The approval of novel orphan drug designations continues to grow, while many existing rare disease therapies are receiving approval for expanded indications. With this increase and broadening class of products, including some that target the same mutation or molecular defect, sponsors face new and significant market access challenges in securing reimbursement.
Leading manufacturers increasingly employ stakeholder research early in development to better identify the needs of patients and providers. This strategy can build in compelling asset value during development, help avoid pitfalls and better inform go/no-go decisions earlier to avoid costly development delays or even dead-ends.
Truly Meeting Unmet Needs?
罕见的疾病药物发育已经面临多障碍：疾病了解，研究设计创作，也许最重要的是，获得有限的患者人口。战略性地纳入了发展的最早阶段的利益相关者研究可以帮助解决最重要的问题之一：是否会使经济意义继续投资这种孤儿药的发展？
Answering this question requires an examination of the current and future treatment landscape to understand a number of key factors. These factors include the needs of the limited patient population, the efficacy of any existing or anticipated future treatments, as well as market reaction to introduction of a new product.
Stakeholder research enables sponsors to “fill in the blanks,” calculate the potential risks earlier in their development process and make more informed go/no-go decisions, before investing significant resources along the orphan drug pathway to market.
揭示了付款人和处方的观点
Even if a product is deemed safe and effective, sponsors must consider how it is positioned against competing products to avoid ending up on the “me-to” product shelf. As more orphan drug applications receive approval, preference for one drug at the expense of another becomes a real commercial risk for sponsors. An example of this is currently unfolding with competing immunotherapies targeting the same type of rare lung cancer.
识别商业上可行的市场利基更可能是通过利益相关者研究定义临床试验的比较者。这项宝贵的练习可以帮助将产品定位有意义的差异体，以便更有可能达到充分的报销和更强大的市场地位。
即使孤儿药使其成为市场，如果他们认为不可接受的成本效益，或者如果有限的临床证据或治疗的比较疗效，则付款人即使拒绝治疗。赞助者面临审查和压力，以证明其定价证明，强调需要考虑其开发和市场访问策略中可能的定价方案。利益攸关方研究可以将这些考虑带到了早期的开发中的前列，提供了提案国时间为应付款者和处方人准备令人信服的沟通，以证明该产品的价值主张。
包括合适的利益攸关方
Depending on the indication, stakeholder research usually encompasses a mix of perspectives including those from key opinion leaders, clinicians and prescribers, health economists, hospital payers, third-party payers and even health technology assessors.
As the voice of the patient, Patient Advocacy Groups are also significant stakeholders in rare disease drug development. Inclusion of this stakeholder group’s perspectives helps to incorporate patient-centered health outcomes into clinical research and strengthens engagement between patients, families, caregivers and drug developers.
随着来自多个群体的利益攸关方的正确组合和平衡，可以收集有价值的现实数据，以提供关键的见解，包括：
- 影响定价理由和报销的政策/指导方针
- Needs for companion diagnostic tests, and whether diagnostic testing confirmation is likely to be required by payers
- Expected efficacy, possible therapy combinations and labeling strategies
虽然没有有效的护理标准many rare diseases, stakeholders often are a source of valuable, if not critical insights into an improved understanding the disease, relevant biomarkers and clinical endpoints.
Applying Research to Maximize Commercial Potential
随着利益相关者研究有助于揭示关键决策点和发展途径，市场准入和毒品开发团队在早期努力工作至关重要。今后的关键产出之一，联合团队的努力是在临床简介和前瞻性试验设计中建立和纳入战略市场建议。与关键利益相关者一起工作，赞助商可以利用现实世界的数据来突出疾病对患者的负担，描述产品的独特功能，并更好地定位产品以实现有需要的患者。
