Rare Disease Patient Experience

Goals

To understand rare diseases patient experience in Asia, Eastern Europe, West Africa, and LATAM in order to help guide training sessions.

Early Findings

  • Bulgaria has a national policy that is aimed at improving the patient experience of those suffering from rare diseases in the country. The national policy was developed in conjunction with health authorities, clinicians and patient representatives.
  • The national policy is expected to improve awareness of the disease in Bulgaria, access to treatment, and "to integrate medical and social approaches to rare disease patients and their families in Bulgaria."
  • However, a study into the rare disease landscape in Bulgaria showed that although the national policy on rare diseases has been adopted, the implementation is still limited leading to poor patient experience.
  • Bulgaria doesn't have a legal provision for early access to rare disease treatment for patients.
  • Turkey passed a national policy that is aimed at improving the patient experience of those suffering from rare diseases in the country, however, implementation of the policy has been delayed.
  • While there is no legal provision for early access to treatment for patients with rare diseases in Bulgaria, there is a "program available for patients with unmet medical needs meeting defined criteria."
  • In addition, Turkey’s national reimbursement scheme "enables both availability (through importation) and access to orphan drugs when these drugs are unavailable, unauthorized and inaccessible."

Proposed next steps:

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