Pediatrics Form 3 Answers

[Giorgina Makara]

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Background: The International Knee Documentation Committee (IKDC) Subjective Knee Evaluation Form is a patient-reported outcome with adult (1998) and pediatric (2011) versions validated at different ages. Prior longitudinal studies of patients aged 13 to 17 years who tore their anterior cruciate ligament (ACL) have used the only available adult IKDC, whereas currently the pediatric IKDC is the accepted form of choice.

Purpose/hypothesis: This study compared the adult and pediatric IKDC forms and tested whether the differences were clinically significant. The hypothesis was that the pediatric and adult IKDC questionnaires would show no clinically significant differences in score when completed by patients aged 13 to 17 years.

Methods: A total of 100 participants aged 13 to 17 years with knee injuries were split into 2 groups by use of simple randomization. One group answered the adult IKDC form first and then the pediatric form. The second group answered the pediatric IKDC form first and then the adult form. A 10-minute break was given between form administrations to prevent rote repetition of answers. Study design was based on established methods to compare 2 forms of patient-reported outcomes. A 5-point threshold for clinical significance was set below previously published minimum clinically important differences for the adult IKDC. Paired t tests were used to test both differences and equivalence between scores. By ordinary least-squares models, scores were modeled to predict adult scores given certain pediatric scores and vice versa.

Conclusion: There was no clinically significant difference between the pediatric and adult IKDC form scores in adolescents aged 13 to 17 years. This result allows use of whichever form is most practical for long-term tracking of patients. A simple linear equation can convert one form into the other. If the adult questionnaire is used at this age, it can be consistently used during follow-up.

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For a medicinal products with multiple marketing authorisations (MAs) or future or ongoing marketing authorisation applications (MAAs), a single PIP decision can be used, provided that the marketing authorisation holder (MAH) or applicant are considered the same according to the Commission communication on the Community marketing authorisation procedures for medicinal products (98/C 299/03) ('Will Article 7 or Article 8 of the Paediatric Regulation apply to my application, taking into account the global marketing authorisation concept?').

This applicant will be the addressee of the Agency's decision; however, an appropriate PIP or waiver Decision can be used by an applicant who is not the PIP addressee, to satisfy the requirements of Article 7 or Article 8.

Before contacting EMA's Paediatric Medicines Office with your question(s), please first consult the questions and answers on this page, as well as the comprehensive information available under Paediatric medicines: applications and procedures: Procedural advice on paediatric applications.

If you cannot see an existing RPI in the list available when drafting a new submission, the most likely cause is that the RPI is assigned to a different organisation, for which you do not have an affiliation as IRIS Industry Manager, or to another individual. Please bear in mind that the organisation could be a different legal entity of the same multinational company.

Applicants can request scientific advice from EMA in preparation of a PIP, which is free of charge for questions relating to the development of paediatric medicines. They can also follow up a PIP with scientific advice, for example on combined adult and paediatric development in light of the PIP requirements.

Specific scientific questions on various aspects of paediatric development, including juvenile animal study design or paediatric formulations continue to be well within the scientific advice (SA) scope. In fact, most scientific questions remain within the remit of SA. The only questions the Scientific Advice Working Party does not address are those that fall under EMA' Paediatric Committee (PDCO) remit, such as:

In essence, the PDCO bears overall responsibility for agreeing, modifying or refusing a paediatric investigation plan. When designing a PIP, however, scientific advice may be requested about specific elements of it (but not about the plan as a whole), whereas paediatric pre-submission interactions assist in practical and regulatory aspects.

EMA is running a pilot for a 'stepwise PIP' agreement which would introduce a partial development programme supporting the authorisation of innovative medicines for children.

This programme would be conditional on the development of a full PIP once sufficient evidence becomes available. It would rely on predefined steps agreed with the EMA's Paediatric Committee (PDCO), such as applicants being able to hold discussions with the PDCO once they obtain more data.

A stepwise PIP would apply to cases where there is a lack of crucial information needed to decide on certain parts of the paediatric investigation plan, such as whether a clinical study for a whole age group is necessary.

Joint procedural information is available from EMA and the United States Food and Drug Administration (FDA) for medicine developers submitting a paediatric investigation plan (PIP) to EMA and an initial paediatric study plan (iPSP) to the FDA.

This joint document aims to make it easier for developers to submit paediatric development plans simultaneously to the regulators, to help speed up the development and approval of COVID-19 treatments and vaccines:

Given the rarity of childhood cancers, this joint guidance aims to speed up the development and authorisation of cancer medicines for children. It describes the information the two regulators typically require for their evaluations to support:

Please consult the IRIS Access & submission guide and the IRIS stakeholder forums for information on a variety of topics, including the communication to industry on data reconciliation relating to decisions on PIPs and product-specific waivers.

Applications for a marketing authorisation in respect of a medicinal product for human use which is not authorised in the European Union at the time of entry into force of Regulation (EC) No 1901/2006 have to comply with the requirements of Article 7 of Regulation (EC) No 1901/2006.

In accordance with Article 8 of Regulation (EC) No 1901/2006, the requirements of Article 7 shall also be applicable to the authorisation of new indications, new pharmaceutical forms or new routes of administration of authorised medicinal products which are protected by a supplementary protection certificate or by a patent which qualifies for the granting of a supplementary protection certificate.

Since Articles 7 and 8 refer respectively to 'a medicinal product for human use which is not authorised in the Community' and to an 'authorised medicinal product', at the time of submitting a new stand-alone application, it is necessary to establish whether the product applied for is considered or not a 'medicinal product for human use which is not authorised in the Community'. In this context, the global marketing authorisation concept, as defined in Article 6(1), 2nd subparagraph of Directive 2001/83/EC applies.

The global marketing authorisation includes the initial authorisation and all variations and extensions thereof, as well as any additional strengths, pharmaceutical forms, administration routes or presentations authorised, through separate procedures and under a different name, granted to the marketing authorisation holder of the initial authorisation. For further reference, see the notice to applicants, chapter 1, section 2.3. Thus, the global marketing authorisation concept applies to products belonging to the same marketing authorisation holder: according to the Commission communication on the Community marketing authorisation procedures for medicinal products (98/C 299/03), applicants belonging to the same mother company or group of companies, or which are 'licensees', have to be considered as one.

The global marketing authorisation (GMA) concept, together with the notion of 'same marketing authorisation holder', is used to determine whether an application concerns a 'medicinal product for human use which is authorised or not in the Community' and whether Article 7 or 8 applies.

For example, if company A holds a marketing authorisation in indication A for a product containing substance x (still patented), and company B (a subsidiary of company A) intends to apply for a new stand-alone marketing authorisation for substance x in a new indication B, the product will be considered as 'already authorised' based on the GMA concept, and company B will be required to cover also indication A in its PIP (i.e. Article 8 applies).

The GMA approach will apply to variations, extensions and new marketing authorisation applications falling under the requirements of Article 7 and 8. Where relevant, you should also consider whether a modification to an agreed or ongoing PIP or waiver decision is required in case the GMA concept had not been applied, in order to avoid difficulties at validation of your subsequent regulatory submission.

When an active substance from the 'same marketing authorisation holder' is or will be the subject of two marketing authorisations, one covering orphan indications and another covering non-orphan indications, those marketing authorisations would not be considered as part of the same GMA in light of Articles 7 and 8 of the Paediatric Regulation. In such situation, it is recommended to liaise with the EMA for further clarification of the regulatory implications.

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