title: Integrating health technology assessment requirements in the clinical development of medicines: the experience from NICE scientific advice.
abstract: OBJECTIVE
  The primary objective of the study was to analyse the proposed clinical development and economic evaluation plans for investigational medicinal products for which pharmaceutical companies have sought health technology assessment (HTA) scientific advice (SA).
  
  
  METHODS
  We have selected and analysed all the scientific advice procedures undertaken by National Institute for Health and Care Excellence (NICE) SA between 1 January 2009 and 3 December 2015 for investigational medicinal products. We have mapped the questions asked by the companies and the areas of advice highlighted in the advice reports to the sections of the NICE methods guide to the technology appraisals (2013).
  
  
  RESULTS
  An overwhelming proportion of SA procedures have addressed questions related to the clinical development and specifically the main pivotal efficacy studies. Approximately a quarter of the questions relate to the approaches to economic evaluation. Questions raised in European Medicines Agency-HTA procedures generally focus on clinical efficacy issues whereas cost-effectiveness ones tend to dominate in NICE-only procedures. Our analysis shows that the issues mostly discussed in the HTA SA are the choice of comparator, the generalisability of the clinical trial evidence to the NHS practice and the impact of the clinical trial outcomes on quality of life and survival. Less disagreement with the developers' plans was seen in the choice of clinical endpoints, population definition, position of the technology in the treatment pathway and study design.
  
  
  CONCLUSIONS
  Scientific advice is designed to improve the quality of evidence and approaches to evidence generation for future regulatory approval and HTA evaluation. Our experience to date suggests that payer requirements are inconsistently integrated in the clinical development programmes. More efforts should be dedicated to demonstrating the clinical value of new medicinal products to patients and key decision-makers.
authors.affiliation: Office of Health Economics Research, Southside, 7th Floor, 105 Victoria Street, London, SW1E 6QT, UK.
authors.forename: François
authors.initials: F
authors.lastname: Maignen
authors.affiliation: Scientific Advice, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence, 10 Spring Gardens, London, SW1A 2BU, UK. leeza.osipenko@nice.org.uk.
authors.forename: Leeza
authors.initials: L
authors.lastname: Osipenko
authors.affiliation: Scientific Advice, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence, 10 Spring Gardens, London, SW1A 2BU, UK.
authors.forename: Pilar
authors.initials: P
authors.lastname: Pinilla-Dominguez
authors.affiliation: Scientific Advice, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence, 10 Spring Gardens, London, SW1A 2BU, UK.
authors.forename: Emily
authors.initials: E
authors.lastname: Crowe
mesh_terms: D003362:Cost-Benefit Analysis; D017408:Guidelines as Topic; D006801:Humans; D013673:Technology Assessment, Biomedical / Q000458:organization & administration* / Q000592:standards; D013812:Therapeutics; D014481:United States
keywords: Health technology assessment; Medicinal products; NICE; Scientific advice