Pharmaceutical innovation

In this section:


We expect to see significant pharmaceutical innovation over the next 20 years, with the potential to improve disease treatment, morbidity and mortality across the population.

Antibody-drug therapies could replace traditional chemotherapy, delivering drugs directly into cancer cells (1); monoclonal antibodies could be used to treat neurodegenerative diseases, such as multiple sclerosis (2) and Parkinson's; and synthetic compounds could be developed that can tackle obesity and diabetes simultaneously (3,4).

Therapeutic vaccines could prevent a wide range of cancers by triggering the immune system to recognise and destroy cancer cells (5,6,7), while preventative vaccines may finally be able to provide lifelong protection against tuberculosis, malaria, HIV, flu and hepatitis C (8,9,10).

Multi-drug therapies will be able to use existing drugs more effectively, preventing drug resistance (11), and treatments for lifelong conditions such as epilepsy will be refined, reducing unwanted side effects (12).

However, there is uncertainty over whether anticipated breakthroughs can be realised given pressures on research and development budgets (13).

Precision medicine

Genetics play a fundamental role in determining health: by the age of 60, six out of ten people are likely to develop a disease that is at least partially genetically determined.

Precision medicine could revolutionise our ability to predict, prevent, monitor and treat conditions, radically improving patient outcomes and overall population health.

We expect use of low-cost genetic sequencing to increase with genetic profiling available to detail a person's predisposition to certain diseases. Genome mapping data could be used to stratify cohorts for preventive screening (14,15,16).

Pre-clinical trials, animal studies and clinical trials are discovering genetic differences in responses to complex cancer therapies. Combined with research to sequence tumours and categorise subtypes, these developments mean clinicians can match patient subgroups to treatments, reducing the risk of side effects and targeting treatments to those patients most likely to respond (17,18,19,20,21). Clinical biomarkers have the potential to assist prognosis and treatment and trials are underway to develop gene therapies for a variety of conditions including HIV (22,23,24,25,26).

However, progress to develop therapies for progressive neurological disorders and validate biomarker tests has been slow. Recent evidence suggests that the effectiveness of biomarkers may have been overstated (27). This vision of the future also raises concerns around ethics and data protection, while lifestyle profiling could lead to over-medicalisation.

Targeted drugs and treatments have implications for the development of pharmaceuticals, potentially reducing both the time required to bring treatments to market and also the pool of eligible patients and potential profits. This trade-off may inhibit the speed at which new therapies enter mainstream use.

Budgetary implications

Total NHS spend on pharmaceuticals increased from £2.3 billion in 1990 to £12.2 billon in 2009 (28,29), with pharmaceutical use within hospitals accounting for an increasing proportion of costs, up from 22 per cent in 2003 to 31 per cent in 2009.

However, the Office of Health Economics (OHE) estimates that NHS spending on pharmaceuticals could begin to fall, or at least be contained, over the next six years because:

  • Cheap generic alternatives to widely prescribed pharmaceuticals, such as statins, will be available as patents expire.
  • Many pharmaceuticals currently in development are specialist drugs targeted at small populations. Although they are costly to develop and expensive to buy, the volumes bought are low, limiting their cost to the system.

Historically pharmaceutical costs have risen year-on-year and this is likely to continue, despite OHE forecasts, if pharmaceutical costs continue to shift away from primary care (where generic versions are entering the system) towards more innovative, specialised and expensive drugs within hospitals.

Contributing to current pharmaceutical spend are drugs that are widely prescribed, but ineffective in a large number of cases. The concept of personalised prescribing will reduce this spend, but could render this market unviable for pharmaceutical companies. Companies may find it more difficult to patent targeted replacements (30): recent rulings in the United States have restricted the patent protections granted to biotechnology companies and the continuation of this trend could fundamentally change the way that drugs are developed and funded, further driving up costs (31).

It is also important to note that research and development expenditure by pharmaceutical companies has grown rapidly, rising tenfold between 1975 and 2006 (32,33,34). The current rate of innovation may stall unless pharmaceutical companies can recoup research and development costs (35,36).

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