The biopharmaceutical industry has an opportunity to permanently adopt regulatory strategies that only a crisis can produce. These processes have the potential to accelerate approval and reduce the cost of drugs in the future, all while increasing the likelihood of their commercial success.

Time will also show that, when events predicate its action, science can move rapidly from an idea to a result. Clearly, the COVID-19 pandemic has done that for vaccine development. The rapid cohesion of established and new technologies by industry and government have already paid off in lives saved. The question to ask now is what has been learned from this public health emergency that can be applied more broadly to accelerate drug development in areas beyond COVID-19.

Keeping Attuned to What Works

The globally shared language of science is ultimately what sped the development of vaccines and treatments for COVID-19. Regulators moved just as fast. In under a year, manufacturers designed, developed, tested, and acquired authorization for multiple safe and highly efficacious vaccines for use in countries around the world. Now is a time to employ what has been learned from this success, realize these same efficiencies in development and manufacturing, and enhance development of better medicines for all diseases.

The duration of development and regulatory review are the 2 most obvious targets for faster approvals, especially if they can overlap. While the COVID-19 vaccines were developed at financial risk, using methods such as concurrent manufacturing scale-up and clinical evidence generation would enable development for all medicines including biologics to be sped up. No compromise in quality is entailed. Compendia can play an integral role in helping with fit-for-purpose bedrock technologies and shared quality priorities. A big game changer with COVID-19 vaccine development was the rapid identification and dissemination of viral sequence data to the global scientific community and the decentralization of this electronic information sharing. In the future, this could enhance communication on the sequences of emerging infectious diseases.

Each country has its own statutory framework, but science is the same. That is what has given us vaccines with worldwide application. As such, the value of regulatory reliance across jurisdictions is becoming more widely recognized—namely use of the same data and increasingly the same analyses to expedite common decisions across multiple regions, often where regulatory capacity is limited.

Leveraging Recent Experiences to Optimize the Future

How dire is the need to accelerate drug approvals?

COVID is clearly a public health emergency that warranted the reappropriation of many resources. However, unmet medical need is important always and can be balanced with the opportunity of a cure or mitigation of the worst symptoms. The role of public health can be formalized within the mission of US health agencies such that contingency planning with Congressionally appropriated resources are solidified. Even questions of debilitation versus death can be considered thoughtfully, especially as access, disability and disparity are confronted more broadly within society. While playing a key role, FDA does not stand alone on this frontier even though their regulatory role is more explicit than those of other sister Agencies such as CDC and NIH.

Can greater use of real-world experience be traded for earlier approval?

Real-world evidence has become ubiquitous in conversations on innovative trial design and drug development, but its effective applications are still unclear. It’s possible to view greater post-market surveillance as part of a fair trade for earlier market approval and use and not as an add on requirement to an already-large data package. That is essentially what the Emergency Use Authorizations (EUAs) for COVID vaccines and therapies represent. There are previous instances of accelerated approval of medicines for diseases with a clear unmet need where lack of benefit resulted in removal from a label. Similarly, hydroxychloroquine’s COVID EUA is a sign that this system is working and not a sign that that the initial access was wrong. This approach does require that the public understand that science is iterative; evidence generation is temporal and dynamic, sometimes confirming initial results and sometimes modifying them. Methods to balance what needs to be known ahead of human use with what can be safely demonstrated during use would vastly improve time to access, offering the potential to save and improve countless lives.

Does reassessing the plethora of priority approval programs have merit?

Multiple special programs at the Food & Drug Administration (FDA) offer expedited development and review. However, it may be time to reassess the complexity and value of the multiplicity of these programs, especially as must-pass industry user-fee legislation that support so many of the activities at the FDA are scheduled to sunset in 2022. Not every product can be at the front of the queue, yet all applications need timely review as they may benefit someone in dire need. Efficient review concurrent with efficient development is an aspirational goal that harmonizes and builds on current practices. If this became the norm, then there would not be a need to prioritize development of fast-tracked products over potentially more impactful products with larger patient populations that didn’t seek or weren’t granted a special designation.

How can manufacturing be scaled to meet demand promptly while maintaining quality?

Certain basics, such as Current Good Manufacturing Practices (CGMPs), are essential. How to expeditiously scale up quality manufacturing is equally critical – and one of the core lessons derived from the COVID vaccine process. Modular facilities, continuous manufacturing, and ways to flexibly move across facilities (and even across companies as needs must) is key to cost-effective and timely capacity to meet demand. The processes by which quality is assured are also important, with greater use of virtual tools and recognition of the value of establishment licenses for biologics (which comprise half of the full license). There are many opportunities in this space to enhance cross jurisdictional collaborations and regulatory reliance around the world.

What Is Possible Now?

The pandemic has shown us that coordinated and collaborative actions can leverage expertise while supporting regulatory alignment and global efficiency. This approach can now be extended well beyond this crisis, as today’s knowledge contributes to the development of tomorrow’s products. Consequently, application of this cumulative experience to know what to look for to anticipate safety and efficacy earlier in development is invaluable. Class effects are expected, especially for platform technologies, and can be appropriately labeled as part of the risk–benefit balance for any given medicine.

The FDA has decades of experience evaluating products with complex technologies, now including medicines made with nanotechnology, cellular and gene therapy products, and vaccines. Given an irrefutable global acknowledgment of the caliber of the agency, the FDA has the potential to “lean in” with confidence after this COVID-19 experience and work with sponsors on new ways to continue to define the pinnacle of regulatory oversight and cooperation in the interest of the patient.

The FDA has worked toward advancing and protecting public health by:

• Promoting innovation through an efficient and predictable science- and risk-based regulatory framework
• Strengthening public outreach and communication through strong, effective and transparent engagement with stakeholders
• Returning to engagement with domestic and international partners

For life science manufacturers, the opportunity is now available to engage with the FDA early to help the agency understand what would facilitate more efficient development and support more expeditious reviews, safely speeding time to approval for much needed medicines. Regulatory and clinical teams need a combination of expert guidance and executive leadership support to devise and successfully execute these new regulatory strategies. Collaborations between life sciences stakeholders—including patients—can ultimately get products to patients more quickly. Enabling faster adoption of state-of-the-art science may even mitigate the next pandemic while helping save and improve lives in the meantime. Learning from and extending the COVID-19 success stories has the potential to be immensely valuable to all stakeholders of the healthcare enterprise, which benefits humanity as a whole. Health is not a zero-sum game.

Avalere brings extensive knowledge and expertise in regulatory strategy to help clients navigate new and emerging environments and maximize the value potential of their assets by:

• Identifying opportunities to drive more efficient clinical development strategies aligned with the FDA’s new policies, objectives, and outlook
• Enhancing regulatory predictability through the integration of all forms of evidence for an FDA approval, as well as what payers, patients and their health care providers are demanding for reimbursement and commercialization purposes in all settings of care
• Delivering more timely market access in the US and global markets in a manner that uses the same data efficiently and concurrently

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