This information is provided in response to a series of questions shared jointly with Roche in writing by UK patient organisations Muscular Dystrophy UK (MDUK), TreatSMA and SMA UK. Unless otherwise stated this information applies to people living across the UK.
1. What is an Early Access to Medicines Scheme (EAMS)?
The early access to medicines scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.
Under the scheme, the Medicines and Healthcare products Regulatory Agency (MHRA) will give a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made.
More information is available on the gov.uk website:
2. Who with SMA can access this scheme? What are the eligibility criteria?
This EAMS is available to people with type 1 and type 2 Spinal Muscular Atrophy (SMA) aged 2 months and older who are not suitable for authorised treatments.
A patient’s eligibility within these criteria is to be determined by their treating physician. If the treating physician is located in England they will also have to complete a Blueteq form provided by NHS England, which may contain additional eligibility criteria.
Further information about this EAMS is available on the gov.uk website:
3. How does this EAMS affect Roche’s existing compassionate use programme in the UK?
EAMS is overseen by the MHRA, who have indicated that EAMS is the preferred method of access to medicines in a pre-license period. Therefore Roche has stopped accepting compassionate use requests for new patients, except in unforeseen and exceptional circumstances.
A very small number of compassionate use applications have been received by Roche and are currently within the evaluation process. Roche will discuss the option of transitioning these few applications to EAMS directly with the individual treating physician.
Patients who are currently enrolled in compassionate use will continue to receive risdiplam provision. Roche will discuss the option of transitioning currently enrolled patients to EAMS with each individual treating physician. Transition from compassionate use to EAMS is not mandatory.
4. Who pays for the actual drug risdiplam via the EAMS? (Is it free to the NHS and patients?)
Risdiplam is supplied free of charge to the NHS for the duration of this EAMS. Roche will also provide oral syringes for administration and cover the cost of homecare for the duration of this EAMS.
5. Will I be eligible for risdiplam under this scheme?
A patient’s eligibility within the EAMS criteria is to be determined by their treating physician. Risdiplam is an unlicensed medicine. All licensed therapies should be considered before an application is made for access to risdiplam via this EAMS.
Treatment with risdiplam should be initiated by a physician with experience in the management of spinal muscular atrophy. The treating physician is required to determine patient eligibility, to make the application to Roche and to oversee the treatment of risdiplam.
If the patient is located in England, the treating physician must be employed by a specialist neuromuscular centre that has been approved for risdiplam provision by NHS England. Your treating physician can obtain this list of approved centres from NHS England. In England they will also have to complete a Blueteq form provided by NHS England, which may contain additional eligibility criteria.
Further information about this EAMS is available on the gov.uk website:
6. What happens once my treating physician has indicated that authorised treatments are not suitable for me, and I would be eligible for risdiplam?
A patient’s treating physician will provide information about risdiplam, including; the treatment protocol for patients, informed consent form, patient alert card, and instructions for use. Patients will be required to read these materials, complete and return the informed consent form to their treating physician. After receiving the completed form the treating physician can submit an application to Roche for risdiplam EAMS provision. The physician is responsible for obtaining internal trust or pharmacy approval if required, and if located in England, completion of a Blueteq form.
If this is the first application from the treating physician, Roche will organise:
- – An agreement between Roche and the treating institution
- – An agreement between the homecare provider and the treating institution
- – Safety training for the treating physician
These agreements and training typically take a few weeks to organise and must be completed prior to risdiplam drug provision.
7. How often, how and where would I take risdiplam treatment?
Risdiplam is supplied as a liquid with a prescribed does based on a patient’s weight. Risdiplam should be taken once daily after a meal, at approximately the same time each day by mouth (orally) or feeding tube, using the provided syringe. Risdiplam will be provided in a box that should be kept in a fridge at a temperature between 2C and 8C.
It may be possible to have risdiplam delivered to a patient’s home by a homecare provider, who will deliver risdiplam as a liquid. The alternative is that risdiplam will be provided to the treating institution as a powder, that is constituted with purified water by the pharmacy. As detailed in Q6, an agreement is required between the homecare provider and a treating institution in both scenarios.
Risdiplam will be supplied via a homecare pharmacy or by the treating institution. If risdiplam is supplied by a treating institution, a doctor will:
- provide sufficient risdiplam to take at home/outside of hospital setting
- provide instructions on how bottles can be transported home
- provide instructions on how to store and take the medication
- supply oral syringes for medicine administration
If risdiplam is supplied by a homecare pharmacy, they will:
- deliver sufficient risdiplam to the patient’s home or a previously agreed address
- provide instructions on how to store and take the medication
- supply oral syringes for medicine administration
Where and how a patient receives treatment is ultimately the responsibility of the clinician and should be discussed with them.
8. Once I start risdiplam under the EAMS, what monitoring, guidance and support will I receive and from whom?
The treating physician will provide information about risdiplam, including; the treatment protocol for patients, informed consent form, patient alert card, and instructions for use. Information about home delivery will be provided by the homecare provider and Roche.
Roche is required to monitor the safety of risdiplam throughout the duration of the EAMS. Therefore Roche requires patients to report any side effects to their clinician, who must report these to Roche. The clinician will discuss what to report with the patient. Any other monitoring is ultimately the responsibility of the treating physician and something that should be discussed with them.
9. What are the possible negative side effects I and my clinician must look for and report?
Like all medicines, this medicine can cause side effects, although not everybody gets them.
The below side effects have been seen in Type 1 SMA patients taking risdiplam:
Very common: may affect more than 1 in 10 people
- upper respiratory tract infection
- pyrexia
- pneumonia
- constipation
- nasopharyngitis
- rhinitis
The below side effects have been seen in Type 2 SMA patients taking risdiplam:
Very common: may affect more than 1 in 10 people
- diarrhoea
- rash
Common: may affect more than 1 in 100 people
- arthralgia
Reporting of side effects: If any side effects are observed, a patient should talk to their doctor or pharmacist or nurse. This includes any possible side effects not listed here.
Side effects should also be reported directly via the Yellow Card Scheme at: www.mhra.gov.uk/yellowcard or call freephone 0800 731 6789 (10am to 2pm Monday-Friday only) or Roche can be contacted on telephone 01707 367554 (24 hours).
The doctor or pharmacist will complete an EAMS adverse event reporting form. Reporting side effects helps to provide more information on the safety of this medicine.
10. How long is the EAMS going to last?
Risdiplam will be provided free of charge for all patients enrolled onto the EAMS until marketing authorisation (until the medicine receives a licence from the European Medicines Agency (EMA) or the MHRA). Following marketing authorisation of risdiplam, the EAMS scheme will close in line with the MHRA regulations and no new patients will be allowed to enroll onto the scheme. The EMA accepted Roche’s risdiplam filing in August 2020.
Free of charge supply will continue for patients who are already enrolled in the EAMS at the point of marketing authorisation.
If the EMA or MHRA does license risdiplam and the UK reimbursement authorities recommend that the NHS funds access to risdiplam for people with SMA, supply of treatment will continue for as long as a patient receives clinical benefit or within the recommendations made by the relevant UK reimbursement authority.
If EMA or MHRA does license risdiplam but the UK reimbursement authorities don’t recommend the NHS funds access to risdiplam, or recommends funding for a subgroup of people with SMA, Roche would continue to supply treatment for people for whom risdiplam is not routinely available for at least five years from first treatment while the treating physician deems a patient to be receiving clinical benefit. Following five years, further drug provision shall be discussed with relevant NHS stakeholders and your treating physician. New findings in regard to the benefit & risk assessment could lead to termination of risdiplam provision.
11. Is there anything else you think I need to know about the EAMS, risdiplam and what is happening next with regulatory approval?
Further information about this EAMS is available on the gov.uk website:
12. I have Type 3 SMA – can I access risdiplam privately?
Risdiplam has been submitted to the EMA for marketing authorisation, however it is currently an unlicensed product and therefore cannot be bought in the UK at this time.
