Scientific Services
Orphan Drug Designation
An important aim of the European regulation on Orphan Medicinal Products is to promote the development of
products for serious rare diseases, or for serious diseases where without incentives it is unlikely that the
marketing would generate sufficient return to justify investment.
An orphan designation can be based on the claim that a condition for which the medicinal product is intended
is rare; i.e. that the condition affects not more than 5 in 10,000 persons in the community.
The following services are provided by mcintoshscientific:
- Advice regarding the suitability of a product for orphan designation
- Preparation of applications for Orphan Designation or review of prepared applications
- Participation in pre-submission meetings and submission of applications
- Preparation and submission of Orphan Drug Annual Reports
Paediatric Investigation Plan/Waiver (PIPs)
Submission of a PIP and/or a waiver request is obligatory for medicinal products previously unauthorised in
the EU and for patent-protected products being developed for use in new indications, routes of administration
or pharmaceutical forms. A PIP and/or a waiver request must be submitted for assessment by the PDCO generally
no later than at the end of human pharmacokinetic studies in adults.
The following PIP-related services are provided:
- Advice regarding the content and logistics of PIP submissions
- Preparation of Paediatric Plan/Waiver Applications or review of documents prepared by client
- Submission of applications and liaison with European Co-ordinators and Reviewers.
- Post-submission liaison with regulatory authorities
Risk and Gap Analyses
Documents can be prepared which identify Risk and hence ensure the control of hazards which are significant
for pharmaceutical quality in the production and supply chain.
The aim of a Gap Analysis is to identify the gap between the technological or regulatory requirements governing
the company's operations and the practices and processes the company has in use. This provides the company with
an insight into areas that have room for improvement. The GA process involves determining, documenting and
approving the variance between requirements and current capabilities.
Services provided include:
- Review of client's documentation describing the sourcing of materials and manufacture of products for the detection of possible hazards
- Review of client's documentation (protocols and reports) which describe the company's operations and practices
- Preparation of Risk Analysis report identifying possible hazardous materials or processes
- Preparation of Gap Analysis report highlighting possible shortfalls in technology or regulatory requirements
Scientific/Technological Due Diligence
The company can help clients considering investment in a company, to evaluate the scientific potential or
problems associated with novel biotech/biological products.
Services provided can include:
- Preparation of a report on the current status of the project, technical and regulatory hurdles to be overcome, the amount of work required to complete the project and the likely timelines to submission of a dossier
- Regulatory implications of alternative production processes or expression systems
Scientific Advice
Experience gained in more than 25 years of experience in biological research enables the company to advise
clients on a range of subjects relating to pharmaceutical science and technology.
Specific areas of expertise include:
- Antibody-Mediated Drug/Toxin Delivery for Cancer Chemotherapy
- Liposome Technology and Targeted Drug Delivery
- Tumour and Cell Biology and Vascular Targeting
- Endocytotic & Transcytotic Trafficking Pathways,
- Ultracryomicrotomy and Transmission Electron Microscopy
Translation and Interpreting
We are able to translate general and technical documents from Spanish or French into English and from English into French. We are also able to provide a qualified interpreter for meetings with Spanish and French-speaking clients.
02088 508808
07984 338945
