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Who are the generic manufacturers?
We have identified a small number of companies that are manufacturing generic versions of cystic fibrosis drugs. One in particular has entered into negotiations with us around the potential price of their products. We have chosen to keep the name of company confidential initially.
How much will the medicines cost?
We have negotiated a sliding pricing scale - with the price dropping the more people who purchase through the buyer’s club, ranging from £18,000 to £23,000 per patient per year. Whilst significantly cheaper than Vertex’s price of £105,000, this is still very expensive.
We will therefore be calling on the NHS to financially support those who need ivacaftor-lumacaftor through personal health budget payments which allow patients to make their own decisions about what health interventions to purchase.
We also hope to negotiate further discounts as the buyer’s club grows, and more suppliers seek to supply through us. The price is likely to drop considerably if the NHS moves to buy the medicine for all NHS patients from a generic supplier.
How do we know the generic is safe and effective?
The generic manufacturer’s labs have been inspected by a number of stringent regulatory authorities including the FDA and a number of other regulatory authorities, and it has achieved the World Health Organisation’s certification for Good Manufacturing Practice. They already supply other medicines to the US and EU health systems.
In order to secure regulatory approval they have conducted bio-equivalence tests which prove that their product has the same biological effect in patients as the Vertex product. This is the methodology used to ascertain if any generic drug is the same as the original product. The vast majority of medicines used on the NHS are generic medicines.
Furthermore, we have worked with a leading London university and a London NHS Hospital Trust to test the generic product in their academic lab. This facility has extensive experience of undertaking pharmacokinetic testing, and they analysed the active pharmaceutical ingredients in the originator (Vertex) product and the generic product to ensure they have comparable levels of ivacaftor and lumacaftor. These independent test results confirm the bio-equivalence tests conducted by the generic manufacturer. The company is also collecting real world data collected from the 100 patients currently being treated using the generic product.
What CF drugs are available generically?
The supplier we are working with currently has ivacaftor for 6 years and older, and ivacaftor-lumacaftor for 12 years and older..
They are awaiting regulatory approval of the 6-11 years old dosage of ivacaftor-lumacaftor and expect to have that agreed in September.
The 2-5 year old formulation made by Vertex is dosed according to weight in the form of granules designed to make them easier to consume. The generic is not available in granule form. Some children with CF within this age bracket will already be taking other kinds of tablets, so taking the 6-11 tablet may be an option for some patients.
As with all decisions on treatment we advise you to speak to your clinical team.
They are currently developing a generic tezacaftor, enabling them to produce a generic Symkevi®, with a view to launching it in the first half of 2020. Additionally the company are in the process of developing a triple therapy.
How are they able to produce generic versions of these drugs?
Patents are granted at a national level. Vertex has not secured patents on their products in the country where the generic supplier is based so there is no legal barrier to them producing the medicines. Beyond this they undertook extensive scientific research and development to produce the active pharmaceutical ingredients in the medicines and testing to ensure their medicines are safe and efficacious.
Is it legal to purchase from them?
What follows is not legal advice. You may however find it useful in making an assessment about the legality of importing CF medications into your country.
UK law - like domestic laws in many countries around the world - includes a personal use exemption allowing patients to bring medicines into the country for personal use. In the UK this is understood as up to a three month supply and can be either brought in on your person when you are travelling into the country, or it can be bought from an overseas supplier and delivered to a patient or their family member in the mail.
This exemption from patent law has been declared by the UK with the World Intellectual Property Organisation (WIPO) which states that private, non-commercial uses of patented products is accepted. Guidance published by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) sets out how patients or family members can avail of the personal use importation of medicines exemption:
“We consider personal use to involve the use of the products by an individual or their immediate family or household; under such importation an individual must not sell or supply imported medicines onward as this would be considered placing the product onto the market and notification and licensing requirements would then apply.
“It is advisable to provide evidence to confirm the medicine is for personal use (prescription, doctor’s letter, or similar). Up to a 3 month supply of a medicine is considered to be an acceptable quantity for personal use (although there is no legally defined maximum). HM Revenue and Customs can prevent importation if large quantities are being imported and/or they have suspicions that the product is not being imported for personal use.
“Anyone posting packages containing medicines are advised to include a copy of the prescription and/or a letter from the patient’s doctor explaining why the product(s) are required; the package should be clearly labelled on the outside stating the contents of the package and that the products are for personal use. Medicines should be kept in their original packaging and should be transported in accordance with storage conditions specified by the Manufacturer (this not only helps identify the medicines, but also helps ensure the product’s stability).
“Medicines that contain drugs controlled under the Misuse of Drugs regulations may additionally require a Home Office licence.”
Furthermore, there is no need to inform them of your intention to pursue this avenue.
This is still very expensive - how can I cover the cost?
We realise that for most people, despite being much cheaper than the Vertex price, the cost of the generic treatment is still an unaffordable amount of money. Nonetheless, for some people this may be an option so we felt obliged to share information on the generic’s availability in the knowledge that some patients could benefit.
We are taking a number of steps to make this something all patients can benefit from:
Demanding our government make it available to all patients - It is a disgrace that patients and parents are having to resort to setting up a buyer’s club to get access to treatment. We are therefore calling on the government to step in to use its power to solve this problem. There are two ways we think they should act - firstly, they should issue a Crown use licence allowing them to buy the generic version of Orkambi. Secondly, they should explore setting up a large scale clinical trial to investigate the wider impacts of Orkambi on patients and their carers - this would also allow the government to use a generic product, through the research exemption in UK law. Please write to your MP asking them to call on the government to take this step at the debate in parliament on the 10th June.
Push for the NHS to help cover the costs of buying the generic with personal health budget payments - The NHS has been rolling out personal health budgets for some patients. We believe that they should allocate resources to every patient that needs Orkambi. The patient or their family can then choose how to spend that money to meet their care needs, including covering the cost of buying the generic via this buyer’s club. We are making the case for this to the NHS.
Undertake fundraising to help cover the cost - While it is very far from an ideal way to cover health costs, people have been successful in fundraising to pay for medical bills. By using crowdfunding sites justgiving.com or gofundme.com you could generate funds to help cover the costs. There are tips on how to do this effectively here: https://www.gofundme.com/c/blog/medical-crowdfunding-guide and we will be running a fundraising webinar to help people who are thinking about this option to develop a strong fundraising plan.
How do I get a prescription and support from my NHS clinical team?
Patients will need to secure the support of their clinical team to monitor their response to the treatment. A number of NHS CF clinical teams have already undertaken to provide this support to members of our community using the generic version of the treatment. There is strong precedent for this within the NHS including the use of generic medicines purchased through buyer’s clubs for hepatitis C and PrEP for HIV. If you have any issues with your clinical team refusing to support use of the generic, please contact us - we can provide further information on quality and safety to help answer any questions they have.
In order to utilise the personal use exemption facilitating the import of the generic medicines you should get a prescription. Your NHS team may provide this, but you may also have to secure a private prescription. We are working to find prescribers who are willing to do this for multiple patients. If you are having difficulties getting a prescription please let us know.