Adriaan Fruijtier speaker of compliance global

November, 2016

Speaker: Adriaan Fruijtier
Duration: 60 Minutes
Product ID: 700472


Orphan Medicinal Product Designation in the EU

The European Medicines Agency plays a central role in the development and authorisation of medicines for rare diseases. These medicines are termed ‘orphan medicines’ in the medical world.

The EMA is responsible for reviewing applications from sponsors for the designation of medicines for rare diseases.

To benefit from the incentives, sponsors intending to develop an orphan medicine must submit an application to the EMA requesting 'orphan designation' for their medicine.

The application is evaluated by the EMA’s Committee for Orphan Medicinal Products (COMP), which provides its opinion on whether or not the medicine qualifies as an orphan medicine for the treatment, prevention or diagnosis of a rare disease. If the COMP issues a positive opinion, the European Commission may then grant the medicinal product orphan status.

Because rare diseases are a global issue, the EMA works closely with its international partners on the designation and assessment of orphan medicines.The EMA works with organisations representing patients with rare diseases through the European Organisation for Rare Diseases (EURORDIS).

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Adriaan Fruijtier speaker of compliance global

August, 2016

Speaker: Adriaan Fruijtier
Duration: 60 Minutes
Product ID: 700342


Scientific Advice in the EU

Scientific advice helps the company to make sure that it performs the appropriate tests and studies, so that no major objections regarding the design of the orphan indication tests are likely to be raised during evaluation of the marketing-authorization application. Such major objections can significantly delay the marketing of a product, and, in certain cases, may result in refusal of the marketing authorization. Following advice from the Regulatory Authorities increases the probability of a positive outcome.

The Regulatory Authorities give scientific advice by answering questions posed by companies. The advice is given in the light of the current scientific knowledge, based on the documentation provided by the company. It is not the role of the Regulatory Authorities to substitute the industry's responsibility for the development of their products.

Scientific advice is prospective in nature. It focuses on development strategies rather than pre-evaluation of data to support a marketing authorization application. 

Scientific advice received from the Regulatory Authorities is not legally binding on the Authorities or on the sponsor with regard to any future marketing authorization applications for the medicine concerned. Protocol assistance is the special form of scientific advice available for companies developing designated orphan medicines for rare diseases. 

In addition to scientific advice, companies developing an orphan medicinal product can receive answers to questions relating to the criteria for authorization of an orphan medicine. These include:

  • The demonstration of significant benefit within the scope of the designated orphan indication;
  • Similarity or clinical superiority over other medicines. This is relevant if other orphan medicinal products exist that might be similar to the product concerned and which have market exclusivity in the same indication.

Companies can request scientific advice or protocol assistance either during the initial development of a medicinal product before submission of a marketing authorization application or later on, during the post-authorization phase. Scientific advice and protocol assistance are particularly useful to companies developing a medicinal product when:

  • There appears to be no or insufficient relevant detail in European Union guidelines or guidance documents, or in Pharmacopoeia monographs, including draft documents or monographs released for consultation;
  • When the company chooses to deviate from the available guidance in its development plan.

Parallel scientific advice with the EMA and FDA may lead to an increased dialogue between the two agencies and sponsors from the beginning of the lifecycle of a new product, a deeper understanding of the bases of regulatory decisions, and the opportunity to optimize product development and avoid unnecessary testing replication or unnecessary diverse testing methodologies. Parallel scientific advice should focus primarily on important breakthrough drugs or important safety issues in the following areas which have been identified as clusters of interest between the agencies: Oncology, Vaccines, Orphan Drugs, drugs in the Paediatric Population, Nanotechnologies, Advanced Therapies, Pharmacogenomics and Blood products. 

Parallel Scientific Advice procedures are conducted under the auspices of the confidentiality arrangement between the European Commission, the EMA, and FDA.

The parallel procedure with the HTA agreement helps to establish the evidence that both parties will need to determine a medicine's benefit-risk balance and value. and has four stages:

  • Pre-notification: Applicants engage early in informal discussions with HTAs and the Agency, announcing their intentions for the procedure, product and timescale, and specifying which HTAs will participate.
  • Pre-validation: Includes a teleconference with HTAs to discuss the scope, wording and clarity of the questions, and whether the material provided is sufficient to answer the questions posed. Applicants circulate their own documents throughout the procedure.
  • Meeting: A face-to-face meeting between all stakeholders, lasting approximately four hours, for which the applicant prepares the agenda according to priorities.
  • Outcome: Meeting minutes are circulated by the company for individual written HTA agreement. Regulatory scientific advice is provided in a CHMP advice letter. Sponsors may choose to share this letter with the participating HTA bodies.

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Adriaan Fruijtier speaker of compliance global

July, 2016

Speaker: Adriaan Fruijtier
Duration: 60 Minutes
Product ID: 700401


Pediatric Investigation Plans (PIP) in the EU

The European Medicines Agency plays an important role in the development of medicines for children. In the past, many medicines authorized in Europe were not studied adequately or authorized in children. This caused difficulties for prescribers and pharmacists treating children, as well as for their patients and careers.

To help to solve this problem, the European Union's Paediatric Regulation gave the EMA new responsibilities in 2007. These responsibilities allowed the EMA to stimulate research into the uses of medicines in children and to lead to their authorization in all ages. The Paediatric Regulation's main aim is to improve the health of children in Europe without subjecting children to unnecessary clinical trial directive, or delaying the authorisation of medicinal products for use in adults. 

The clinical trials regulations came into force on 26 January 2007. Its main impact was the establishment of the Paediatric Committee (PDCO), which is responsible for coordinating the EMA's work on medicines for children. The Committee's main role is to determine the studies that companies must carry out on children as part of paediatric drug development plans. The PDCO replaced the Agency's previous Paediatric Working Group. 

Several incentives for the development of medicines for children are available in the EU directive for clinical trials and in Member States:

  • Medicines that have been authorized across the EU with the results of PIP studies included in the product information are eligible for an extension of their patent protection by six months. This is the case even when the studies' results are negative;
  • For orphan medicines, the incentive is an additional two years of market exclusivity;
  • Scientific advice and protocol assistance at the Agency are free of charge for questions relating to the development of medicines for children;
  • Medicines developed specifically for children that are already authorized but are not protected by a patent or supplementary protection certificate, can apply for a paediatric-use marketing authorization (PUMA). If a PUMA is granted, the product will benefit from 10 years of market protection as an incentive.

The EMA has drawn up a list of off-patent medicines that are priorities for development as pediatric legislation.  Funding from the EU is available for EU clinical trials and studies into these medicines. 

The EMA maintains a database of studies carried out in children that were completed before the Paediatric Regulation came into force in 2007. The database contains the details of each study, including its title, its aims, the medicines studied and the ages of the patients included. 

The EMA works closely with its international partners on medicines for children:

  • It has agreed to share information with the United States Food and Drug Administration (FDA) on scientific and ethical issues related to the development of medicines for children, to foster global development plans;
  • It participated in the launch of the World Health Organization (WHO) initiative make medicines child size;
  • It is a member of the Paediatric Medicines Regulators' Network (PmRN). This is a network of national medicines regulatory authorities set up by the WHO in 2010, which promotes the quality and availability of medicines for children.

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Adriaan Fruijtier speaker of compliance global

October, 2015

Speaker: Adriaan Fruijtier
Duration: 60 Minutes
Product ID: 700193


Development regulatory affairs for Oncology Medicinal Products

Because malignant tumours are life-threatening, the death rate from these diseases is high, and existing therapies have limited effectiveness, it is desirable to provide new, effective anticancer drugs to patients more expeditiously.

Therefore the requirements for non-clinical studies and clinical studies are different compared to other medicinal products. These are described in various guidelines.

Confirmatory clinical trials should be designed with the aim to establish the benefit - risk profile of the experimental medicinal product, including supportive measures, in a well-characterised target population of relevance for clinical practice.

The EMA uses three categories:

  • Reduced or similar toxicity
  • Increased toxicity and 
  • Major increase in toxicity

No precise definition is given due to heterogeneity of the conditions. ”Major increase in toxicity”, however, in most cases refers to a fear that the experimental regimen might be associated with an increase in treatment related deaths, irreversible adverse events with a long-term impact on quality of Life, or severe impairment to patient condition. The requirements in each of these categories are different.

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Location: New York

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