Jerome Charton, chief executive of Clinigen, explains how to accelerate action for equitable access to essential medicines beyond international women’s day.
Women’s health still faces a critical challenge: entrenched gender disparities that impact women’s access to essential medicines, especially for those affected by rare diseases or living in low to middle-income countries. These disparities are not mere statistics; they represent lives impacted, potential unrealised, and a powerful call to action.
Equitable access to medicines is a deeply personal matter for me. As chief executive of Clinigen, I believe we have a strong responsibility to address the systemic barriers that unnecessarily deny women access to essential medicines.
As a father, I envision a future where my daughter, and all women regardless of their circumstances, have access to the best possible treatment options. As a leader, I’m committed to inspiring others to join this critical mission.
The World Economic Forum estimates gender parity is achievable in 2158 at the current pace. We cannot accept this. “Accelerating Action” demands swift, decisive steps, and for us, accelerating action means accelerating access to essential medicines for those who need it the most.
Consider this: in cardiovascular research, despite heart disease being the leading cause of death in women, they represent only 40% of trial participants. This lack of representation has deep historical roots. In the mid-20th century, due to flawed scientific practices and biases, women were nearly universally excluded from clinical trials. This legacy of exclusion has led to under-researched and underdeveloped treatments, particularly in rare diseases where women are disproportionately affected.
Underlying these access disparities are numerous influences: economic factors, social norms, and biases in the medical system, which contribute to delays in diagnosis and a lack of access to treatment.
For patients in low-to-middle-income countries, these challenges are amplified by limited healthcare infrastructure, cultural barriers, and economic constraints.
At our recent Clinigen Insights conference in Windsor, where we convened leading pharma and biotech experts to tackle the critical issues shaping global patient access, we polled them on the biggest barriers to rare disease treatment distribution and patient access and 74% of respondents identified managed access programmes as “the greatest opportunity to improve patient access to treatment, particularly for rare diseases”, whereas 52% thought “limited healthcare infrastructure” was the biggest barrier.
In addition to the access gap, there is a significant disparity in diagnostic timelines, with women waiting on average 46% longer than men to receive a correct diagnosis for a rare disease.
We cannot remain complacent about the diagnosis and access barriers women face. There are multiple pathways to expand ethical access to treatment to close the access gap.
The gender gap in rare disease treatment access
Globally, women with rare diseases face unique challenges. Studies show women are more likely to suffer from delays in diagnosis, with women waiting on average 5.4 years for a diagnosis compared to 3.7 years for men, and women are also underrepresented in clinical trials for rare diseases.
For example, autoimmune diseases, many of which are rare, affect women disproportionately, with women accounting for approximately 75% of those affected according to the National Institutes of Health (NIH), Office of Research on Women’s Health, yet research and treatment protocols can often fail to address this gender bias.
Economic barriers further compound these challenges. Women in the UK are more likely to face poverty, impacting their ability to afford healthcare, medications, or time off work for treatment. Studies indicate women are more likely to report cost-related non-adherence to medications, with the greatest disparity found in the US.
This is especially critical in rare diseases, where treatments are often costly and specialised.
A missed opportunity
Symptoms of menopause such as fatigue, joint pain and cognitive issues often overlap with those of rare diseases, leading to misdiagnosis and delayed treatment. This is particularly concerning in rare autoimmune diseases and cancers that disproportionately affect women but are often misattributed to hormonal changes.
A significant portion of women report waiting up to three years for menopausal symptoms to be taken seriously. This pattern of misdiagnosis and dismissal, combined with underrepresentation in clinical research, creates a dangerous lack of data on how diseases and treatments affect women, particularly in the rare disease space.
Accelerating action
At Clinigen, we’re not just acknowledging these disparities; we’re actively disrupting them. We recognise that empowering women is key to driving innovation and improving patient outcomes. We’re committed to accelerating action both within our organisation and for patients needing access to innovative medicines.
Internally, we’re taking concrete steps to foster a culture of gender equality and empower women in their careers. We are proud that 60% of our workforce is female, and we actively work to support the progression of women.
This commitment translates into tangible action: mentorship programs providing guidance and support for career advancement, our future shapers programme and the Clinigen Management Academy nurturing current and emerging female leaders, transparent DE&I policies (including our published gender pay gap report), and a supportive work environment with flexible options to meet diverse needs, recognising the importance of work-life balance, especially for women who often bear the primary caregiving responsibilities.
Outside our organisation, we’re leveraging our expertise in managed access, regulatory affairs, and clinical trial management to pioneer innovative solutions that support women’s access to essential medicines.
We must challenge the historical exclusion of women from clinical trials, which has created a dangerous knowledge gap in rare disease diagnosis and treatment. We must ensure women are not only represented but also prioritised in clinical research. This means designing trials that are culturally sensitive and accessible, considering factors like childcare responsibilities, flexible participation options, and healthcare infrastructure.
Furthermore, we need to move beyond traditional clinical trial models. Clinigen is supporting approaches such as decentralised and personalised trials and real-world evidence studies, to gather more comprehensive data on women’s health in rare diseases. We are also working with our partners to address the unique needs of diverse populations affected by rare conditions.
Bridging the access gap to rare disease treatments
Addressing gender disparities in rare diseases requires a collective effort. We must foster stronger collaboration between pharmaceutical companies, healthcare providers, patient advocacy groups and policymakers. By sharing data, best practices, and innovative solutions, we can accelerate progress and create a more equitable healthcare ecosystem. Collaboration is especially crucial in regions with limited resources, where partnerships can help overcome infrastructure and logistical challenges in rare disease management.
Technology can be a powerful catalyst for change. We need to explore how artificial intelligence, telehealth and other digital tools can be used to improve access to care and personalise treatment for women with rare diseases, particularly in areas where access is limited.
Beyond International Women’s Day, I urge the healthcare industry to move beyond rhetoric and take concrete action. Let’s work together to dismantle gender disparities in access to rare disease diagnosis and treatment, empower women, and create a healthier future for all. At Clinigen, we’re committed to leading the charge.
