Putting compassion into compassionate use

Compassion can be defined as “ feeling or showing sympathy and concern for others” 

RareiTi is founded on the premise that the best way to enable patients to access the medicines that they need, at the earliest opportunity, is to develop our programs from empathy and patient experience, rather than through the prism of the regulatory process.  

Yes, the regulators (FDA, EMA, ANSM, MHRA, PMDA, Minzdrav etc.) have effective regulatory pathways in place to FastTrack early patient access on compassionate grounds. However, this compassionate approach often does not materialise within the patient experience. 

Alarmingly, patients with rare diseases and their carers have their mental health and psychosocial needs overlooked. According to recent Findacure essay winner Anna-Lucia Koerling’s  ( “No Friends 1” – Orphanet Rare Disease Journal) “In a survey conducted by Rare Disease UK, 46% of patients, and 58% of their carers report never being asked about their mental health/wellbeing.” 

Furthermore, Global Genes reported “Rare disease patients report their disease caused;- Depression (75% in the US, 69% in the UK) , Anxiety and stress (86% in the US, 82% in the UK), Isolation from friends/family (65% in the US, 57% in the UK), Worry based on future outlook of disease (90% in the US, 91% in the UK)”

It is well-established that living with a rare disease has an impact on a patients’ mental health which should be taken into account when planning managed access programs and patient support services. 

It is our aim to co-develop RareiTi managed access solutions with patient groups holistically, and build person-centered solutions with long term benefits of real-world evidence data collection for the whole patient community.

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