Forty-eight per cent of rare disease patient groups represent patient interests to regulators. Participation in HTA and reimbursement reviews is most common (26%), while 14% facilitate equitable healthcare access and 11% influence healthcare policy and legislation.
48% of respondents from Rare diseases ‘Regularly’ or ‘Sometimes’ represent patient interests to drug regulators
Patient groups representing each Rare diseases sub-type
478 patient groups across 27 sub-types
interactions
The inclusion of AME [Atrofia Muscular Espinhal – Spinal Muscular Atrophy] in the expanded newborn screening programme [newborn heel-prick screening programme], under Brazilian Federal Law 14,154/2021, together with the relevant legislation at the municipal level in Curitiba and across the state of Paraná — and now its full implementation in Paraná.
Inclusão da AME na expansão do teste do pezinho, Lei 14154/21, além da legislação em Curitiba e no Paraná. E agora a efetivação no Paraná.
National rare-disease patient group, Brazil
interactions
TSA [Tuberous Sclerosis Association] works with NICE [National Institute for Health and Care Excellence], SMC [Scottish Medicines Consortium], and AWMSG [All Wales Medicines Strategy Group] to ensure patient voice is at the heart of any decision-making by regulatory bodies.
The TSA works with NICE, SMC, and AWMSG to ensure patient voice is at the heart of any decision-making by regulatory bodies.
National rare-disease patient group, UK
actions & advocacy
Letters were co-authored and submitted to AIFA [Agenzia Italiana del Farmaco – Italian Medicines Agency] at the request of paediatric nephrologists, seeking compassionate use authorisation for investigational medicinal products [for rare kidney conditions].
Lettere scritte ad AIFA insieme e su richiesta di nefrologi pediatrici per chiedere uso compassionevole di farmaci sperimentali.
National rare-disease patient group, Italy
actions & advocacy
…The organization’s flagship event - the annual Indo US Bridging RARE Summit brings together rare disease stakeholders, including scientists, patient advocates, government officials, biopharma executives, and policy experts who share a common goal: transforming the future of care for the 400 million people worldwide living with rare diseases.
International rare-disease patient group, USA
The percentages below highlight how the 227 patient groups interact with regulators and advocate for patient interests. As respondents may be represented in more than one area, percentages may not total 100%.
interactions
139 mentions
59 mentions
45 mentions
21 mentions
8 mentions
6 mentions
39 mentions
26 mentions
13 mentions
19 mentions
7 mentions
7 mentions
3 mentions
2 mentions
19 mentions
15 mentions
4 mentions
7 mentions
7 mentions
3 mentions
2 mentions
1 mention
actions & advocacy
49 mentions
31 mentions
13 mentions
5 mentions
46 mentions
25 mentions
12 mentions
6 mentions
3 mentions
23 mentions
9 mentions
8 mentions
2 mentions
2 mentions
1 mention
1 mention
12 mentions
6 mentions
6 mentions
8 mentions
5 mentions
3 mentions
2 mentions
2 mentions
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Our research covers 2,262 patient groups representing 100 countries and 45 therapy areas. The sample report shows you the depth of analysis for a single country or therapy area. To access any other reports, please get in touch.
Visit patient-view.com to gather more information or email: Alex Wyke, alexwyke@patient-view.com
