Research & Development Strategy
At Proveca we want to develop and license medicines for children. Currently, over half of the medicines children take are in the wrong format and/or are not licensed for their use. What do we mean by wrong format and not licensed?
- wrong format: this means that the drug given is not designed to be taken by children. This can result in issues with administration of the medicine and poor compliance. There might be also be excipients (additives) in the medicine which could be harmful to children. Examples of administering the wrong format to children include using medicines which contain alcohol or using adult tablets which need to be crushed.
- not licensed: this means that there may not be any clinical data to support the use of a product in children and the product has not been approved for such use. Many children using unlicensed medicines have chronic conditions which require long term drug treatment.
To find out more about excipients, the EuPfi (European Paediatric Formulation Initiative) provides a database (called STEP) for health care practitioners, where they can look up information for different excipients.
What is the STEP Database
STEP stands for Safety and Toxicity of Excipients for Paediatrics. The STEP Database is a user-designed resource that brings together the safety and toxicity data of excipients, from many various sources, and present them in one freely accessible resource. It holds (1) General Information, (2) Clinical Data, (3) Non-Clinical Data, (4) In Vitro Data, (5) Regulatory References and (6) Reviews in relation to safety and toxicity.
Click on the image to find out more:
To explain more about ‘How to identify and manage ‘problem’ excipients in medicines for children, please watch this white board explainer video (Please note at the time the paper was published and this video was created, all products mentioned contained the relevant excipients).
At Proveca we identify products which require any of the following:
- a new paediatric licence (new indication);
- an improved format of administration for better patient compliance for an existing indication
We then build the evidence base through clinical and pharmaceutical development. By working with clinicians, parents/carers and children, we are able to tailor the features and benefits of our products to the specific requirements of children.
We have a strategy to work with leading children’s hospitals and academic groups across Europe to identify unmet needs and to collaborate on research to help with our mission. Our research focus has the following themes:
- Design, develop and licence medicines specifically for children;
- Develop knowledge to inform better decisions and create novel paediatric formulations;
- Contribute to the education of healthcare professionals in paediatrics.
An example of our collaboration is a PhD research project we have with Aston University:
- The project focuses on the development of a novel excipient screening platform for Paediatric Oral formulations.
- Background: The lack of suitable medicines for the paediatric population means that unlicensed formulations are often prepared and administered by healthcare professionals, which entails exposing this vulnerable population to risks in terms of safety and clinical efficacy. Another limitation in paediatric medicine development is the direct application of knowledge and principles from adult studies, thereby completely disregarding the specific needs and requirements of paediatric patient and prescriber preferences and acceptability of medicines. Compounded with this problem is the lack of translatable dosage form development technologies that offer acceptable taste, high drug loading, convenience and minimal excipient load for paediatric medicine. The multitude of challenges restricting progress in paediatric medicine development include lack of understanding of dosage forms that offer dose flexibility, acceptability, desirability, safety and commercial feasibility. In addition, a limited choice of excipients for safe inclusion in paediatric medicines, together with taste acceptability issues, presents an additional layer of formulation difficulty. The overarching hypothesis of the proposal is that the establishment of an excipient screening platform will accelerate paediatric formulation development.
Created April 2021