Drug Delivery Outlook: 7 Waves To Ride In 2025 And Beyond

By Fran DeGrazio, executive editor, Drug Delivery Leader

Looking out across the ocean of trends in healthcare needs and advancements, as well as the drivers for them, I see seven strong currents on the near horizon that should draw sustained focus from biopharmaceutical companies and their commercial partners in 2025 and beyond. Each of these key influences on attention and effort will bring challenges, to be sure. Nevertheless, given the goal of reaching patients effectively while achieving market success, the benefits outweigh the risks in every case. For each of the seven waves to ride, I offer here some directional guidance for drug and biological product developers and deliverers:

Wave #1: The Need To Heed Health Policy

Health policy, which encompasses the broad set of decisions, plans, and actions underpinning societal goals for effective and efficient healthcare, will become increasingly important. Central to that increased focus on health policy is the concept of Health Technology Assessment (HTA) – a multidisciplinary approach to evaluating new technologies in terms of clinical effectiveness, safety, and cost. More so than ever before, HTAs will be leveraged to inform or respond to health policy decisions. Biopharma sponsor companies, in particular, will need to apply them to drug and biologics product development, including delivery device and system development, with greater diligence and intention.

Take, for example, cost effectiveness, a key consideration of an HTA. Consider such scenarios as the delivery of a drug product via an on-body device, either by way of adding the device to the drug product or by way of developing the drug and device as a combination product. On the way to commercialization, justifying cost in relation to need and effectiveness will be critical to general acceptance in the marketplace from a health policy perspective.

Even more prone to scrutiny around cost justification are new and emerging ATMPs (advanced therapy medicinal products), which reflect a growing desire for more personalized approaches to treatment. The corresponding growth in the cell and gene therapy market is unmistakable: In 2024 the FDA approved 20 gene therapies. Also on the rise are Instances of CAR-T (Chimeric Antigen Receptor T-Cell) therapies for treating certain cancers and of stem-cell transplantation for treating blood disorders. Across the landscape of ATMP development, a significant and representative example of addressing a wider swath of heretofore unmet needs is the FDA’s approval in 2024 of the first genome-edited, hematopoietic stem cell-based gene therapy for sickle cell anemia patients older than 12.

To make these life-changing and lifesaving technologies more universally available without breaking the bank of cost, so to speak, there will need to be continued innovation, not only from a therapeutic standpoint but also in the consideration of effective and efficient processes, delivery approaches, and supply chains.

Given that biopharma companies may need to address input emerging from HTAs, they should infuse such thinking into their organizations, starting with early stage, cross-functional discussions of potential technologies to be employed in product development and, ultimately, delivery. Thinking about potential HTA-uncovered risk early helps provide sufficient time for identifying – and perhaps avoiding – the need for mitigation.

Wave #2: The Amplified Voice Of Patient Communities

The development of drug delivery devices, including combination products, demands diligent attention to Human Factors (HF), which takes into consideration patient use scenarios and related goals regarding convenience, adoptability, and effectiveness. In fact, attending to HF is a regulatory requirement for drug and device product developers. When assessing and applying technologies, especially new ones, it is essential for biopharma companies to honor the tacit agreement about priority focus reflected in the increasing use by industry of the term patient centricity.

Of growing influence and importance in the consideration of human factors are patient communities. These are groups of patients with similar medical/therapeutic conditions and needs who meet in person or online. There, they share what it is like to live with the illness all are experiencing, discuss various treatments, and provide emotional support.

Diabetes and various cancers are among the therapeutic areas around which formally organized patient communities have been formed. One of the world’s largest patient communities, PatientsLikeMe, serves as a kind of online consortium or hub of communities serving patients across more than 2,800 reported conditions.

Because they are already formed and active, such communities can and should be leveraged as unique and credible sources for biopharma companies to understand patient challenges and to receive feedback on and suggestions for innovation. Currently some of these communities are funded by industry.

Those working in the biopharmaceutical space, including and perhaps especially in the delivery device area, should eagerly and actively engage with patient groups. Leveraging these kinds of resources will better inform those involved in developing innovative drugs or devices and delivering them to the market.

Wave #3: The March Toward Global Harmonization

Biopharmaceutical companies looking to establish or grow a global footprint currently face significant challenges stemming from distinctly different regulatory requirements across jurisdictions. Chief among those challenges is the task of overcoming the resulting process inefficiencies in two fundamental areas: 1) product development, including technical aspects of drug delivery, and, of course, 2) regulatory activities on the way to approval and commercialization.

For those reasons and others, there has been a tremendous amount of effort toward global harmonization of regulatory standards and of related requirements for technical components. Those efforts will continue with the goal of accelerated convergence wherever possible.

A critical contributor to efforts toward greater alignment is participation by industry in standards organizations such as ISO (International Organization for Standardization) and ICH (International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use). Having industry provide resources to these organizations is fundamental to forwarding their missions.

As an example of the current challenges and the drivers for global harmonization, consider a typical drug product development process. When a biopharmaceutical company is applying Quality by Design (QbD) principles, it is likely leveraging several ICH guidances. If, along the way, a robust target product profile (TPP) with critical quality attributes (CQAs) has been established for the drug product, all manufactured iterations of it should be able to be validated as technically equivalent regardless of where on the globe and in how many locations production is occurring. Furthermore, technical equivalence should be provable even across multiple locations, including those manufacturing with slightly different equipment than is being used elsewhere.

That example illustrates the need for increased and sustained efforts toward global harmonization. While the FDA has indeed encouraged the use of QbD in drug development, it has done so by way of a recommended best practice, not as a formal regulatory requirement. To contribute their part to harmonization, biopharma companies should diligently and intentionally echo the FDA recommendation by incorporating these internationally applicable and proven best practices into their R&D and manufacturing sites. Such activity includes educating personnel and defining owners to ensure execution.

Wave #4: The Maturation Of Quality As Culture

The future of biopharmaceutical product development and delivery was infused with a significantly underscored emphasis on quality in January 2024. That’s when FDA published its Final Rule amending the approach to quality as outlined in 21 CFR 820. Now referred to as the Quality Management System Regulation, or QMSR, the guidance echoes the drive toward global harmonization I commented on above. (I offered my take on the QMSR in a previous commentary.) One of the FDA’s main goals in issuing updated and expanded guidance on quality is to align with other global regulatory authorities and promote consistency in the regulation of devices and drug/device combination products.

To a large degree, regulatory attention to quality management is not new. The QMSR reflects a continued FDA desire and expectation that biopharmaceutical organizations build cultures of quality. Since late 2022, FDA has been strongly focused on encouraging Quality Management Maturity (QMM) within the industry. The agency sees it as a way to help stabilize the pharmaceutical supply chain and avoid the potential of drug shortages due to inferior quality. FDA has executed several pilots and has engaged industry in various workshops and other related activities. As of the time of this writing, FDA has identified  111 drugs facing shortages.

For biopharmaceutical organizations not yet familiar with or not yet having fully operationalized a QMM approach, I strongly recommend learning about this initiative and actively defining a plan for taking it forward. The first step would be assessing the current state of your organization’s quality management.

Along the way of helping to guide and build a culture of quality within a biopharmaceutical company, QMM can also be utilized in the evaluation, qualification, and management of suppliers who also incorporate QMM as a best practice. One fundamental goal of assessing biopharmaceutical manufacturers’ commitment to quality is to incent them to invest in QMM. Investing in more mature quality practices reduces the likelihood of disruptions and product losses.

Those reductions should result in gains in operational efficiency, in customer safety and satisfaction and, hopefully, in revenue. Frankly, for companies that demonstrate a fully operationalized and, therefore, “mature” culture of quality, business growth associated with market differentiation should follow.

Wave #5: Efficiency In Product Development And Regulatory Activities  

Along the way of getting safe and effective products to patients, the industry needs to execute in an increasingly efficient manner. That imperative comes from regulatory agencies and therefore should inform communications and interactions with them.

In listening to FDA representatives at recent conferences, I have heard the agency’s consistent expression of support for generating efficiencies in both operational and regulatory processes. On the latter front, the goal of streamlining regulatory review processes without impacting product quality and safety stems from the acknowledged reality that neither industry nor the FDA has unlimited resources.

With that encouragement and acknowledgement in mind, biopharmaceutical organizations will want to consider applying “lean” thought processes to, for example, the CMC work executed by internal regulatory staff. They will want to be increasingly thoughtful, clear, and logical about the data being generated during product development and in the story presented during the submission process. Of critical importance will be the ability to demonstrate technically sound reasons for the decisions made, the data generated, and documentation submitted.

Frankly, It’s not about the volume of work or documents. Rather, it’s about logic, science, and clarity. Demonstrating those priorities with efficiency – supported by just enough documentation to paint a credible and trustworthy picture – will be the expectation of regulators.

Wave #6: The Application Of Digital Tools And Artificial Intelligence

We are living and working amid significant and understandable excitement around digital transformation and artificial intelligence. While I share much of the enthusiasm, I also recommend thoroughly vetting any decision or justification for bringing a digital or AI solution forward. Is the market need well understood? I have seen IT technologists generate many digital healthcare concepts that have gone nowhere due to the lack of true understanding of the benefits that could be delivered, as well as the relative cost and value of each.

In that regard, we find ourselves at the inflection point I highlighted above – that is, doing more to leverage HTAs (health technology assessments) within a landscape in which health policy will have increased influence on product development and commercialization. For example, would the benefit of having data gathered by software added to a medical or delivery device be sufficient for health policy stakeholders to endorse the expansion of the products and for health insurance payers to cover the cost?

In general, explorations of the use of software, AI, and other digital solutions – whether to inform product development and/or to become a component of the product itself – should begin early in the development process. The knowledge gained – particularly from human feedback loops – will be usefully dynamic and iterative. As a result, it can be actively used to guide risk management and process controls, including during manufacturing scale-up and throughout the product lifecycle.

One of the most significant industry challenges, particularly in combination product development, is understanding the intersection of various products and components (e.g., drug and device), as well as their critical process parameters and the changes that may occur over time. Given the complexity, biopharmaceutical organizations should commit to maintaining a central knowledge repository that brings together process data (e.g., sensors), process analytical technology data (e.g., near-infrared, or NIR, data) and human input (e.g., observations,  notes, and QC testing).

Whether or not your organization is developing drug delivery products or other devices that incorporate digital and AI software capabilities, a minimum goal should be to soon identify and implement (if not already done) digital tools for bolstering product development with data collected and crunched from a comprehensive set of relevant inputs.

Wave #7: The Agility Imperative

As has been said by many a wise prognosticator, the only constant is change. As the six trends already outlined above remind us, that is also true of the biopharmaceutical industry – increasingly so. For that reason, change management remains an even more critically fundamental component of product development and related regulatory activities. Being consistently successful at change management requires organizational agility.

Whether it be the introduction of innovative technologies or the publication of new or updated regulations and guidance, expeditious adoption of the new and the emerging can provide real efficiencies and true differentiation. Of course, there are many challenges in behaving and performing as an agile biopharmaceutical organization. Agility must move from concept to culture.

To get there, I suggest undertaking a diligent, honest appraisal of the flexibility of current, critical processes. Speak with colleagues and employees to understand the most significant bottlenecks. Focus on understanding whether faster and improved decision-making is necessary and, if so, how to streamline it in the spirit of accelerated change. Commit to removing bureaucratic silos and protocols to clear the path for a nimbler, more collaborative and, ultimately, more entrepreneurial mindset across the organization.

I believe that addressing the seven waves outlined above can be business transformative and patient health transformative. Frankly, it’s time to ride them.