Guiding the development of MND therapeutics with a target product profile
We are establishing a patient-focused target product profile (TPP) to act as a blueprint to guide the development of treatments for MND.
Overview
MND remains an area with significant unmet medical need. Despite advancements in research and investment, an effective disease-modifying therapy is still out of reach for people living with MND. We are collaborating with experts to develop a TPP for MND that is aligned with the needs and priorities of people with MND.
What is a target product profile (TPP)?
A TPP is a strategic planning tool that defines the ideal characteristics of a product for a specific disease or condition. It specifies the product’s intended use, target population, and key attributes, such as safety and efficacy. TPPs establish R&D goals for funders and developers and steer therapeutic development to address unmet needs.
Aims and impact of the target product profile (TPP)
Define desirable drug profiles
Establish clear, patient-centred standards for MND drugs’ desirable and minimally acceptable therapeutic qualities, including efficacy, safety, and ease of use.
Focus on patient needs
Ensure treatment goals meet the specific needs of MND, from dosing regimens to practical considerations, such as treatment monitoring and caregiver support.
Guide drug development
Serve as a foundation for developing patient-focused therapeutics that can quickly integrate into healthcare systems and reach diverse patient populations.
Stimulate innovation
Provide an opportunity to build consensus for future MND treatment requirements, steer industry efforts and shape funding calls and other translational initiatives.
MND target product profile (TPP) development roadmap:
Phase 1: defining the scope and objectives
- Landscape analysis: we are performing this analysis to understand the unmet need, limitations, care pathways and priorities for MND drugs.
- Stakeholder identification: we are identifying stakeholder groups to steer the development of the MND TPP.
- Stakeholder engagement: through workshops, focus groups and surveys, we will develop the TPP framework to include the target patient population, desired treatment regimen and clinical outcomes. Drug development metrics, including clinical trial endpoints, may be included to inform research goals.
Phase 2: drafting and refining
- Initial draft: we will develop the preliminary draft of the TPPs to include the minimally acceptable and desirable therapeutic criteria.
- Iterative refinement: using this draft, we will gather feedback from key stakeholder groups and refine the TPP through interviews, surveys, and workshops.
Phase 3: consensus building
- Finalisation: we will host a roundtable with experts to build a consensus TPP.
- Communication strategy: we will develop a dissemination strategy to maximise the adoption and influence of the TPP.
Phase 4: launch and ongoing refinement
- Launch and integration: disseminate the TPP and integrate into ongoing R&D and funding efforts.
- Future updates: to reflect advances in research and the therapeutic needs of people with MND, we will regularly revisit the TPP with consensus surveys and working groups as required.
Want to help us develop the MND TPP?
We are looking for stakeholders to participate in workshops, surveys and focus groups.
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