Editorial
Christina Koci Hernandez/San Francisco Chronicle/Corbis
Rare diseases in children: towards better and fairer treatment
For the FDA report see http:// www.fda.gov/downloads/ RegulatoryInformation/ Legislation/FederalFoodDrug andCosmeticActFDCAct/ SignificantAmendments totheFDCAct/FDASIA/ UCM404104.pdf
Rare disease, defined by the US Orphan Drug Act of 1983, is any disease that affects fewer than 200 000 people. According to a 2009 estimate, more than 25 million Americans were affected by nearly 7000 rare diseases; almost half of these rare diseases affect children. Research to produce effective treatments is hampered by the small number of patients with each type of rare disease, ethical concerns about clinical testing, and the diversity of clinical manifestations of each disease. In recognition of the challenges, the US Food and Drug Administration (FDA) published the agency’s strategic plans for accelerating development of treatments for paediatric rare diseases last week. The report outlines four objectives: enhanced basic and translational science; strengthened communication, collaboration, and partnership; regulatory science to advance clinical trial design and performance; and improvements in the FDA’s review process for any new treatment. These are all laudable and important initiatives. But an important further issue is the relationship between public and private stakeholders. In any collaboration,
benefits must accrue to both parties. Once a new orphan drug is developed and marketed, it should be recognised that its success depended on publicly funded research and voluntary participation of patients in clinical trials. Any new treatment must therefore be priced in ways that are appropriate and sustainable for patients and health systems. Too often, this is not the case. Ivacaftor is a drug for a subgroup of patients with cystic fibrosis. Government and charitable research funding were crucial to its development. The price of the drug? Around US$300 000 annually—for a medicine that is needed for a patient’s entire life. For many diseases, drug development is still a long way from potential clinical use. But rare diseases in children need more than new drugs and devices. Individual care plans, up-to-date information in ageappropriate language for children and carers, links to communities and patient organisations, and guidance for health-care professionals will be just as important to save and improve the lives of children with rare diseases. The Lancet
STRINGER/ITALY/Reuters/Corbis
Europe’s responsibility to refugees: health beyond borders
For the UN report on Syrian refugees in Europe see http://www.refworld.org/ docid/53b69f574.html For the Amnesty International report on Europe’s borders see http://www.amnesty.org/en/ library/asset/EUR05/001/2014/ en/48cb6136-cefc-4fd0-96cdcd43b46eb5a8/ eur050012014en.pdf
208
For Syrian refugees, life is bleak. 2·8 million people have fled the country since the start of its civil war, with 1 ·1 million travelling to Lebanon, according to the UN Refugee Agency (UNHCR). Half a million of Turkey’s Syrian refugees live outside government camps, at risk of homelessness and exploitation; in Lebanon, health services are under such strain that some refugees are returning to Syria for treatment. Through the UNHCR resettlement programme, Europe has pledged to accept only 30 000 refugees; the UK, which declined to participate in the UN programme, has resettled just 24. No wonder, then, that Syrians are joining economic migrants in attempting to reach European Union (EU) countries directly, accounting for the most illegal EU border crossings in 2013. Since the reinforcement of land borders in Greece and Bulgaria, asylum seekers have few opportunities to legally enter EU countries, and many attempt the perilous journey across the Mediterranean by boat. In The Human Cost of Fortress Europe, Amnesty International documents the effects of EU border
enforcement on migrants, including allegations of illegal forced expulsion of migrants by Greek, Bulgarian, and Spanish border patrols. EU funding for border controls dwarfs that for refugees and asylum procedures, with inadequate monitoring of human rights abuses. Refugees are a particularly vulnerable group. In 2013, Europe received more than half of the 25 300 asylum claims made worldwide for unaccompanied children. Many refugees have mental health problems, disabilities, or are elderly, but some countries do not have the capacity to identify and support these individuals. What are Europe’s responsibilities to refugees and migrants? All refugees seeking asylum should be identified and assessed quickly and fairly. Governments should not exclude undocumented migrants from health care, or use health systems as immigration screening services. Sadly, the UK seems to be one country that has put rigid rules of border security before a compassionate approach to victims of intolerable brutality. The Lancet www.thelancet.com Vol 384 July 19, 2014
Christina Koci Hernandez/San Francisco Chronicle/Corbis
Rare diseases in children: towards better and fairer treatment
For the FDA report see http:// www.fda.gov/downloads/ RegulatoryInformation/ Legislation/FederalFoodDrug andCosmeticActFDCAct/ SignificantAmendments totheFDCAct/FDASIA/ UCM404104.pdf
Rare disease, defined by the US Orphan Drug Act of 1983, is any disease that affects fewer than 200 000 people. According to a 2009 estimate, more than 25 million Americans were affected by nearly 7000 rare diseases; almost half of these rare diseases affect children. Research to produce effective treatments is hampered by the small number of patients with each type of rare disease, ethical concerns about clinical testing, and the diversity of clinical manifestations of each disease. In recognition of the challenges, the US Food and Drug Administration (FDA) published the agency’s strategic plans for accelerating development of treatments for paediatric rare diseases last week. The report outlines four objectives: enhanced basic and translational science; strengthened communication, collaboration, and partnership; regulatory science to advance clinical trial design and performance; and improvements in the FDA’s review process for any new treatment. These are all laudable and important initiatives. But an important further issue is the relationship between public and private stakeholders. In any collaboration,
benefits must accrue to both parties. Once a new orphan drug is developed and marketed, it should be recognised that its success depended on publicly funded research and voluntary participation of patients in clinical trials. Any new treatment must therefore be priced in ways that are appropriate and sustainable for patients and health systems. Too often, this is not the case. Ivacaftor is a drug for a subgroup of patients with cystic fibrosis. Government and charitable research funding were crucial to its development. The price of the drug? Around US$300 000 annually—for a medicine that is needed for a patient’s entire life. For many diseases, drug development is still a long way from potential clinical use. But rare diseases in children need more than new drugs and devices. Individual care plans, up-to-date information in ageappropriate language for children and carers, links to communities and patient organisations, and guidance for health-care professionals will be just as important to save and improve the lives of children with rare diseases. The Lancet
STRINGER/ITALY/Reuters/Corbis
Europe’s responsibility to refugees: health beyond borders
For the UN report on Syrian refugees in Europe see http://www.refworld.org/ docid/53b69f574.html For the Amnesty International report on Europe’s borders see http://www.amnesty.org/en/ library/asset/EUR05/001/2014/ en/48cb6136-cefc-4fd0-96cdcd43b46eb5a8/ eur050012014en.pdf
208
For Syrian refugees, life is bleak. 2·8 million people have fled the country since the start of its civil war, with 1 ·1 million travelling to Lebanon, according to the UN Refugee Agency (UNHCR). Half a million of Turkey’s Syrian refugees live outside government camps, at risk of homelessness and exploitation; in Lebanon, health services are under such strain that some refugees are returning to Syria for treatment. Through the UNHCR resettlement programme, Europe has pledged to accept only 30 000 refugees; the UK, which declined to participate in the UN programme, has resettled just 24. No wonder, then, that Syrians are joining economic migrants in attempting to reach European Union (EU) countries directly, accounting for the most illegal EU border crossings in 2013. Since the reinforcement of land borders in Greece and Bulgaria, asylum seekers have few opportunities to legally enter EU countries, and many attempt the perilous journey across the Mediterranean by boat. In The Human Cost of Fortress Europe, Amnesty International documents the effects of EU border
enforcement on migrants, including allegations of illegal forced expulsion of migrants by Greek, Bulgarian, and Spanish border patrols. EU funding for border controls dwarfs that for refugees and asylum procedures, with inadequate monitoring of human rights abuses. Refugees are a particularly vulnerable group. In 2013, Europe received more than half of the 25 300 asylum claims made worldwide for unaccompanied children. Many refugees have mental health problems, disabilities, or are elderly, but some countries do not have the capacity to identify and support these individuals. What are Europe’s responsibilities to refugees and migrants? All refugees seeking asylum should be identified and assessed quickly and fairly. Governments should not exclude undocumented migrants from health care, or use health systems as immigration screening services. Sadly, the UK seems to be one country that has put rigid rules of border security before a compassionate approach to victims of intolerable brutality. The Lancet www.thelancet.com Vol 384 July 19, 2014
