A rare diseases conference held in London in March has focused on issues related to market access, pricing and the reimbursement of orphan drugs.
Organisers said one of the aims of Orphan Drugs and Rare Diseases Global Congress 2019 Europe was to ensure companies operating in the rare diseases space were well aware of the potential challenges facing clinical development.
Some of these included the misdiagnosis of patients with rare diseases (which represent missed opportunities of positive diagnosis), a focus on centers of excellence which, while it enables effective utilisation of various resources, may also mean that some patients may not be able to access these treatment centres due to logistical challenges leading to other issues such as patient recruitment and retention.
Equity and VC funding
Conference delegates heard that smaller biotech companies who are funded by equity firms and venture capitalists experience challenges in the form of pressure to meet major milestones, for example, first patient recruitment.
Meanwhile, the Congress was reminded that different stakeholders have a critical role to play at each stage of clinical development and it is critical that clinical trial studies are designed in a way so as to overcome as many challenges as possible, and by keeping it patient-centric and focused on the end requirements.
Adaptive clinical trial designs, education of physicians to increase their awareness of rare diseases, having a feasibility study with practical timelines, as well as the early engagement of regulators and funding agencies all offer different ways to improve the chances of an orphan drug reaching a wider patient population.
One point raised by patient groups was the need to publish results and details from failed clinical trial studies as this information would help researchers to avoid obvious mistakes or duplication efforts.
Pricing and reimbursement
A panel discussion on pricing and reimbursement of orphan drugs highlighted yet again the complexity surrounding the market access of such drugs. Patient advocacy groups play a critical and positive role in the lives of these patients and while collaborating with such groups is expected to help companies at different stages of their drug’s clinical development, industry representatives highlighted the challenges imposed by a regulatory framework that did not account for the rarity of the diseases.
Underscoring the discussion was an understanding that developing a sustainable funding solution for orphan drugs represents a major unmet need in the area of rare diseases.