Thursday, 10 April 2014

Mesothelioma - making sense of recent developments

April 2014 is shaping up to be a significant month for the mesothelioma community and other people with rare and life threatening diseases.  

With announcements about new regulations, draft legislation, new schemes and consultations coming thick and fast, it's easy to get confused or overlook something.  I thought this would be a good time - between Steve's end of trial scan and hearing the results next week - to take stock of all these changes, join the dots and look at the bigger picture.  

So here goes....

Clinical Trials Regulation in Europe
For mesothelioma and other diseases for which there is currently no cure, the way forward has to be through research and clinical trials.  The problem at the moment is that there is no legal requirement to register clinical trials or publish results.  

On 2 April, the European Parliament voted to adopt a new Clinical Trials Regulation.  This will require, amongst other things,
  • All new trials to be registered on a publicly accessible EU clinical trials register before they begin
  • A summary of the results to be published within a year of the trial's completion, including a summary understandable to non-medical people
  • Clinical Study Reports to be publicly available 
There are often not enough patients in one country alone to develop new or improved treatments for rare diseases. The new regulation will make it easier to conduct cross-border, multinational trials within the EU by cutting red tape and simplifying the current rules by having:
  • One straightforward authorisation procedure for all EU Member States, resulting in one single assessment outcome
  • Simplified reporting procedures, with one report for all Member States
  • Applying the same rules about protection of subjects to trials performed outside the EU where they are to be applied to trials within Member States
  • A lighter touch regulation for "low intervention trials", for example trials comparing medicines which are already authorised  
The new regulation will also strengthen transparency and, in the process, avoid the redundancy and duplication which can happen when trials are not conducted and reported on in the public domain.  

As far as I can see, this is all good stuff.  There are only two downsides.  Firstly, although approved, the regulation won't come into effect until mid-2016, when the EU portal and database (currently under development) ought to be ready.  

Secondly, it is only concerned with new trials. It does not address the existing problem that we do not have full reporting on all trials for the medicines currently being used. Evidence is routinely and legally withheld, making it difficult to make an informed decision abut which treatment is best for a particular individual.  This harms not only patients but the pharmaceutical industry's reputation.  Shame on you!  

To find out more about All Trials which has campaigned for this change CLICK HERE . There are other useful links on this page.

The Early Access to Medicines Scheme (EAMS) 

This UK government scheme came into effect on 7 April. 

EAMS will see doctors working with patients to make innovative and promising drugs available as soon the Medicines and Healthcare Products Regulatory Agency - the UK’s regulator - has signalled that the benefits outweigh the risks following an initial scientific assessment.  Severely ill patients with life-threatening and seriously debilitating conditions will be offered the lifeline of trying ground-breaking new medicines years before they would normally reach them.

The scheme will be funded by pharmaceutical companies which develop innovative treatments, meaning patients will benefit from world-class breakthroughs at no cost to the NHS. Once drug manufacturers have received a scientific opinion from the Medicines and Healthcare products Regulatory Agency, doctors will work with patients to prescribe the drugs when appropriate.
In return, the companies will be able to gain experience of their medicines being used in the NHS and work closely with regulators to look at the value of the drugs, gaining guidance and advice much earlier in the regulatory process.
As a result, the process of patient access will be speeded up and new drugs could be made available to patients months or sometimes years before the treatment is licensed.
The scheme involves a 2 stage approach:

  • Stage 1: Promising Innovative Medicines (PIM) Designation - Based on early clinical data (for example, a Phase 2 trial) where a drug shows early promise, the maker can apply for a PIM.  This can happen several years before the full clinical trial is complete and the drug licensed
  • Stage 2: Early Access to Medicines Scientific Opinion - This will describe the benefits and risks of the medicine and support doctors make a decision as to whether the drug might be suitable to help a patient with a life threatening disease like mesothelioma, for whom there are no other treatment options
Full details of EAMs are now on the website of the Medicines and Healthcare products Regulation Agency, the body responsible for regulating the scheme.  You can read about it in more detail by CLICKING HERE .  The scheme is now up and running, and the deadline for submitting the first applications for a Scientific Opinion is 5 May.  

As far as we are concerned, this is wonderful news.  There have been a number of promising clinical trials involving immunotherapy drugs which Steve is unable to take part in because his medical history of autoimmune disease (arthritis) which might skew the drug trial results.  However, if one of more of these drugs achieved a PIM designation and the Scientific Option indicated that in his case, the benefits would outweigh the risk, that would open up treatment options for him in future, which can only be a good thing!

The Medical Innovation Bill aka Saatchi Bill
One of the things which holds back the medical profession from taking risks associated with new, innovative treatments is fear of litigation if it all goes horribly wrong.  This is something that the Medical Innovation Bill seeks to address, while still protecting the patient's interests.  

The law as it currently stands dictates that patients are only offered standard treatments.  However innovation departs from standard treatment.  The Bill supports doctors who endeavour to act in the best interests of the patient without fear of litigation.  

It deters irresponsible experimentation but encourages a much needed attitude change in favour of innovation in the provision of patent care. 

The Health Minister has said the Government will support the draft Bill but ONLY if it has public support.  You can read more about the Bill and add your support by CLICKING HERE!  The consultation closes on 25 April.  

Please, please, please support this Bill.  It's an important piece of the jigsaw.  

The EAMS scheme described above will only work to patients' benefit if the medical profession is bold enough to take advantage of it and prescribe promising, but unlicensed, drugs to patients who need a lifeline, having exhausted all other treatment options.  Doctors are far more likely to be prepared to make such decisions if the fear of litigation is removed.  

The Bill proposed by Maurice Saatchi (who lost his wife to cancer) would give the medical profession the reassurance it needs that innovation won't lead to litigation if decisions are made with care, the best interests of the patient at heart, and based on the best information available.  

Moreover, it will cover innovative medicine of all types - not just drugs, but also surgery and other medical procedures, and therefore benefit a wide range of patients who might otherwise die before today's innovation becomes tomorrow's gold star treatment or cure.

Mesothelioma Priority Setting Partnership (PSP)
With avenues for research, clinical trials and innovative treatment opening up as a result of the developments described above, it is of the utmost importance that patients, their families and carers have an input into setting priorities for research into the disease.  The Mesothelioma PSP has been consulting widely with this group and with the healthcare professionals who treat them.  

The consultation is due to close 30 April!  If you fall into this group and have yet to give your views on what research would make a difference to you and what researchers should be working on, then please go the the PSP website and complete the questionnaire before it's too late.  This is your chance.  Use it!  

To access the survey, please CLICK HERE!  

So what does it mean to us?
If you managed to get this far, well done you - and thank you!  

As I've been writing this, it's helped me gather my thoughts on our situation....

Steve had his end-of-trial scan on Tuesday.  Next Monday, we will find out the results of the scan and whether taking the combination of vandetanib and selumetanib in the VanSel Early Phase clinical trial has stopped his cancer growing or spreading.  If the side effects of treatment are anything to go by, something must be happening, but whether it's happening to the cancer cells as well as normal healthy cells we will have to wait until Monday to find out.

We are both aware that these drugs are primarily aimed at people with non-small cell lung cancer.  It remains to be seen whether they act the same way on mesothelioma.  However, if you don't test, you will never know.   So if it doesn't work out, at least we will not be plagued by thoughts of "what if...."

When Steve started the drug trial back in early February, we had no idea that the developments summarised above were even a twinkle in someone's eye.  The fact that they have reached these various stages by the end of the drug trial brings me hope that Steve will have other treatment options in the not too distant future, if the Vansel combination doesn't work for him.  Those of you in a similar position will know how good that feels.  

Take away treatment options, and you are likely to take away hope - at least until a cure is found.  And it will be found, one day.  

A huge and heartfelt hank you to all those who are working on it! 

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