History shows that well-designed incentives drive innovation in under-represented areas of research. The US Orphan Drug Act was introduced in 1983 to accelerate innovation in treatments for rare, or “orphan”, diseases by providing strong incentives for drug development. Since its passage, the US Food and Drug Administration (FDA) has approved more than 600 orphan drug indications from more than 450 distinct drug products compared with only 10 such product approvals in the decade prior to enactment.21 The Act grants the FDA authority to designate orphan drugs, offering sponsors key benefits such as market exclusivity for seven years, tax credits of up to 25% for clinical trial expenditures and waived prescription drug user fees.22 Comparable incentive structures have been introduced across the globe to drive innovation in rare diseases. For example, in 1993, Japan launched the Orphan Product Development Support Program to promote the development of therapies for rare diseases by providing various incentives for developers, such as financial subsidies for research, market exclusivity and prioritized scientific consultations.23 Similarly, even though the US Congress has not renewed the FDA’s Rare Pediatric Disease Designation and Priority Review Voucher Program recently, the programme’s success demonstrates how strong incentives can lead to scientific innovation.24 Between 2012 and 2024, the programme drove innovation by accelerating treatment development for rare paediatric diseases, with more than 560 designations, 53 priority review vouchers, 47 awarded for indications that had no approved treatment prior to the programme and 39 new treatments – 36 of which previously had no approved options.25 These incentives have driven investment and innovation in previously neglected areas of medicine and can serve as a blueprint for advances in women’s health research. Finally, antimicrobial resistance (AMR) is another example of how incentivization can affect global health. AMR is a pressing global health threat, yet the development of new antibiotics has lagged due to limited financial incentives.26 To address this, policy-makers in some jurisdictions introduced market entry rewards, priority review vouchers and extended market exclusivity, which successfully spurred investment in antibiotic innovation. These incentives, which began in 2012, encouraged pharmaceutical companies to develop novel treatments despite high research costs and uncertain returns. The AMR model demonstrates that targeted incentives can drive innovation and bring life-saving advances to market, offering a valuable blueprint for accelerating progress in women’s health research.1.2 Success in orphan and paediatric diseases: Incentives drive investment and innovation Regulatory as well as financial incentives are critical to accelerating innovation in women’s health. Streamlined approval pathways, tax incentives and public–private partnerships can encourage R&D. Past successes, such as the orphan drug and paediatric research incentives, as well as the AMR initiatives, have demonstrated the power of incentives in driving investment and scientific breakthroughs. Aligning policy, investment and innovation can drive meaningful improvements in women’s health outcomes globally. The following policy recommendations address the underfunding to drive innovation in women’s health (Figure 3): –Introduce regulatory incentives to drive innovation in women-specific conditions. Innovation in women-specific conditions should be incentivized through the introduction of programmes such as female disease designation and priority review vouchers. Like the Rare Pediatric Disease programme, this would grant sponsors priority review vouchers for developing treatments for women-specific conditions. These vouchers could be redeemed for expedited approval of another product. Additional incentives, such as extended data or market exclusivity – for example, through a transfer exclusivity voucher – can further encourage investment in research. –Create financial incentives to drive innovation in women’s health. Increased government funding can be achieved through grants, prizes and dedicated women’s health research initiatives. Tax credits, deductions and exemptions can further incentivize innovation. Together, these financial incentives can address the significant funding gaps reported by agencies such as the NIH. Additionally, public–private funding matches can heighten the impact of philanthropic and private-sector contributions, driving investment in innovative treatments and technologies. 1.3 Policy recommendations to unlock innovation in women’s health Prescription for Change: Policy Recommendations for Women’s Health Research 10

Prescription for Change 2025