👁️‍🗨️ Rare Retinal Disease

Westminster Hall

🌶️ 🌶️ 🌶️ 🌶️ 🌶️

In a passionate parliamentary session, MPs debated the urgent need for innovation in treating rare retinal diseases, highlighting the profound impact on individuals and families. Jim Shannon emphasized the necessity for quicker diagnoses and improved access to treatments, while Shockat Adam shared heart-wrenching stories illustrating the life-altering effects of these conditions. The session stressed the importance of NICE’s role in approving new treatments and the government’s commitment to advancing research and care, with a focus on gene therapies like Luxturna. MPs urged for continued momentum in the UK’s rare diseases framework to ensure those affected are not left behind.

Summary

  • Debate Focus: The session focused on innovation in the treatment of rare retinal diseases, which affect around 25,000 people in the UK.

  • Personal Impact: MPs shared personal stories highlighting the life-changing effects of these diseases. For example, stories included a mother worried about pregnancy outcomes and a boy whose dream to play rugby was shattered due to his condition.

  • Importance of Eye Health: The debate emphasized the critical role of regular eye tests, as they can lead to early detection not only of eye-related issues but also other serious health conditions like tumors.

  • Research and Development: The need for continued research and development into treatments like gene therapies was discussed. Gene therapy, Luxturna, was cited as a recent successful example.

  • UK’s Role in Research: The UK is considered a world leader in genomic diagnosis, with active research projects funded by the National Institute for Health and Care Research aimed at developing new treatments for rare retinal diseases.

  • Challenges in Accessing Treatment: The debate touched on the difficulties in accessing treatments for rare diseases, especially concerning NICE’s (National Institute for Health and Care Excellence) appraisal pathways and the need for a more tailored approach to rare diseases.

  • Mental Health Support: There was a call for increased mental health support for those affected by these conditions, as many experience anxiety, stress, and a loss of confidence.

  • Government Commitment: The government expressed its commitment to improving the lives of those with rare diseases through the UK rare diseases framework, which focuses on faster diagnosis, increased awareness, better care coordination, and enhanced access to treatments.

  • Future Plans: The government plans to refresh the UK rare diseases framework in 2025 and continue to engage with the rare diseases community to address challenges and improve outcomes.

  • Role of NICE: NICE’s role in approving treatments was discussed, along with a need for transparency and consistency in evaluating treatments for very rare diseases. A public consultation on these matters is ongoing.

Divisiveness

The session demonstrates minimal disagreement, with participants largely aligned on the importance of addressing rare retinal diseases. Key observations include:

  • Consensus on urgency: All speakers emphasized the need for improved funding, research, and regulatory flexibility to support patients.
  • Shared critiques of NICE processes: Multiple MPs highlighted concerns about the HST pathway criteria, but these critiques were framed as collective challenges rather than partisan disputes.
  • Cross-party collaboration: Contributions from DUP, Labour, Lib Dem, and Conservative members focused on shared goals (e.g., genetic testing expansion, mental health support) without ideological clashes.
  • Ministerial alignment: The Minister acknowledged concerns and outlined ongoing governmental efforts, avoiding defensive posturing.

The session prioritized problem-solving over conflict, with differences limited to nuances in policy implementation rather than fundamental disagreements.