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Policies to promote affordability and access across the life cycle of costly new drugs. Strategic collaboration among countries and sectors on policies that prioritise equity throughout the full medicine life cycle could make innovative therapies more affordable and accessible globally, argue Krista Kruja and colleagues

Public funding is critical to drug research and development, but without built-in access and equity measures, resulting health products can remain unaffordable or inaccessible, ultimately limiting the societal value of new drugs. For example, an estimated $187bn (£142bn; €162bn) of US government funding contributed to the development of 99.4% of the 356 drugs approved by the US Food and Drug Administration from 2010 to 2019, but few safeguards ensured equitable access.9 10 Even where legal provisions exist, such as march-in rights in the United States, which can enable government to remove barriers to generic companies’ use of publicly owned drug patents, they are rarely used.11 Similar issues have also occurred in the context of health emergencies (box 1), raising questions about whether and how public funders can secure better access when supporting innovation. A Lancet Commission concluded that the lack of coordinated global governance severely undermined equitable vaccine access, especially for low and middle income countries.12 During covid-19, vaccine makers received over $31.9bn in US government funding alone,13 14 but equitable access was not guaranteed, and company approaches varied widely.15

A Lancet Commission concluded that the lack of coordinated global governance severely undermined equitable vaccine access, especially for low and middle income countries.12 During covid-19, vaccine makers received over $31.9bn in US government funding alone,13 14 but equitable access was not guaranteed, and company approaches varied widely.15 AstraZeneca, funded by the Coalition for Epidemic Preparedness Innovations and subject to a vaccine licensing agreement with Oxford University,16 engaged with COVAX, a global initiative aimed at equitable access to covid-19 vaccines, and supplied a large volume of vaccines to low and middle income countries. By contrast, Pfizer-BioNTech declined public research and development funding tied to access conditions, contributing less to COVAX despite BioNTech benefiting from public subsidies and advance purchase agreements, in which high income countries signed contracts reserving doses before vaccine approval.13 16 Moderna similarly relied on significant public grants and advance purchases while retaining commercial control over pricing and supply.15 As a result, these companies delivered relatively few doses to COVAX in the first year. Vaccine distribution reflected both manufacturers’ commercial priorities and the political priorities of high income countries that secured first access contracts, leaving little room for early engagement with COVAX.16 A lack of shared global standards or joint negotiation mechanisms meant that access conditions were inconsistent and largely driven by bilateral negotiations.

cial priorities and the political priorities of high income countries that secured first access contracts, leaving little room for early engagement with COVAX.16 A lack of shared global standards or joint negotiation mechanisms meant that access conditions were inconsistent and largely driven by bilateral negotiations. A potential solution is for public and philanthropic funders to adopt common standards for access and intellectual property, including affordable pricing, supply commitments, and licensing. While such provisions exist, they are inconsistently applied and often depend on intermediary organisations.17 In South Africa, a review of tuberculosis research and development showed that access clauses tied to public funding are often vague, poorly enforced, and hindered by weak oversight and limited transparency.18 Policy makers should test stronger, enforceable access conditions, supported by greater transparency and independent oversight. Recent international commitments, for example, through the Pandemic Agreement to link access conditions to public research and development funding, provide a timely opportunity to implement such mechanisms for pandemic products and could be extended to other therapeutic areas.

er transparency and independent oversight. Recent international commitments, for example, through the Pandemic Agreement to link access conditions to public research and development funding, provide a timely opportunity to implement such mechanisms for pandemic products and could be extended to other therapeutic areas. Policy makers could support innovation by building researcher capacity, establishing patient registries, and streamlining clinical trial approvals across jurisdictions. Another complementary strategy is repurposing off-patent drugs to provide affordable solutions. Initiatives such as REMEDI4ALL (https://remedi4all.org) and RePo4EU (https://repo4.eu), funded by the European Horizon Europe framework, aim to advance this approach by identifying and testing new therapeutic uses for existing drugs. Making research data publicly accessible in open databases could support this effort by enabling the systematic screening of existing drugs for beneficial side effects. However, it is too early to evaluate whether these ongoing programmes could ensure affordable, equitable access to repurposed drugs.

or existing drugs. Making research data publicly accessible in open databases could support this effort by enabling the systematic screening of existing drugs for beneficial side effects. However, it is too early to evaluate whether these ongoing programmes could ensure affordable, equitable access to repurposed drugs. Finally, drug development is costly, and especially for novel therapies, often financed through venture capital in early stages. This can drive pressure for high returns and contributes to high launch prices. Tackling affordability requires acknowledging these structural incentives. There is growing evidence that drugs can be developed successfully through innovation models that depart from the mainstream profit driven system. For example, product development partnerships such as the TB Alliance, Medicines for Malaria Venture, and Drugs for Neglected Diseases initiative have developed dozens of products by mobilising public and philanthropic funding for research and development, coordinating partnerships, and de-risking private, academic, and non-profit research and development.19 Such approaches have brought to market critical drugs, like the first all-oral cure for sleeping sickness. These approaches could also be applied to other areas of market failure­—for example, rare diseases, antibiotics, or pandemics.20 Wider adoption of alternative innovation models in the pre-market research and development phase could ensure drugs are more affordable by design.21

irst all-oral cure for sleeping sickness. These approaches could also be applied to other areas of market failure­—for example, rare diseases, antibiotics, or pandemics.20 Wider adoption of alternative innovation models in the pre-market research and development phase could ensure drugs are more affordable by design.21 Realising this potential also means grappling with deeper tensions in pharmaceutical financing. Public funds support early stage research, but profits reward the venture capital and corporate investors that finance later clinical development. These investors include pension funds, among others, meaning ordinary savers indirectly benefit from industry returns. Governments themselves face trade-offs: they benefit from pharmaceutical tax revenues but also carry the political responsibility of ensuring affordable access. The covid-19 vaccine experience underscored this tension, as countries that invested heavily in research and development also sought to secure priority access for their own populations, valuing domestic priorities over global equity goals. Tackling these pressures requires structured coordination—for example, shared platforms where governments, industry, and patients weigh trade-offs openly and negotiate solutions that balance national interests with global equity.

Robust regulatory review ensures the quality, safety, and efficacy of drugs and safeguards patients but also affects how quickly new therapies reach market and eventual affordability.22 Regulatory processes can support affordability by promoting efficiency, expediting access to generics and biosimilars, supporting the entry of me-too drugs, which can increase market competition and reduce prices,22 and fostering collaboration with payers and developers to align evidence generation with value based decision making. Recent health emergencies, including covid-19 and Ebola, highlighted the need for early engagement between manufacturers and regulators.23 Regulatory pathways can accelerate the development and approval of drugs for unmet medical needs, in emergency and other contexts.23 These can include23 24 conditional or early approvals based on limited clinical data, with additional data requirements post-launch; communication based approaches, including rolling reviews or ongoing engagement programmes between regulators and manufacturers; and priority review and expedited review timelines.

xts.23 These can include23 24 conditional or early approvals based on limited clinical data, with additional data requirements post-launch; communication based approaches, including rolling reviews or ongoing engagement programmes between regulators and manufacturers; and priority review and expedited review timelines. However, many countries lack capacity for these pathways. Reliance based approaches, which enable authorities to leverage assessments from trusted regulatory counterparts, could help fill this gap, by enabling faster access in emergencies and supporting regulation of complex therapies where local expertise or frameworks are limited. A reliance based model was used by the European Medicines Agency to help accelerate national decision making during covid-1925 and is being explored as a regulatory solution for enabling access to advanced therapy medicinal products.26

ing regulation of complex therapies where local expertise or frameworks are limited. A reliance based model was used by the European Medicines Agency to help accelerate national decision making during covid-1925 and is being explored as a regulatory solution for enabling access to advanced therapy medicinal products.26 The WHO Good Reliance Practices framework supports risk based, context specific implementation of reliance models.27 Equitable reliance requires low and middle income country regulators to shape and adapt external decisions to local needs. This depends on medicine developers and funders investing in locally relevant trials and on public and private stakeholders building platforms for real world data and pharmacovigilance, so that post-launch evidence can continuously inform regulatory decisions in low and middle income countries. Well resourced regulatory authorities should provide transparent assessments, and alongside partners like WHO should support capacity building in countries with underdeveloped regulatory infrastructure. The European Medicines Agency’s Article 58 offers one model, which enabled approval of the malaria vaccine after trials in seven African countries,23 by allowing the agency, in collaboration with WHO, to assess medicines for use outside the EU, involving low and middle income country regulators in reviews.

Advanced therapy medicinal products and products critical in public health emergencies, such as vaccines or novel therapeutics, face accessibility challenges owing to complex, costly, and proprietary manufacturing. Manufacturing remains concentrated in a few high and middle income countries owing to the high capital investment needed to build and maintain facilities compliant with good manufacturing practice, especially for advanced therapy medicinal products; long infrastructure lead times; and the need for highly specialised staff. Additionally, manufacturers’ exclusive control over patented processes limits broader production. These factors reinforce global health inequities and can limit access to new drugs in countries where they are not manufactured. Localised manufacturing can advance equity and strengthen supply chain resilience by reducing import costs, improving supply chain responsiveness, and facilitating local access programmes.28 Localised manufacturing can help countries respond faster in crises and expand access to specialised care; examples include covid-19 vaccines (box 2) and chimeric antigen receptor T cell therapies (box 3).

ience by reducing import costs, improving supply chain responsiveness, and facilitating local access programmes.28 Localised manufacturing can help countries respond faster in crises and expand access to specialised care; examples include covid-19 vaccines (box 2) and chimeric antigen receptor T cell therapies (box 3). During the covid-19 pandemic, local manufacturing in India and China enabled early, large scale vaccination campaigns without waiting for international shipments.29 In China, strong government support to partnerships between public research institutes and pharmaceutical firms29 enabled nationally based companies to scale up production even before clinical trials concluded, helping China meet both domestic and export demands. By contrast, in India limited early public investment slowed research and development and manufacturing of local vaccines. It was only after a severe wave of domestic cases that the government provided significant financial assistance needed to scale up local manufacturing.29

hina meet both domestic and export demands. By contrast, in India limited early public investment slowed research and development and manufacturing of local vaccines. It was only after a severe wave of domestic cases that the government provided significant financial assistance needed to scale up local manufacturing.29 Small molecule drugs, manufactured in centralised facilities that enable mass production and distribution, benefit from economies of scale.30 By contrast, personalised medicines, like chimeric antigen receptor T cell therapies, which are advanced therapy medicinal products that use the body’s immune system to destroy cancer cells,31 may involve patented, complex, and costly manufacturing processes that are difficult to scale using traditional industry models. Most current approaches to developing chimeric antigen receptor T cell therapies rely on centralised manufacturing, which contributes to prohibitively high costs in many low and middle income countries. Recent efforts show that decentralised manufacturing of chimeric antigen receptor T cells is practical and can reduce costs. In India, researchers produced clinical grade chimeric antigen receptor T cell therapies in a non-profit setting, with good results and an estimated cost of $35 000 per treatment, significantly lower than commercial prices.32 Decentralised T cell therapy was also shown to be a less costly and more efficient alternative in a non-profit setting in Germany.33

l grade chimeric antigen receptor T cell therapies in a non-profit setting, with good results and an estimated cost of $35 000 per treatment, significantly lower than commercial prices.32 Decentralised T cell therapy was also shown to be a less costly and more efficient alternative in a non-profit setting in Germany.33 Inequalities in access to therapeutics and vaccines during recent global health emergencies such as Ebola and covid-19 have prompted greater interest in building localised production capacity for drugs and health technologies.34 The 2025 WHO Pandemic Agreement calls for geographically diverse manufacturing, particularly in low and middle income countries, supported by regional hubs and technology transfer mechanisms.6 Achieving this requires governments to tackle patent barriers through knowledge sharing and by activating legal tools such as voluntary or compulsory licensing mechanisms.30 Initiatives focused on overcoming patent related restrictions have expanded drug access. For example, in Egypt, strict patentability criteria supported policies that enabled localised production of direct acting antiviral drugs for hepatitis C.35 In 2014, Egypt had a limited supply of branded direct acting antivirals, which cost US$900 for a 12 week treatment course and so were restricted to patients deemed most in need. However, the Egyptian government rejected patent applications for the antiviral drug sofosbuvir under its strict patentability criteria. Doing so enabled local production and the supply of generics. As a result, treatment became available for US$84 per treatment course from generic manufacturers.35

Translating regulatory approval into equitable access depends on what happens in the post-market phase: how health systems generate and use evidence, anticipate demand, and strengthen delivery capacity. Pricing36 and procurement37 are tackled elsewhere in this BMJ Collection. Effective post-market planning requires robust evidence to assess clinical value, inform demand forecasting, and optimise distribution. Covid-19 showed the costs of fragmented information sharing and misaligned production planning, which impeded vaccine access.34 In response, initiatives like the Access to COVID-19 Tools Accelerator and its COVAX initiative emerged to promote rapid scale-up and equitable distribution.38 Regional efforts, such the African Medicines Agency centralisation of regulatory oversight and HTAsiaLink’s facilitation of knowledge sharing in health technology assessment in the Asia Pacific region, underscore a growing recognition of the need for international collaboration in evidence generation and analysis to improve access to drugs.38 Permanent, coordinated mechanisms for demand forecasting and pooled procurement could mitigate some of the delays and challenges seen during covid-19.

n the Asia Pacific region, underscore a growing recognition of the need for international collaboration in evidence generation and analysis to improve access to drugs.38 Permanent, coordinated mechanisms for demand forecasting and pooled procurement could mitigate some of the delays and challenges seen during covid-19. Covid-19 also showed that coordination fails when evidence is fragmented, underscoring the need to treat evidence itself as a global public good. Regulators and policy makers, like companies, should disclose data on benefits and harms; this information should not be considered commercially sensitive. Real world evidence from conditional regulatory approvals, risk sharing agreements, and coverage with evidence development schemes should be made accessible for healthcare professionals, patients, and policy makers worldwide. Patients should be engaged not just as data contributors but as partners in defining value, priorities, and trade-offs when public funds and health systems bear financial risk.

, and coverage with evidence development schemes should be made accessible for healthcare professionals, patients, and policy makers worldwide. Patients should be engaged not just as data contributors but as partners in defining value, priorities, and trade-offs when public funds and health systems bear financial risk. Demand forecasting and horizon scanning activities are also critical for reducing uncertainties related to drug development for manufacturers.39 Early planning by health systems is particularly important for clinically advanced or cost intensive therapies. Cross country collaboration, such as the International Horizon Scanning Initiative, can help health systems to plan for and manage resources,40 but it requires early dialogue among regulators, payers, manufacturers, and patients.39 Early and continuous communication on evidence expectations by regulatory bodies, health technology assessment, and pricing and reimbursement bodies to drug developers can also reduce uncertainty and support optimal evidence generation from clinical trials; this also supports regulatory and payer decisions and serves patients by facilitating faster access.22 41 Enhanced global access also depends on increased availability of generic and biosimilar medicines, which can be achieved through mechanisms such as intellectual property flexibilities, compulsory or voluntary licensing, and large scale regional or global procurement.

serves patients by facilitating faster access.22 41 Enhanced global access also depends on increased availability of generic and biosimilar medicines, which can be achieved through mechanisms such as intellectual property flexibilities, compulsory or voluntary licensing, and large scale regional or global procurement. Strong health systems are also essential to scaling access. Beyond tools like health technology assessment and demand forecasting aiming for more efficient use of existing resources, countries need robust infrastructure, skilled workforces, effective procurement, and innovative delivery models. Health system strengthening has emerged as a key development strategy, recognising that resilient, well funded systems are vital for equitable healthcare.40 Though resource intensive, such systems enable efficient allocation through tools including reimbursement mechanisms, market based models, pharmacovigilance, and regulatory oversight, ensuring new (and older) treatments reach patients when they are needed.40 Persistent systemic challenges in the medicine life cycle hinder equitable access to novel therapies, whether for ongoing health challenges or acute health emergencies Tackling these challenges requires actions that prioritise equity from research and development through to post-market use Strategic, coordinated collaboration across countries and sectors is essential, as is the need to embed equity considerations from the start of the medicine life cycle

Persistent systemic challenges in the medicine life cycle hinder equitable access to novel therapies, whether for ongoing health challenges or acute health emergencies Tackling these challenges requires actions that prioritise equity from research and development through to post-market use Strategic, coordinated collaboration across countries and sectors is essential, as is the need to embed equity considerations from the start of the medicine life cycle Effective solutions include enforceable pricing and access provisions in publicly funded research and development, alternative innovation models, global collaboration on regulatory processes, and expanded localised manufacturing