A couple of weeks ago HCD staff attended an interesting online event: ‘Health Technology Assessment of Gene Therapies: Are Methods Fit for Purpose’. Facilitated by the Office of Health Economics (OHE). This was non-promotional meeting discussing a recent Pfizer funded, consulting report.

The OHE report finds 4 key challenges for Health Technology Assessment (HTA) bodies including:

  1. Initial assessment of clinical effectiveness using non-RCT trial design and appropriate outcome measures.
  2. Uncertainty regarding long-term outcomes measures. Which could be further evaluated by Value of Information, (VOI) analyses.
  3. Incorporating additional elements of value, especially severity but also accounting for any Disability Paradox.
  4. Assessment of costs for potentially transformative therapies delivered in maybe only one dose or a short regime. With the need for innovative payment schemes and budget impact models covering a lifetime perspective.

Highlighting that in 2021 over 1600 potential gene therapies were in companies research pipelines. 10 years ago, that number was only about 200. Most gene therapy research focuses on oncology, rare disease and metabolic disorders.

The report recommends improving the HTA through:

  • Broader acceptance of other forms of evidence
  • Use of expert input regarding treatment outcomes
  • Better appraisal of uncertain of missing evidence
  • Wider use of observational data e.g. RWE collected in patient registries
  • Developing a framework to elicit support from experts to inform model parameters

The report cites a Disability Paradox study in Haemophilia as an example of the importance of evaluating quality of life using alternative methods. In the cited paper the HCD team found patients had a higher preference value for 99% of EQ-5D-5L health states compared to the general public. The OHE report states: “It is important that this phenomenon is considered and accounted for during HTA evaluations”.

The online meeting also discussed Project Hercules as best practice in developing RWE collaboratively. Set up by the charity Duchenne UK, this initiative developed tools and evidence to support HTA and reimbursement decisions for emerging treatments. HCD were proud to have been involved with developing Burden of Illness studies in this large global effort to improve understanding in Duchenne’s Muscular Dystrophy (DMD).

HCD Economics has a range of methods that can be used to evaluate and elicit key information at an early phase. Including Early Economic Analysis such as VOI and Sheffield Elicitation Framework (SHELF) technique to elicit key data distributions and using Disease Proxies. These approaches are highly applicable for Rare Disease & Paediatric Patients.

For an informal discussion and overview slide deck detailing these methods please contact Anthony Woodhead: anthony.woodhead@primeglobalpeople.com