New drug dosage formula for the treatment of hypertension and heart failure in children

由于当今制药公司的主要研究和开发活动侧重于满足日益老龄化的人口的需求，占全球依赖人口的大部分的儿童仍然是一个被广泛忽视的利基市场。对儿童获得安全有效药物的权利的认识以及医疗保健提供者获得适龄药物信息的需求继续对儿科药物的所有领域产生重大影响。为了应对这一挑战，普罗维卡的目标是开发一种治疗儿童高血压和心力衰竭的非专利药物的新剂量配方，并申请相关授权。目前获得许可的药物主要以片剂形式提供；然而，儿童要么不能或不愿意吞下片剂，证明长期使用存在问题。替代药物以液体形式提供，然而，缺乏官方监管机构的正式许可引发了几个问题。由于药物配方是确保最优剂量水平和最小毒性的重要参数，目前在英国分发给儿童的无证液体配方中存在较大差异，这引发了与患者安全和药物疗效相关的重大问题。随着对药物安全的压力越来越大，以及对风险评估和缓解策略(REMS)的新要求，越来越需要向患者提供准确和一致的药物供应。普罗维卡的新配方有可能改善儿童的生理状态、生命质量和生命长度。这一市场证明的目的是使普罗维卡能够通过提供深入的市场研究来调查新药配方的市场需求和潜力。

With today’s major research and development activities of pharmaceutical companies focusedon meeting the needs of an increasingly aging population, children who represent the bulk ofthe dependent population worldwide still remain a widely neglected niche market Therecognition of the rights of children to have access to safe and effective drugs and the needs ofhealthcare providers to receive age-appropriate drug information continues to have a majoreffect on all areas of pediatric pharmaceuticals. In line with this challenge, Proveca’sobjective is to develop a new dosage formula of an off-patent drug for the treatment ofhypertension and heart failure in children and apply for the relevant authorisation. Thecurrently licensed medicine is mainly available in tablet form; however children are eitherunable or unwilling to swallow tablets, proving problematic in the long-term use. Alternativesof the drugs are available in liquid form, however the lack of formalised licensing with anofficial regulatory body causes several concerns. With the formulation of medicines being asignificant parameter to ensure optimal dosing levels with minimal toxicity, the high variationin the currently unlicensed liquid formulations dispensed to children in the UK raisesignificant issues related to patient safety and drug efficacy. With a growing pressure on drugsafety and new requirements on risk evaluation and mitigation strategies (REMS), there is anincreasing need to deliver accuracy and congruence of medication supply to patients.Proveca’s new formulation has the potential to improve children’s physiologic status, qualityof life and length of life. The aim of this Proof of Market is to enable Proveca to investigatethe market requirements and potential for the new drug formulation through the delivery of anin-depth market research.