a licence from the EMA. People who are already enrolled in the scheme may continue taking the medicine provided free of charge by the manufacturer for as long as their doctor considers it to be beneficial until the NHS has made decisions about funding. Santhera Pharmaceuticals intends to make Raxone available through EAMS free of charge to local NHS services for a minimum of one year for each individual patient, if recommended by a doctor s prescription. Santhera may extend this period at its discretion, or shorten it if the NHS agrees to fund Raxone sooner, or if important new information emerges. How and when is NHS funding of a new medicine decided? These decisions are made by different organisations in the different countries of the UK according to their own evaluation processes: England (National Institute for Health and Care Excellence (NICE) and NHS England), Wales (NICE or All Wales Medicines Strategy Group (AWMSG) and NHS Wales), Scotland (Scottish Medicines Consortium (SMC) and NHS Scotland) and Northern Ireland (NICE and NHS Northern Ireland). It is not possible to predict if the NHS will fund a newly-licensed medicine in each country or when these funding decisions may be made. The evaluation processes usually involve clinical specialists and representatives from patient charities as well as the manufacturer of the medicine, and they may take many months to complete. 1-L V1-1 Date of preparation: June 2017