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AbbVie Pedro Valencia ADCs

PE: How is AbbVie using novel cancer biomarkers to develop potential first-in-class ADCs for the treatment of advanced solid tumors?

We're constantly looking for biomarkers that can help us to do two key things. The first is to tackle cancer cells. and spare normal cells. We’re looking for specific antigens that are over-expressing tumor cells, and you see limited expression in normal cells. We want to make sure that target that’s over-expressed is critical for the survival of the cancer cell. Once you block it, you can actually drive a cancer cell death. That’s what we're looking for. In that perspective, c-Met is one of those biomarkers that is over expressing multiple tumor types such as lung, colorectal, gastric, and other GA tumors. We as an industry have tried to tackle c-Met in the past but have been unsuccessful. It would actually be successful to tackle what we call met, which is a gene inside the cell.

There’s this thing called met amplification, where the cancer cell produces multiple copies of the gene, and around 30% of patients have it. We can actually tackle that with small molecules. But once the gene becomes a protein and gets over-expressed in the cell, that over-expression is something we haven’t been able to target. For example, we know that on lung cancer, c-Met over-expression is about 25% to 50% in colorectal cancer, and it could be anywhere from 30% to 70% depending how you measure it, so it’s quite prevalent.

With ADCs, we’re trying to tackle c-Met over-expression on the surface of cancer cells. That was novel, and we can do it through ADCs. 706 has a target called SEZ6, and it’s a target that is over expressed in neuroendocrine cells or neuroendocrine neoplasms. When you think about it, multiple tumor types kind of have a subset of cells that are neuroendocrine driven. Small cell lung cancer is one of the few that has everything driven by neuroendocrine cells. The beauty of SEZ6 is over-expressing those cells and limited expression in normal cells. So, we can tackle it with an ADC and drive the results that we're seeing today.

PE: ADCs have redefined the treatment of cancer in recent years. Can you please discuss the promise of this emerging class and what the future may hold?

At the end of the day, ADCs work like a trojan horse. You have a good antibody that cancer cells are happy to receive, but once you hook into the cancer cell receptor, you deliver the payload, which is pretty much chemotherapy. It’s actually a highly potent version of the regular chemotherapy cancer patients are given on a regular basis. So, from that perspective, our goal is to replace chemotherapy. Our grand goal with ADCs is to replace good old, decades old chemotherapy that is really toxic for some patients. That’s how we see the redefinition of cancer treatment in the future with ADCs. So, what do we see in the future? Well, if you think about it, chemotherapy is being given in combination with other agents. In the future, we see ADCs being given in combination with chemotherapy. There will be one with other agents such as immuno-oncology agents. So, if you combine the ADC and the PD1s, you can start seeing that synergy come together and kind of start really dreaming about cures for the patients. All in all, we're super excited about ADCs replacing chemotherapy. 

In an interview with Pharm Exec Associate Editor Don Tracy, Pedro Valencia, VP, Solid Tumor Pipeline Strategy & Execution, AbbVie, discusses using novel cancer biomarkers to develop potential first-in-class antibody drug conjugates (ADCs) and the promise that they offer. 

Utilizing Biomarkers to Develop First-in-Class Antibody Drug Conjugates to Treat Advanced Solid Tumors

A more harmonized formula and schedule for administering anti-COVID-19 vaccines gained strong support from FDA’s expert advisory panel last week, with agreement on the need to reduce confusion over which shot to take and when in order to increase vaccination rates among both adults and children in the US. The 21 members of FDA’s Vaccine and Related Biological Products Advisory Committee (VRBAC) voted unanimously for a more simplified approach, setting the stage for the agency to authorize a common bivalent shot for all patients that targets the original COVID strain and the Omicron subvariants. The new program would end use of Pfizer and Moderna monovalent vaccines that only target the original COVID strain.

FDA received less support for setting a schedule for annual vaccination against COVID, as the advisors expressed uncertainty about the effectiveness of the current shots and questioned what time during the year would be best for a national COVID vaccination campaign. Several panel members urged additional research on the effectiveness of the new formulations on frequently changing variants and the possible need for different vaccination schedules for both children and older adults with health impairments. The experts called on FDA to provide additional data on vaccine effectiveness and timing to better determine whether all patients would need an annual shot.

The strategy to set a common formula for anti-COVID vaccines across the country aims to increase vaccination rates overall. New data from the Centers for Disease Control and Prevention (CDC) indicates that less than half of all older Americans (over age 65) have received the updated booster, and that vaccination rates are a meager 10% for children under age 4. The hope is that a shift away from differing vaccine compositions and varying immunization schedules, along with clearer alignment between US and European vaccination recommendations, will encourage more people to get such protection.

Pfizer, Moderna, and Novavax reported to VERBAC on efforts underway to produce reformulated bivalent vaccines and supported the plan to establish a common formulation for all manufacturers of the adopted shot to simplify vaccination recommendations and reduce patient uncertainty over effectiveness and safety of vaccines from different companies. The manufacturers emphasized the need for specifics from FDA on the process for updating vaccine strains, including the timing, process, and methodology for producing a data package for new licensure. While such filings are expected to include new preclinical and manufacturing data, sponsors opposed requirements for additional clinical trials, indicating that it will be more productive to assess post-authorization real-world data to confirm effectiveness.

FDA officials proposed to meet with the advisory panel in May or June to decide on which COVID strain to target in vaccines for later this year, anticipating a fast reformulation by manufacturers of mRNA vaccines. But Novavax officials indicated that it might need to identify the strain earlier, probably in March, to produce a new vaccine by fall with its more traditional vaccine production process. All three shot manufacturers reported that they were already shifting to use prefilled syringes and single-dose vials, which are important for physicians, clinics, and pharmacies planning to administer the new vaccines more widely in the wake of reduced federal government involvement in the program.

A related question is whether FDA will ask manufacturers to produce combination influenza/COVID vaccines to further simplify annual vaccination programs. The three firms reported that they are already studying such combination shots, but any shift toward such products will involve much further assessment of the need for and effectiveness of annual COVID vaccines or combination products.

While advisory committee experts and officials from FDA and other health agencies acknowledged the value of utilizing mRNA platforms to produce anti-COVID vaccines so quickly and effectively at the start of the pandemic, some panel members called for further research on how well the new formulations provide long-term protection and avoid safety issues, urging continued examination of whether more traditional vaccine production methods may have broader benefits moving forward. In its presentation, Novavax presented data indicating a “robust breadth of immunity” and strong manufacturing consistency for its more recently approved preventive.

In concluding the meeting, Peter Marks, director of the Center forBiologics Evaluation and Research (CBER), reiterated the need for a “data-driven approach” to achieve a vaccine regimen that is as simple as possible. But he also noted the importance of monitoring vaccine safety and assessing product effectiveness in shifting to a next generation of COVID vaccines able to provide “greater depth of protection.” 

Agency panel backs the use of a common bivalent shot for all patients.

FDA Seeks to Simplify COVID Vaccination Program

The European Parliament’s voluminous report

 says a little about COVID, and a lot about the challenges that Europe faces in continuing to host a thriving medicines industry. In particular, the report and its attendant debates reveal the depths of the split in public—or at any rate parliamentary—opinion about science, medicines, and management. The episode also suggests some fragility in the centrist tendencies that–largely to the benefit of the pharmaceutical industry—have dominated European policy for decades, of particular significance given the impending change in EU leadership due in 2024.

First, what does the report say about COVID and the EU? Plenty of self-congratulation about the management of a crisis, about valiant researchers and doctors, about the massive delivery of vaccines in the EU, and about sterling efforts to provide for the rest of the world. All rather predictable and rather anodyne.

It also makes plenty of positive comments on the contribution of the healthcare industries. Scientific research and innovation “allowed the development and roll-out of COVID-19 vaccines in record time, therefore saving millions of lives worldwide,” says one of its clauses. “The speed at which researchers developed effective vaccine protection was unprecedented,” says another.

The report also carries stout defenses of orthodoxy. The benefits of the vaccines “far outweighed their potential risks,” and “complemented by the use of conditional marketing authorization, contributed to a remarkable reduction in deaths and hospitalizations in the EU, as well as overall protection from the most severe effects of COVID-19.” It “underlines the importance of decision-making based on scientific evidence.” And the report makes plenty of suggestions for more innovation-friendly regulation, even urging the EU to explore the same approach beyond pandemic situations.

So what’s not to like? Perhaps this: “COVID-19 has laid bare the ways in which the market-driven model of drug development and production can work against the equitable and affordable distribution of emergency medical products.” The report “notes with concern the lack of preconditions attached to public investments in the COVID-19 vaccines, as well as medicine development that could have facilitated greater public returns on public investments.” Or this: the report also “notes with concern the transfer of financial risks related to liability for serious adverse effects of COVID-19 vaccines to the member states and the risk of this becoming a standard practice.” Also this: “National pricing should be based on fully transparent factors, such as the real costs of public and private R&D and added therapeutic value.”

These are just some of the critical observations that made their way into the final report. But they are in many respects only a pale shadow of what did not get in—the dozens of amendments from skeptical MEPs who accused the mainstream political groups of conniving to produce a report that is “a whitewash,” “a cover-up,” “a farce.”

The debate reflected a deep distrust of imposed lockdowns and novel treatments. It articulated widely-held anger in the parliament at the refusal of the EU to divulge details of payments and liability arrangements in the contracts with industry. And it resurrected the long-standing concerns about the role of the European Commission president in exchanging private messages with the head of Pfizer outside the formal vaccine contract negotiation procedures.

 The failure to disclose the messages “has critically deprived” the parliament of essential information for its evaluation, according to the parliament’s ECR Group.

 Confidence was not helped by “the outright refusal of Pfizer’s Albert Bourla to participate in a hearing” and “the refusal of Commission President Ursula Von der Leyen,” which “raises grave concerns about the Commission’s commitment to transparency and its lack of democratic accountability,” said the group.

Although much of the criticism has been sidelined by the main political groups, who claim to be taking a noble stand against “populism,” there is little doubt that the reputation of the EU and its handling of the pandemic remains tarnished and that serious doubts have been implanted about EU credibility. As the EU gears up to elect a new parliament and appoint a new European Commission next year, the risk rises of political fragmentation—fed in part by resentment. That cannot be a helpful scenario for the pharmaceutical sector.

1. Report on the COVID-19 Pandemic: Lessons Learned and Recommendations for the Future. European Parliament. June 26, 2023, 

https://www.europarl.europa.eu/doceo/document/A-9-2023-0217_EN.html  

2. Verbatim Report of Proceedings. European Parliament. July 11, 2023, 

https://www.europarl.europa.eu/doceo/document/CRE-9-2023-07-11-ITM-010_EN.html

3. EU's von der Leyen Can't Find Texts With Pfizer Chief On Vaccine Deal -Letter. Reuters. June 29, 2022, 

https://www.reuters.com/world/europe/eus-von-der-leyen-cant-find-texts-with-pfizer-chief-vaccine-deal-letter-2022-06-29/

4. Amendment 17, Robert Roos, On Behalf of the ECR Group. European Parliament. July 5, 2023, 

https://www.europarl.europa.eu/doceo/document/A-9-2023-0217-AM-017-017_EN.pdf 

Report on lessons from crisis includes—and ignites—some criticism. 

Do Findings Point to a Tarnished COVID Victory for Europe?

Amid the search for effective COVID-19 treatments and vaccines, the pandemic will have long-term side effects for the global pharmaceutical industry. Drug manufacturers will need to focus on change in three areas—portfolio reprioritization, accelerated R&D, and technology transformation—if they are to position their businesses successfully for the future.

To that end, organizations should address major strategic questions now. In this article, we look at the impact of the coronavirus pandemic on the life sciences and the long-term prognosis, and suggest some priorities for executives to consider in order to position their companies best.

Our assessment is that the life sciences industry will see significant change in at least those three key areas mentioned.

Companies that are allocating significant resources to COVID-19 are forced to reprioritize their limited resources, reducing their focus on other activities.

 The urgency to identify treatments (from either new or approved medicines) and vaccines has caused health authorities to turn things around with unparalleled speed. This will raise expectations for shorter timelines in the future.

Rapid adoption of technology and transforming operations.

 This involves digital innovation, especially to solve the dilemma of the pharma “innovation paradox,” whereby highly innovative companies in R&D are quite conservative in operational innovation.

Refocusing significant efforts on COVID-19 will not be without consequences. A dollar can be spent only once, and even though people and systems can be more productive, this will inevitably lead to deprioritization of R&D activity in other areas.

A significant number of companies are allocating substantial resources to either developing or identifying treatments or developing vaccines for COVID-19. This will require reallocating resources (people, facilities, budgets), which will lead to other programs being delayed or cancelled. As it is difficult to cancel an ongoing clinical trial, this will mostly impact programs that have not yet started. This means, for some patients, it will take longer for treatments to become available, or it may never happen. Clinical programs have expiration dates linked to patents, and must be executed by certain times to be attractive commercially.

Although the industry is generally cash rich, there are plenty of early stage companies that will find it hard to survive this crisis, and others that are currently vulnerable due to other factors. This may mean good M&A opportunities for private equity, venture capital, and larger pharma companies.

The pandemic is forcing companies, regulators, and governments to enhance collaboration and speed up R&D activity in multiple ways. Regulators have demonstrated that they can act fast if they need to, and this potentially sets new standards for the future.

The urgent drive for treatments and vaccines is forcing companies to do things in ways they haven’t done before at this scale—although the Ebola and H1N1 scenarios hold learnings that are being leveraged. Organizations are also compelling regulators to (re)act much faster than they are used to. Clearly these are extraordinary circumstances, and some of this progress will fall away when the situation normalizes. However, some of the lessons that companies and regulators are learning should stick, which will result in a smoother and faster process. Certain caveats remain—a clinical trial that takes two years to perform cannot feasibly be delivered in less time. The randomized clinical trial will remain preeminent.

The pharmaceutical industry is home to an interesting paradox. It has a well-deserved reputation for innovation, based on its R&D output and scientific prowess. However, when you shift from science to operations, there is generally resistance to innovation and doing things differently. This means that while organizations have been looking at options for telehealth, virtual trials, wearables, etc., progress has been cautious and slow.

From an operational perspective, most of the pharma industry has rapidly adopted working from home, opening quite a few eyes along the way. Remotely selling medicines or conducting clinical trials is a lot harder, but significant efforts are being made to adapt operations. Research labs and manufacturing facilities need to maintain core operations on-site, but the overall impact has been enough for pharma executives to start making plans for inventive new ways of working.

Pharma has been reluctant to adopt digital beyond iPads for sales reps and other logical applications. COVID-19 has dramatically changed this attitude, with future concepts now up and running. With pharma company associates not allowed into hospitals to monitor clinical trials, sales reps not welcome at doctors’ offices, and patients unwilling or unable to go to trial sites to get their medications or undergo their assessments, companies have had to be agile to maintain the integrity of trials and protect their major investments. This has opened the door to home visits (a key component of virtual trials) and implementation of telemedicine, which are expected to continue.

The same applies to working arrangements, with companies concluding that they can safely rethink these quite radically.

Beyond innovation and operations, these changes may have a dramatic impact on culture. Taking risks in operations, rather than just in major development programs, will become acceptable. Companies will, therefore, need to develop new capabilities to manage these.

Many, when discussing the pandemic, now use the already cliché phrase “the new normal.” Pharma executives, who are used to thinking long term while, at the same time, providing shorter-term updates to Wall Street analysts, are looking well beyond the crisis and planning how to best position their companies to take advantage of the changed world.

Currently, it appears the life sciences sector is not going to be hit very hard financially. Although there is significant longer-term uncertainty, compared to other sectors, pharma is positioned to come out well. There will be strategic opportunities for new operating models, increased productivity from new and accelerated technology, and potentially more efficient ways to market as regulators change how they operate.

However, the overall economic environment will change significantly and impact how well pharma companies can recover and grow. Healthcare costs are an obvious target, which means drug prices will be under close scrutiny. This will impact the top line, and executives have to look at how to maintain margins, including new opportunities uncovered during the crisis. Hospitals have been hard hit financially, which will impact their spending and ability to support clinical trials going forward.

 Having been forced into wholesale adoption of technology much faster than anticipated or desired, life sciences companies will come out of the crisis with a new set of capabilities and a thirst for putting them to work. Telemedicine, artificial intelligence and machine learning, data science, and a range of digital tools are now being used in clinical trials, sales activities, and other areas. The drive to implement and innovate outside R&D has become stronger. Full transformation will not happen overnight, but the industry will not return to pre-crisis operating methods.

Most experts expect that the current coronavirus, and potential new ones, will not depart soon. This means the pharma industry has a role to play in the long term, and a commercial model is needed—one that aligns with public expectations to avoid impacting the industry’s reputation. Most of the companies engaged in COVID-19-related trials will just come out of it with experience and goodwill. A few will be successful, and given the size of the market for both therapy and vaccines, blockbuster status seems assured. Scaling fast and globally will be a challenge, but it is a core competency of big pharma.

Who “wins” could impact the whole industry—as shown by the share price of Moderna Therapeutics quadrupling in less than three months on high hopes for its vaccine development.

The crisis is an opportunity for the pharma industry to do what it excels at—developing innovative therapies and vaccines by using its scientific and operational capabilities. A side benefit would be enabling the sector to reshape its public image into something much more positive. In our view, the life sciences sector will come out of this crisis stronger, but this won’t happen automatically. There are critical takeaways for executives as they position their companies.

Leaders need to consider current strategies relative to the new environment. What is changing in the therapeutic areas in whichtheir company is active, and does it need to adapt? Are there any assets out there that are complementary to internal portfolios and can be acquired for good prices? Is the risk landscape changing?

If the R&D timeline is shortened, what does that mean for a company specifically? Where in the organization do leaders need to change and bolster capabilities so it can take maximum advantage of shifting landscapes and timelines? If companies make those choices now, they will have a head start when the “new normal” becomes actually normal.

Pharma needs to step up the development of data science and digital capabilities. Companies understand this, and although some have made progress, it needs to become ubiquitous. This will push pharma over the hump and turn it into the data industry it really wants to be.

Lastly, if an organization is not in the coronavirus rat race of developing therapeutics or vaccines, it should build an understanding of the areas deprioritized by others, and execute its strategy to take advantage.

Manager and US Lead for Energy & Utilities, Both with 

In adjusting to the long-term aftershocks from the coronavirus pandemic, pharma companies will need to focus on change in three core areas.

Strategic Realignment for a Better Future 1

COVID-19 brought in enormous attention to Pharma resilience as the world faced a global pandemic and prepared for an unprecedented vaccine rollout across the world. But the importance of this topic is not limited to the pandemic. Climate changes, geopolitical tensions/wars, cyber-attacks, reputational damages, financial risks, currency risks, natural disasters, solar flares, and many unforeseen issues can cause massive supply chain network disruptions and impact the availability of life-saving drugs and critical therapies. Many similar disparate incidents caused disruptions in the global pharmaceutical supply chain. In addition to this constant challenge of counterfeiting, regulatory changes, etc., are challenges that a pharma organization needs to plan for. A study by Cytiva highlights that global pharma resilience has weakened since 2021.

The potential consequences of not investing in resilience and recovery are not just severe but can be catastrophic, including loss of brand reputation, compromised consumer safety, and financial instability. Hence, in this modern age, it has become more critical for any pharma organization to have a robust strategy for any disasters to protect its brands and ensure its consumers' safety. Pharmaceutical organizations bear a crucial responsibility for patient safety and ensuring the uninterrupted production of drug substances for the final product is not just a business imperative but an ethical obligation.

This article will focus mainly on the business continuity and recovery of Information Technology (IT) systems, but other aspects of resilience are equally important. One stark example of the impact of IT on global pharmaceutical operations is the cyberattack that Merck reported in 2017, which caused revenue impacts of $260 million in 2017 and $150 million in 2018.

It is crucial to be prepared for supply-chain disruptions caused by malicious activities such as cyberattacks and accidental issues such as the recent CrowdStrike, which caused major disruptions worldwide. Such issues can lead to significant data loss for businesses, with 96% of victims experiencing more than a day of downtime, which impacts production.

There are crucial steps for pharma organizations to practice resiliency. The first step for a pharma organization is to assess its vulnerabilities and strengths. The second step is prioritizing initiatives to address the gaps, reinforce strengths, and monitor critical external events. The final step is to implement, monitor, and adapt the initiatives and learnings to the resilience and recovery plans, making this an ongoing process. Preparedness, response, and recovery strategies must be meticulously laid out to face any eventuality and to reduce the impact, with patient safety at the forefront of these efforts. Overall, these steps can be encapsulated in a robust Information Security Management System (ISMS). A pharma organization should do the following to establish ISMS:

Establish policies with clear roles and responsibilities.

Identify and assess information security risks.

Implement controls and safeguards to mitigate identified risks and protect information assets.

Develop procedures for detecting and responding/recovering from information security incidents.

Monitor the effectiveness of information security controls through regular reviews and audits.

Ensure compliance with relevant legal, regulatory, and contractual requirements related to information security.

Now, pharma executives must dive into other common risks and how they can be managed and mitigated.

Pharmaceutical companies typically maintain high inventory levels to minimize supply chain vulnerabilities, but these are not immune to risks. Diversifying material sources, localizing supply chains, and using scenario planning and digital twin simulation to gauge the impact of production slowdowns/ shutdowns help mitigate these risks.

Capacity buffers and considering geopolitical developments are also crucial for minimizing drug shortages. Pharmaceutical manufacturers are subject to strict regulations regarding traceability and batch serialization. Any downtime during a batch run would likely result in the entire batch being discarded, disrupting the continuity of the batch data. This waste has significant cost implications regarding materials, time, and profit. Additionally, backlogs at shipping ports can lead to short-term disruptions, while global events, trade wars, and geopolitical risks pose long-term threats to capacity and capability. Businesses in heavily regulated sectors such as healthcare and personal finance face severe fines and penalties for data breaches due to the critical nature of the data they handle. Implementing a robust disaster recovery strategy can help reduce response and recovery times after an unplanned incident, which is particularly critical in sectors like pharmaceuticals, where the financial penalty often depends on the duration of the breach.

These risks encompass the potential for new drugs not being approved by regulatory agencies, alterations in regulations, and heightened scrutiny of drug pricing. Companies can mitigate these risks by staying informed about regulatory changes and ensuring compliance with current regulations. Investing in research and development can help companies ensure that their products meet the required safety and efficacy standards.

Legal risk may arise from product liability, intellectual property, and antitrust issues, potentially leading to lawsuits. Companies can reduce these risks by seeking legal advice and establishing strong compliance programs. These financial risks include declining revenues, heightened competition, and the high costs associated with research and development. To reduce these risks, companies can diversify their product portfolio, implement cost-cutting measures, and invest in new technologies to enhance their operations.

Reputational risks encompass negative publicity and harm to brand image. To address these risks, companies should prioritize transparency, clear communication, and upholding high ethical standards. In the pharmaceutical industry, managing above risk is of paramount importance. Having disaster resilience and business continuity systems in place is essential to minimizing the impact of these risks. The main goal of BCDR (Business Continuity and Disaster Recovery) is to minimize the impact of outages and disruptions on business operations. Planning documents are crucial for an effective BCDR strategy and aid in resource management. They provide information such as employee contact lists, emergency contacts, vendor lists, test procedures, equipment lists, alternate shipment routes, technical diagrams of redundant systems and networks, etc.

Business continuity (BC) mainly focuses on the organization, while disaster recovery (DR) emphasizes the technology infrastructure. A business continuity plan includes contact information, change management procedures, guidelines for plan usage, step-by-step procedures, and a schedule for reviewing, testing, and updating the plan.

Disaster recovery is a component of business continuity planning that deals explicitly with quickly retrieving data following a disaster. A disaster recovery plan includes crucial action steps during a disaster, contact information, DR team responsibilities, plan usage guidelines, policy statement, geographical risk information, and incident response and recovery steps.

The primary goal of pharmaceutical companies' resilience strategy is to use their risk management functions to enhance patient outcomes. To achieve this, an internal risk committee should be formed, consisting of the company's operational leadership team and risk experts, potentially at different levels of the organization. This helps ensure accountability for assessing and managing risk across the company. The committee should have strong analytic capabilities and access to tools that provide useful data. It's important to establish strong connections with existing governance forums to secure ownership of risk by functional leaders. Leadership teams should map out production sites, distribution centers, and material flows to increase transparency against potential hazards. Regular employee training on Disaster Recovery Plans, roles, and responsibilities is crucial. Conducting mock drills periodically to assess and improve response is recommended. In addition to backing up computer data, keeping an offsite list of insurance policies, banking information, phone numbers, and email addresses of employees, key customers, vendors, suppliers, and other key contacts is essential. There should also be an inventory of business equipment, supplies, and merchandise. By digitizing and centralizing data related to resource allocation and utilization, pharmaceutical companies can optimize resource planning, prevent shortages or overages, and improve operational efficiency.

, Head of Healthcare and Life Sciences, Zensar

2023 Global Biopharma Resilience Index, by Cytiva. Link: 

https://cdn.cytivalifesciences.com/api/public/content/dfNVgLAyxEGcOE2XCy8Ydg-original

Merck Announces Fourth-Quarter and Full-Year 2018 Financial Results. Link: 

https://www.merck.com/news/merck-announces-fourth-quarter-and-full-year-2018-financial-results/

To safeguard patient access to life-saving medications, pharma companies must prioritize building robust resilience strategies that encompass IT systems, inventory management, risk mitigation, and regulatory compliance.

A Roadmap to Pharma Resilience

The pharmaceutical industry stands at a crossroads of innovation, science, and stringent compliance. Predicting the regulatory pathway for any newly developing drug has never been an exact science. Consequently, achieving approval into the pharmaceutical market has always been expensive, time-consuming, and stressful for all stakeholders. Navigating this intricate landscape presents challenges for stakeholders—from global corporations to emerging biotechs. These hurdles arise from varying regulations across regions, rapidly advancing technologies, and the need to balance safety with innovation. In the United States, the biomedical industry encounters numerous regulatory challenges in conducting nonclinical and clinical studies and managing chemistry, manufacturing, and controls (CMC) activities. While the industry faces several obstacles, drug manufacturers can adopt strategies to address them effectively.

As recognized gatekeepers to the global marketplace, the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) are the starting points for many drugs introduced today. According to the Centre for Innovation in Regulatory Science,

 the FDA approves the highest number of new active substances among the world’s six major regulatory agencies, which, aside from FDA and EMA, include Canada’s Therapeutic Products Directorate, China’s National Medical Products Administration, Australia’s Therapeutic Goods Administration, and Japan’s Pharmaceuticals and Medical Devices Agency. This number continues to increase. In 2023, the FDA approved 55 new drugs, a 50 percent increase from the previous year (37 new drugs) and the second-highest number of new drugs approved over the past three decades. Despite the large caseload, FDA officials boast various efficiencies that highlight the overall success and validity of the administration’s review process, including a 76 percent rate of novel drug approvals by the administration’s Center for Drug Evaluation and Research on the first cycle. Furthermore, of the 37 novel drugs approved by the FDA in 2022, 25 (or 68 percent) were first approved for use in the United States, according to the FDA.

Nonclinical studies and regulatory challenges

While limited in early development, nonclinical studies provide critical insights into potential risks compared to the evaluation of safety, efficacy, dosage, and side effects that clinical studies can offer. There is no set number or specific typeofnonclinical studies required to satisfy safetyassessments. Flexibility exists for each new compound. The traditional approach is outlined in guidance documents from the FDA and the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use. These guidelines typically involve a combination of in vitro assays and whole-animal testing. Recent scientific advancements have introduced new approach methodologies for nonclinical testing,

 which are currently used across various stages of drug development. While traditional nonclinical testing methods can predict clinical outcomes, there is ongoing encouragement for improvements to enhance their predictive capabilities.

Nonclinical studies, such as preclinical toxicology and pharmacology, must comply with FDA standards, including Good Laboratory Practice.

 Demonstrating safety through animal studies presents ethical and scientific challenges, as it is critical for these studies to strike a balance between ethical considerations and the scientific rigor required to meet regulatory standards. Companies face high costs and extended timelines when generating the necessary nonclinical data to support investigational new drug (IND) applications. The use of innovative approaches, such as in vitro methods and computational toxicology, introduces regulatory uncertainties, since these methodologies may not always align with established guidelines.

Regulatory challenges in clinical studies

Clinical trials are inherently intricate, demanding precise planning and execution. The evolving FDA guidelines have made this endeavor even more challenging. In June 2024, the FDA introduced draft guidance highlighting the significance of promoting inclusivity in clinical research. This guidance urges sponsors to establish diversity action plans to recruit participants from diverse racial and ethnic groups and other demographic categories, such as older adults and individuals with complex medical conditions. Fulfilling diversity objectives remains challenging due to the need to balance FDA requirements for demographic representation and the competitive landscape for participants in rare disease trials, which can lead to delays. Additionally, the FDA requires sponsors holding IND applications to include the initially planned and actual enrollment figures in annual reports, categorized by race, gender, and age. This requirement underscores a growing regulatory emphasis on demographic transparency.

In September 2024, the FDA released guidance

 advocating decentralized clinical trials (DCTs). These trials aim to enhance accessibility and efficiency by incorporating telehealth visits and remote assessments. Implementing DCTs presents obstacles, such as achieving regulatory compliance, ensuring robust infrastructure, and maintaining data quality from remote sources. Sponsors are also required to address complex requirements for IND submissions, safety protocols, and amendments, further increasing administrative burdens. The rising reliance on real-world evidence

 adds another layer of difficulty, as it demands meticulous data validation and interpretation. Ensuring compliance through HIPAA and other privacy laws, along with securely managing data from digital health devices, such as wearables, is crucial to maintaining data integrity.

With trial costs increasing and resources constrained—particularly for smaller sponsors—it has become essential to innovate trial designs, engage with the FDA proactively, and adopt advanced technologies. By focusing on ethical and patient-centered practices, sponsors can effectively navigate the challenges of modern clinical trials while meeting regulatory standards.

Regulatory issues in executing CMC strategies

Executing CMC strategies involves managing challenges to ensure compliance with evolving regulations. Organizations must stay vigilant and adapt proactively, updating filings, aligning compliance measures with new requirements, and revising testing protocols. Having systems in place to monitor regulatory updates, assess their impact, and implement changes quickly is critical. Non-compliance can lead to delays, rejections, or legal and financial consequences, underscoring the importance of responsive compliance strategies.

Transitioning to large-scale manufacturing can reveal gaps that need to be addressed to meet regulatory standards.Regulatory bodies require proof that processes consistently produce quality products.Extensive testing to confirm quality, safety, and efficacy is resource-intensive and can be difficult to meet consistently.

Variability in raw material quality or availability poses risks to production, requiring robust risk-mitigation strategies,

 including assessments and remediation plans. Regulatory bodies mandate the documentation and reporting of manufacturing deviations, with non-compliance potentially resulting in delays or recalls. It’s crucial for companies to also remain inspection-ready at every development stage to meet CMC requirements.

Maintaining data integrity is essential. This involves ensuring data accuracy and uniformity throughout the product lifecycle. Challenges arise from generating data across various development stages and managing complex systems with cross-functional teams. Any errors or gaps in data handling can undermine submissions that invite regulatory scrutiny or delays. Organizations can adopt robust data management systems to address this, adhere to best practices for maintaining data integrity, and conduct regular audits to ensure compliance with regulatory standards.

Mitigation strategies for meeting regulatory challenges

Creating a regulatory strategy that spans the entire drug lifecycle is crucial. Anticipating future issues and evaluating options early can determine product approval success. Early CMC focus ensures drug quality and supply. Mitigation strategies must align with the clinical development plan from a multidisciplinary perspective. These strategies cover pre-submission, regulatory approval, and post-market monitoring. Key baseline strategies include

Collaboration is crucial to managing nonclinical, clinical, and CMC risks. This proactive engagement helps ensure compliance with regulatory standards, addresses safety concerns early, and aligns the development process with established guidelines. By maintaining open communication, companies can gain valuable insights into regulatory expectations, receive guidance on innovative approaches, and resolve potential challenges that may affect the advancement of drug development.

 Assess drug benefits versus risks and identify the most significant anticipated regulatory hurdles.

Defining the patient population to be targeted.

In the United States, the biomedical industry encounters numerous regulatory challenges in conducting nonclinical and clinical studies and managing chemistry, manufacturing, and controls (CMC) activities.

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