WP6 will group stakeholders into clusters and will be working to identify and address any evidence gaps which might delay decision-making by regulatory agencies, HTA bodies, and payers. Such activities will identify those factors which prevent patient access to innovative treatments for HM. The group’s work, building upon previously gained experience and other EU-funded projects and international initiatives, will lead to consensus-based decisions and facilitate interaction and consultation between all the relevant stakeholders.
Read about the Delphi Survey to Develop a Core Outcome Set (COS) for Acute Myeloid Leukemia. A COS constitutes the minimum set of outcomes that should be collected and reported in future clinical trials. Download the Delphi AML Study Protocol document to read more and to have an overview of the full outcome list in the annex.
- Providing support and advice to other WPs to ensure that all relevant perspectives (from HTA bodies, patients, regulators, policy-makers, etc.) are reflected;
- Engaging all stakeholders to collect input and ensure the long-term implementation of the project's outcomes;
- Creating an evidence framework to enhance decision-making processes, thereby facilitating centralized regulatory approval and national/regional HTA;
- Developing a clinical value framework to assess how effective new clinical interventions are and to assess their relative therapeutic value;
- Inviting input in terms of multiple viewpoints and requirements on the part of stakeholders;
- Fostering consensus among the stakeholders, thus facilitating the development of and access to innovative solutions and therapies for HMs;
- Integrating stakeholder participation and evidence;
- Providing guidance on methodologies for evidence generation and analysis of real-life evidence.
AEMPS, Amgen, Bayer, BFArM, Celgene, EHA, HULAFE, IBSAL, Janssen, Leukanet, MediUni Wien, Menarini, NICE, Novartis, Synapse, University of York.
WP6 aims to identify and address evidence gaps which can delay decision-making by regulatory agencies, HTA bodies, and payers, thus compromising timely patient access to innovative treatments for HMs. For this, a Policy Health Stakeholder Feedback Forum (PHSFF) has been established. PHSFF will act as a key platform for interaction and consultation between stakeholder groups. Collecting input and discussing viewpoints from patient organizations, hematologists/clinicians, regulators and HTB bodies will be crucial to shaping the work of HARMONY.
- The Policy Health Stakeholders Feedback Forum (PHSFF) was established and a community e-consultation tool for stakeholders was created;
Successfully implemented were:
- The active working group met in face-to-face sessions during two full-day workshops;
- Elements of the Phase 2 previous reimbursement decisions were implemented (products were selected across disease pillars; submission documents were identified; an extraction tool was developed; and pilot methods and tools were created);
- Data was successfully extracted from 11 HTAs, EMA, and the EUnetHTA (the European Network for Health Technology Assessment): 86 assessment/submission documents;
- In terms of the “proof of principle” study, stakeholders’ guidelines were created, and results began to be disseminated at international conferences;
Work on the development of the HM-specific evidence framework was initiated and a working group was established.
- Developed the Stakeholders Feed back Forum for the clusters: Patient organizations, Hematologists/clinicians, Medicines Authorities, HTA bodies and Pharmaceutical Industry;
- Completed a scoping document describing the HM-specific Evidence Framework.
In 2018, the Stakeholder Forum will continue to bring its support to WP2 for the development of overarching or common outcomes and specific outcomes to the individual HMs. It is the aspiration of WP2 and WP6 to see the development of a Core Outcomes Set for HMs. To be successful we need to obtain the viewpoints from patients’ organizations, hematologists/clinicians, regulators and HTA bodies.It will also provide input into WP7 (communication and dissemination) and WP8 (ethics and legal).
- Improving communications tools and strategies between members and WPs;
- Expanding coordination among all partners and stakeholders;
- Increasing collaborative tools in order to enhance planning, consultation, and communication, thus establishing the means of aligning expectations;
- Increasing support for WP2 as it continues its process of consulting with stakeholders;
- Continuing the Policy Health Stakeholders Feedback Forum activities;
- Populating the access value framework and work with WPs 2 to 5 to establish content and crossover;
- Initiating the assessment of multiple clinical-value frameworks;
- Organizing Masterclasses for patient, nurses and parent organizations in collaboration with WP7 and including the development educational materials;
- Taking steps to work on a proof-of-concept project on Big Data related to the European Medicines Agency’s qualifications for novel methodologies.
The other Stakeholder Forum objectives are to:
- Support any stakeholder consultations requested by all WP leads
- Involve pan-European patient organizations in myeloma, acute leukemia, childhood cancers, CLL, MDS and lymphoma to make sure HARMONY’s work in e.g. WP2, WP, and WP8 stays focused around patients’ needs and evidence.
The Access Evidence Value Framework aims at developing an HM-specific clinical and economic evidence framework. In 2018, steps to consider are:
- Conduct and analyze interviews done with Regulators/HTA/Payers;
- Extraction and analysis of key information from HTA/Regulatory dossiers related to HM drugs.
The Clinical Value Framework aims at developing an instrument able to quantify the added therapeutic value of new technologies for the treatment of HMs.
- This task will be kicked-off in 2018 with the involvement of all Task partners.
- From the kick-off meeting, plans and milestones for 2018 will be more detailed.
Download the PDF with the WP6 outlook for 2018.
Work Package Leadership