Friday 28 March 2014

The Medical Innovation Bill

What happens when you reach the end of the road with the standard cancer protocols? A patient starts with first-line treatments, usually a combination of chemotherapy, surgery and radiotherapy, depending on diagnosis and disease staging. If the response to that first-line isn’t positive, the disease continues to spread, then there’s a second-line and possibly a third-line treatment, depending on the type of cancer and how the patient responds to these often toxic treatments. And if the disease is still there at the end of this gruelling process? At this point the prognosis is pretty grim. What happens then?

At this point I should say that I’m speaking from direct personal experience with my son, George, who died almost three years ago from metastatic osteosarcoma that resisted every treatment thrown at it. And from the experience gained in the years following his death in trying to help other people in a similar situation, often also suffering from Li Fraumeni Syndrome as George did.

What happens? In the first instance the lead oncologist will often look for a clinical trial, hopefully a Phase II or Phase III trial. But often the patient will have already been on a trial and come out the other side with the disease still active. In practice if there are trials available, and often they are not, it will be a Phase I trial. The aim of a Phase I trial is to find the right dose to use in a Phase II trial, which is looking to see how well a drug works. Efficacy – how well the drug works – isn’t normally the primary outcome from a Phase I trial.

And if there’s no trial, or you don’t want to go on a Phase I trial with a new drug that might have toxic side effects and no effect on your disease? Then you are in the realm of palliative care. If there’s no standard treatment and there’s trial, then the emphasis is on trying to keep a lid on the symptoms and trying to preserve what quality of life you have given the advanced state of the disease.

But what if there’s something that doctors are using in another country? What if there’s a drug that has been shown to have some effect in your type of disease? What if there are other options that aren’t covered by the standard protocols?

This was the situation we found ourselves in with George. But the problem was that the oncologist in charge of the case at the time felt constrained. We found a protocol that had been tried in Germany, using well-known low-cost drugs in combination with low-dose oral chemotherapy. I even got the doctor from Germany who had used these drugs to write and offer to help with George’s case. But it didn’t happen. In the end we were offered a different protocol, one that the hospital had tried before and therefore it was considered OK. This wasn’t what we had asked for, had lower evidence in its favour and had been abandoned by the people who had originally tried it (in the US).

Now, being realistic it’s unlikely that the protocol we had discovered would have saved George’s life. But the protocol we were given was poor, and caused considerable side-effects which led it to be abandoned. I think it was a poor clinical decision, but the fact is that the institutional mind-set meant that the oncologist was constrained and limited in the options available.

The Medical Innovation Bill, also called the Saatchi Bill, is a proposed change in the law that will free doctors from that kind of mind-set that stops them from trying things outside of the standard protocols or clinical trials. It’s not about giving free reign or abandoning science or the need for evidence. It’s about recognising that each patient deserves a chance to have what’s best for him or her, even if it’s not part of a standard protocol.

There are many innovative treatment options that involve repurposed non-cancer drugs, for example the anti-diabetic drug metformin or the anti-histamine cimetidine or the anti-inflammatory drug celecoxib. These generally have low toxicity and can be combined with existing chemotherapy drugs. Or there are new metronomic protocols that use established chemotherapy drugs, such as oral cyclophosphamide or capecitabine, in low doses but with no treatment breaks. Often these kinds of treatments have to be adapted and personalised and tweaked, which requires a clinician to closely monitor and adapt treatment in a way that a standard protocol doesn’t. These kinds of treatments are used in other countries where doctors have greater freedom to try out innovative approaches in patients for whom standard treatment options are no longer available.

When we have tried to get these adopted for patients here we have met with resistance or a flat out refusal to consider them, even in cases where we have oncologists from abroad offering to help – just as we did with George’s case.

Based on our experience the issue isn’t so much to do with a fear of litigation, but an institutional mind-set that does not encourage creative approaches, collaboration with doctors from other countries and which relies on the clinical trial for innovation. In particular moving patients to Phase I clinical trials is not a good substitute for trying adaptive and metronomic protocols while the patients are in good physical condition. It does those patients a grave disservice as Phase I trials have a greater risk of adverse events and a lower chance of clinical benefit compared to the low-dose and low-toxicity approaches that are available.

Therefore, I strongly support this Bill in the hope that it can encourage oncologists and other clinicians to become more creative in their responses to treatment failure. There is currently a public consultation, so if you’ve been affected by this, if you have a view, then please take the opportunity to make your voice heard.

The website for the consultation is here:

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