Jeanne Hecht, Chief Executive Officer and Executive Chairwoman, Lexitas Pharma Services
(Bio)pharmaceutical companies can improve global access to essential medications in resource-limited settings by adopting several strategies. They can implement tiered pricing models to make drugs more affordable in low-income regions and collaborate with governments and non-governmental organizations (NGOs) to strengthen healthcare infrastructure and distribution networks. Additionally, companies can invest in local manufacturing facilities to reduce costs and improve supply chain efficiency.
Partnerships with global health initiatives and participation in programs like the World Health Organization’s (WHO) prequalification can ensure quality and safety standards. Finally, fostering innovation in drug formulations, such as heat-stable or single-dose therapies, can enhance accessibility and compliance in challenging environments.
Spandan Mishra, Global Director Technical Marketing Biopharma Solutions, Evonik
Biopharma companies play a major role in improving global access to essential medications by introducing low-cost versions of off-patent, high-value biological drugs, commonly known as "biosimilars." Many of the best-selling essential medications, like monoclonal antibodies (mAbs), which have important applications in oncology and other chronic diseases, will lose patent protection after 2023. According to various market studies, many more innovative mAbs will come off patent in the period 2023–2032 and will be replaced by low-cost biosimilar mAbs.
Medium and small manufacturers working in resource-limited settings are encouraged to offer such affordable drugs essential for global healthcare. Cost cutting and performance boosting cell culture and bioprocess supplements help biosimilar developers and manufacturers to keep the biosimilar drug prices as low as possible.
At Evonik, we offer cQrex® peptides, which contribute to the reduction of the production and operation costs of biopharmaceuticals. These work by enhancing cell metabolism, increasing protein (mAbs) titers, reducing production costs, and enabling the highly efficient production of biopharmaceuticals. Thanks to their high stability and solubility at neutral pH, cQrex® peptides also allow the supply of (key) ingredients with one main pH-neutral feed, lower risks for bioprocesses and operators, by ensuring the addition of all essential cell culture ingredients in one pH-neutral main feed, leading to lower risks for bioprocesses and operators and reduced bioprocess complexity.
Ryan Guest, Ph.D., Senior CMC Translational Consultant, eXmoor Pharma
Cell and gene therapy (CGT) developers can enhance global access to essential medications in resource-limited settings through several key strategies.
First, partnering with governments, NGOs, and local healthcare providers enables investment in R&D tailored to diseases prevalent in low-income regions, ensuring therapies address significant unmet health needs. These partnerships can facilitate infrastructure development, ensuring necessary medical facilities and trained personnel are available to administer advanced treatments, which often require more complex knowledge and, in some cases, specialist cell/tissue collection and treatment centers.
Second, collaborating with international health organizations and global funding initiatives for targeted diseases can support tiered pricing models, which offer subsidies or donation programs to make therapies more affordable for low-income populations. Global regulatory adoption by low-income populations streamlines regulatory processes and enables more efficient approval and distribution of therapies.
Third, developers can focus on scalable, cost-effective manufacturing processes, such as allogeneic cell therapies, which can be produced in larger quantities and distributed more widely, or decentralized manufacturing, which can be geolocated to minimize logistics and turnaround time.
Finally, investing in education and training for healthcare professionals in these regions ensures the safe and effective use of these advanced therapies, ultimately leading to better health outcomes and increased access to life-saving treatments.
Stella Vnook, Chief Executive Officer, Likarda
Biopharmaceutical companies already play a crucial role in enhancing global access to essential medications, particularly in resource-limited settings. We can do more of what works, such as tiered pricing models based on the economic status of different regions, voluntary licensing agreements with generic manufacturers to increase the availability of affordable medications, and local investment and strategic partnerships to reduce production and distribution costs and support local economies.
This also includes establishing access programs that provide medications at low or no cost, donating lifesaving drugs, and allocating resources toward R&D for neglected diseases that disproportionately affect low-income countries.
International regulatory agencies can support us through simplified processes for expedited approval and distribution.
It will be important to strengthen local healthcare systems through training and support to ensure effective usage of these medications. Developing robust distribution networks and leveraging technology can overcome logistical challenges in reaching remote areas. Collaboration with governments, NGOs, and international organizations can advocate for supportive policies. We can also adapt our corporate structures to better promote ethical imperatives, including sustainable business models that prioritize long-term health outcomes over short-term profits.
Trevor Smith, Senior Manager, Regional Marketing - Cell & Gene Therapy, Terumo Blood & Cell Technology
With new innovations in the biopharmaceutical segment, the World Health Organization Model List of Essential Medicines has grown, causing the already extant gap in global access to widen. There are, however, a few strategies developers may leverage from the industry’s approach to chimeric antigen receptor (CAR)-T cell therapies, which have initially been difficult to bring patients — inspiring a concerted effort to improve access.
For instance, licensing IP has shown some success in bringing CAR-T therapies to China. Additionally, so-called “home grown” approaches — supported by knowledge transfers with non-resource-limited groups like the National Institutes of Health — have also been celebrated this year, as in the case of India’s NexCAR19 CAR-T cell therapy. Efforts are also underway to develop decentralized cell therapy manufacturing approaches; on-demand drug manufacturing wherever they are needed is a broadly appealing concept.
Natalia Elizalde, Ph.D., Chief Business Development Officer, VIVEbiotech
The best way that (bio)pharmaceutical companies, and more specifically CMOs, can contribute to improving global access of medications is developing cost-effective, productive processes that are very well optimized to increase the productivity and enhance the biologics functionality to exert corresponding therapeutic effect.
Working at VIVEbiotech, which is fully specialized on lentiviral vectors, I have observed this firsthand in very advanced fields, such as cell and gene therapy.
In this industry, the effort is focused on improving the bioprocess in such a way that the manufacturing costs per patient are significantly decreased, impacting directly on the overall treatment cost. Altogether, this has a direct impact on the coverage of these innovative treatments by public health systems and hopefully also on the access to these cell and gene therapies in less-developed countries.
VIVEbiotech, as a CDMO specialized in lentiviral vectors, contributes to improving global access to essential medications through several key strategies. First, being experts in manufacturing and virology, VIVEbiotech is committed to innovation in automation and continuous monitoring, which not only enhances production efficiency but also ensures consistent quality. Furthermore, VIVEbiotech focuses on developing scalable and cost-effective manufacturing processes that ensure high-quality production of lentiviral vectors. By optimizing these processes, the company can reduce production costs, making advanced therapies more affordable and accessible, all of which translates into treating more patients.
Through these efforts, VIVEbiotech exemplifies how (bio)pharmaceutical companies can play a pivotal role in enhancing global access to essential medications, addressing the needs of vulnerable populations, and supporting sustainable healthcare solutions.
Carol Houts, Chief Strategy Officer, GermFree
Biopharmaceutical companies can adopt decentralized or regional manufacturing models with agile platforms, such as fully autonomous modular or mobile facilities. These flexible units can be deployed quickly and tailored to meet regional needs, ensuring a swift response to local demands. Working closely with local healthcare providers, governments, and non-profits can enhance the effectiveness and sustainability of these facilities.
Companies should develop agile supply chains to ensure efficient distribution, particularly in remote areas, with digital tracking technologies and mobile cleanroom units for localized vaccine and therapy production. Additionally, strategic alliances and public–private initiatives can lead to robust solutions for global health challenges and can also support large-scale vaccination programs and the distribution of essential medications.
Companies should advocate for regulatory harmonization to simplify approval processes and participate in global health policy discussions. Emerging country regulatory agencies need support to expedite key approvals required to work regionally. Investment in research and development of cost-effective production methods that can be scaled up or out can ensure treatments remain affordable and accessible. It is vital to establish access programs with organizations like Gavi, the Vaccine Alliance, and Caring Cross for cell and gene therapies, for low-cost medications, and engage with local communities to address specific healthcare needs.
Priya Baraniak, Ph.D., Chief Business Officer, OrganaBio
A holistic and collaborative approach is crucial to improving global access to medicines and advanced therapies. Biopharmaceutical companies, working alongside government agencies and international organizations, should prioritize equitable access as a core principle. Key strategies include:
Implementing tiered pricing models based on a country's economic status to allow lower-income nations to access essential medications at significantly reduced costs.
Granting voluntary licenses to generic manufacturers in developing countries to empower local production and distribution of affordable versions of patented drugs, reducing reliance on imports.
Partnering with governments, NGOs, and international organizations to develop and implement comprehensive access programs that encompass supply chain management, healthcare infrastructure development, and training of healthcare workers to strengthen local healthcare systems and ensure sustainable delivery of medications.
Advocating for reducing trade barriers, strengthening intellectual property rights protection, and promoting regulatory harmonization to create a favorable environment for expanding access and fostering innovation.
By embracing these strategies, biopharmaceutical companies can significantly contribute to reducing global health inequities and ensuring lifesaving medications reach those who need them most, regardless of their location or economic status. This approach not only fulfills a moral imperative but also fosters a sustainable industry that thrives by prioritizing patient needs on a global scale.
Matthew Hewitt, Ph.D., Vice President, CTO Manufacturing, Charles River Laboratories
We see this in various therapeutic areas, and one thing therapeutic developers can do to properly address the market is to partner with CDMO/CMOs who are experienced in manufacturing at scale for their therapies, similar to what’s been asked in the prior question. If therapeutic developers continue to have challenges, then they may need to consider having multiple external manufacturing relationships.
Ludovic Brellier, President Biotechnology Integrated Services, Cytiva
As our population ages, the demand for innovative medicines grows ever stronger. The healthcare industry is navigating through the challenges of a complex therapeutic pipeline, which includes the rise of personalized therapies and mounting cost pressures. In interviews conducted with over 1,000 industry professionals, Cytiva identified two main areas of concern. The first is the need for a flexible workforce, and second is the need to strengthen and expand the supply chain to deliver uncompromising quality for local populations.
If we are to meet global demand with limited resources, security of supply and speed will be key. Embracing automation can truly transform the future. Automating processes can reduce the demands on workforce expertise, allowing manufacturing to happen anywhere without the need for specialized or advanced degrees. The geographic footprint for manufacturing is expanded while simultaneously improving quality and reducing waste and cost. Another geographic equalizer is virtual training. This technology opens the doors to a more diverse and inclusive workforce by providing equal learning opportunities to individuals no matter where they call home.
Wade Macedone, Chief Executive Officer, Andelyn Biosciences
Andelyn was spun out of Nationwide Children’s Hospital in 2020, and many of our staff were involved in not only pioneering the manufacturing technology and methodology but also in the lived experience of the families who were the recipients of the very first approved treatments for Duchenne muscular dystrophy and spinal muscular atrophy. This sense of closeness to the patient is the inspiration for the very name of our company, and we are still in regular contact with the families to this day and are delighted to watch them grow into adulthood.
Many of the disease areas that Andelyn’s clients are working on are in the rare or ultrarare category, meaning there is a very small pool of patients. A lot of these diseases are fatal or severely debilitating, and many affect children, so there is an added sense of urgency. At the same time, researching, developing, and ultimately manufacturing gene therapies to address them is expensive. Often, funding for these treatments is raised through family or charitable foundations, so it’s imperative that we make every dollar count along this journey.
So, we’re always cognizant of the balance between developing manufacturing processes and executing them as efficiently and expeditiously as we can, while also acknowledging that being part of the for-profit biopharmaceutical ecosystem with private investment is ultimately a great motivator and driver of innovation. That’s why we have worked hard to develop manufacturing systems like our AAV CuratorTM platform, which redefines what a manufacturing platform is by taking advantage of standardization as much as possible while being flexible and optimizable by design with a modular approach.
Andre Unglert, Director of Commercial Development, Lonza
Antibody–drug conjugates (ADCs) are revolutionizing cancer therapy with their highly specific mechanism of action, offering new hope to cancer patients worldwide. To date, 13 ADCs have received market approval to treat various cancers, and over 200 are currently in clinical development.
Four crucial steps can enhance global access to essential medications, and CDMOs play a pivotal role in this process. A benchmark CDMO should provide integrated end-to-end drug development and manufacturing services as the first key step. The second step involves defining the right scale at the right time in order to accelerate and simplify market supply. The next step is to provide the necessary flexibility to ensure time to market. Finally, CDMOs must ensure overall cost consistency and full regulatory and operational compliance. By providing support to small and emerging (bio)pharmaceutical companies, CDMOs enable them to concentrate on understanding the market and differentiating their products, thereby facilitating the launch of new drugs.
Masoud Toloue, Ph.D., Chief Executive Officer, Quanterix Corporation
As the industry witnesses groundbreaking advancements in Alzheimer's disease (AD) therapies, including the recent approvals of Leqembi and Kisunla, it’s imperative to address the issue of global access to testing and these essential treatments, including in resource-limited settings. One of the most promising avenues to achieve equity in this capacity is through the innovation of non-invasive diagnostic tools, such as blood-based testing.
FDA-approved anti-amyloid treatments for AD are most beneficial to patients diagnosed in earlier stages of the disease. Traditionally, diagnosing AD has relied on invasive and expensive procedures like PET scans and CSF draws. These methods, while effective, pose significant barriers to widespread access in high-resource settings due to cost, complexity, and the need for specialized facilities, which can delay a diagnosis and efficacy of the drug.
Technologies like ultrasensitive biomarker detection platforms make it possible to pinpoint early signs of the disease with fewer resources, allowing for earlier intervention and better patient outcomes. Additionally, by collaborating with health systems and researchers across the country, it’s possible to build a global infrastructure for widespread blood testing, overcoming barriers of access for patients. This democratization of access ensures all patients, especially in resource-limited settings, have accurate diagnosis and treatment.
Fady Boctor, President and Chief Commercial Officer, Petros Pharmaceuticals
Fundamentally, improving access to essential medications globally via biopharmaceutical company contribution requires a multidimensional platform that produces a quantifiable, visible, and tangible value proposition for biopharmaceutical companies and their stakeholders, individually and as a consolidated global industry. We believe that disparate and individual biopharmaceutical contributions will not likely endure the breadth or depth of need globally or have the ability to establish a universal standard capable of fortifying change. This proposed multidimensional platform may include a broad collaboration between biopharmaceutical companies, state-governmental entities, international-governmental, and non-governmental organizations, and the establishment of a global sociopolitical healthcare culture that champions and reveals the existential interdependencies of global healthcare essentially driving a correlation of markets, consumer, and sociopolitical competitiveness among biopharmaceutical companies worldwide. In addition to aggressively developing affordable biosimilars and generics, along with global distribution partnerships and capabilities, biopharmaceutical companies, as an industry, will need to be induced and enticed to seek the competitive advantages of addressing a global population as a consolidated patient demographic of value.
Justin Kong, Associate Director of Commercial Manufacturing Project Management, Samsung Biologics
Adaptability is a critical capability that biopharmaceutical companies, whether limited in resources or not, need to hold on to their concerted mission — increase the chance of a healthier life for people — in the most erratic healthcare environment. Being readily prepared to tackle healthcare challenges requires resources, such as lab facilities for turning molecules into high-titer proteins, scalable bioreactors that suit best for culturing distinguished cells, experts ready to cope with unexpected problems while in bioprocessing operations, and regulatory expertise to handle time-sensitive queries from the FDA, to name a few.
The caveat is that not all companies have such resources in abundance.
This is where partnerships with CDMOs can play a role. Those who find it difficult to adapt to challenges can offset their relative lack of resources by partnering with a flexible CDMO.
Amid the COVID-19 pandemic, when vaccine manufacturers were troubled delivering enough vaccine shots on time, Samsung Biologics offered a line of readily available bioreactors, with knowhow to handle multi-layered supply chains, to help manufacture and deliver tens of thousands of COVID-19 vaccines to where needed in time per its client’s urgent demand.
Reports say more and more biopharmaceutical companies have asked for support from CDMOs to counterbalance resource deficits in recent years. Diseases advance too quickly, and time doesn’t wait for patients. In a fast-paced situation like this, collaborations between knowledge-rich biopharmaceutical companies and resource-rich CDMOs are critical for the former to maintain their readiness to overcome challenges and continue with their commitment to patients.
And a competitive CDMO, like Samsung Biologics, will be their trusted partner and work together to improve global access to essential medications as one team.
Jason Bock, Ph.D., Co-Founder and Chief Executive Officer, CTMC
The considerable focus on the high cost of cell and gene therapy ignores the potential transformation to patients. These therapies are designed as single doses with curative intent compared to traditional protocols requiring ongoing medications and a series of treatments over months or even years.
The prospect of providing single-dose treatments to patients with aggressive, refractory cancers is revolutionary. The potential long-term savings from cell and gene therapy could be significant if weighed against the cost of non-curative chemotherapies and radiation, along with the cost and burden of long-term and delayed adverse health effects due to traditional treatments.
As these modalities advance, the ability to administer them in remote settings will improve. First-line cancer treatments typically require multiple rounds of therapy, which are challenging both financially and logistically when patients — and their caregivers — live long distances from a major hospital. The availability of a single-dose curative therapy versus frequent front-line immunotherapy regimens would provide opportunity for patient populations facing barriers to receive cancer treatments, ultimately expanding access and reach.
Steve Amoussou-Guenou, PharmD, Technical Developer Manager Europe, Roquette
In short, by constantly improving production practices to shield vital supply chains from disruption. The World Health Organization (WHO) adopted its concept of “essential medicines” in 1973 from principles originally set out by military tradition as to which treatments were essential for soldiers to carry into battle. Fittingly then, healthcare systems which follow today’s WHO guidelines hold (bio)pharmaceutical companies to highly regimented standards. Producers are required to continually review business continuity plans to ensure patients can access medications that are both high quality and cost effective. Expectations in this area have only sharpened in the wake of the COVID-19 pandemic too. But with heightened pressure comes increased innovation — efforts to shore up the supply of essential medications have undoubtedly helped advance the field of drug delivery as a whole. Examples include the adoption of continuous manufacturing (CM) techniques that increase production speed, while reducing the out-of-specification (OOS) or product defect rate, the implementation of multiple ingredient sourcing, development of convenient new dosage forms and exploration of technologies like pharmaceutical compounding or 3D printing that help ensure the supply of niche, but nevertheless essential, medications.
Akihiro Ko, Chief Executive Officer and Co-Founder, Board Chairman, Elixirgen Therapeutics
Considering the significant need in so many communities, it’s great when companies are able to think about improving global access to medications throughout the drug development process. Part of the approach involves working hand-in-hand with the local government, non-profit organizations, and with other companies. One of the first things to think about is what specific needs a community may have, which requires being in close contact with experts who are working in those areas.
Also, when thinking about where to conduct clinical trials, companies can expand past the usual pool of sites and give underserved communities an opportunity to participate in these trials. In addition, when thinking about regulatory applications, companies can consider applying outside of the U.S. if they have data from ex-U.S. trials, which allows for greater access to medications upon approval. Lastly, a key component to increasing access to medications is to reduce the cost of drug development. There are many ways to do so, including automation of certain processes in the lab as well as in manufacturing.
I look forward to continuing to see how the industry evolves in its work to expand global patient access.
Jason King, Ph.D., Director, Business Development, Ascend Advanced Therapies
The price, particularly of cell and gene therapy medicines, is currently too high, even in relatively wealthy healthcare systems. This can make the challenge of serving less affluent regions even more daunting.
CDMOs can do several things to generally reduce the per-dose cost of these new medicines. These include developing more effective scaled-up or scaled-out manufacturing processes to generate more product. This is already needed for some indications during the clinical trial phase where larger amounts of vector are required per patient but must continue to improve as these new therapies make it into the commercial phase.
In addition to the scaling considerations, CDMOs can look to improve the volumetric or per-cell productivity of viral vector manufacturing so that more patient doses can be generated from the same sized bioreactor. In AAV vector manufacturing, vector productivity depends heavily on both the size and nature of the therapeutic cassette and the capsid type doing the actual delivery. The introduction of new production platforms and process development and intensification with existing ones has greatly improved AAV productivity, which a few years ago was around 1011vg/mL in the bioreactor and is now edging closer to 1012vg/mL levels.
Focusing on disease indications that require the smallest active vector doses may help establish gene therapy workflows in low-income regions. Ophthalmic indications for example may only need about 5 x 1010 vg per eye, which can result in a lot more patients being treated per bioreactor run.
Anil Kane, Ph.D., Senior Director, Global Technical Scientific Affairs, Pharma Services, Thermo Fisher Scientific
Essential medicines can save lives, reduce suffering and improve health. But to do so, they must be available and affordable, of good quality, and properly used. That still poses problems for several countries, due to the rising prices of new medicines, persisting shortages, and stock-outs, as well as increasing numbers of substandard and falsified medical products.
Having a trusted CDMO/CRO partner can help pharma companies in identifying the needs, planning and execution to bridge that accessibility gap — avoiding shortages and delivering these essential drugs to the patients. For example, as an integrated, end-to-end service provider, our teams at Thermo Fisher Scientific can coordinate between drug substance and drug product manufacturing, packaging, and quality/regulatory compliance to ensure a seamless, cost-effective manufacture and supply of medicines to regions, countries, and patients in need. Working with a CDMO that offers a global network of manufacturing sites, a proven regulatory track record, quality systems, infrastructure, experience, capacity, and global supply chain distribution solutions also helps facilitate flexible resource and capacity allocation in emergency and critical situations, bringing speed and agility to essential medicine development and manufacturing — and, ultimately, helping to facilitate access to emergency medicines.
