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“Orphan” diseases - those that affect only small numbers of people - have over the past few decades gone from neglect to the mainstream. They now account for an estimated 16 per cent of global prescription medicine sales, providing considerable improvements in quality and length of life for a growing range of conditions.

Genome analysis is accelerating progress. Much of the modern industry is due to the pioneering work of Henri Termeer, who died last year but left a powerful legacy with his company Genzyme, from which many of his protégés went on to create or join other orphan drug specialists. His innovations included establishing very high prices for treatment to support research for diseases affecting very small numbers of patients.

Yet this week’s Rare Disease Day comes at a time of debate over the costs to healthcare systems of expensive personalised and rare drugs. In countries such as China, that is a significant brake on providing therapies. The shadow of Brexit is also raising concerns over progress as highly effective networks of patients and doctors risk fragmenting.

Despite advances, there are still many rare diseases for which treatment options are scant. New drugs are not always the limiting factor. A historical condition such as leprosy continues to infect 200,000 people a year despite the existence of simple and effective cures. Improved advocacy, diagnosis and research on transmission remain essential.

Read our full report on the future of rare diseases.


Three questions

We spoke to Sheila Tlou, co-chair of Nursing Now, a global campaign to raise the profile and status of the profession.

Why are nurses so important?

As we discuss wellbeing for all, and the growth in non-communicable diseases, nurses are the most important health personnel. In primary healthcare, and in the context of universal health coverage, they are the linchpins. They operate alongside community health workers and in primary care where doctors are thinly spread.

How many more nurses does the world need?

The aim is 9m by 2030. [Of the 43.5m health workers in the world, it is estimated that 21m are nurses and midwives]. It’s an ambitious target but I’m really glad the International Council of Nurses is supporting this aim. We also need to standardise training so that anyone who moves between countries will be at the same standard.

Why are they often neglected and undervalued?

Their role has not been that well appreciated in lots of countries. It depends on the history. Different countries have had different approaches. Some people go into medicine not because they wanted to but because they had no other options. Nurses should come out of their training with empathy and self-esteem.


Chartwatch

HIV/Aids deaths This interactive graphic shows the decline in mortality by age group as the tide turned in the war on HIV/Aids in 2006. A notable exception to the current positive trend is Russia, where critics blame a socially conservative approach influenced by President Putin's embrace of the Orthodox church. (Our World in Data, Politico)


News round-up

Anti-vaxxers on the march The vaccine backlash in the US is partly due to a discredited study by Andrew Wakefield, a British researcher, which linked autism and the MMR vaccine. Mr Wakefield is now a prominent anti-vaxxer in Texas — where opt-outs of vaccines have rocketed. The UK has published its methodology for vaccine funding. (FT, Guardian, Department of Health)

Aid and health security As funds for surveillance are pared back, there is a pressing need to strengthen the investment case for health security. A “moderate pandemic” could cost the global economy more than $570bn or 0.7 per cent of GDP, not to mention lives lost. Changes are being mooted in the US but new approaches are needed for allocation. (Global Health Now, Devex, LSHTM, below: Economist/Twitter)

Opioid crisis The US will target opioid manufacturers and distributors in a new push to curb the epidemic sweeping the country. President Trump went a step further at a White House summit, suggesting the execution of drug dealers. (Washington Post, ABC News) 

Dealing with diesel A landmark ruling means German cities — 70 of which exceeded EU nitrogen oxide limits last year — can ban older diesel cars, while Fiat said it would kill off all diesel models by 2022. Brussels plans to make public transport free on days of high air pollution. Britain's chief medical officer issued a detailed report on its effect on health: those in poorer areas suffer the most. (FT, Guardian, UK government) 

Healthier eating Calorie counts on menus help reduce intake by 8-12 per cent per meal. The WHO is examining fiscal policy and diets and there are labelling initiatives in France and Canada. In the UK, millennials are set to be the fattest Britons ever. (Cochrane Library, WHO, Lancet, Canadian government, BBC)

Tailored diets Dietary guidelines are usually based on average population data but it is known, for example, that some ethnic groups are more prone to high blood pressure and excess body fat on certain diets. To combat this, foods adapted to a person’s genes could soon be on the way. (WSJ)

Whose data? Drugmakers are making huge efforts to gather medical information as it is digitised. But this growth in “real-world evidence” raises many questions about data access and patient privacy. The same goes for medical device makers. (Reuters, Stat)

Disease X Among the usual suspects on the WHO's updated priority list of diseases is “Disease X”. The inclusion of a "known unknown" on the list is a recognition that brand-new pathogens — such as human-to-human bird flu — could come out of nowhere. (UN Dispatch)

Avatars for health The Health EU project hopes to give people their own virtual twin to monitor their health. The concept — already used in aerospace and automotive industries — enables processes to be tested that would be impossible in the real world — and would reduce testing on animals. (Eureka)

Dating with genetics Who says romance is dead? Pheramor, a new dating app, gets users to swab their cheeks, mail in their samples and browse profiles of genetically-compatible matches. Researchers are not convinced this is the path to true love. (Stat, The Smithsonian)

Cat (poo) scans Research into toxoplasma gondii — better known as the “cat parasite” and usually passed to humans through cat faeces — could lead to more effective treatment for toxoplasmosis and malaria. (The Conversation)

Try a coffee . . . Still struggling with that new year exercise regime?A cup of coffee could help you run faster and lift more weights — depending on your genetic make-up and how you make your brew. (The Conversation UK)

. . . or a MoCA The interest shown in President Trump's MoCA (Montreal Cognitive Assessment) test has led researchers to develop a DIY version that could speed up the detection of mild cognitive impairment (MCI), the earliest stage of dementia. Up to one in five over 65s have MCI and more than half progress to full dementia within five years. (Kaiser Health News)


FT conference

US Healthcare & Life Sciences Summit From potential changes to the ACA to managing the opioid crisis, the US healthcare system is on the cusp of a major shift. But can we afford it? Join us to discuss the issues in New York on May 10. Use the link below and code Health for your 20 per cent discount. More information here.


Best from the Journals

Diagnosing diabetes The fastest growing disease worldwide is better understood if examined as five categories rather than just type 1 and type 2, an approach which could enable better-targeted treatment. Diabetes diagnoses in the UK have doubled in 20 years. (The Lancet, Guardian)

The Global Virome Project Fighting epidemics can be undermined by poor understanding of viral threats. A project launches this year to identify these threats sooner and provide data for public health interventions before they hit. (Science)

Mapping malnutrition New maps show the extent of stunted growth due to malnutrition among African children. It is unlikely any single country will meet the UN's Sustainable Development Goal of ending malnutrition by 2030. (Nature)

Shining a light on drug prices An editorial calls for greater transparency on drug pricing — something that would also produce a more sustainable pharma business. “An industry with such profound implications for the public interest should not be shrouded in so much secrecy.” (BMJ) 

Antibiotic alert Almost a third of antibiotic prescriptions issued in primary care in England have no clinical justification. The most common reasons for prescription are respiratory and urinary infections. (Journal of Antimicrobial Chemotherapy)


Podcast of the week

US aid and global health Alma Crumm Golden of USAID discusses maternal and child health and the priorities for USAID in the face of potential cuts to global health funding from President Trump. (CSIS Take as Directed, 19m)


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Final thought

Sport for all? Are countries such as Britain right to focus sports funding on Olympic medal prospects? Or should they concentrate on less elite activities with more potential for participation and improving the health of the young? (The Guardian)

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