Access to Medical Treatments (Innovation) Bill

Lord Freyberg Excerpts

Friday 26th February 2016 Lords Chamber

[Lord Freyberg (CB)]

My Lords, I, too, am delighted to support the Bill. I speak today, as many noble Lords do, as one touched by the tragedy of cancer. As some noble Lords may know, my sister died two years of mesothelioma. Recent data from the Office for National Statistics show that 43% of cancers are less common cancers such as mesothelioma. While each is small, collectively they account for well over half of cancer deaths. There is insufficient commercial incentive globally for the current innovation processes to bring new hope to sufferers of these terrible diseases. We can see this in the rate of improvement in annual mortality ratios, which are the number of deaths linked to a cancer divided by the number of incident cancers. In the top five cancers, mortality ratios have improved 12% since 1999, from 49% to 37%. In the less common cancers the improvement was only 4%, and today’s mortality ratio is a shocking 56%, 19% worse than the 37% found in common cancers. That is three times less innovation in less common cancer.

So where have the modern precision drugs been launched? They have been launched for common cancer, of course. What is that? It is economics. At $75,000 per patient, cancer clinical trials are immensely expensive. It can cost billions to bring a drug to market. Where are rational economic agents going to place their research bets? They will do so in the big markets of common cancer, not the small, such as mesothelioma.

What is so unfortunate is that less common cancer has the same underlying genetic causes as common cancer. Modern precision drugs developed for common cancer often, but not always, work in these rarer diseases, but the costs of trials and the challenge of finding patients in rare disease mean that little development happens. That does not matter much in the US, the largest market in the world. There, a physician can prescribe off-label. There is no rationing watchdog like NICE breathing down his or her neck. US insurers will generally pay for the drug, with very limited data, such as a small case series in that cancer. As a result, the largest market in the world has extensive off-label use and does not require costly trials in rarer cancers. By implication, most of the economic incentive for formal label expansion vanishes. Pharma can get revenue without much R&D cost. Sadly, the research potential of this off-label use is not captured in the US, as its balkanised healthcare system does not capture the outcome data needed to close the loop and to discover what works for whom.

It is in this global context that we must evaluate this Bill. In particular, concerns about the Bill overriding UK due regulatory process ignore these economic realities. This is not the 19th century; there is no sea of pink on the maps. The regulatory and reimbursement process that matters globally for the pharma industry is the US, not the UK. We are a paltry 3% of the global market; it is 40%. Also, the US has a system that allows promiscuous off-label use. We must deal with the consequences of those global incentives and not be distracted by due process, as the noble Lord, Lord Ryder, stated.

Let us explore this global context further. As the noble Lord, Lord Saatchi, mentioned, US Vice-President Joe Biden is leading Obama’s $1 billion moonshot on cancer, after his son died of a rare brain cancer. A key part of that initiative looks set to be a database similar to that proposed by this Bill, implemented in willing but small US integrated care systems such as the Veterans Association. These databases have a name: stratified outcome registries. They are large-scale, real-world clinical databases, but upgraded for the 21st century with molecular diagnostic information, treatment outcomes and electronic consents. They capture not only what works for whom but, as importantly, what does not work. They close the learning loop.

I have consulted the supporters of the Bill and will be looking to see that it is appropriately amended—or not, depending on how we come to it in the time limit—to this effect in Committee. That is in line with the recent input from the Association of the British Pharmaceutical Industry on the importance of capturing both the good and the bad in such a database.

I also commend to the House the work that is being done by a small and innovative US not-for-profit, Cancer Commons, in helping to design such registries. In the US, it pioneers the application of these registries for optimising the use of existing drugs in common cancer and extending their use into less common cancer. It points out that such databases have a number of other benefits. They can discover which patients will not respond to costly treatment, saving those patients side-effects for no gain and saving the system money.

As an example, my sister had a molecular test that showed that cisplatin would not work for her cancer and would be more toxic for her than for most. As it is the NHS’s standard cancer treatment for the disease, she was given it. The toxicity occurred as predicted. She suffered horrendously and became ineligible for trial. The NHS faced significant costs in managing those unnecessary side-effects and complications. My sister might have lived a few months longer but for the side-effects.

Another benefit is that these databases have enough molecular information for scientists to hypothesise why a drug worked or did not work and so pump-prime basic research. Where we cannot explain the response, we can feed those exceptional patients to Genomics England, which will make that superb basic research engine more efficient. Finally, over time, the database would generate the information to derisk formal trials in less common disease. That will make such trials more economically attractive to industry and so change drug availability globally.

What may not be known to the House is that the UK is well placed to lead globally on developing these databases. In particular, we have a secret weapon in the National Cancer Intelligence Network registry in England. This covers 280,000 new cancer patients a year and today captures classical presentation, treatment and outcome data and small amounts of molecular data. The best US outcomes registry today covers fewer than 30,000 patients a year.

That volume of patients with clinical data gives us huge power globally. It is the largest in the world by a factor of 10, housed in the country that led the human genome initiative and with an enviable clinical research tradition. We should aim to use it to recraft the NHS to be the workshop for the world in precision oncology. This would win back a significant share of pharma’s global R&D spend, create a huge number of high-value precision medicine research jobs, and have a positive impact on investment flows into the UK. In particular, if we deployed low-cost generic panel-based cancer molecular diagnostics proactively into the NHS and ahead of approved drugs, pharma would supply late-stage research drugs in less common cancer. It would do this because the clinical data generated would both encourage off-label use in the US and give it future expansion options.

The evidence for this can be seen in France, where the Institut National du Cancer has been driving a national molecular pathology programme for over a decade. It is the preferred European partner for large pharma like Pfizer for late-stage clinical trials in precision oncology. That investment also created equality of access to modern molecular diagnostic technologies and prevented a testing postcode lottery. Today in the UK, we have an appalling postcode lottery in molecular testing, as recently highlighted by the Independent Cancer Taskforce and the NHS Atlas of Variation in Healthcare. We need the political will to change how we fund molecular diagnostics in the NHS to alter this. France drove the adoption of such tests by top-slicing the hospital care activity budgets to create a dedicated pool of money for a national testing programme and taking on entrenched vested interests to drive reform. We need to be similarly brave and creative if we are to achieve this here but it will save money and lives.

If we upgraded NCIN to being a stratified registry through the investment in a national molecular pathology service, I am confident that quickly some, but not all, UK patients would also live longer as a result of the increased access to innovative drugs. Over time we will also close some, but not all, of the yawning gap in mortality burden in the less common cancers, and we would have an intelligence system that could manage the risks of real-world innovation.

If such a system had existed when my sister had been ill, it might have helped her. Half of us will get cancer—it might well help you. In short, we can do well as a nation by doing good for the world. We can make the UK the preferred destination for the US drug companies for their clinical development, but only if we reshape the NHS to be a cradle for the sorts of innovation supported by the Bill, not a barrier. Let us use the global drug development system and its perverse incentives to our national advantage. Let us support the Bill and its database of innovation and work together on the many important details that may need ironing out in Committee.