Your understanding is my understanding Hyperopia. They are not licensed, and often coming from overseas. The idea is to allow patients to access drugs from overseas, if they are not yet in the U.K. but their doctor believes they will address an acute need. They actually are not licensed so licensing price I believe is beyond the scope of the exception.
If it were a license to market the product, which they can’t do, THEN pricing is relevant. But these are drugs from elsewhere that are not licensed, that a patient’s doctor has determined that they need. I do not believe there is really any particular focus on the price since it is paid for OUT OF POCKET, and is not funded by the National Healthcare system in the U.K.
I think people are mixing up programs. Compassionate Use programs and the specials program are different though both are both often referred to as compassionate use. The strict compassionate use programs are more like individually licensed drugs for particular patients, they legally are fundamentally different and THOSE programs often require domestic manufacturing (e.g. the German program) and they frequently set guidelines on profit and they often forbid profiting. Supposed patient advocates have worked to forbid tang profit from an experimental drug (ethical concerns) but I would guess that that is big pharma also, tricking folks into believing that it is the most ethical thing, undermining small and innovative companies in the process, just like they trick people into believing other kinds of programs won’t suit their interests and are “bad”. The ethical argument has merit, but it undercuts the entire concept. A compromise would make it more sustainable and possible for these patients with life threatening conditions that are desperate to get access to the latest science. There should be a better way to provide it. The UK specials program actually seems to work.
But yes, there is a difference between the true compassionate use programs and the U.K. specials program. NWBO with it’s designation as an innovative drug COULD have gone the traditional compassionate use route in the UK, but from what I see of the big pharma who have gone that route, they do it temporarily for the PR bump, and then do not maintain the designation because it’s just not financially sustainable to go the regular compassionate use route.
Also, there are some products that need to be assembled by pharmacists who are not truly manufacturing anything, so there may be some limits there.
And as for manufacturing some products, like DCVax, they have to meet specific GMP requirements.
In the UK ATMPs (Advanced Therapy Medical Products) may be manufactured under a UK ‘Specials’.
It requires a bona fide, unsolicited request from the patient’s surgeon, typically in the form of a prescription, I before they can commence the manufacturing process. The legal responsibility for use of the ATMP in this case rests with the surgeon.
ATMPs manufactured as ‘Specials’ must meet the expectation of UK laws, as we all know and are waiting for the GMP certification.
