Economic burden of rare bone diseases

Anmerkungen:

• Want to keep track not only of diagnosis (FD, etc) but also of clinical symptoms because they can be very wide, "from incidental single bone cysts to symptomatic multiple bone cysts with severe hormonal disorders requiring lifelong orthopaedic and endocrine care".  If we later find a way to identify severity somehow, even by resource use, that could be useful.

1. Patient dairies
   1. Financing

      Anmerkungen:

      •  Might want to run a pilot to get an idea of whether private instance or payments play an important role, and if so, when collecting use of resources, would want to ask who bears the cost: out-of-pocket, private insurance or NHS
   2. Consultations
   3. Drugs
   4. Other?
   5. Frequency
      1. Weekly?
   6. Medium
      1. Web
      2. Post?
   7. Prospective
2. GP records
   1. Retrospective
3. Carers
   1. Questionnaire
4. Types
   1. Orthopaedic
   2. Endocrine
   3. Pharmacological
   4. Rehabilitation
      1. Physio
      2. OT

1. Patient dairies
   1. Interventions
2. HES
   1. Validate Patient dairies

1. EQ-5D-5L
2. SF-36
3. VAS during previous 5 years?

   Anmerkungen:

   • Interesting to correlate to resource use over time

1. Funding source
2. Consultations / Medication
   1. Type of health care staff
3. Primary/Secondary/Home care
4. Proximity to centre of excellence?
5. Severity?
6. Deprivation?
7. Productivity loss / Absentiism

1. Overall economic burden
2. By clinical symptom or severity?

   Anmerkungen:

   •  Idea would be to be able to produce general economic burden of the disease, but also to be able to tell how much of that burden comes from the care of different groups according to clinical symptoms or severity? 
3. By who bears burden

   Anmerkungen:

   •  Produce economic burden and then discriminate by who bears the cost (NHS, patient, private insurance?)

1. Patients
2. Carers
3. NHS

1. Absentiism
2. Carers
3. Welfare

1. Costs by disease by funding source

   Anlagen:

   • Graph 1
2. Cost (by disease) (by funding source) by proximity to centre of excellence
3. Percentual distribution of costs amongst funding sources by severity deciles (by disease)

   Anlagen:

   • Graph 3
4. Percentual distribution of costs amongst funding sources by disease
5. Histogram of costs identifying different diseases inside the distribution

   Anmerkungen:

   • Include OA as a reference? Problem would be number of observations... so mention the mean (median, range) value as a reference point

   Anlagen:

   • Graph 2
6. Histogram of costs for each disease identifying different patient subgroups inside the distribution

   Anlagen:

   • Graph 2

1. Glasgow
2. Edinburgh
3. Manchester
4. Birmingham
5. Bristol
6. Cambridge
7. Oxford
8. Southampton
9. Stanmore
10. London