Biologic Therapies in Inflammatory Joint Diseases: Models for
Decision Making
This workshop was held as a collaboration between Arthritis
Research UK and the MRC Network of Hubs for Trials Methodology
Research.
Date: September 1, 2010
Venue: Royal Institute of British
Architects, London
Summary
This workshop was attended by rheumatologist clinicians,
statisticians, health economists, representatives from NICE, and
Hub members. Presentations and discussions focussed on the health
economic models used to model the outcome of treatment of
inflammatory joint diseases with the newer biologic drugs for
rheumatoid arthritis (RA) and related disorders. One of the major
drivers for this workshop from the rheumatological community was
the lack of randomised ‘head to head’ trial data between the
licensed agents. There was discussion of the use of indirect
comparison approaches to existing data and the use of new
methodologies such as Value of Information analysis, in the
prioritisation and design of further research.
The group acknowledged that Value of
Information (VOI) analysis has the potential to inform decisions on
research priorities, and on the design of future studies. For
example, VOI could be used to assess the value of developing an
Early Rheumatoid Arthritis National Database to capture data on
treatment of patients. It could also be used to inform
investigators about the value of head-to-head trials of biologics,
and the respective value of small, non-inferiority trial compared
to larger trials intended to demonstrate superiority.
The group appreciated, however, that in RA
there is no consensus on how the natural history of inflammatory
joint diseases should be modelled. What are the key outcomes? And
how are they inter-related both in terms of state and change?
Without reliable cost-effectiveness models which clinicians
understood and could sign up to, research priorities and study
designs suggested by VOI analysis would be unlikely to command wide
support in the academic arthritis community. Specific areas that
need to be addressed are the incorporation of drug safety in the
models, the changes during treatment in one of the key current
outcomes in cost effectiveness analyses: the Health Assessment
Questionnaire (HAQ) for disability, and the impact of withdrawing a
treatment. Modellers agreed that cost-effectiveness results were
sensitive to the way these particular aspects were modelled. There
was also uncertainty about the best way of defining response to
treatment, and considerable lack of agreement on the need for
individual patient models.
As there is currently no consensus view on
model assumptions, structure or outcomes chosen, the group agreed
that it would be reasonable to form a Clinical Studies Group that
could work towards finding consensus on the model inputs and
methodology. This should be a collaborative exercise involving
modellers and clinicians, with involvement of NICE.
Click on the links below to view the
presentations from the workshop:
Use of
Biologic Therapies in RA
Indirect and Mixed Treatment Comparisons in Arthritis
Role of Value of Information Methods in Arthritis
Economic Models of Biologic Therapies
The Sheffield Model
The
Birmingham Model
Cost effectiveness of anti-TNF's for psoriatic arthritis
A
clinican's view of the models