Fibrotic diseases, which involve the harmful deposition of excessive amounts of connective tissue within organs such as the lungs or the heart, can be seen as a perversion of healthy scarring. The difference is that while the latter repairs the body after damage has taken place conditions like, for instance, cystic fibrosis, Crohn’s disease and liver cirrhosis have destructive effects that can ultimately kill. The various mechanisms involved are not as yet fully understood but are linked to abnormal inflammatory, immune and allied responses to external challenges, coupled with genetic vulnerabilities.
Pulmonary fibrosis (PF, or ‘lung scarring’) is a relative rare fibrotic illness. It is frequently of unknown origin, albeit that some cases are linked to factors like, for instance, the use of medicines such as methotrexate and certain antibiotics, or the occurrence of ‘background’ diseases like heart failure or chronic bronchitis. As it develops PF causes constant coughing and increasingly severe breathing problems. Ultimately – typically after considerable physical and mental distress – it results in death.
Commonly quoted figures on its incidence could well be under-estimates, in that milder PF cases may pass undiagnosed. This might in part be because of the limited treatment available. Until today the main therapeutic choice has – short of lung transplants – been to provide interventions such as giving oxygen and anti-inflammatory and immuno-suppressant medicines. Doctors have had little reason to find early cases, and may have feared counter-productively labelling those they suspected of beginning to develop PF.
However, it appears that the incidence of PF in countries such as the US and those of Western Europe is currently approaching 10 per 100,000 population, and that the prevalence is 30-40 per 100,000. This implies an average survival time from diagnosis of around three years, less than that for many cancers.
Pulmonary fibrosis is an example of a rare disease as defined in the US Orphan Drugs Act and similar legislation across the world, albeit that at the global level there are in total over 2 million people living with it at any one time. The goal of orphan drug legislation is to incentivise private (and linked public) investment into rarer disease research and treatment development. Yet low volume sale, high science based, therapies are inevitably relatively expensive when they are first marketed. Many health care funders/providers tend to be unwilling to pay the prices asked for by innovators during the ten to fifteen year periods in which intellectual property rights apply.
In the last year two new medicines, nintedanib and pirfenidone, have become available for the treatment of idiopathic (unknown origin) pulmonary fibrosis. Their full potential is as yet unknown. But there is evidence that both drugs (which have differing modes of action) can slow the progression of the disease and may in time, notwithstanding side-effect risks, prove to be of value in other contexts. These include not only – perhaps – early stage or ‘pre’ PF treatment but also modifying other fibrotic disease processes.
Critics of what are at the time of launch highly priced and potentially health service cost increasing pharmaceutical innovations may argue that drugs such as nintedinab are not worth the money currently being asked for them. This may be because the projected ‘cost per incremental QALY’ is above the level assumed to be affordable. Less overtly, it may also be because of fears that they will extend the survival of seriously disabled individuals and hence prove costly in overall care provision terms.
Such negative judgements threaten to undermine the moral purpose of orphan drug legislation. They may also be ill-founded in that despite all the drug advances made since the 1940s pharmaceutical costs have in recent decades stayed reasonably constant as a proportion of health spending. As new medicines are introduced older ones become low cost generics.
Narrow Health Technology Assessments (HTA) fail to reflect the long term value of bio-scientific innovations which over time will lead to fully effective, low cost, treatments for savage diseases like pulmonary fibrosis. Despite the ‘austerity’ pressures on health care funders and the current unpopularity of pharmaceutical companies that are seeking premium returns for what may seem to be treatments of merely marginal additional value, the marketing of products such as pirfenidone and nintedanib for me represent profoundly important stepping stones to a better world.
Health care organisations have to work within their budgets. This means making hard choices. But at the same time reductionist HTA calculations are too simplistic to reveal the true worth of advances which are individually limited but nevertheless important parts of humanity’s continuing progress towards understanding biology in its entirety. In the long term biomedical progress is leading not only to technologies for preventing or curing ill-health but to wider gains that will contribute to both individual and collective survival and well-being in the coming century.
My fear is that desires for short term savings and the political pursuit of the immediate approval of the greatest number of voters will pervert public priorities, and in the coming decade drown this message out in settings like Europe. This could lead to much needless suffering. But my hope is that people and organisations of good-will, from patient and carer groups to health professionals, will be able to understand the important opportunities becoming available, and be successful in communicating the case for continuing to invest in developing and using better treatments for conditions such as pulmonary fibrosis.

Contrary to popular political opinion, neither population ageing nor new medicines have been the main drivers of rising health and social care spending in recent decades. As life expectancy has grown because death rates from events like strokes and heart attacks have fallen, so age specific disability rates have also fallen. In essence, the events and diseases that can kill you young can also take your independence early. As their incidence falls and life expectancy goes up, the age when people start to need more costly forms of care tends to rise in parallel.

With drug treatments, total costs have also stayed surprisingly stable in richer countries, at around 15% as a proportion of all health spending. As expensive new products arrive so older ones lose intellectual property protection and become low price generic therapies. The costs of human labour, by contrast, rise over time as people become more educated and their expectations naturally grow.

However, some conditions, including dementias such as Alzheimers Disease and eye disorders like macular degeneration, have long seemed directly linked with ageing. Their observed prevalence has increased more or less in line with the average ages of populations across the world, albeit Japan, the major nation with the longest recorded life expectancy, claims the world’s lowest age standardised dementia rate. Some may question such observations, but there is evidence that in countries like the UK the average age of dementia onset has shifted backwards in recent decades. Continue reading

PSA based screening has been described as an expensive public health disaster. But new approaches offer more accurate diagnoses and better outcomes.

Prostate cancer kills over 300,000 men a year world-wide. As it mainly affects older males (only about 10 per cent of those who die of it are aged under 65) these figures, together with those for the disability and distress caused by prostate cancer and its emasculating treatments, are set to rise as populations across the globe become both older and wealthier – sex hormone linked cancer rates are higher in well fed people than in poorer ones. Continue reading

A similar version of this blog was published in The Hindu Business Line on August 25, 2014.

India’s National Pharmaceutical Pricing Authority has recently announced new medicine price reductions, intended to improve the care available to people with diabetes and heart disease. But Professor David Taylor says that cheap medicines alone cannot solve India’s health care problems, and that driving down drug prices to unsustainably low levels damages public interests. Continue reading

On July 16 2013, Lord Tim Clement-Jones convened a meeting at the House of Lords in London to mark the publication of the report: Health and Health Care in India: national opportunities, global impacts, co-authored by Professor David Taylor and Dr. Jennifer Gill of University College London. The meeting was co-chaired by former Health Minister Lord Philip Hunt.

A round table discussion was led by David Taylor. He highlighted the fact that India currently spends only a little over 1% of its GDP on publicly funded healthcare and only about 0.1% of GDP on publicly funded medicines for the Indian people. Despite the fact that India is now the world’s largest generic medicines exporter many citizens still lack assured free access to good quality generic medicines and the support needed to use them to best effect. Continue reading

This report was researched and written by Dr Usman Kahn and Stephan Kreutzer of Matrix Insight and Dr Jennifer Gill and Professor David Taylor of the UCL School of Pharmacy.

Medicines counterfeiting (or falsification) presents a risk throughout the world that requires effective preventive responses. Falsification involves the knowing misrepresentation of a pharmaceutical product’s characteristics and/or provenance, and the deliberate avoidance of legally instituted regulatory processes. Even if they contain the right ingredients, medicines which have been produced in unregulated circumstances are inherently hazardous.

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Debate about VBP for innovative pharmaceuticals will continue throughout 2013, prior probably to a new system of NHS medicines pricing for novel medicines being introduced in 2014. Despite the claims of some authorities, there is no ‘scientific’ method for determining ‘the correct’ price for patented and allied medicines. The reality is that in contexts such as NHS medicines pricing and supply, political and other decision makers must seek to balance pragmatically the perceived harm resulting from paying ‘too much’ for new and more effective treatments against the dangers of failing to invest in the future, and under-incentivising activities that will advance biomedicine and increase patient and public wellbeing.

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Sue Sharpe, the Chief Executive of the PSNC, presented today a blue‐print for community pharmacies to serve as a ‘third tier’ in the NHS, alongside GP practices and hospitals. Innovations like ‘Healthy Living Pharmacies’ can provide better and more affordable care in areas ranging from the prevention and early detection of obesity and smoking related problems such as cancers, strokes and heart disease through to ensuring good treatment for people with raised blood pressure, type 2 diabetes and some emergency needs.

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Medicines wastage in the NHS and pharmaceutical falsification (counterfeiting and allied crimes) across the world are controversial topics which receive frequent media coverage. There have been allegations that poor prescribing, unnecessary dispensing and inappropriate patient use of medicines are leading to levels of wastage that are undermining health service finances.

However, research undertaken at the UCL School of Pharmacy indicates that although both medicines wastage and medicines counterfeiting represent significant problems, there are dangers in exaggerating their scale and impacts. This could distort priorities and lead to counter-productive public policies. There is in fact no evidence that NHS medicine users behave less responsibly than those who pay for treatments, or that levels of drug wastage are higher in Britain than in other relatively rich countries. At the same time it has been observed that charges can lead vulnerable people (and others) to stop taking therapies.

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