The European Medicines Agency has published a draft guidance that gives timelines early dialogue between regulators, health technology bodies, and pharmaceutical companies.
IHS Life Sciences perspective | |
Significance | Following the EMA-HTA workshop on parallel scientific advice held in November 2013, the European Medicines Agency has published best-practice guidance on parallel scientific advice with health-technology assessment (HTA) bodies. |
Implications | The draft guidance provides a clear timeline for pharmaceutical manufacturers to approach regulators and HTA bodies in order to receive early advice on medicine development programmes. |
Outlook | The early advice represents a good opportunity for manufacturers to potentially reduce development costs and diminish delays. |
The European Medicines Agency (EMA) published yesterday (8 May) draft best practice guidance on parallel scientific advice with health technology assessment (HTA) bodies. The document, which will be open for consultation until 14 July, aims to provide tools to enable early dialogue between pharmaceutical manufacturers, regulators, and HTA bodies. In particular, the guidance is designed to define best practices for the parties involved in the process of regulatory and HTA approvals and set the timelines for action. It follows a workshop held in November 2013 on parallel scientific advice in drug development that involved discussions among key stakeholders. Key outcomes of the workshop have fed into the development of the draft guidance. Full text of the guidance is available here.
The scope of the guidance is wide: it includes all medicinal products for human use which are laid out in the Directive 2001/83, with no restrictions in terms, for instance, of drugs' eligibility for centralised procedure. Advices provided include clinical trial design, safety, and efficacy and quality of product. Advices given in the framework of the parallel scientific advice are not binding for the applicant. The guidance sets three main phase of scientific advice with the EMA and the HTA bodies: pre-notification phase, pre-validation phase, and meeting phase.
Pre-notification phase
The EMA strongly advices pharmaceutical manufacturers to establish early contacts with HTA bodies and the EMA in order to inform the authorities through a letter sent to the EMA about their intention to file for procedure as well as to provide information regarding the candidate product, timeline, and potential stakeholders involved. The guidance recommends engaging in early contact with the authorities approximately six to four months before the meeting.
Pre-validation phase
The EMA has provided two different options for applicants for the pre-validation phase:
- The first option is a 60–80 day pre-validation phase which foresees a teleconference (TC) between HTA bodies, the EMA, and the pharmaceutical company to enable discussions for complex procedures. This would be most suitable for inexperienced applicants or very complex applications. The TC is aimed at discussing scope, questions, and whether the wording used and information provided by the applicant meet the requests of the EMA.
- The second option is an approximately 45-day pre-validation phase where no TC is allowed and comments from the EMA and HTA bodies are communicated in written form.
Meeting phase
During the face-to-face meeting, applicants are able to discuss with the EMA and HTA bodies the list of issues delineated by the scientific advice working party (SWAP). Within two weeks before the face-to-face meeting, the applicant is requested to provide the presentation for the face-to-face meeting and to include written responses, when necessary. The presentation should generally focus on the introduction of the programme, its rationale, as well as its status. The session also includes open discussion and questions.
Outlook and implications
The first draft of the proposals was established in 2010 when a pilot project for parallel scientific advice with HTA bodies was created in the European Union. The guidance published yesterday by the EMA formalises the opportunity for pharmaceutical companies to establish early contacts with the EMA as well as the HTA bodies and to receive simultaneous early feedback concerning the development programme of new medicine. This may enable speeding up the regulatory authorisation process at European Union level as well as the evaluation of drugs for reimbursement.
Early dialogue is likely to involve discussions on relevant clinical trial data required for drug approval, such as relevant comparators and clinical endpoints. This may enable companies to design clinical development programmes able to meet requirements of regulators and HTA agencies. This opportunity would certainly be appealing for pharmaceutical companies that may be equipped with the opportunity to streamline the process and reduce clinical trials costs. In addition, early dialogue may also help to reduce risks of regulatory application rejections as information required by the evaluating authorities is discussed beforehand. Tomas Salmonson, chair of the Agency's Committee for Medicinal Products for Human Use, said during the November workshop, "This guidance can be a major tool for medicines development, which will help new medicines with a positive benefit-risk balance and expected added value to reach patients in a faster and more transparent way."