- 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 (NASDAQ: VPHM) 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 Agency's 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.
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. 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 children's-only medicines for several years in recognition that many adult medicines are offered to children in cut-down doses".
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.
Hypersensitivity to midazolam, benzodiazepines or to any of the excipients may occur. Midazolam should be used with caution in patients with chronic respiratory insufficiency because midazolam may further depress respiration. Midazolam should be used with caution in patients with chronic renal failure, impaired hepatic or cardiac function. Midazolam may accumulate in patients with chronic renal failure or impaired hepatic function whilst in patients with impaired cardiac function it may cause decreased clearance of midazolam.
The most common adverse reactions in clinical trials associated with oromucosal midazolam were sedation, somnolence, depressed levels of consciousness, respiratory depression, and nausea and vomiting. No severe adverse events were reported. The safety profile was similar to rectal or intravenous diazepam in the comparative clinical trials.
About Epileptic Seizures
Seizures occur because of sudden and abnormal electrical activity in the brain. There are many causes of seizures affecting children; triggers can include the use of medicines, head injuries and certain diseases.
Epilepsy is a chronic condition in which a person has a tendency to have recurrent seizures, normally diagnosed after greater than or equal to 2 seizures > 24 hours apart. Epilepsy is among the most common childhood neurological disorders in developed countries, affecting nearly one percent of the population. There are approximately six million people affected by epilepsy in Europe; nearly one million European children and adolescents have active epilepsy. It is anticipated that 25-30% of children will experience breakthrough seizures, despite treatment with anti-epileptic drugs (AEDs). Seizures can last from a few seconds to several minutes and in most cases will cease spontaneously. However, seizures can become self sustaining, and if left untreated after 5 minutes, can lead to prolonged or established status epilepticus (SE), which may require hospitalisation and intensive care.
About ViroPharma Incorporated
ViroPharma Incorporated is an international biopharmaceutical company committed to developing and commercializing novel solutions for physician specialists to address unmet medical needs of patients living with diseases that have few if any clinical therapeutic options. ViroPharma is developing a portfolio of therapeutics for rare and Orphan diseases including C1 esterase inhibitor deficiency, Friedreich's Ataxia, and adrenal insufficiency; and recurrent C. difficile infection (CDI). Our goal is to provide rewarding careers to employees, to create new standards of care in the way serious diseases are treated, and to build international partnerships with the patients, advocates, and health care professionals we serve. ViroPharma's commercial products address diseases including hereditary angioedema (HAE), seizures, adrenal insufficiency and C. difficile-associated diarrhea (CDAD); for full U.S. prescribing information on our products, please download the package inserts at http://www.viropharma.com/Products.aspx; the prescribing information for other countries can be found at www.viropharma.com.
ViroPharma routinely posts information, including press releases, which may be important to investors in the investor relations and media sections of our company's web site, www.viropharma.com. The company encourages investors to consult these sections for more information on ViroPharma and our business.
Forward Looking Statements
Certain statements in this press release contain forward-looking statements that involve a number of risks and uncertainties. Forward-looking statements provide our current expectations or forecasts of future events, including the estimated number of patients in the EU that may experience non-epileptic and epileptic seizures. There can be no assurance that our commercial launch of Buccolam in the EU will be successful. The commercial success of Buccolam in the EU will depend on a number of factors including the actual number of patients in the EU that may experience non-epileptic and epileptic seizures, physician and patient acceptance of Buccolam, the timing and level of pricing approvals obtained in EU member states and the level of manufacturing and supply of Buccolam produced by third party manufacturers. These factors, and other factors, including, but not limited to those described in our annual report on Form 10-K for the year ended December 31, 2011 and 10-Q filings for the quarters ended March 31, 2012, June 30, 2012, and September 30, 2012 filed with the Securities and Exchange Commission, could cause future results to differ materially from the expectations expressed in this press release. The forward-looking statements contained in this press release are made as of the date hereof and may become outdated over time. ViroPharma does not assume any responsibility for updating any forward-looking statements. These forward looking statements should not be relied upon as representing our assessments as of any date subsequent to the date of this press release.
 Buccolam European Summary of Product Characteristics. June 2011.
 MHRA Press release: A big step forward for better children's medicines. 7 September 2011. Available at
 Epilepsy in the WHO European Region: Fostering Epilepsy Care in Europe. 2010. Available at: http://www.ibe-epilepsy.org/downloads/EURO%20Report%20160510.pdf. Last accessed July 2012.
 Ekinci O, et al. Depression and anxiety in children and adolescents with epilepsy: Prevalence, risk factors, and treatment. Epilepsy Behav 2009;14:8-18.
SOURCE ViroPharma Incorporated