The Government has announced the intention to fast-track breakthrough drugs to seriously ill patients under a new Early Access Scheme, as promised in the 2011 Life Sciences Strategy. It is aimed at innovative drugs for serious illnesses for which there is no existing treatment or where the current medicines are inadequate – like dementia. The idea is that once promising drugs have been proven to be safe and show signs of efficacy or are effective in certain parts of the population they can be prescribed, contingent upon patient consent and doctor approval. This could allow drugs to be given to patients years earlier than currently happens.
We must not lose safeguards
The impact for patients could be significant if this is implemented properly. But we must interject a note of caution, as the Faculty of Public Health have in response to this announcement. There will be concerns, driven not least by high profile historical scandals of drugs such as thalidomide. Even though the reasons for these catastrophic failures may not have been the result of this specific policy, the idea that drugs are given to people before they have been through the commonly recognised development process will raise concerns. The big pharmaceutical companies have previously faced flack for not reporting negative outcomes of new drugs, and only reporting the positive outcomes in trials. It’s fantastic to see a number of pharmaceutical companies have signed up to alltrials.org, but the public must be confident safeguards are in place and crucially individuals, or their families, are given the right information to weigh up potential risks. Of course this can be a specific problem for people with late stage dementia, because they often cannot make an informed decision.
More can be done to encourage drug development for dementia
At Alzheimer’s Research UK we’ve been asking for some time that the major regulatory authorities create new avenues to encourage industry investment in potential treatments for dementia. At the moment it takes so long to develop drugs for dementia that there is often little sense in drugs companies investing because the licence they have to sell the drug exclusively is likely to finish before they have had time to recoup the huge development costs. It also means smaller pharmaceutical companies, who don’t have the huge budgets of big operators, simply cannot afford to develop many promising treatment candidates. This means that people with dementia are currently getting a raw deal, and is part of the reason that we have no effective disease modifying therapy for the condition.
We would like to see further action
Fast-tracking is potentially good news, but we would like to see more action on regulation.
Fast-tracking is potentially good news, but we would like to see more action on regulation. This includes allowing conditional licensing, contingent upon phase 4 trial data (when a drug is tested properly and has been proven to be effective), and increased data exclusivity periods as currently afforded for so-called “orphan drugs” (for rare diseases that affect a small number of the population). Although not a rare disease, future dementia treatments would benefit from rules similar to orphan status, not to line the pockets of the pharmaceutical industry , but to encourage drugs companies to be more willing to attempt to develop promising treatments in the first place.