In this section:
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).
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.
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).
- Ledford H (2011). Journal article. Toxic antibodies blitz tumours. Nature 476, p380–381
- Graham-Rowe D (2011). Online journal article. Antibody offers hope for multiple sclerosis treatment. Nature, 24 October
- Cho H, Zhao X, Hatori M, Yu RT, Barish GD, Lam MT, Chong, L-W, DiTacchio L, Atkins AR, Glass CK, Liddle C, Auwerx J, Downes M, Panda S, Evans RM (2012). Journal article. Regulation of circadian behaviour and metabolism by REV-ERB-α and REV-ERB-β. Nature: 485, p123–127
- Couzin-Frankel J (2011). Online article. Novel Drug Shrinks Monkeys' Waistlines. Science: 9 November 2011
- Koski GK, Koldovsky U, Xu S, Mick R, Sharma A, Fitzpatrick E, Weinstein S, Nisenbaum H, Levine BL, Fox K, Zhang P, Czerniecki BJ (2012). Journal article. ‘A novel dendritic cell-based immunization approach for the induction of durable Th1-polarized anti-HER-2/neu responses in women with early breast cancer’. Journal of Immunotherapy, vol 35, pt 1, pp 54–65.
- Kovjazin R, Volovitz I, Kundel Y, Rosenbaum E, Medalia G, Horn G, Smorodinsky NI, Brenner B, Carmon L (2011). Journal article. ImMucin: a novel therapeutic vaccine with promiscuous MHC binding for the treatment of MUC1-expressing tumors. Vaccine, vol 29, pp 4676–86.
- Telegraph. Science news. Universal cancer vaccine developed
- Balazs AB, Chen J, Hong CM, Rao DS, Yang L, Baltimore D (2011). Journal article. 'Antibody-based protection against HIV infection by vectored immunoprophylaxis'. Nature, vol 481, no 7379, pp 81–4.
- Rappuoli R, Aderem A (2011). Journal article. 'A 2020 vision for vaccines against HIV, tuberculosis and malaria'. Nature, vol 473, pp 463–9.
- Agnandji ST, Lell B, Soulanoudjingar SS (et al) (2011). Journal article. ‘First results of phase 3 trial of RTS,S/AS01 malaria vaccine in African children’. New England Journal of Medicine, vol 365, pt 20, pp 1863–75.
- Ledford H (2011). Online article. New drug targets raise hopes for hepatitis C cure. Nature, 26 April 2011
- Mestre-Ferrandiz J, Mordoh A, Sussex J. Updated report. The many faces of innovation. A report for the ABPI by the Office of Health Economics, March 2012
- Riccaboni M (2012). Seminar briefing. Is There a Productivity Crisis in Pharmaceutical R&D? Office of Health Economics
- Hayden EC (2012). Online article. Sequencing set to alter clinical landscape. Nature, 15 February 2012
- Chen et al (2012). Journal article. ‘Personal “omics” profiling reveals dynamic molecular and medical phenotypes’. Cell, vol 148, pp 1293–307.
- Dennis C (2012). News and comment. The rise of the 'narciss-ome'. Nature, 16 March 2012
- Dennis C (2012). News and comment. Mouse 'avatars' could aid pancreatic cancer therapy. Nature, 21 March
- Chen Z, Cheng K, Walton Z, Wang Y, Ebi H, Shimamura T, Liu Y, Tupper T, Ouyang J, Li J, Gao P, Woo MS, Xu C, Yanagita M, Altabef A, Wang S, Lee C, Nakada Y, Peña CG, Sun Y, Franchetti Y, Yao C, Saur A, Cameron MD, Nishino M, Hayes DN, Wilkerson MD, Roberts PJ, Lee CB, Bardeesy N, Butaney M, Chirieac LR, Costa DB, Jackman D, Sharpless NE, Castrillon DH, Demetri GD, Jänne PA, Pandolfi PP, Cantley LC, Kung AL, Engelman JA, Wong KK (2012). Journal article. ‘A murine lung cancer co-clinical trial identifies genetic modifiers of therapeutic response’. Nature, vol 483, no 7391, pp 613–7.
- Curtis C, Shah SP, Chin SF, Turashvili G, Rueda OM, Dunning MJ, Speed D, Lynch AG, Samarajiwa S, Yuan Y, Gräf S, Ha G, Haffari G, Bashashati A, Russell R, McKinney S, Langerød A, Green A, Provenzano E, Wishart G, Pinder S, Watson P, Markowetz F, Murphy L, Ellis I, Purushotham A, Børresen-Dale AL, Brenton JD, Tavaré S, Caldas C, Aparicio (2012). Online article. ‘The genomic and transcriptomic architecture of 2,000 breast tumours reveals novel subgroups’. Nature. Published online 18 April 2012.
- Villarroel MC, Rajeshkumar NV, Garrido-Laguna I, De Jesus-Acosta A, Jones S, Maitra A, Hruban RH, Eshleman JR, Klein A, Laheru D, Donehower R, Hidalgo M (2011). Journal article. ‘Personalizing cancer treatment in the age of global genomic analyses: PALB2 gene mutations and the response to DNA damaging agents in pancreatic cancer’. Molecular Cancer Therapeutics, vol 10, no 1, pp 3–8.
- Juergens RA, Wrangle J, Vendetti FP, Murphy SC, Zhao, M, Coleman B, Sebree R, Rodgers K, Hooker CM, Franco N, Lee B, Tsai S, Espinoza Delgardo I, Rudek MA, Belinsky SA, Herman JG, Baylin SB, Brock MV, Rudin CM (2011). Journal article. ‘Combination epigenetic therapy has efficacy in patients with refractory advanced non-small cell lung cancer’. Cancer Discovery, vol 1, no 7, pp 598–607.
- Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S (2012). Journal article. ‘Therapeutic hemoglobin levels after gene transfer in beta-thalassemia mice and in hematopoietic cells of beta-thalassemia and sickle cells disease patients’. PLOS One, vol 7, no 3, 32345.
- Science Now (2011). Article. Gene Therapy May Thwart HIV
- Stieger K, Lorenz B (2010). Article. ‘Gene therapy for vision loss – recent developments’. Discovery Medicine, vol 10, no 54, pp 425–33.
- Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, Down J, Denaro M, Brady T, Westerman K, Cavallesco R, Gillet-Legrand B, Caccavelli L, Sgarra R, Maouche-Chrétien L, Bernaudin F, Girot R, Dorazio R, Mulder GJ, Polack A, Bank A, Soulier J, Larghero J, Kabbara N, Dalle B, Gourmel B, Socie G, Chrétien S, Cartier N, Aubourg P, Fischer A, Cornetta K, Galacteros F, Beuzard Y, Gluckman E, Bushman F, Hacein-Bey-Abina S, Leboulch P (2010). Journal article. ‘Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia’. Nature, vol 467, no 7313, pp 318–22.
- Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S (2012). Journal article. ‘Therapeutic hemoglobin levels after gene transfer in beta-thalassemia mice and in hematopoietic cells of beta-thalassemia and sickle cells disease patients’. PLOS One, vol 7, no 3, e32345.
- Ioannidis JP, Panagiotou OA (2011). Online article. Comparison of Effect Sizes Associated With Biomarkers Reported in Highly Cited Individual Articles and in Subsequent Meta-analyses. JAMA. 305(21):2200-2210. Accessed 24.09.2012
- Hawe E, Yuen P, Baillie L (2011). OHE guide to UK health and health care statistics. Table 4.8. Office of Health Economics.
- Health and Social Care Information Centre (2011). Report. Hospital Prescribing, England: 2010.
- Greenbaum D (2012). Journal article. Regulation and the Fate of Personalized Medicine. Virtual Mentor: American Medical Association Journal of Ethics 14 (8): 645-652
- Greenbaum D (2010). Journal article. New rules, different risk: the changing freedom to operate analysis for biotechnology. North Carolina J Law Tech 11(1):139-160
- Health Select Committee evidence (2009)
- Association of the British Pharmaceutical Industry submission to the Health Select Committee inquiry into public expenditure
- Sussex, J (2010). Report. Innovation in Medicines: Can we value progress?. Office of Health Economics.
- Mestre-Ferrandiz J, Mordoh A, Sussex J (2012). Report. The many faces of innovation. A report for the ABPI by the Office of Health Economics, March 2012
- ABPI UK NHS medicines bill projection 2012-2015