The report presents what the advocacy organization believes should be included in the U.K.’s coming new governmental framework for dealing with rare diseases. It also builds upon its 2019 National Neurology Patient Experience Survey, in which 55% of respondents reported delays in healthcare access.
“Poor public awareness and a lack of investment in services for rare conditions has meant that, for too long, people with rare neurological conditions have experienced long waits for a diagnosis, poor care coordination, and insufficient information provision,” said Georgina Carr, CEO of Neurological Alliance, in a press release.
“We hope that this report will bring about a sea change in how rare neurological conditions are viewed, both in the medical profession and in wider society. It’s time that rare neurological conditions came out of the shadows,” Carr said.
According to the 89-page report, “Out of the Shadows: What needs to change for people with rare neurological conditions,” individuals in the U.K. with rare neurological disorders are too often left behind when it comes to care and treatment.
The report calls for speedier access to specialists and a diagnosis. For one thing, there needs to be broader awareness among primary care doctors of rare neurological conditions, so that patients are quickly referred to a specialist for treatments that are generally more effective when started early, it states.
By the year’s end, according to the report, more than 200,000 patients likely will be awaiting an appointment with a doctor who specializes in their disease. What’s more, the COVID-19 pandemic could exacerbate delays and gaps in treatment and care caused by doctor visit waiting lists and overburdened services.
The Neurological Alliance report also calls for changed perceptions around rare neurological conditions. Just because a disorder is complex does not mean that it cannot be well-managed, the report states, adding that patients with such conditions are entitled to parity in access and care.
The U.K. often lags behind other European countries when it comes to approving and funding new therapies for rare neurological conditions, according to the report, which seeks faster access to new treatments.
“The paediatric rollout of Nusinersen (Spinraza) has been quite good, but the treatment rollout for adults hasn’t progressed at all well,” said a statement by Spinal Muscular Atrophy UK that was included in the report.
Spinraza (nusinersen), by Biogen, in 2016 became the first disease-modifying therapy approved by the U.S. Food and Drug Administration to treat SMA in both infants and adults. The following year, the European Union approved Spinraza to treat SMA throughout Europe.
England’s National Institute for Health and Care Excellence has begun a review process to decide whether to include SMA type 3 patients who are unable to walk unaided in the country’s conditional reimbursement program for Spinraza.
Based on last year’s patient experience survey, which found that less than one-third of people with such disorders felt they were getting the mental health care they need, the report also recommends improved mental health support.
Finally, the report asks that disease information be consistently provided to patients upon diagnosis to help them understand more about their condition and care choices. In the survey, only one-third of respondents said they were given informational literature when they were diagnosed.
People with rare neurological conditions also need government help in funding home adaptations, and high-quality and well-funded community programs to help with social care, the study states, adding that such provisions should be more than afterthoughts as they currently appear to be.
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