6 Recommendations for further data collection

6 Recommendations for further data collection

6.1 As a condition of its positive recommendation, the committee instructed that the following data should be collected using the BILAG registry to resolve uncertainties in a future review of this technology appraisal guidance:

  • Efficacy data:

    • clinical response measured by BILAG Index 2004 and SLEDAI‑2K

    • organ damage accrual using the SLICC Damage Index and BILAG Index 2004

    • use of corticosteroids.

  • Safety data:

    • incidence of serious adverse events: hospitalisation for infection, malignancy and death, other serious adverse events.

  • Patient-reported outcomes:

    • EQ‑5D

    • SF‑36

    • LupusQoL.

  • Other data:

    • demographics (age, gender, ethnicity)

    • belimumab treatment details: date started/stopped/restarted, dose and intravenous frequency, reason for discontinuation (as appropriate)

    • lifestyle questionnaire (for example, drinking, smoking, employment status)

    • patient diary (recording hospital admissions, visits to outpatients and medications)

    • clinical serology – autoantibody profiles

    • prior therapy

    • concomitant medications

    • comorbidities

    • laboratory parameters.

6.2 The committee specified that initial assessment in the BILAG registry should take place before starting a new biological treatment, or at re-treatment. Further clinical assessment should be undertaken at 3, 6 and 12 months then annually thereafter. Questionnaires should be given to patients every 6 months for 2 years then annually thereafter.

6.3 The committee expects the proposed managed access arrangement to include a section detailing specific requirements for patient consent.

6.4 The committee acknowledged the company's post-marketing commitment to investigate intermittent treatment with belimumab including time to flare from withdrawal of treatment and response to belimumab at re-treatment, and considered that these studies would be of value.

  • National Institute for Health and Care Excellence (NICE)