Reforming legislation is rarely a straightforward task. In the context of legislation governing medicines, countless stakeholders have a diverse range of interests and ambitions: patients need to be protected, and all without stifling future research and development. The European Commission’s proposed reform of European pharmaceutical legislation introduces several important changes to the way paediatric medicinal products (PMPs) are regulated in the EU in terms of the requirements and incentives to study medicines in children. We have already looked at proposals affecting, for example, data protection, environmental risk assessments, orphan products and antimicrobial resistance.
There are two main legislative provisions before the Commission: a new Directive (replacing 2001/83/EC and 2009/35/EC) and a new Regulation (replacing 726/2004, 1901/2006 (the Paediatric Regulation), and 141/2000/EC). Chapter VII of the proposed new Regulation sets out procedural requirements concerning the agreement and management of Paediatric Investigation Plans (PIPs), including deferral and waiver schemes. Obtaining a product or class waiver for diseases/conditions occurring only in adults becomes more limited as this will not cover situations where the product has the same mechanism of action in children, even in a different condition in the same therapeutic area, as in treatment of the adult condition applied for. Provision for a “Paediatric Use Marketing Authorisation” (PUMA) is maintained in the draft Regulation. The possibility of submitting a simplified “initial” PIP in specific cases is introduced, but overall PIP scrutiny is strengthened.
Rewards for the completion of PIPs are set out in the proposed new Directive. This specifies when marketing authorisation applications will need to contain the results of a completed PIP (or evidence of an agreed waiver/deferral); variation applications covering new strengths of authorised products covered by a SPC or a patent qualifying for a SPC are newly included, as are “hybrid” applications for such products. The proposal also contains specific provisions on checking PIP compliance and on the data derived from PIPs and sets out the rewards for PIP compliance; the current six-month supplementary protection certificate (SPC) extension reward is maintained (provided that a 1 year market protection extension is not obtained for the new indication), but the current separate 2-year market exclusivity extension for orphan medicines has been abandoned so the SPC extension will now also apply to orphan products (we discuss the new orphan incentive proposals here). This could give originator companies a total of up to five and a half years of additional protection via an extended SPC.
Many of the new proposals are born out of the Commission’s 2020 evaluation of medicines for rare diseases and children, which paved the way for legislative reform; revealing that, although the Paediatric Regulation fosters development and availability of PMPs, there is inadequate development in areas where the need for medicines is greatest (i.e. products for less profitable therapeutic needs). This problem is not new or limited to PMPs (see for example orphan drugs, which are also incentivised in the new legislation).
The Commission intends to streamline various measures and obligations, aiming to reduce patient access time by two to three years and commercialising at least three paediatric products every year. The Commission claims that originator companies would gain EUR103 million annually in gross profits by developing new PMPs. The Commission also believes that generics will benefit from the simplification of the rewards scheme because it will be easier to predict when their products could, in theory, enter the market. As with all new legislation, the proof is in the pudding.
The Commission believes that the revised incentives will lead to an increased number of medicinal products (in particular where there is an unmet clinical need) which are expected to reach the bedside faster than under the current legislation.