The European Medicines Agency (EMEA) has adopted a list of symptomatic conditions that will qualify for class waivers from paediatric investigation plans (PIPs) under the EU’s regulation on medicinal products for paediatric use (No. 1901/2006).

Under the paediatric regulation, companies seeking approval either for a new medicinal product or for a new indication, route of administration or pharmaceutical form of an existing patent-protected product must submit a PIP detailing their strategy for developing the drug in all subsets of the paediatric population. The trade-off for meeting the requirements of an agreed PIP is a six-month extension to the product’s supplementary protection certificate.

Waivers from these obligations are available where there is evidence that the drug or class of drugs is likely to be ineffective or unsafe in part or all of the paediatric population; the disease or condition targeted by the product occurs only in adult populations (these are the ‘class waivers’); or the drug concerned does not present a significant therapeutic benefit over existing treatments for paediatric patients. The first opinions on product-specific waivers (three in total) were issued by the EMEA’s Paediatric Committee (PDCO) at a meeting in late October.

After reviewing comments from a public consultation on its proposed list of class waivers under Regulation No. 1901/2006, the PDCO adopted a positive opinion on the list at its meeting on 21-23 November. The EMEA subsequently endorsed the list in a final decision on 3 December. Further guidance for applicants intending to develop a drug for one of the listed conditions will be available shortly.

Symptomatic conditions
The list of symptomatic conditions – 17 in all – for which class waivers from a PIP are available includes a number of cancers (e.g., treatment of lung, breast or prostate carcinoma, hairy cell leukemia and multiple myeloma), as well as neurodegenerative diseases (e.g., Alzheimer’s, Parkinson’s) and age-related conditions such as macular degeneration or menopausal disorders. The waivers relate to the treatment of these conditions and not to medicines for their prevention or diagnosis.

The PDCO acknowledges that there may be anecdotal cases of these conditions occurring in the paediatric population. Moreover, the EMEA’s decision does not rule out voluntary submission of a PIP for a waived condition. The publication of the class waiver list “should not prevent an applicant from considering the development of a medicinal product in different or related conditions/indications for use in the paediatric population”, the agency stresses.

A further product-specific waiver was adopted by the PDCO at its meeting last month. This was for Pfizer’s lasofoxifene tartrate (Oporia), where indicated for bone diseases. The waiver releases Pfizer from the obligation to conduct clinical trials with the drug in the paediatric population for the waived condition(s).