Orphan medicinal product meaning

In this issue: Medical device Research and development Post-market Competition in life sciences Intellectual property Pharmaceuticals—regulatory framework Advertising of medicines LexTalk®Life Sciences: a Lexis®Nexis community Daily and weekly news alerts New and updated content Trackers Useful information Medical device MedTech Europe leads industry call for EU to provide medical device regulatory relief by early 2026 MedTech Europe, alongside 35 national associations, has pressed the European Commission to act quickly on regulatory hurdles impacting the medical device and diagnostic sectors. Although EU officials are gathering evidence to underpin future reforms of the medical device and in vitro diagnostic regulations—aimed at simplification and greater efficiency—the signatories to the open letter contend that prompt relief is also crucial. They urge a blend of short-term actions with longer-term legislative change. In particular, the letter requests an implementing act to standardise Notified Bodies’ practices, pilot pathways for orphan, paediatric and breakthrough devices, and a targeted extension...

(OMPs) are medicines that help prevent, identify or treat rare illnesses and medical conditions. Because the number of people affected is very small, without incentives, pharmaceutical companies may doubt whether sales would cover the research and development (R&D) costs of medical products to detect, prevent and treat such disorders. In the relevant EU law, 'rare' is defined as affecting fewer than five in 10,000 people in the EU. Yet most rare conditions impact fewer than one in 100,000 people. Although each individual rare disease is uncommon, they carry major public health importance and significance. There are thought to be more than 6,000 distinct rare diseases; so, while each is infrequent on its own, together they account for a substantial patient population, roughly one in every 12 people in the EU (all rare diseases combined). In the absence of incentives, it is frequently not commercially feasible for pharmaceutical companies to develop and market OMPs, and only about 5% of rare diseases currently have an authorised treatment option available...

A–B | C–D | E–H | I–N | O–P. Qualified person (QP) The qualified person (QP) must confirm that every batch of medicinal products is manufactured and checked in line with UK or EU Member State law and in keeping with the marketing authorisation (MA) terms. See also: batch release marketing authorisation (MA) medicinal product Practice Note: Manufacturing of medicinal products for human use Qualified person for pharmacovigilance (QPPV) A qualified person for pharmacovigilance (QPPV) is the individual who, by law, holds personal responsibility for the safety of a human medicinal product. See also: adverse reaction marketing authorisation (MA) medicinal product Practice Note: Pharmacovigilance Rare disease See: orphan medicinal product (OMP)...

Directive 2001/83/EC (the Pharmaceutical Code) introduces a period of data exclusivity starting from the first authorisation of innovative medicinal products, during which the pre-clinical and clinical trial evidence produced to support that approval cannot be relied upon by any applicant seeking a marketing authorisation (MA) for a generic medicine. During this time, such data may not be cited or cross‑referred to by others at all. Accordingly, data exclusivity provides innovative companies with assurance that the scientific material prepared for the MA of the innovative product is safeguarded and will not be used to evaluate dossiers submitted by makers of generic or biosimilar medicines until the exclusivity enjoyed by the innovative product has expired. This protection is also termed regulatory data protection (RDP). This Practice Note sets out an overview of the relevant EU rules that govern RDP for MAs covering innovative medicinal products and considers how those rules affect the timing of market entry for generic and biosimilar medicines. It further outlines how RDP is addressed in the...

Introduction This due diligence questionnaire on medicinal products regulation concerns the intended purchase by [ insert buyer name ] (the Buyer) of the [ entire share capital of [ insert name of target company ] Limited, incorporated in England and Wales under number [ insert company number ] (the Company) OR [ insert description of the business to be acquired ] (the Business) as a going concern, together with specified assets used in the Business ] from [ insert seller name ] (the Seller) (the Proposed Acquisition). Its purpose is to equip the Buyer, the Buyer’s solicitors and other professional advisers with the legal information on UK and EU medicinal products regulatory compliance of the [ Company OR Business ], to support the Buyer’s valuation of the [ Company OR Business ] and to facilitate negotiation of the Proposed Acquisition. Please answer every question in full. Set out your responses in italics directly beneath each question and attach copies of all relevant documents. Ensure that each answer...