Our news coverage in 2023 continued with Prime HCD’s focus on our commitment to generating evidence to inform decisions that deliver health equity.

Throughout the year we have highlighted areas that are often overlooked components of Health Technology Assessment such as informal care:

  • Prime HCD believe that where family responsibility is predominant in social care, it is crucial to acknowledge the importance of factoring in informal care costs.
  • This care involves various tasks, such as aiding with daily activities and medication, consuming considerable time and commitment from caregivers.
  • Valuing this unpaid care poses challenges, with commonly used methods often neglecting the relational aspect.
  • The National Institute for Health and Care Excellence (NICE) recommends considering carer Health-Related Quality of Life (HRQL) in economic evaluations, yet only a small percentage of NICE assessments incorporate it.

Care for those with dementia often falls on informal caregivers, primarily family and friends, with an estimated 700,000 informal carers in the UK in 2019. On World Alzheimer’s Day we reported:

  • Dementia is estimated to affect 944,000 people in the UK, with prevalence projected to rise significantly by 2050.
  • Despite its widespread impact, formal diagnoses remain low indicating under recognition.
  • Providing routine care for dementia patients takes a toll on caregivers' mental and physical health, leading many to give up work.
  • The economic burden of dementia in the UK was estimated at £25 billion in 2021, with a predicted rise to £47 billion by 2050. Despite this, detailed evaluations of unpaid care and the broader societal impact of dementia are lacking in economic assessments, highlighting a need for more comprehensive research.

Throughout 2023 we continued to publish news articles on areas of unmet need in the following rare diseases:

1. Gaucher Disease Type 3 (GD3) – Quality of Life Challenges
Research reveals substantial negative effects on the quality of life (QoL) for both Type 3 GD patients and their caregivers, covering social support, sleep disturbances, overall QoL, mental health, and treatment burden. Despite progress in understanding GD, unanswered questions remain, urging tailored research for Type 3 GD to address unique challenges, including comprehensive impact assessment and understanding patient preferences for improved well-being.

2. Sickle Cell Disease (SCD)
Ensuring accessibility and affordability of promising gene therapies, particularly in low and middle income countries, remains crucial in SCD. It is essential the global burden of SCD is understood comprehensively.

3. Glioblastoma Multiforme (GBM) – Challenges in therapy development
Key challenges include difficulties in drug delivery due to the blood-brain barrier, recruitment challenges in clinical trials, and a lack of specific patient-reported outcome measures. In addition, neuro-cognitive side effects further complicate health-related quality of life data collection, hindering the evaluation of treatment cost-effectiveness and potentially limiting support services for patients and their families. Overcoming these challenges is essential for advancing successful GBM treatments.

4. Haemophilia
We also continued in our commitment and development of research in haemophilia. On World Haemophilia Day we said a big thank you to everyone who has helped us with our CHESS studies.

With the approval of the first two gene therapies for Haemophilia A and B, we highlighted the increasing complexity of the haemophilia treatment landscape and the posed challenges for healthcare systems in terms of access and reimbursement. Prime HCD has explored:

  • The challenges faced by payors, addressing topics such as the high cost of cell and gene therapies & pricing strategies.
  • Accounting for indirect and societal costs
  • Research initiatives in Latin America and Asia where haemophilia and rare disease populations in general may be underserved.

We’d like to extend our gratitude to all our research and industry partners, plus all the amazing co-authors we’ve collaborated with over this last year. Thank you! We look forward to continuing collaborative and engaging research in 2024.