Orkambi rationed for Cystic Fibrosis

It is expensive to develop new drugs. Britain and the UK has a 12 year patent rule, after which the drugs can be made competitively in a “generic” form by any company that can master the production. Development is expensive and a long term risk for companies and investors. Many products have been refused funding by the health services of the UK, until their patent is about to expire, or has expired. Other countries have similar rules. This all means that the payback for investment must be covered in 12 years…….

If governments want to share in the pricing decision, then they need to share in the risk of the research. And of course this will include many failures. 12 years seems a reasonable time frame for payback, until you or your next of kin is the potential beneficiary, and the treatment is effectively rationed. There are many new drugs to come, especially genetically engineered ones, but risky investment will cease if the payback time is reduced. It is a great shame that patients with cystic fibrosis cannot be funded for a new drug. It is, however, reasonable rationing, or tough love. But those who need it in 12 years time can be hopeful that it will be affordable then.

Cystic fibrosis: Company urged to lower cost of life-changing drug – BBC News 21st April 2018

Health ministers have urged a pharmaceutical company to drop the price of a life-changing cystic fibrosis (CF) drug for NHS patients.

Vertex Pharmaceuticals’ Orkambi costs £100,000 a year per patient, and has been deemed too expensive for the NHS.

The company rejected an NHS England counter offer, saying it was not enough to fund research into future medicines…..



This entry was posted in A Personal View, Rationing, Stories in the Media on by .

About Roger Burns - retired GP

I am a retired GP and medical educator. I have supported patient participation throughout my career, and my practice, St Thomas; Surgery, has had a longstanding and active Patient Participation Group (PPG). I support the idea of Community Health Councils, although I feel they should be funded at arms length from government. I have taught GP trainees for 30 years, and been a Programme Director for GP training in Pembrokeshire 20 years. I served on the Pembrokeshire LHG and LHB for a total of 10 years. I completed an MBA in 1996, and I along with most others, never had an exit interview from any job in the NHS! I completed an MBA in 1996, and was a runner up for the Adam Smith prize for economy and efficiency in government in that year. This was owing to a suggestion (St Thomas' Mutual) that practices had incentives for saving by being allowed to buy rationed out services in the following year.

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