HOW HAS THE SMC PATIENT AND CLINICIAN ENGAGEMENT (PACE) PROCESS BEEN USED IN ASSESSMENTS OF END-OF-LIFE MEDICINES AND MEDICINES TO TREAT VERY RARE CONDITIONS?

Author(s)

Akesson C1, Jones C2, McKendrick J3, Grant C1
1PRMA Consulting, Fleet, UK, 2PRMA Consulting, Hampshire, UK, 3PRMA Consulting Ltd, Fleet, UK

OBJECTIVES: To identify decisions by the SMC using the PACE process since the scheme was introduced and identify issues that influence a successful outcome. METHODS: Assessments that used the PACE process were identified from SMC briefing notes between May 2014 and June 2015. Key points expressed were classified into clinical issues or added value to the patient or carers. RESULTS: Twenty assessments were identified that used the PACE process: 17 in oncology indications and one each in hypertension, infection in cystic fibrosis, and myelofibrosis/splenomegaly. Ten were accepted (seven with a patient access scheme [PAS]) – three that met orphan criteria, one that met end-of-life criteria, and six that met both.  Five products were accepted with restrictions (four with a PAS) and five were rejected (one with a PAS). All assessments discussed clinical issues, including health-related benefits (17/20), health-related quality of life (HRQL) benefit related to symptoms (13/20), and reduction in adverse events (11/20). Sixteen provided data on the severity of the condition (impact on patients, carers and families, and/or assessment of severity); 9 characterized the unmet need; 13 considered the most appropriate position for the medicine in the pathway of care and/or identified specific patient groups. Seven assessments argued for added value to patients (convenience of treatment) and value to patient’s family and carers in terms of impact on family life (6), requirements for carers (2), and carers’ ability to work (1). CONCLUSIONS: PACE assessments to date have largely focused on clinical issues and patient’s quality of life, with limited attention as yet to the benefit to patients’ families/carers. PACE appears to be meeting the SMC’s objective of providing a forum for patients and clinicians to present arguments regarding the wider benefits of a drug that may not be easily captured in the standard assessment process.

Conference/Value in Health Info

2015-11, ISPOR Europe 2015, Milan, Italy

Value in Health, Vol. 18, No. 7 (November 2015)

Code

PCN331

Topic

Health Service Delivery & Process of Care

Topic Subcategory

Health Care Research

Disease

Multiple Diseases, Oncology, Rare and Orphan Diseases

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