PP216 Indirect Treatment Comparison Assessment: An Improvement Intervention In The Scottish Medicines Consortium

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of the National Health Service Scotland based on pharmaceutical company submissions. As the appraisals are conducted close to the point of marketing authorization, there is often a lack of direct head-to-head data. In 2019, assessment of relevant comparative efficacy was informed via indirect treatment comparisons (ITC) in 55 percent (36/66) of submissions. While the ITCs are essential to the decision-making process, they are frequently incomplete.MethodsA focus group was conducted with the clinical assessment team (n = 11) to explore problems in the submission process and to identify areas for improvement. It was agreed that providing improved guidance to companies prior to submission may prevent future inconsistencies. A working group (n = 5) was tasked with identifying and implementing potential solutions. The group reviewed the focus group findings, relevant literature, and guidance from other organizations. Draft guidance was developed that was reviewed by two pharmaceutical industry representatives (SMC subcommittee members).ResultsFindings from the focus group highlighted issues broadly related to the incomplete presentation and reporting of ITCs. The improved guidance document outlined specific requirements in a checklist format for reporting and presenting the results of different ITC data. This guidance was published in February 2020. To evaluate the impact of the updated guidance and to identify any further changes required, a follow-up focus group and survey of industry representatives is planned for March 2021.ConclusionsThe aim of the ITC guidance is to provide pharmaceutical companies with direction to improve the quality and transparency of reporting, which will in turn improve the quality of HTAs and thus strengthen the recommendations provided by the SMC. The follow-up focus groups and survey will assess the impact of the guidance. It is acknowledged that the results of this process may be limited by the small sample size and short duration of the assessment.

Health in Digital World: A Regulatory Overview in United States

Adaptation towards digitalization in pharmaceuticals leads to the utilization and development of Artificial Intelligence (AI). Significantly it is reducing human workload with the help of an algorithm. Already AI is acting as a key in clinical trial, health care, quality management, manufacturing, product development, and management. Top pharmaceutical companies have adopted AI in different applications within the pharma sector. Different AI models like Machine learning, Artificial Neural Network, Deep Learning, robotics, and Natural Language Processing are being used in pharmaceuticals and healthcare systems. The Worldwide AI market is growing remarkably with a compound annual growth rate of 49.6% and is expected to reach $18,119 million by 2025. So, for better regulation, concerning safety, privacy regulatory strategy is heading towards a better framework. Different regulatory authorities like China, Europe, and United States (US) have adopted AI for economic and policy aspects. Emerging countries are using these tools for administrative work. US has begun implementing frameworks for AI adaptation, research, and development. The AI policy strategy started in 2016 with a series of workshops conducted under the Obama administration. Federal Food and Drug Administration (FDA) has also published draft guidance for regulatory oversight of AI and Machine Learning. In 2021 FDA published a draft regulation for software as a medical device. This review article provides a snapshot of AI implementation in pharmaceuticals and health care with the regulatory approach in the US.

Who are engaging in the nano-specific partner expert groups? An analysis of partner expert groups vs. expert groups

Background Consultation with partner expert groups (PEGs) is an important step in updating guidance under European chemical legislation concerning nanomaterials. Here, we briefly review the differences between PEGs and the broader use of expert groups in general, and scrutinise the five closed - and one ongoing-nano-specific PEGs to investigate stakeholder composition, level of engagement and the extent to which stakeholder comments resulted in revisions being implemented in ECHA’s draft guidance. Results Thirty-six different stakeholders were identified as having been involved in the closed PEG consultations, and an additional nine are currently involved in an ongoing PEG. For the closed PEG consultations, industry and trade associations (I&Ts) and member or associated member states (MSCAs) were the most represented groups, accounting for 15 and 13 members, respectively, whereas non-governmental organisations (NGOs) and European Union bodies (EUB) accounted for four members each. Interestingly, Academia was not represented. A total of 2700 comments were provided to ECHA’s draft guidance updates. Of these, MSCAs, I&Ts, EUB and NGOs accounted for 924, 876, 771 and 126 comments, of which 678, 494, 547 and 70 were adopted by ECHA, respectively. Eight stakeholders did not provide a comment. Conclusions Even though EGs and PEGs are not fully comparable, we find that they hold many similarities. The nano-specific PEGs are influenced by a few very active stakeholders that have the time, resources and motivation to engage extensively while some stakeholder groups are partly or completely missing. We recommend that ECHA provides funding opportunities for less resourceful stakeholders, in order to minimise the effects of scarce funding on engagement. Furthermore, we recommend broadening the list of accredited stakeholder organisations, thereby allowing for more diversity among stakeholders involved, e.g. Academia, and that ECHA provides a justification for inclusion of the PEG members.