Repurposing Medicines Toolkit - Guidance for navigating the process

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Parties working together on a project to develop a technology will ensure that they keep ownership of, and control over, their knowledge and intellectual property. They may license the IP to another party if required.

Collection of human tissue or other biological material, often associated with personal data, which is stored for potential research use.

Highly similar and clinically equivalent to an existing biological medicine (protein-based medicines).

The BGMA is a member of the European Generic medicines Association (EGA) and a member of the International Generic Pharmaceutical Alliance (IGPA). Promotes intellectual property, regulatory, pricing and market environment that enables the reasonable creation, development, and sustainability of generic markets in the interests of members, patients, and the United Kingdom National Health Service (NHS).

Provides up to date authoritative and practical information on the selection of medicines, prescribing, dispensing and administration of medicines to doctors, pharmacists, and other healthcare professionals. BNF Publications provide national policy decisions for both on and off label medicine use.

Describe products or materials that are suitable for direct therapeutic use. The use of clinical grade material in clinical trials requires regulatory approvals to ensure safety and efficacy for human use.

A legal contract between parties that outlines confidential material, knowledge, or information that the parties wish to share with one another for certain purposes but wish to restrict access to. Also known as NDA - Non-Disclosure Agreement.

CROs can provide clinical trial management, clinical development services, including biologic assay development, commercialisation, pharmacovigilance, and data collection. Additionally, they can provide a broad range of R&D support services. They can often undertake studies to GLP (Good Laboratory Practice) standard.

The process of developing medicines and new pharmaceutical products to market after drug discovery. Drug development includes preclinical development, clinical trials; phase I, II, III and regulatory approval. Not all phases will be relevant to all development programmes.

• Preclinical development:
  Also known as pre-clinical or non-clinical studies is the stage before beginning clinical trials.
• Phase I (i.e. Phase I is it safe?):
  Involves testing a new drug in a small number of healthy volunteers to determine the safety. At what dose can they achieve therapeutic effect with minimal adverse side effects.
• Phase II (i.e. Does it work?):
  Once the safety has been established in phase I, the aim of this phase II is to establish the effectiveness of the drug in people with the condition. 
• Phase III :(i.e. is it more effective than the current standard treatment?):
  • Phase III, compare the safety and effectiveness against current stand treatment.
  • In controlled study: the drug is compared with the standard treatment or placebo.
  • In double-blind: neither the investigators nor the participants know which treatment they are receiving
  • And in Randomised study: participants will be randomly allocated to receive drug or placebo.
  • The use of placebo in Phase III, will be given along with a treatment that is available and is known to work. Additionally, patients are closely monitored for any adverse side effects.

The regulators who commission recommendations and approve new medicines and licences in European and in Northern Ireland.

Freedom to operate refers to being able to carry out activities which do not infringe on the Intellectual property rights of someone else. A freedom to operate search should be conducted as early as possible to ensure that no IP rights in the field of technology would block the route to patient benefit.

New know-how arising from a project. The parties will need to agree what is to be done at the end of a relationship with any confidential information developed in the collaboration.

A generic medicine contains the same active ingredient as the equivalent original branded product authorised (the 'reference medicine') and can be produced by other companies once the patent and regulatory protection periods have expired.

Good Clinical Practice (GCP) is the international ethical, scientific and practical standard to which all clinical research is conducted. It is important that everyone involved in research is trained or appropriately experienced to perform the specific tasks they are being asked to undertake.

In the experimental (non-clinical) research arena, good laboratory practice or GLP is a quality system of management controls for research laboratories and organizations to ensure the uniformity, consistency, reliability, reproducibility, quality, and integrity of products in development for human or animal health (including pharmaceuticals) through non-clinical safety tests; from physio-chemical properties through acute to chronic toxicity tests.

Good Manufacturing Practice (GMP) is a system for ensuring that products are consistently produced and controlled according to quality standards. It is designed to minimize the risks involved in any pharmaceutical production that cannot be eliminated through testing the final product.

A process used by decision makers and other stakeholders (NICE) to support the decision-making process in health care at the policy level by providing evidence about given technologies. The assessment considers the safety, clinical effectiveness, economic considerations, budget impact analysis, organisation impact, equity issues, ethical issues, feasibility considerations acceptability to health care providers and acceptability to patients.

The UK's National Institute for Health and Care Excellence (NICE) is responsible for conducting health technology assessment (HTA) on behalf of the National Health Service (NHS).

Intellectual property is a category of property that includes intangible creations of the human intellect. There are many types of intellectual property, and some countries recognize more than others. The best-known types are copyrights, patents, trademarks, and trade secrets.

A new licensing and access pathway, managed by the Medicines and Healthcare products Regulatory Agency (MHRA), aims to accelerate the time to market, facilitating patient access to medicines. These medicines include new chemical entities, biological medicines, new indications, and repurposed medicines. The ILAP is open to both commercial and non-commercial developers of medicines (UK based and or global). It comprises of an Innovation Passport designation, a Target Development Profile (TDP) and provides applicants with access to a toolkit to support all stages of the design, development, and approvals process

The licence or marketing authorisation for a new drug is granted by a regulatory authority. The regulatory authority reviews data from all the clinical research to check that the drug is effective, safe and meets manufacturing quality standards. If they are satisfied, a marketing authorisation or licence is issued. This allows the product to be sold by the licence holder in the regions covered by the regulatory authority.

Legal contract to permit the transfer of material or data between the provider and recipient with or without a fee. Outlines the terms and conditions between the parties and other legal obligations.

Market authorisation sets out the conditions for a new medicinal product to reach market. Approval of medicinal products is regulated by the Human medicines Regulatory 2012.

A person or organisation that has the authorisation to market a medicinal product. MAH are responsible for providing continuous and competent information on how to use the medicine in the licensed setting.

An identified health problem or disease that will benefit from a therapy. Once such benefit has been established and approved by the regulatory authority the therapy is said to be approved for a specified indication.

An executive agency of the UK government's Department of Health. Responsible for granting national marketing authorisation, regulating the UK medicines supply chain (for example issuing manufacturer and wholesaler dealer licences), educating the public and healthcare professionals about the risks and benefits of medicines, and helping to develop the UK, EU and international regulatory frameworks for medicines.

Medicines repurposing also known as drug repositioning, reprofiling or re-tasking. Repurposing medicines is a strategy for identifying new uses for approved or investigational medicines that are outside the scope of the original medicinal indication and marketing authorisation (MA).

A significant event in the schedule of a project, usually the completion of a deliverable. If specified properly, it is the only way that a project manager can be sure that work has been done satisfactorily. Milestones have associated success or no-go criteria.

Medicines Repurposing Programme is a national multiagency initiative which identifies and progresses opportunities to use existing medicines in new ways, outside the current marketing authorisation.

An executive non-departmental public body, responsible for publishing guidance on the new and existing medicines used in the NHS, outlining clinical standards for healthcare professionals in treating patients.

Strategy to improve and enable humane use of animals in scientific research.

• Replacement - Avoiding or replacing the use of animals in areas where they otherwise would have been used.
• Reduction - Minimising the number of animals used consistent with scientific aims.
• Refinement - Minimising the pain, suffering, distress, or lasting harm that research animals might experience.

Where a medicine is administered outside the scope of the Market authorisation. Clinicians may consider using medicines off label when there is no other alternative licenced product available. There must be sufficient evidence on safety and patient informed consent is required. Example of Off-label prescribing; using a different dose, administering in a different way or using a medicine licenced only for adults for a child.

Where a medicine is prescribed in line with the scope of the medicine market authorisation (or licence).
This will include the approved use of the medicine for a specified therapeutic indication. Appropriate administration, dosing recommendations, and provision of standardised patient information with listed contraindications and precautions.

An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.

Orphan Regulation grants a ten-year period of market exclusivity to orphan medicinal products such that they are protected from market competition with similar medicines. An additional two years is extended for paediatric indication.

A right that allows the owner to prevent others from practising the invention. The patent system encourages public disclosure of inventions in exchange for a monopoly of 20 years. Patents are a national right granted by national or regional offices.

Basic prototyping and testing or providing demonstrations of basic proof of technical feasibility. Proof Of Concept can also include market studies, IP protection, investigation of production and assembly options.

Newly-licensed medicine will be carefully monitored for safety. All medicines have a patient information leaflet (PIL), which gives instructions on how the medicine should be used, and on side effects and reporting.

Also known as Paediatric study plan (PSP), is a development plan to support the authorisation of medicines in children. The MHRA will assess PIP and decide on waiver opinions modification and compliance statements to support market authorisation particularly with respect to any areas of unmet therapeutic need.

A leaflet included in a pack of a medicine to provide patients and healthcare professionals more information about the medicine and will include information on the active ingredient, instructions on how to take the medicines, possible side effects, storage, marketing authorisation and manufacturer.

The European Union defines a disease or condition as rare if it affects fewer than 1 in 2,000 (1) people within the general population.

Template used for non-commercial research studies in the UK. Its primary purpose is to ensure costs are appropriately attributed to AcoRD principles at the time of applying for research funding, to ensure that Research Costs are met via that funding.

Provides advice to NHS Scotland and reviews newly licensed medicines before they can be prescribed routinely in Scotland. Additionally, carry out horizon scanning to ensure NHS boards are aware of new medicines expected to come to market over the next financial year.

A scientific tool that supports interpretation of study data and informs decision making on the next steps in the drug development process. Compare the response of the drug-treated group(s) of patients to the response of the placebo-treated group. Phase 2 and Phase 3 clinical trials, the statistical analysis serves demonstrate the efficacy and safety.

Description of the eventual product being developed. Includes the key characteristics of the target product aimed at treating disease or diseases. They are intended to be used to support product design, research, development planning and to facilitate discussions with regulators

TTOs have the primary responsibility for the identification, assessment, development, protection, and exploitation of research outputs arising at Universities or Public Research Institutions

Store the data generated from various sets of trials use as a tool for analysis and reporting.