A New Rare Disease Action Plan for England

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An estimated 3.5 million people in the U.K. are affected by a rare disease. But, with more than 7,000 different rare diseases discovered thus far, clinicians and patients can find it difficult to keep up.

To improve care for rare disease patients, England recently released its first Rare Disease Action Plan. Part of the larger UK Rare Diseases Framework published in 2021, England’s framework outlines four key national priorities:

  1. Faster diagnoses: Accurate and timely diagnoses can improve access to treatment and care, potentially improve disease outcomes, and end the “diagnostic odyssey” that many rare disease patients face.
  2. Increased awareness among health care providers: It is unrealistic to train clinicians on all 7,000 different rare diseases. But it is important to raise awareness on a broader level, and to ensure that clinicians are aware of specialists, support opportunities and care pathways for rare disease patients.
  3. Better coordination of care: Due to the complex nature of rare diseases, health care teams for these patients often include multiple specialists and clinicians. The burden typically falls on the patient or their carer, however, to manage communication among these providers. It is important to minimise this burden and to ensure that communication runs smoothly so that the right care can be provided in a timely manner.
  4. Improving access to specialist care, treatment, and medications: For those rare diseases that have established treatments, patients need timely access to achieve successful outcomes. As new and innovative treatment options are developed, it is also important that patients have access to them.

England’s plan allows the country to take steps towards achieving its overall goal: delivering improvements in diagnosis, awareness, treatment, and care – and creating lasting positive changes for those living with rare diseases.