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Glossary of Regulatory Deliverables

Here is a glossary of common regulatory deliverables a medicine developer should be aware of. The list includes regulatory deliverables to be completed for the following regulatory authorities:

  • The European Medicines Agency (EMA)
  • The Food and Drug Administration (FDA)
  • The Medicines and Healthcare products Regulatory Agency (MHRA)

Accelerated assessment (EMA)

Accelerated assessment reduces the timeframe for the Committee for Medicinal Products for Human Use (CHMP) to review a marketing authorization application from 210 to 150 days (excluding clock-stops for responses). An accelerated assessment may be granted if the CHMP decides the product is of major interest for public health and therapeutic innovation.

Accelerated approval (FDA)

The U.S. Food & Drug Administration (FDA) instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on a surrogate endpoint – one considered likely to predict clinical benefit but is not itself a measure of clinical benefit. Post-approval studies are required to confirm the anticipated clinical benefit – resulting in traditional approval for the drug. Otherwise, the FDA could remove the drug from the market.

Breakthrough Therapy Designation

The U.S. Food & Drug Administration’s Breakthrough Therapy Designation is intended to expedite the development and review of drugs that treat serious conditions and demonstrate substantial improvement over existing therapies. There must be preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

Conditional marketing authorization

A conditional marketing authorization may be granted based on less complete data than is normally required, if all of the following criteria are met:

  • the benefit-risk balance of the medicine is positive;
  • it is likely that the applicant will be able to provide comprehensive data post-authorization;
  • the medicine fulfils an unmet medical need;
  • the benefit of the medicine's immediate availability to patients is greater than the risk inherent in the fact that additional data are still required.

It is recommended that the clinical development (both pre- and post-authorization studies) and regulatory plans are discussed prospectively with the Committee for Medicinal Products for Human Use via scientific advice.

Exceptional circumstances approval

A marketing authorization under exceptional circumstances may be granted if the applicant can demonstrate that comprehensive data on the efficacy and safety cannot be provided, because:

  • the indications for which the product in question is intended are encountered so rarely that the applicant cannot reasonably be expected to provide comprehensive evidence, or
  • in the present state of scientific knowledge, comprehensive information cannot be provided, or
  • it would be contrary to generally accepted principles of medical ethics to collect such information.

An applicant wishing to seek exceptional circumstances approval should seek scientific advice from the Committee for Medicinal Products for Human Use on the inability to provide comprehensive data.

Fast Track Designation

The U.S. Food & Drug Administration’s Fast Track Designation is designed to expedite the development and review of drugs that treat a serious or life-threatening condition and address unmet medical needs. The drug must demonstrate the potential to address an unmet medical need by providing a therapy where none currently exists or offering a therapy that is potentially better than existing treatments. Evidence of activity in a non-clinical model, a mechanistic rationale or pharmacological data could be used to demonstrate such potential.

Innovative Licensing and Access Pathway (ILAP)

The MHRA lunched ILAP in 2021 as an initiative to accelerate marketing authorization in the UK and provide additional support to medicine developers throughout the process. ILAP is open to commercial and non-commercial organisations developing new chemical and biological products, as well as those applying for new indications for repurposed medicines.

Orphan drug designation

Orphan drug designation in the European Union (EU) offers some important benefits for the sponsor and their medicinal product – including European Medicines Agency fee reductions; 10 years of market exclusivity; and more favourable pricing in some EU countries. Products must meet all the following criteria:

  • intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Orphan medicinal products must continue to satisfy these criteria at the time of marketing authorization application submission – scientific advice on the maintenance of significant benefit is advisable.

The U.S. Food & Drug Administration’s orphan drug designation is intended to encourage the development of drugs for rare diseases. The drug must be intended to treat, diagnose or prevent a rare disease or condition. A rare disease is defined as one that affects fewer than 200,000 people in the United States (US) at the time of the designation request (or which otherwise would not recoup development costs from sales in the US). There must be a scientific rationale demonstrating that the drug may be effective for the rare disease or condition. Benefits of orphan drug designation in the US include tax credits for qualified clinical testing expenses; waiver of Prescription Drug User Fee Act fees; and 7 years of market exclusivity upon approval.

PRIME

PRIME (PRIority MEdicines) is intended to facilitate the development of innovative medicines that target an unmet medical need, i.e. offer a major therapeutic advantage over existing treatments, or benefit patients with no current treatment options. Products granted PRIME status benefit from various forms of early and enhanced support from the Europe Medicines Agency/Committee for Medicinal Products for Human Use.