Devashish Singh, Co-founder and CEO, MrMed highlights that there are more than 7,000 recognised rare diseases, and new ones continue to be identified. Yet, one of the most pressing concerns in the realm of healthcare is ensuring that those afflicted with rare diseases can obtain the necessary medications
Uncommon illnesses, often referred to as rare diseases or orphan diseases, affect a smaller segment of the population when compared to more prevalent health conditions. Despite their infrequency, the impact of these rare diseases on individuals and their families is far-reaching. In fact, there are more than 7,000 recognised rare diseases, and new ones continue to be identified. Yet, one of the most pressing concerns in the realm of healthcare is ensuring that those afflicted with rare diseases can obtain the necessary medications.
The unique challenge posed by rare diseases
Rare diseases are characterised by their low prevalence and typically affect around 70 million people in India. Due to their rarity, developing medications for these conditions can present a formidable challenge for pharma companies. The smaller number of patients means that the potential for profit is limited, which renders the development of treatments for rare diseases less enticing for drug manufacturers compared to drugs targeting more common ailments.
Hurdles in accessing medications
Lack of Research and Development (R&D): A primary impediment to improving access to medications for rare diseases is the inadequate investment in research and development. Pharma companies often hesitate to allocate resources to develop treatments for rare diseases due to the high costs and uncertain chances of success. Consequently, this results in a shortage of treatment options for many rare diseases.
Financial barriers: When treatments for rare diseases are available, they often come with a hefty price tag. Research, development, and manufacturing expenses are typically spread thinly across a small patient population, leading to inflated costs. As a result, many individuals and families find it financially burdensome to afford these life-saving treatments.
Regulatory complexities: The regulatory framework surrounding medications for rare diseases can be intricate and burdensome. While regulatory agencies recognise the unique challenges associated with rare diseases, the approval process can still be demanding and time-consuming, delaying access to medications for those in need.
Limited participation in clinical trials: Clinical trials are essential for the development of medications for rare diseases, yet not all patients have the opportunity to partake in these trials. Geographic barriers, eligibility criteria, and other factors can limit participation, leaving some individuals without the chance to receive experimental treatments.
Strategies for overcoming barriers
Incentives for R&D: Governments and regulatory bodies should consider offering incentives to entice pharma companies to invest in research for rare diseases. This may encompass tax incentives, grants, or expedited regulatory pathways for orphan drug designations. By making it more financially appealing for companies to develop treatments for rare diseases, we can increase the availability of medications.
Promoting affordability: Policymakers should strive to make medications for rare diseases more affordable. This could involve implementing price controls, offering subsidies, or initiating reforms in insurance to ensure that no one is denied access due to the high costs of treatment. Patient advocacy organisations can also play a crucial role in raising awareness and advocating for policies that improve access to medications.
Streamlined regulatory processes: Regulatory agencies should continue to refine and expedite the approval process for medications designed to treat rare diseases. This could entail special designations for orphan drugs, accelerated pathways, and increased flexibility in the design of clinical trials. These efforts can help reduce the time required to bring new treatments to the market.
Improved access to clinical trials: Enhancing access to clinical trials should be a priority. This can be achieved through increased funding for patient transportation, improved communication between healthcare providers and research institutions, and the removal of restrictive eligibility criteria. Ensuring that more individuals can participate in trials increases the likelihood of discovering effective treatments.
Access to medications for rare diseases is a pressing issue that requires immediate attention. By removing these barriers, we can significantly enhance the quality of life for individuals living with rare diseases and provide hope for patients and their families. Collaboration among pharma companies, regulatory agencies, governments, and patient advocacy groups is essential to ensure that treatments for rare diseases are not only developed but also accessible to those in need. With the right strategies in place, we can expand access to medications for rare diseases and make significant strides in addressing the challenges associated with these conditions. This is not just a matter of public health; it is a matter of compassion for our fellow humans.