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Research & Development

Research & Development Strategy

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At Proveca we develop and license medicines for children with a core focus on treatments within cerebral palsy, epilepsy and heart failure. We have also collaborated with the European Medicine Agency’s Paediatric Committee to gain a Paediatric-Use Marketing Authorisation (PUMA) for the symptomatic treatment of severe sialorrhoea (chronic drooling) in children and adolescents aged 3 years and older with chronic neurological disorders.

Currently, over half of the medicines children take are in the wrong format and/or are not licensed for use in children. 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 may also be excipients (additives) in the medicine which could be harmful to children. Examples of administering a medicine in the wrong format to children includes using medicines containing alcohol, or using adult tablets which need to be crushed, risking inconsistent dosing and under or over dosing.
  • Not Licensed: this means that there may not be any clinical data to support the use of a product in children and as such, the product has not been approved for use in children. 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 compiles the safety and toxicity data of excipients from a range of sources and presents them in a single, 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 for administration, to improve patients compliance, for an already 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 our products features to meet the specific requirements of children.

Our current strategy is to work with leading children’s hospitals and academic groups across Europe to identify unmet needs and collaborate on research projects to help with our mission. Our research focus has the following themes:

  • Design, develop and licence medicines specifically for children;
  • Develop our 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 risks exposing this vulnerable population to excipients which may not be safe for them. Another limitation in paediatric medicine development is the vast majority of clinical studies are conducted in adults, thereby completely disregarding the specific needs and requirements of paediatric patients, and therefore limiting suitable prescribing options. Compounded with this problem is the lack of translatable dosage form development technologies that offer acceptable taste, innovative dose design, convenient dosing and minimal excipient load for paediatric medicine. The multitude of challenges restricts progress in paediatric medicine and 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.

Dedicated to improving children’s medicines

Ways you can work with us:

Contact us at dream@proveca.com to help us identify and meet unmet paediatric needs

Can you think of any of the following:

  1. product suggestions
  2. therapeutic indications in paediatrics which require attention
  3. paediatric products which you feel could be in a much better format for  children and why
  4. potential collaborative research projects or publications
  5. other thoughts you have. Please just get in touch with us and let’s see what we can do together

UK-NON-22-0036

Reviewed September 2022

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