Coming together to address unmet medical needs in children (Guest blog)
18.02.20
Unmet medical need (UMN) describes situations where treatment options can be improved or worse, where no treatment options exist. Discovering, developing and delivering innovative and effective treatments to meet these needs is what drives our industry. When it comes to unmet needs in children everyone feels that drive even more intensely.
With that in mind, EFPIA partnered with EFGCP to organise a multi-stakeholder workshop to explore how to best define and tackle unmet needs in children, in order to provide a basis for strategic decision making on paediatric medicine development.
The objective behind this one-day workshop was to find a broadly accepted approach to defining unmet medical needs among paediatric patients, in order to make better and safer medicines for children, and to make sure they are available and affordable. This is not in itself a new ambition, but the workshop was novel in its approach, with a primary focus on patients, involving all stakeholders and mapping out a plan for action. The participants were united in their determination to go beyond discussion to agreeing a pragmatic process for all stakeholders to take effective action.
The design and participation at the workshop was centred around a collaborative approach, coming together to evolve the process and have real impact. The timing was important as over the last two years, the EU reviews its paediatric and orphan medicine incentive schemes, with a focus on paediatric needs as part of the Commission/EMA action plan.
The outcome of the workshop was an outline of potential guiding principles for defining UMN in children, and a programme for testing the principles in selected therapeutic areas over the course of 2020.
Among the key recommendations were:
- As no one-size-fits-all approach can work at present, progress should be made through addressing UMN by speciality, with an accent on treatment categories or gaps, rather than individual products.
- The therapy area models should be explored in further workshops, under academic leadership, but with full engagement by all stakeholders.
- They should take account of treatment developments in each specialty, but go beyond issues of PIP* and PSP**, in the EU and US respectively, to a broader review of drug research and diagnosis.
- Their aim should be to formulate and validate principles for defining UMN in that speciality – high, low or intermediate, and irrespective of feasibility.
- The results of the workshops would feed into the generation and subsequent agreement on a more widely valid framework by the end of 2020.
Defining unmet medical needs in children is one of the building blocks in meeting the needs of children across Europe and beyond. This is one of the ongoing activities to address the Commission/EMA action plan for better implementation of the Paediatric Regulation.
That’s an aim we are committed to achieving globally and look forward to working with stakeholders in future workshops.
*Paediatric Investigation Plan; **Paediatric Study Plan.
