Tobias Polak

Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 111 6♦ and the maximum dose (2 x 200 mg) given resulting in an ICER of £88,788 per QALY gained.’ Manufacturer submission, Intervention and comparators costs, TA268 Treatment experience NHS professionals share their opinions and experience on the technology appraised in expert committee meetings. In the appraisal of patisiran for treating hereditary transthyretin amyloidosis, the Head of the National Amyloidosis Centre (NAC) is asked ‘how data on real-world experience in this condition compare with clinical trial data?’. His response is: ‘The experience of my colleagues at the NAC treating patients through compassionate access (over one year) and Early Access to Medicine Schemes has been extremely favourable. Remarkable clinically significant improvements of well-being and function have occurred in a majority of cases, including regaining the ability to walk unaided.’ Clinical expert statement, HST10 Patients, caregivers, or patient group representatives are also provided the opportunity to share their experience with the appraised treatment. The assessment of nusinersen for treating muscular atrophy sparked comments from parents with children that suffer from this disease: ‘My son is currently receiving Spinraza at Gosh for type 1c SMA. He was lucky enough to be included into the expanded access program for a select group of children. Since receiving his treatment we have watched the transformation of a seriously weakening child to a thriving boy who has gained significant progress in his motor function and health, we are continually amazed by his progress. He starts preschool in the coming weeks, an achievement we never thought possible. (…)’ Patient/caregiver stakeholder comment, TA588 The above provides qualitative examples of EA usage in NICE appraisals. To further illustrate how expanded access data are appraised by the manufacturer, ERG and NICE committee, and what the advantages and limitations of its use may be, a detailed discussion of the top five appraisals in which the search terms most frequently occurred can be found in the Supplementary Material. This includes representative examples in the areas of hemato-oncology (e.g., prostate cancer, follicular lymphoma) and rare diseases (e.g., spinal muscular atrophy).

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