- Health Technology Assessment (HTA) Bodies may need to review their requirements when assessing products that have been granted a PUMA -
- Buccolam (Midazolam, Oromucosal Solution) was the first medicine to be granted a PUMA and has received positive recommendations by two HTA bodies in Europe -
BRUSSELS, Nov. 5, 2012 /PRNewswire/ -- ViroPharma Incorporated today announced the presentation of a poster on cost effectiveness considerations in submissions for therapeutics targeting small patient populations, including those considered under the new Paediatric Use Marketing Authorisation (PUMA) process. These data were presented at the 15th Annual Congress of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), held November 3 - 7 in Berlin, Germany.
The PUMA approval process differs from a traditional marketing authorisation, as it will often be based upon population subsets, as is often seen in orphan drug product development. In the case of Buccolam (Midazolam, Oromucosal Solution), in order to help inform the HTA, primary data gathering was required to gain information on cost effectiveness, the results and approach of which have been accepted by two HTA bodies (the Scottish Medicines Consortium and All Wales Medicines Strategy Group). These data included expert views on the treatment pathways and relevance of clinical data, patient / carer views on the treatment pathways and the frequency and locale of seizures.
HTAs need to consider that in countries where reimbursement is linked to the strength of comparative data, it may be challenging to gather sufficient data to demonstrate value for a PUMA product, or an orphan drug. As a result, the authors of the poster suggest that HTA bodies may need to review their requirements when considering products licensed through the PUMA process in order not to inadvertently undermine the European Medicines Agencys objectives that originally led to the PUMA initiative, specifically to encourage the development of paediatric medicines for well-established off-patent products. The PUMA is intended exclusively for medicines to be used in the paediatric population.
"The regulatory process for granting a PUMA has been created with the hope that this would encourage the development of further paediatric products, " commented Lead Author, Dawn Lee, BresMed. "In order for the process to be effective, HTA bodies need to give consideration of the regulatory process followed while assessing a product that has been through the PUMA process."
About the new Paediatric Use Marketing Authorisation (PUMA) process A PUMA is a new type of marketing authorisation designed to incentivise the development of age appropriate formulations of medicines that are already licensed but are no longer covered by a supplementary protection certificate (SPC). The PUMA initiative was designed to ensure that medicines used to treat children are subject to high-quality, ethical research and are appropriately authorized, and to achieve its objectives without subjecting the paediatric population to unnecessary clinical trials and without delaying the authorisation for other patients.
Buccolam, approved in September 2011, was the first medicine to be granted a PUMA when it was licensed for treatment of prolonged, acute, convulsive seizures in infants, toddlers, children and adolescents, from three months to less than 18 years of age.[1]
About Buccolam (Midazolam, Oromucosal Solution) Buccolam is oromucosal midazolam provided in an individual dose formulation for buccal delivery. It is provided as convenient, portable, ready to use, pre-filled oral syringes containing age-specific doses. Buccolam is approved throughout the European Union and the EEA via the PUMA procedure for treatment of prolonged, acute, convulsive seizures in infants, toddlers, children and adolescents, from three months to less than 18 years of age.[1] The PUMA is designed specifically to encourage the development of medicines for children. The Medicines and Healthcare products Regulatory Agency (MHRA) has been advocating for the increased availability of "specific childrens-only medicines for several years in recognition that many adult medicines are offered to children in cut-down doses".[2]
Buccolam must only be used by parents/carers where the patient has been diagnosed to have epilepsy. For infants between 3-6 months of age, treatment should be in a hospital setting where monitoring is possible and resuscitation equipment is available.[1]
Originally posted here:
New Data On The Challenges Of Cost Effectiveness Modeling In Paediatric Use Marketing Authorisation (PUMA) HTA ...