Press releases Monday 15 November to Friday 19 November 2010

Please remember to credit the BMJ as source when publicising an article and to tell your readers that they can read its full text on the journal's website (http://www.bmj.com).

(1) Experts urge prime minister to act on "massive" rises in the prices of drugs for rare diseases

(Letter: Open letter to prime minister David Cameron and health secretary Andrew Lansley)
http://www.bmj.com/cgi/doi/10.1136/bmj.c6466
(Feature: What makes an orphan drug?)
http://www.bmj.com/cgi/doi/10.1136/bmj.c6459
(Editorial: The problem of orphan drugs)
http://www.bmj.com/cgi/doi/10.1136/bmj.c6456
(Analysis: Orphan drug pricing may warrant a competition law investigation)
http://www.bmj.com/cgi/doi/10.1136/bmj.c6471

An open letter from 20 consultants and a patient group published on bmj.com today, calls on the prime minister to take action over a legal loophole that allows drug companies to make easy profits by licensing existing treatments for rare (orphan) diseases.

They argue that the current situation concerning orphan drugs is not in the best interests of patients or the NHS and that the cost to the NHS is likely to be above £10m a year.

The original purpose of this legislation was to encourage drug companies to conduct research into rare diseases and develop new treatments. But, as the rules are currently enacted, many companies simply modify existing drugs and obtain a licence for one or more orphan diseases. This then gives the company sole rights to supply the drugs and to charge what many consider "exorbitant" prices.

One example of the effect of these rules is the drug 3,4-diaminopyridine (3,4-DAP), which doctors have been using for over 20 years to treat two rare muscle diseases at a cost of £800 to £1,000 per patient per year. The company BioMarin has now been issued with a licence to supply the drug (marketed as Firdapse) throughout Europe and charges £40,000 to £70,000 per patient per year - a 50-fold to 70-fold increase. Although Biomarin argues that Firdapse is more stable and reliable than 3,4-DAP, clinicians have not found instability to be a significant problem with 3,4-DAP.

Other examples include hydroxycarbamide (hydroxyurea) licensed for chronic myeloid leukaemia. Using 500 mg capsules, it costs £160 a year to treat a patient with sickle cell disease on an unlicensed basis, but it costs £14,900 a year using 1 g tablets of hydroxycarbamide licensed as an orphan drug for sickle cell disease.

Oral ibuprofen for analgesia costs £0.08 per gram, but intravenous ibuprofen for patent ductus arteriosus (a congenital heart disorder) costs £6,575 per gram, tens or even hundreds of times more than the cost of producing sterile ibuprofen solution for intravenous injection in an NHS facility.

"In the present economic situation it seems vital to ensure that systems are in place to prevent excessive commercial profits being made at the expense of patients and public spending," say the signatories.

They conclude: "Legislation on orphan drugs, far from encouraging the development of new treatments for orphan diseases, is severely limiting the availability of existing treatments. We believe that the Medicines and Healthcare Products Regulatory Agency and Department of Health should not just state the rules but should act now to progress the issue of unfairness upwards, so as to instigate change."

In a BMJ investigation, also published today, Dr Sam Richmond, a consultant neonatologist at Sunderland Royal Infirmary, and a signatory of the open letter, argues: "If drug companies are undertaking research where nobody else was interested – and some are – then a monopoly may be justified. But if it's a product already in use, they should clear off, or sell at a price comparable with the existing price."

Dr Daphne Austin, chair of the UK Commissioning Public Health Network, says: "It disgusts me, it really does. [Amifampridine] is one of a number of drugs that are not new, but under the legislation have been licensed so that they can be sold for much more money, which is pure profit." She believes the price set for the drug is "indecent" and points out that the extra cost of amifampridine in the UK "is equivalent to that of kidney dialysis for 323 patients."

This view is supported in an editorial, which says that current incentives to licence drugs for rare diseases "are now too generous." Authors Robin Ferner and Dyfrig Hughes believe the NHS "could, and should, make and distribute 'specials' (unlicensed medicines) for rare diseases" and that the GMC "should allow doctors to prescribe a drug that meets the individual patient's needs, but is not licensed for the specific indication, even if a licensed medicine exists for the same indication."

In an accompanying analysis, Timothy Cox and colleagues at the University of Cambridge argue that pricing for orphan drugs hinders access to treatment and may warrant a competition law investigation. They add that current high pricing contravenes the aim of the Orphan Regulation, which states: "Patients suffering from rare conditions should be entitled to the same quality of treatment as other patients."

Contacts:
Open letter: David Nicholl, Consultant Neurologist, City Hospital and Queen Elizabeth Hospital, Birmingham, UK
Email: david.nicholl@nhs.net
BMJ investigation (feature): Deborah Cohen, Investigations Editor, BMJ, London, UK
Email: dcohen@bmj.com
Editorial: Robin Ferner, Director, West Midlands Centre for Adverse Drug Reactions, City Hospital, Birmingham, UK
Email: r.e.ferner@bham.ac.uk
Analysis: Tim Cox, Department of Medicine, University of Cambridge, UK
Email: tmc12@medschl.cam.ac.uk

(2) Government's personal health record project meets with limited enthusiasm from patients
(Research: Adoption, non-adoption, and abandonment of a personal electronic health record: case study of HealthSpace)
http://www.bmj.com/cgi/doi/10.1136/bmj.c5814

HealthSpace, the internet-accessible personal health organiser developed as part of the National Programme for IT in the National Health Service, was significantly less popular than anticipated, a research team from the University of London has found.

Instead of the predicted 5-10per cent of the population signing up, only 0.13 per cent of those invited got as far as activating the full functionality of their personal health record, according to the study published on bmj.com today.

Professor Trisha Greenhalgh from Barts and the London School of Medicine and Dentistry led the team and was joined by researchers from University College London and Raft Consulting. They studied uptake of HealthSpace from its release in 2007 until mid-2010.

HealthSpace is an internet-accessible personal health record. Its features include the facility to enter and store health data such as blood pressure or weight; a calendar to store health appointments; email-style communication with the person’s general practitioner; and the ability to access their summary care record (a centrally stored summary of key medical details drawn from a person’s general practitioner record). HealthSpace is free but users must register for a basic account (online) or an advanced account (in person at a front office run by their local Primary Care Trust)

Early business plans anticipated that between 5 and 10 per cent of the over-16 population would become HealthSpace users. It was also predicted that patients would access their summary care records and notify their GP of errors, thereby driving up data quality in NHS medical records. In fact, only 2,913 people (0.13 per cent of those invited to open an advanced HealthSpace account) actually did so, and the research team did not find a single person who had accessed their summary care record.

Problems included a complex registration process, a "clunky" user interface (some aspects of which have been upgraded since the research was undertaken), the need to enter much of the data oneself, limited functionality which did not seem to match how people actually looked after their own health, and the low levels of knowledge about, or interest in, the product from healthcare staff.

The recent emergence of rival products from commercial IT companies including Microsoft's HealthVault (released in the UK in June 2010) and Google's GoogleHealth (released in 2008) have raised questions about the place of a standard-issue personal health organiser from the NHS.

Contact:
Trisha Greenhalgh, Director, Healthcare Innovation and Policy Unit, Centre for Health Sciences, Barts and the London School of Medicine and Dentistry, London, UK
Email: p.greenhalgh@qmul.ac.uk

(3) Medical journals come together to put health at the heart of the climate change agenda
(Editorial: Health and climate change)
http://www.bmj.com/cgi/doi/10.1136/mj.c6357

Several leading medical journals have come together to urge health professionals everywhere to put health at the heart of climate change negotiations.

An editorial published simultaneously in the BMJ, the Lancet and the Finnish Medical Journal today, warns that the links between climate policy and health policy must not be overlooked. The editorial has also been made available for publication in all peer reviewed medical journals worldwide through the World Association of Medical Editors.

Written by Robin Stott and Ian Roberts on behalf of the Climate and Health Council, it is a call to action for health professionals across the world to help tackle the health effects of climate change.

Failure to agree radical reductions in emissions spells a global health catastrophe, they say.

Later this month, representatives from countries around the world will meet at the 2010 United Nations Climate Change Conference in Mexico. Stott and Roberts argue that "if the delegates at this conference think that obesity and climate change are unrelated, they would be wrong. The planet is getting hotter, its people are getting fatter, and the use of fossil fuel energy is the cause of both."

They argue that moving to a low carbon economy "could be the next great public health advance." For example, a low carbon economy will mean less pollution and a need for more physical activity. A low carbon diet (especially eating less meat) and taking more exercise will mean less cancer, obesity, diabetes, heart disease, and even depression. A reduction in car use and meat consumption would also cut world food prices, they add.

They believe that health professionals everywhere have a responsibility to put health at the heart of climate change negotiations. "Responding to climate change could be the most important challenge that health professionals face," they say. "We invite colleagues everywhere to join us in tackling this major public health scourge of the 21st century."

They are also urging health professionals to commit to action by signing the Climate and Health Council pledge (www.climateandhealth.org/pledge), and by contacting their health minister "to ensure that the links between climate policy and health policy are known and fully taken into account in all climate change negotiations."

Contact:
Ian Roberts, Professor of Epidemiology and Public Health, London School of Hygiene and Tropical Medicine, London, UK
Email: ian.roberts@lshtm.ac.uk

Emma Dickinson
Tel: +44 (0)20 7383 6529
Email: edickinson@bma.org.uk

Press Office telephone : 020 7383 6254 (Weekdays : 0900hrs - 1800hrs)
British Medical Association
BMA House, Tavistock Square, London WC1H 9JP

and from:

the EurekAlert website, run by the American Association for the Advancement of Science (http://www.eurekalert.org)
http://intranet.bmj.com/departments/dept-bmj/bmj-team-resources/web-team-resources/General_blogging_principles.doc