QUALITATIVE CONTENT ANALYSIS OF RESPONSES TO THE NICE PUBLIC CONSULTATION ON EXA-CEL FOR THE TREATMENT OF PATIENTS WITH SEVERE SICKLE CELL DISEASE
Author(s)
Adeline Durand, PhD1, Josef Paris, MSc1, Chloe Bost, PharmD1, Emily Evans, MPA2, Nancy Touba, MPH2, Gabriela Vega-Hernandez, BSc, MSc1.
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2OPEN Health, New York, NY, USA.
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2OPEN Health, New York, NY, USA.
OBJECTIVES: NICE received a significant (280) number of responses to their consultation on the draft guidance of exa-cel for the treatment of patients with severe sickle cell disease (SCD). The objective of this qualitative content analysis was to define the key domains and concepts captured during the consultation.
METHODS: Responses submitted during the public consultation period (14th March to 11th April 2024) were extracted from the NICE committee papers for TA10441 and imported into Atlas.ti software2. Attribute identification was conducted targeting relevant domains and concepts in SCD. Respondents were categorised by their relationship to SCD. Domains were defined as higher-order categories and concepts as sub-codes within domains; this framework was validated through manual testing on a sample of 7% of responses before all available responses were analysed.
RESULTS: Of 280 responses, 148 (52.9%) reported their relationship to SCD: 49 (17.5%) were people living with SCD, 42 (15.0%) were family members, and 22 (7.9%) were caregivers. Other respondents included clinicians (5%), patient advocacy groups (3.9%) and parliamentary groups (1.1%). Eight domains were identified: quality of life, complications, unmet need, mortality, caregiver/family burden, health inequalities, disease burden and systemic concerns. The most frequently reported concepts across all domains were ‘Requests for NICE to make exa-cel available’ (n=179/280, 63.9%), ‘Current inequality’ (n=95/280, 33.9%), ‘Feedback on NICE evidence assessment’ (n=92/280, 32.9%), ‘Need for improved QoL’ (n=80/280, 28.6%), ‘Reduced life expectancy’ (n=80/280, 28.6%) and ‘Pain’ (n=79/280, 28.2%).
CONCLUSIONS: The analysis showed that the main concern of the SCD community during the consultation was access to exa-cel. Responses emphasised the burden of SCD complications, the impact on emotional and physical wellbeing and the lack of effective new therapies in a population already affected by persistent health inequalities. This public consultation highlights the critical role of patient communities in decision making and describing the realities of a rare disease.
METHODS: Responses submitted during the public consultation period (14th March to 11th April 2024) were extracted from the NICE committee papers for TA10441 and imported into Atlas.ti software2. Attribute identification was conducted targeting relevant domains and concepts in SCD. Respondents were categorised by their relationship to SCD. Domains were defined as higher-order categories and concepts as sub-codes within domains; this framework was validated through manual testing on a sample of 7% of responses before all available responses were analysed.
RESULTS: Of 280 responses, 148 (52.9%) reported their relationship to SCD: 49 (17.5%) were people living with SCD, 42 (15.0%) were family members, and 22 (7.9%) were caregivers. Other respondents included clinicians (5%), patient advocacy groups (3.9%) and parliamentary groups (1.1%). Eight domains were identified: quality of life, complications, unmet need, mortality, caregiver/family burden, health inequalities, disease burden and systemic concerns. The most frequently reported concepts across all domains were ‘Requests for NICE to make exa-cel available’ (n=179/280, 63.9%), ‘Current inequality’ (n=95/280, 33.9%), ‘Feedback on NICE evidence assessment’ (n=92/280, 32.9%), ‘Need for improved QoL’ (n=80/280, 28.6%), ‘Reduced life expectancy’ (n=80/280, 28.6%) and ‘Pain’ (n=79/280, 28.2%).
CONCLUSIONS: The analysis showed that the main concern of the SCD community during the consultation was access to exa-cel. Responses emphasised the burden of SCD complications, the impact on emotional and physical wellbeing and the lack of effective new therapies in a population already affected by persistent health inequalities. This public consultation highlights the critical role of patient communities in decision making and describing the realities of a rare disease.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA109
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)