Our industry is witnessing increasing growth in the rare disease market, thanks to financial and regulatory incentives to developorphan drugs。这对赞助商和患者来说都是个好消息，但事实仍然是稀有疾病试验本质上挑战奔跑。此外，完成复杂的研究并达到监管批准并不一定转化为市场成功。
John D. McDermott，Jr.，Covance Market Access Services副总裁副总裁，最近分享了他的见解market access稀有疾病药物开发中的挑战，并讨论了赞助商和利益攸关方的关键因素。
为疾病提供早期教育
尽管整个罕见的疾病正在得到更多的关注，但赞助商不能假设他们的潜在付款人对他们所瞄准的特定状况以及对患者的重要性表示了解。
By developing evidence that covers the natural history of the disease and highlighting patients’ burden of illness, sponsors can inform payers about the unmet medical need and its consequences for patients. These data can also define the subsequent value proposition. Sponsors should consider “marketing” the disease to payers to overcome potential payment hurdles and ensure greater acceptance at launch because payers have to understand the disease that they are providing drug coverage for.
专注于患者的观点
Rare disease patients are critical for gathering clinical trial data, but data about their personal experiences while on the treatment can be equally valuable. Sponsors are in a unique position to learn more about the patient perspective in rare diseases to better understand their needs beyond the clinic.
Patient-reported outcomes (PROs) and other health-related quality-of-life instruments can help sponsors generate crucial evidence to appeal to patients. These tools improve understanding of the treatment’s benefits and inform patients about the everyday impact of the drug on their lives. Coupled with partnerships with patient advocacy groups, a strong connection with patients can ease future recruitment burdens, guide the choice of relevant endpoints in clinical studies, and build strong demand for the drug after launch.
Evaluate potential payment hurdles and payer sensitivity
当孤儿药物发射时，它们通常比非孤儿药物价格高，以抵消治疗非常小的人群的开发成本并产生足够的投资回报。即使以全面的保险，罕见疾病治疗的共同支付或口袋费用也可能是令人无尊的。赞助商需要仔细考虑患者的财务负担，并确保他们能够支付药物。
共同支付援助计划可以将患者指向患者，如非营利基金会，协助共同支付，有助于改善进入和保留。制造商还可以考虑为无法承受购股票费用的被保险人或未经保险的患者提供自己的患者援助计划。无论最终价格如何，赞助商都应创建强大的市场访问支持计划，以考虑患者对这些关键的报销案例管理服务的需求。
了解到市场的路线及其影响
Sometimes, sponsors have the option to pursue either orphan or non-orphan indications for the same product. Orphan drug development can be very attractive to sponsors due to potentially expedited approval, longer exclusivity, and lower levels of investment needed to take a drug to market. However, an orphan pathway to market is not always the optimal choice.
Sponsors choosing between orphan and non-orphan indications must evaluate multiple dimensions of their market to choose the path with the highest commercial potential. Some factors to consider are the comparative sizes of the patient populations, the expected prices, the investment needed, the duration of development, and the impact on follow-on indications. Careful planning and investment modeling can help sponsors select the most attractive route to market.
* * * * * * *
Effectively running a rare disease clinical study and bringing a treatment to patients is a challenging process. At Covance, we offer over 25 years of market access consulting experience for rare and orphan products. We can leverage our track record supporting more than 50 indications to help you realize the full market potential of your rare disease drug development program.
Learn more about ourrare disease efforts和我们的市场准入解决方案。
