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Madeira Therapeutics’ Peter Joiner Presents a Case for the Pediatric Market

In the pediatric setting, I believe that Madeira as well as others committed to this arena have the responsibility of educating physicians as to how a drug should be used and what role it plays in a patient’s life. Peter Joiner is CEO and President of Madeira Therapeutics. He has a 30-year history in sales, sales management, and marketing in the pharmaceutical industry. His positions have included executive-level salesforce management, strategic business planning, business analysis, and large customer account management. He has launched more than 15 major pharmaceutical products and established significant partnerships and relationships with key organizations in the healthcare industry. Before starting Madeira he was VP of managed markets at Alliant Pharmaceuticals. Before that, he spent 27 years at Sanofi-Aventis in various capacities. Mr. Joiner has a B.S. in electrical engineering and an MBA in industrial management from the University of Cincinnati. Uncovering deterrents What are the major deterrents to conducting pediatric studies and entering this market? joiner: The challenges facing the pediatrics market fall into four categories: ethics, economics, logistics, and technical considerations. These categories were indentified in 2006 at a workshop — Addressing the Barriers to Pediatric Drug Development — held by the Institute of Medicine’s Forum on Drug Discovery, Development, and Translation. The ethical dilemma relates to physicians. Traditionally, companies have not developed pediatric labeling information, which means physicians have to prescribe an adult medication for a child and have to guess at the dosage and determine whether the formulation is right for the child, or they forgo prescribing a therapy and the child goes without a much-needed medication. There are processes for testing drugs for adult usage; children should have the same advantages. And right now most of the time they don’t; when they leave the doctor’s office with a prescription, 75% of the time it’s for an off-label use. So, children aren’t getting the same deal as adults. In any industry, product development is driven by economics. The reality is that in evaluating children as a patient population, pharmaceutical companies take into account that children grow up, their metabolism rates change as they grow, and before long they will outgrow the drug, so it’s unlikely there would ever be a blockbuster drug in this category. And large pharmaceutical companies are really interested in big blockbuster drugs. But for a smaller-scale company, there can be an economic return for servicing this market. From a logistical standpoint, companies need to have extensive infrastructure in place to conduct pediatric drug trials, which is a barrier. From a technical point of view, compound stability is a big issue. Certain compounds may not work in a liquid format, but for children this type of formulation might be the most appropriate. And it has to taste good; the child may take the drug the first time, but if the taste is bad, it’s difficult to get him or her to take it again. Finding the Needs What are the most pressing needs in the pediatric market? joiner: Traditionally, pediatric pharmaceuticals have focused on the therapeutic areas of respiratory, including asthma; attention deficit disorder; and antibiotics. But because of the obesity epidemic, the most pressing need now is in the area of metabolic compounds — drugs such as lipid lowering statins, hypertension products, and diabetes medicines. Research shows that 30% of children are overweight and 17% have been categorized as obese. Unfortunately, as a result we’re starting to see more younger people develop Type 2 diabetes, which was rare in the past. Discovering the Risks What are some of the biggest risks with regard to the way children are currently treated for a variety of conditions? joiner: There are always risks when using a drug that hasn’t been tested to be efficacious in children. For example, in the area of pain, research in the past few years has shown that younger patients who are given codeine, which is one of the choices for severe pain, aren’t able to metabolize the drug appropriately because of their liver function; therefore, they don’t receive the necessary relief. When using an adult drug in a child, because of different metabolism and clearance rates, they are often not effective. In fact, sometimes the child needs more of the active pharmaceutical ingredient than an adult would because their liver clearance or metabolism rate is greater than an adult’s. Also using a drug in a child before it’s been tested means that it is impossible to know what side effects may occur. Research shows that an estimated 10% of children suffer an adverse event from a drug while in the hospital setting. Dennis Quaid’s newborn twins were given a blood thinner in the wrong dosage form at Cedars-Sinai Medical Center in 2007. There are many other high-profile cases such as this. Shifting the Balance What is Madeira doing to address the imbalances in pediatric drug development? joiner: As a Kansas-based company, we work with Children’s Mercy Hospitals and Clinics in Kansas City, which is a leader in this area, to develop appropriate clinical settings for testing drugs. Children’s Mercy President and CEO Rand O’Donnell has set up a group called the IPI, the Institute for Pediatric Innovation, in which five children’s hospitals are working together to help commercial companies bring pediatric drugs, as well as medical devices, to the marketplace. The FDA and NIH have a list of about 400 compounds for which they would like to have pediatric labeling information developed. Certainly Congress and the FDA have done their part, enacting legislation over the last several years, but this still has not encouraged many pharmaceutical companies to get into this marketplace. (To read more about the role companies can play to address child health issues, see PV’s bonus digital text.) In Madeira’s case, we will take adult drugs that are off-patent and put them through a formulation change, following the 505(b)(2) process to make them compatible for children. These are not new chemical entities, so there are safety and efficacy data already known. F PharmaVOICE welcomes comments about this article. E-mail us at feedback@pharmavoice.com. June 2009 Last Word

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