About PROINSIGHT

To help sponsors build their endpoint strategies, Mapi Research Trust launched the PROINSIGHT™ database in 2017 as a way to inform users about recommendations characterized in terms of Clinical Outcome Assessments (COAs). This database centralizes and summarizes published regulatory recommendations concerning the use of COAs to determine treatment benefits in drug development and approval. The PROINSIGHT™ database is a supplementary option within the PROLABELS™ subscription.

In some clinical settings, COA strategies should reflect both regulatory and payer perspectives. Therefore, the scope of PROINSIGHT™ was extended to include guidelines that give recommendations on the assessment of health technologies. These guidelines consider effectiveness and safety dimensions, but also costs and economic implications, ethical, social, cultural and legal issues, organizational and environmental aspects.

Objectives:

• To give access to guidelines on the development of drugs, drug treatments, and methods for economic evaluation
• To provide information on concepts and COAs that should be considered to assess the impact of the disease on the patients’ life
• To easily capture regulatory COA requirements in clinical settings

The landscape of drug approval and pricing reporting is a complex one. Private, non-profit and governmental organizations, each following diverse missions, are releasing reviews and recommendations which can help pharmaceutical industry and researchers to better understand or define their relevant COA strategies in clinical settings.

Guidelines released by the following entities are reviewed for inclusion in PROINSIGHT™:

Drug Approval regulatory agencies:

• Europe - EMA: EMA Clinical Efficacy and Safety Guidelines
• USA - FDA
  • FDA Clinical/Antimicrobial Guidance
  • FDA Clinical/Medical Guidance
• Canada - Health Canada: Application and Submissions for Drug Products

Health technology assessment agencies:

• Canada - Canadian Agency for Drugs and Technologies in Health (CADTH): Methods and Guidelines - It provides health care decision-makers with reviews, recommendations on new and existing health technologies, and health technology assessments. It releases drug reports, Guidelines (disease-specific and generic) and Health Technology Assessments.
• Europe - European Network for Health Technology Assessment (EunetHTA): Assessments REA - Guidelines released by EunetHTA report a set of European-wide and non-disease specific recommendations for the selection and assessment of clinical endpoints when completing a Relative Effectiveness Assessment (REA).

Healthcare stakeholders’ boards:

• UK – NICE (National Institute for Health and Care Excellence): NICE Clinical Guidelines - Centre for Guidelines (CfG) releases guidelines on the appropriate treatment and care for people with specific diseases and conditions.
• Germany – Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWIG): IQWIG Systematic Reviews of Guidelines - IQWIG compiles scientific reviews to facilitate the decision-making of the Federal Joint Committee (G-BA).

Guidelines are reviewed according to the following criteria:

Inclusion criteria:

• Guidelines on the development of drugs, drug treatments, and methods for economic evaluation
• Mention of any COA (PRO, ClinRO, ObsRO, PerfO) strategy in a clinical trial setting: concept (quality of life, symptoms, functioning etc.) and COA, if stated, that should be considered to assess the impact of the disease on the patients’ life
• Documents in English (For IQWIG, our team of experts review ‘extracts of the final report’ drafted in English)

Exclusion criteria:

• Health technology assessment reports of drugs or devices
• Guidelines on devices

Following this review process, the selected guidelines are scanned and the identified concepts (quality of life, symptoms, functioning etc.) and/or COA are described and made accessible through a PROINSIGHT™ subscription. As in PROLABELS ™, objective measures such as biomarkers and survival are listed if at least one COA is mentioned in the guidelines.

For included guidelines, the following information can be accessed according to its availability in the source document:

Guidelines Description

• Health authority, date of publication, guideline status (draft, final/adopted), guideline category, link to the source document
• Therapeutic area and therapeutic indication
• Guideline’s purpose
• Target population
• Efficacy/study design, statistics and results presentation considerations

Targeted Patient Population

For each targeted technology, the following can be retrieved:

• Population inclusion and exclusion criteria
• General consideration on efficacy/study design
• Information about special populations
• Statistical considerations
• Guidelines for the presentation of the results

For each concept of interest:

• Endpoint positioning and definition
• Type of Outcome Assessment (OA)
• Name of the COA (if stated)
• Type of measure recommended (VAS, NRS, Questionnaire, other, etc)
• Rating: for each concept of interest (excluding those representing either biomarkers or survival), a rating provided by our researchers is assigned according to the following categories: 
  • A. The COA is specifically identified and is publicly (not necessarily freely) available with documented measurement properties in the recommended context of use
  • B. The COA is specifically identified, is publicly available, but documented measurement properties are NOT available in the recommended context of use
  • C. The type of instrument (e.g. a visual analog scale) is recommended, but no specific COA is recommended;
  • D. No COA recommendations appear for this concept of interest
  • E. The COA is recommended but no concept of interest was identified
• Guidance citations

Safety

Safety COA considerations are included if available.

Related Guidelines

In this tab, any guidelines for the same indication published by other regulatory bodies can be accessed.

The oldest guideline is dated 1977 and the on-going review includes new and revised guidelines, released by the agencies listed above, since then.