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Pharmaceutical Executive APEX Awards 

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Executive Director of the Tufts Center for the Study of Drug Development (CSDD)

Framework proposes three strategies designed to address the unique challenges of personalized and genetic therapies for rare diseases—and increase the probability of economic success for a new wave of potential curative treatments for these conditions. 

ROI and Rare Disease: Retooling the ‘Gene’ Value Machine 

New research sheds fresh light on a trend with serious consequences for the public health-not only in the United States but for patients worldwide.

The Scoop on Drug Shortages

Sponsor sourcing requirements are diverging from the operating needs and long-term strategies of the major CROs.

Profound Changes in the Outsourcing Landscape

Big Pharma is finally making a commitment to partner-based outsourcing.

Make a Match

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

Jeffrey Bernstein is the director of cybersecurity and data privacy for Kaufman Rossin’s risk advisory services. He recently spoke with Pharmaceutical Executive about the various risks pharma companies are facing and the ways they can protect themselves.

Pharmaceutical Executive: What are the main mistakes that healthcare companies make when building their software?


 One of the most significant errors is neglecting security-by-design and privacy-by-design principles. These approaches ensure that security and privacy are integral to the software development process. The 2017 Equifax breach is a cautionary example of the consequences of not embedding security into design, where a failure to address a known vulnerability led to the exposure of sensitive data of 147 million individuals. Systems need to be thoroughly tested not only for compliance but for actual security–using penetration, red team and purple teams to test security, and including effective bug-bounty programs to prioritize finding and eliminating or mitigating possible threats and data leaks. It is also critical to perform secure code reviews and to review code modules for functionality updates to any specific application prior to software being put into live production situations. At each step of the design, programmers must also consider whether access to data sets are necessary, and how such access may impact not only patient care, but patient privacy.

Healthcare organizations often fail to map data flows accurately, leaving gaps in understanding how and where sensitive information is stored and transmitted. This oversight can lead to vulnerabilities, flaws, and other deficiencies. You cannot protect data if you do not know where that data is. Moreover, pharmaceutical and healthcare entities rely on dozens if not hundreds of third-party applications, vendors, suppliers, hardware developers, etc., through which sensitive data flows. Robotic and tele-medicine, EHR’s and increasing reliance on predictive systems which use artificial intelligence (including large data sets) only complicate the problem.

A significant mistake is not thoroughly understanding and controlling third-party access to systems and data. Many recent healthcare breaches have been attributed to phishing attacks on third-party vendors, highlighting the risks of insufficient oversight. You are only as secure as your weakest partner that has access to your data. While healthcare entities may be diligent in requiring third parties to execute business associate agreement (BAA’s), they frequently do not police or enforce the terms of the BAA’s. Moreover, most entities simply do not know who has access to data or systems or maintain an adequate list of all of their vendors–from insurers, accountants, regulators, to HVAC providers, cleaners, and food services. Anyone who touches the network or data must have adequate security and auditability.

Many pharmaceutical and healthcare organizations fail to require regular security audits for third-party vendors, partners, and consultants. This often introduces vulnerabilities and other exposure points. Many security compromises are the result of a third-party vendor’s compromised access. This underscores the importance of enforcing strict security audits of these sources. Many providers take a set-it-and-forget-it approach to vendor management. True security requires constant vigilance.

While AI and automation can enhance operational efficiency, over-reliance without proper oversight can lead to significant risks. For instance, IBM Watson faced challenges in cancer diagnosis accuracy, demonstrating the potential pitfalls of over-relying on AI in healthcare. The same is true for automated billing systems, robotic surgery, telemedicine, remote access, and embedded software in everything from drug manufacturing equipment, oxygen delivery systems, and blood pressure monitoring devices. Modern healthcare often looks and feels less like a patient provider than an IT system that happens to deliver healthcare as a product.

Human error, often due to insufficient training, remains a leading cause of cybersecurity incidents. Most expert sources say that approximately 90% of data security compromises involved a human error. Many recent high-profile healthcare breaches involving disruption to operations highlight the importance of comprehensive cybersecurity education for all users. This challenge is exacerbated by several factors–physicians’ resistance to change, overreliance on predictive models and insurance models to determine the appropriate procedures, an understandable desire to put patient care above all other priorities (including privacy and security), and a general failure to understand the nature of the risks. When it comes to drugs and healthcare, providers often place a priority on ease of use and access. Unfortunately, ease of use and access often comes at the cost of security.

A lack of redundancy and resilience can lead to severe operational disruptions, as seen during many recent high profile pharmaceutical and healthcare compromises including several recent compromises which led to prolonged outages and impacted patient services. Recent hacks of this kind included those that targeted blood providers resulting in supply chain disruption and the activation of emergency protocols at hundreds of hospitals. One of the reasons healthcare providers are so vulnerable to ransomware attacks is the lack of truly resilient systems that back up critical data to offsite and security locations.

Offshoring critical components introduces additional risks, particularly when offshore vendors do not adhere to the same security standards as the parties that they serve. The 2020 SolarWinds breach, which affected multiple sectors (including pharmaceutical and healthcare), exemplifies the dangers of offshoring without stringent oversight. In fact, in most enterprises, IT directors may not even know the identity of the hundreds of companies upon which the entity relies for day-to-day operations.

A common issue in healthcare is the lack of clear individual accountability for cybersecurity. Many breaches from the pharmaceutical and healthcare sectors where sensitive data was exposed may be credited to the actions of a single employee. This emphasizes the importance of establishing clear accountability. This is particularly a problem where healthcare personnel have competing demands for their time and priorities. Having documented security policies and socializing them among staff can help these organizations better govern security internally.

Pharmaceutical and healthcare organizations often retain large amounts of data for extended periods, increasing the risk of breaches. This may be understandable since information that may be decades old may be relevant to diagnosis or treatment, and information relevant to family history that may be even older may similarly be relevant. As a result, healthcare providers tend to retain massive amounts of data permanently.

PE: What unique threats does the healthcare industry face when it comes to cybersecurity?


 Ransomware remains a significant threat due to the critical nature of healthcare services. Many ransomware attacks on healthcare have caused widespread disruption, demonstrating the sector’s vulnerability. Threat actors target healthcare entities for ransomware attacks because the data and availability are both time and mission critical. Lives are also at stake and therefore, they are much more willing to pay ransom to get data returned and maintain healthy operations.

Business email compromise (BEC) frauds have become a significant threat to the healthcare industry, often leading to misdirected payments and substantial financial losses. In BEC schemes, attackers typically gain access to or spoof legitimate email accounts of executives or financial personnel, using this access to deceive employees into transferring funds to fraudulent accounts. Healthcare organizations, with their complex billing processes and numerous transactions, are particularly vulnerable to these types of attacks. The consequences can be severe, as funds intended for critical operations or payments to vendors can be diverted, leading to operational disruptions, financial strain, and reputational damage. Additionally, the sensitive nature of communications in healthcare means that BEC frauds can also expose confidential patient information, compounding the impact of the breach. This highlights the importance of implementing stringent security measures on email and other communication channels, thorough verification processes for financial transactions, and regular employee training to recognize and prevent such fraudulent activities.

Insider threats are particularly challenging in healthcare, where employees often have access to vast amounts of sensitive data. Many recent publicly disclosed breaches have shown to be the result of an employee improperly accessing patient records, exemplifying this kind of risk.

The growing use of connected medical devices introduces new security challenges. The FDA’s recall of certain Medtronic insulin pumps due to cybersecurity vulnerabilities highlights the risks associated with IoT devices in healthcare.This is particularly true for robotic medicine and remote medicine (telemedicine) which permits access to sophisticated medical technology remotely. Moreover, providers may not know or have access to information about how these systems work and may inadvertently expose these systems when they place data or access online.

State-sponsored groups often target healthcare organizations for their valuable research data, particularly in areas such as drug development and disease research. The increase in cyberattacks on vaccine research facilities during the COVID-19 pandemic emphasizes the strategic importance of this data.

PE: What sort of data are hackers looking to steal?


 Personally identifiable information (PII) and protected health information are prime targets due to their value on the dark web. Many recent high-profile healthcare and hospital system breaches have resulted in the exposure of the PII and PHI of many millions of individuals, highlighting the high stakes involved in protecting this type of information. This permits threat actors to impersonate patients, access other patient accounts, commit identity fraud or identity theft, and other types of criminal fraud.

Healthcare organizations manage significant amounts of financial data, making it a target for cybercriminals. Many recent breaches of pharmaceutical and healthcare organizations have resulted in the theft and/or leakage of sensitive financial data alongside medical records and other sensitive information. This illustrates the risks involved. In addition, hackers may use this data to commit medical insurance fraud, piggybacking medical care off the insurance information gleaned from a data breach or data theft.

Hackers target medical records for their potential use in fraud and blackmail. Many recently disclosed healthcare network breaches demonstrate the importance of securing medical records. These records may be used for extortion, blackmail, research, and other fraudulent exploits.

Research data and intellectual property related to medical advancements are highly sought after by cybercriminals, especially those backed by nation-states. Hacks targeting COVID-19 vaccine research data during the pandemic demonstrates the critical importance of protecting this information.

PE: How important is it for companies to stay up to date on their cybersecurity systems?


 Staying up to date with cybersecurity systems is essential for compliance with regulations like HIPAA, GDPR, and HITECH. Failure to comply can result in severe penalties, as seen in multiple recent HIPAA settlements which followed many recent security breaches.This means not only keeping up with regulations but understanding your threat environment. As of June 2024, the U.S, Department of Health and Human Services’ Office for Civil Rights (OCR) has imposed 145 cases related to HIPAA. As mentioned within the introduction section of my response, settlements have resulted in approximately $150,000,000 being paid. This data may be found on the OCR Breah Portal which is also known as the OCR Wall of Shame.

Cyber threats are continually evolving, requiring healthcare organizations to keep their cybersecurity systems updated to defend against new threats. Many security compromises may be credited as the result of failing to apply critical security patches. AI and other cutting-edge technologies are being used by threat actors to further sophisticated attacks, including virtual impersonation of personnel, predictive attacks, and constant probes.

Healthcare data is incredibly sensitive, and maintaining up-to-date cybersecurity systems is crucial for its protection. Many recent ransomware attacks against healthcare systems compromised many millions of patient care and data records, underscoring the importance of maintaining current defenses. Patient data remains a valuable target for hackers and the results of a loss can be catastrophic.

A strong cybersecurity posture is vital for maintaining trust with patients and regulators. Many breaches have led to significant reputational damage and regulatory scrutiny. Moreover, reputational damage and losses may not be fully covered by cyber insurance policies, and may persist years or decades after a breach.

Building redundancy and resilience into healthcare systems is crucial for ensuring continuity of operations during and after a cyberattack. The Scripps Health ransomware attack in 2021 highlighted the consequences of insufficient resilience and redundancy in critical healthcare systems. For healthcare entities, it is critical that data and services remain online and available, and that there are robust and redundant backup systems.

Pharma companies are facing a higher number of incredibly destructive cyber-threats than ever before.

Digital Protection: Q&A with Jeffrey Bernstein

The biotech XBI index has broken out from a four-month narrow trading range with a deciding win for President-elect Trump, an initial rate cut of 50 basis points, and the more modest cut of 25 yesterday. The recent strength in macro data, and outlook for inflation, has investors in a wait and see mode on when/if and how much rates might fall from here.

Investors look to M&A as the most likely path to higher valuations in the sector. Lundbeck’s recently announced acquisition of Longboard for $2.5 billion is clearly a positive, but the fact is we are still well off of last year’s pace; at 10 deals over $1 billion thus far in 2024, vs. 20 last year. That said, eight of the 20 deals last year came in the fourth quarter and six of them came in December, so investors are hoping for a strong fourth quarter.

According to EY, the largest pharma companies now have a record $1.6 trillion to fund M&A and as most are approaching large patent exclusivity losses, which will create a growth gap; acquiring external innovation will be necessary to achieve targeted growth expectations.

With Trump now having decisively won the Presidential election, the biotech sector has traded up to recent highs, despite a bit of concern/uncertainty surrounding the potential for Robert F. Kennedy to play a significant role in influencing the FDA and CMS. According to an analysis by Brian Gleason, managing director at Raymond James, and his team, biotech investors are having a solid year with most crossover investors ahead 15% (in a range of down 12% to up 28%)—lagging the S&P 500, which is trading near all-time highs with a gain of 22% year-to-date.

Raymond James’ recent survey work indicates "sentiment continues to be steady with the vast majority of investors:"

More bullish on biotech vs. beginning of the year.

Believe generalist capital is either beginning to return or will return in 2025.

View the Presidential election largely as a non-event for biotech.

Believe that issues in the IPO market during the first half of 2024 were primarily related to companies going public too early.

That the shift in acquisition volume to more clinical stage companies is a function of higher quality post-POC assets and pharma wanting to acquire prior to valuations getting too rich.

Are currently most focused on rare disease and targeted oncology opportunities and less in CNS and metabolic disease.

Believe that the market has become asset -centric in terms of how biotech companies are evaluated.

The IPO market reemerged this year with positive momentum. The post Labor Day IPOs are all trading above launch price with a median performance of 25%, according to Gleason. Several more are expected before year end and it is anticipated that 2025 will be a busy year.

Capital raised so far this year remains on pace to approach record levels achieved in 2020, with 199 transactions priced with an aggregate of gross proceeds of roughly $35 billion.

A continued high level of innovation underpins the flow of capital into the sector and the positive performance.

The biotech market remains a tale of the “haves” and the ”have-nots.” According to Tim Opler, managing director, Stifel Investment Banking, the population of $1 billion-plus biotechs (“the haves”) has doubled this year, while in contrast, the population of “have-nots” with market caps of less than $100 million has fallen only slightly. The sixty-four percent of biotech companies(about 125) with market caps of less than $100 million (“micro-caps”) remain undercapitalized with less than 12 months of cash. These companies are potential merger partners for private companies seeking a public vehicle. It is encouraging that aftermarket performance of these reverse mergers has been positive—with 10 of the past 12 successfully closing on a PIPE (private investment in public equity) in excess of $40 million. Strategic reviews, restructurings, and liquidations have also become pervasive among this group, and these trends are expected to continue. 

In SMID cap, generally restructurings and cost cutting announcements are historically high, with 81 new initiatives already this year.

Barbara Ryan is Founder, Barbara Ryan Advisors, and a member of Pharm Exec's Editorial Advisory Board

The sector's XBI is up 15% year-to-date; rate cuts, election outcome, and M&A are viewed as tailwinds.

Biotech Investor Sentiment Climbing Steadily, with Growing Enthusiasm Heading into 2025

AstraZeneca's Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses:

Results of the NIAGARA trial in bladder cancer

The significance of the five-year overall survival data from the HIMALAYA trial, specifically the long-term efficacy of the STRIDE regimen for unresectable liver cancer

In this episode of the Pharmaceutical Executive podcast, Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses the findings of the NIAGARA trial in bladder cancer and the significance of the five-year overall survival data from the HIMALAYA trial, particularly the long-term efficacy of the STRIDE regimen for unresectable liver cancer. 

Key Findings of the NIAGARA and HIMALAYA Trials

Johnson & Johnson (J&J) has submitted regulatory applications to the FDA and EMA for the approval of Darzalex Faspro (daratumumab and hyaluronidase-fihj) in the United States and subcutaneous formulation in the EU as monotherapy for high-risk smoldering multiple myeloma (SMM). According to the company, the submissions are based on data from the Phase III Aquila trial, which is currently evaluating Darzalez Faspro as an early intervention treatment for the disease.

“There remains an unmet need for early interventions and treatments that are both effective and well tolerated in people living with smoldering multiple myeloma at high-risk of progressing to active multiple myeloma,” said Yusri Elsayed, MD, MHSc, PhD, global therapeutic area head, oncology, innovative medicine, Johnson & Johnson, in a press release. “Darzalex has changed the standard of care in multiple myeloma, and with these submissions to the FDA and EMA, this therapy could become the first approved treatment for patients with high-risk smoldering multiple myeloma, potentially shifting the treatment paradigm.”

The randomized, multicenter AQUILA trial enrolled 390 patients with high-risk SMM. The primary endpoint of the study is progression-free survival (PFS), with secondary endpoints that include time to progression, overall response rate, and overall survival. All patients involved with the trial were diagnosed with SMM at least five years before the start of the study and weren’t included if they had prior exposure to approved or investigational treatments for SMM. The data are expected to be presented at the 2024 American Society of Hematology Annual Meeting next month.

, the FDA approved Darzalex Faspro in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for patients with newly diagnosed multiple myeloma (NDMM) who are eligible for an autologous stem cell transplant. In this indication, the therapy can be administered right after diagnosis and could potentially lead to significantly improved outcomes for NDMM patients. The approval was based on positive results from the Phase III PERSEUS study, which compared D-VRd induction and consolidation therapy to the traditional VRd regimen in transplant-eligible NDMM patients.

"Multiple myeloma has a highly varied clinical course among patients and in each individual patient, and there is a continued need for innovation and therapies that employ different targets and combinations to provide patients with treatment options at diagnosis and throughout the course of their disease," said Amrita Y. Krishnan, MD, professor, director, Judy and Bernard Briskin Multiple Myeloma Center, City of Hope, in a press release. "The efficacy data supporting this new quadruplet regimen, combined with its established safety and tolerability profile, provide compelling evidence that adding D-VRd upon initial diagnosis as compared to VRd can deepen responses and prolong remissions in the context of autologous stem cell transplantation."

SMM affects about 15% of new multiple myeloma cases, and those with high-risk SMM face a 50% likelihood of progressing to active disease within two years. Currently, high-risk SMM is generally managed through observation until signs of active multiple myeloma appear, but emerging evidence suggests early intervention could benefit patients at high risk.

 According to the Cleveland Clinic, SMM typically affects people over 60 years of age, with a median diagnosis between 62 and 67 years of age.

J&J stated that if the treatment is approved, Darzalex Faspro could become the first treatment indicated for high-risk SMM, potentially transforming the current management approach.

1. Johnson & Johnson submits applications in the U.S. and EU seeking approval of DARZALEX 

® / DARZALEX® as subcutaneous monotherapy for high-risk smoldering multiple myeloma. 

November 8, 2024. Accessed November 8, 2024. https://www.jnj.com/media-center/press-releases/johnson-johnson-submits-applications-in-the-u-s-and-eu-seeking-approval-of-darzalex-faspro-darzalex-as-subcutaneous-monotherapy-for-high-risk-smoldering-multiple-myeloma 

2. FDA Approves Johnson & Johnson’s Darzalex Faspro in Combination with Bortezomib, Lenalidomide, and Dexamethasone for Newly Diagnosed Multiple Myeloma. 

July 31, 2024. Accessed November 8, 2024.

Accessed November 8, 2024. https://my.clevelandclinic.org/health/diseases/23911-smoldering-multiple-myeloma

Submissions to the FDA and EMA for new indication of Darzalex Faspro are based on results of the Phase III Aquila trial in patients with high-risk smoldering multiple myeloma. 

Johnson & Johnson Submits Regulatory Applications for Darzalex Faspro for High-Risk Smoldering Multiple Myeloma

Johnson & Johnson Innovative Medicine developed a guide to bring clarity around expanded access for terminally ill patients who have run out of treatment options and aren’t eligible for clinical trials. The Guide to Expanded Access was created based on requests from patients and patient representatives. Christine MacCracken, executive director of patient strategies & solutions and leader of the team that developed the guide, spoke with 

 about the guide and broader issues surrounding expanded access.

Pharmaceutical Executive: How difficult is it for patients to access expanded access treatment options?

 One of the challenges that patients encounter, which can hinder their access to investigational medicines outside of clinical trials, is a lack of clear and transparent information. Expanded access varies by country, so it can be difficult for patients, caregivers, and physicians to navigate, especially without resources. Every country has a Health Authority with different requirements and processes for its expanded access programs. Understanding if expanded access is available in your country, the eligibility requirements, and the request process are all important when determining if it’s a potential treatment option. A lack of easily available information is a major obstacle for those exploring expanded access, making a resource like the Johnson & Johnson (J&J) Guide to Expanded Access vital.

PE: How many patients are in need of expanded access?

 This is difficult to assess for a variety of factors. However, any patient who is seriously or terminally ill, has a disease which has exhausted all available treatment options, and is not eligible for a clinical trial, is a potential candidate for expanded access. These programs are designed for this subset of patients who are unable to wait for the medicine to be available and are intended to be a bridge to when the medicine is available to them.

A common challenge for patients and their treating physicians is that expanded access is a just-in-time need during an already highly stressful time; therefore, access to readily available and transparent information is critical.

Because expanded access is an important potential option for patients, J&J built our expanded access program upon a bioethical framework that helps ensure we approach each request consistently and equitably. In 2015, we established two significant items which laid internal and external foundations to support those in need. First, we codified a policy and standard operating procedures to support the JNJ Innovative Medicines organization in considering expanded access as part of the development continuum. Second, in collaboration with New York University, we developed a first-of-its-kind committee to support our decision making, known as the Compassionate Use Advisory Committee (CompAC). We continue to evolve this external panel of ethicists, physicians, and patient representatives to support both development of our expanded access strategies as well as single patient requests. Since 2015, J&J has had the privilege of making investigational medicines available to more than 10,000 patients around the world.

PE: What led to the creation of the Guide to Expanded Access?

 In short, patient requests for information drove this effort. Information can be particularly difficult to find and, if available, may not always be in a format friendly to those not familiar with this option.And sometimes, even for those of us who are educated in this area, the requirements may not always be obvious. The Guide to Expanded Access is a comprehensive educational resource that was co-created with patient representatives, providing information in a people-friendly format for not only patients, but also their caregivers and treating physicians.

Anyone with a serious illness who has been through several lines of treatment and/or procedures can face many obstacles throughout the course of their treatment, but a lack of information should never be one of them. The power of having the right resources and information can’t be underestimated – something I learned as a nurse and firmly believe to this day.

As I mentioned earlier, expanded access processes vary by country, which can be very challenging to understand and navigate. The original Guide to Expanded Access, a first-of-its-kind resource released in 2021, was inspired by patient calls for greater clarity and resources and was designed to address this significant information gap. It was also important that this be geographically focused and company agnostic.

Now, three years later, we’ve released a revamped version after receiving positive feedback from advocates and patients alike. Patients not only led to the creation of this guide–their insights and experiences helped to bring it to life.

PE: What are the new elements in the updated version for 2024?

 The revamped Guide to Expanded Access builds upon the original, maintaining key features while adding new ones to further support patients. 

The guide now includes country-specific expanded access information for the United States, South Korea, and Switzerland, increasing the total countries covered to 30. Guided by patient and advocate feedback, we’ve also enhanced the language throughout for greater clarity.

Like the initial version, the updated guide is designed for easy navigation. It includes a table of contents map with all featured countries identified and linked to their respective pages, enabling patients to quickly locate country-specific information. Once on their desired country page, patients can find details spanning expanded access requirements, the request process, and more.

To increase patient understanding of the information presented, key terms are highlighted in red and linked to a glossary at the end of the guide. Links to external resources, such as a country’s regulatory agency site, are also included throughout.

The Guide is available for anyone to view at 

https://www.janssen.com/sites/www_janssen_com/files/guide_to_ea_june_2024.pdf

The executive director of patient strategies & solutions discusses J&J’s new Guide to Expanded Access.

Kenneth Getz, MBA

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