Wednesday, September 19, 2012
On Wednesday the 12th, we attended a panel debate regarding the lack of drug development for patients with rare or life threatening diseases. The debate was driven by a campaign named ‘Empower: Access to Medicine’ and it is based around the aim to speed up the development and availability of drugs that treat these diseases and conditions.
The campaign was founded by Les Halpin, a statistician that has been diagnosed with Motor Neurone Disease. He has committed himself to a personal legacy of accelerating drug approval and licensing for those with rare and life-threatening illnesses.
The current research and development model for drug development has been termed ‘broken’. Simply, there are too many phases involved in getting a drug approved. Currently, there are six stages within approving a drug, with three of these being clinical trials known as ‘phases’. Empower wishes to get rid of ‘Phase 3’. Phase 3 is the third round of Clinical Trials, after the drug has been trialled for safety and then efficacy in Phase 1 and 2.
A recent report by the Office for Health Economics found it takes five years on average, after launch, for a new drug to win NICE approval. This timescale can be more than doubled when added to the time taken for a new drug to go from development stage through to Phase 3 and beyond.
The current drug development model is expensive and uneconomic. Three phases of clinical trials are neither cost of time effective, meaning pharmaceutical companies do not have a financial incentive to invest in development of new drugs for rare or ‘orphan’ diseases. This is due to the perceived small number of the population who are affected by such diseases, meaning companies won’t get return in investment.
One way to encourage pharmaceutical companies is to show them that the drugs they develop could be applied to a wider number of conditions, combined with a simpler testing regime.
Empower wishes to abolish Phase 3 of clinical trials. Safety and Efficacy (Phase 1 and Phase 2) are necessary in establishing if the drug is fit to unveil to a larger market, but Phase 3 is arguably ‘more of the same thing’. The proposal is that after Phase 2, patients could sign a legally binding indemnity against ill effects. Unfortunately, this currently conflicts with the Strict Liability Provisions of the Consumer Protection Act 1987. The industry sees how ‘ridiculous’ this is, as medicine is one of the only consumer products still under jurisdiction by this law. Therefore, for this campaign to work, the law must be changed.
Further to this, Empower wishes to abolish the ‘no win, no fee’ arrangements. This is because it directly discourages medical professionals and pharmaceutical companies getting involved in drugs that could have side effects; that have been un-tested or un-developed previously. The argument is that if patients sign a legally binding contract after Phase 2 they cannot sue in the future.
Geoffrey Clifton-Brown MP has put forward for a debate in parliament when it resumes in autumn. Subject to approval of the debate, he will argue in favour of this campaign, putting forward the points raised. This hopes to get the campaign moving forward, and possible laws abolished. Without government sign off, the campaign cannot go ahead, as all governing bodies (such as NICE) are answerable to the government.
An opinion of the panellists, and one shared by us, is that patients need to be able to make the decisions themselves, or at least part of it. It is argued that a patient can be made fully aware of the risks and understand them, therefore they should not be stopped from voicing their own opinion regarding new drug development and involvement in clinical trials. Part of the campaign is to dictate less what a patient can decide for themselves, allowing more patient involvement in drug development.
Hopefully this campaign will pave the way to a future which means easier and cheaper drug development, with more access for rare disease patients such as those with AKU.