The concept of drug repurposing has emerged as a luminous thread, offering new perspectives and methodologies in the quest for therapeutic breakthroughs.

Leveraging the latent potential of existing drugs for novel applications represents not merely an innovative strategy but a profound reevaluation of the very foundations upon which drug development is predicated.

The allure of drug repurposing lies in its promise to transcend traditional barriers—reducing both the financial burdens and the temporal expanse typically associated with bringing new drugs to market. As we stand on the precipice of this burgeoning frontier, it becomes imperative to explore the multifaceted implications, challenges, and opportunities that drug repurposing presents to researchers, investors, and the broader healthcare ecosystem.

The rationale for this shift is compelling — existing drugs have already traversed the rigorous pathways of safety evaluations, thereby mitigating a significant portion of the risk and uncertainty that characterizes novel drug development. In a landscape marked by the daunting statistics of drug development — where the journey from discovery to market can span over a decade and cost upwards of $2.6 billion — drug repurposing emerges as an expedient route to therapeutic innovation.

However, the journey of drug repurposing is not without its challenges. Intellectual property rights, regulatory nuances, and market dynamics present intricate puzzles that must be navigated with acumen and foresight. The question of profitability, particularly for generic drugs, looms large, shaping the contours of research and development strategies.

Today we delve deep into the heart of drug repurposing, shedding light on its scientific, regulatory, and commercial dimensions. From the basic principles that underpin drug repurposing strategies to the nuanced challenges of navigating the commercialization landscape.

Let’s dive in …

Here IQVIA shed light on drug repurposing’s potential to revolutionize the pharmaceutical industry by enhancing efficiency, reducing costs, and lowering the inherent risks associated with traditional drug development processes.

Sommerford, a Global Lead in AI for Drug Discovery & Development, underscores the critical importance of repurposing existing drugs to address the urgent needs of patients, particularly those suffering from cancers and rare diseases.

Not only does he highlight the economic and temporal advantages of drug repurposing but he also presents a nuanced discussion on the various systematic approaches and the emerging role of artificial intelligence (AI) in propelling this field forward.

The traditional path to drug discovery and development is depicted as a daunting and costly endeavor, characterized by low success rates and prolonged timelines necessary for securing FDA approval. Sommerford contrasts this with the more attractive proposition of drug repurposing, where the approval rates are significantly higher due to the pre-existing clinical safety data of the drugs being repurposed.

By leveraging the known pharmacological profiles of these drugs, researchers can bypass many of the initial stages of drug development, accelerating the process of bringing critical treatments to the market.

Sommerford delves into the systematic approaches to drug repurposing, which include disease-centric, target-centric, and drug-centric methodologies.

A focal point of Sommerford’s analysis is the transformative impact of AI and machine learning (ML) technologies on drug repurposing.

These technologies enable the identification of novel drug-disease relationships by mining vast amounts of biomedical data, including literature, genomic data, and electronic health records. AI-powered methodologies are particularly adept at uncovering hidden patterns and evidence that can lead to the repurposing of drugs for new indications. Sommerford illustrates how AI and ML can expedite drug development processes, offering a “second chance” to drugs that were previously withdrawn or failed in clinical trials.

IQVIA

This piece meticulously outlines the critical questions that researchers must consider, encompassing molecular targets, drug efficacy, clinical evidence, safety profiles, and commercial viability. Each question is intricately linked to the broader objective of drug repurposing: to expedite the delivery of safe, effective, and economically viable treatments to patients.

Molecular Targets and Pathways: Understanding the molecular target or pathway implicated in a disease is fundamental to identifying drugs that could be repurposed. This requires a comprehensive analysis of disease pathology, omics data, and scientific literature to establish a connection between the drug and the disease of interest.

Efficacy Over Existing Therapies: A key consideration in drug repurposing is whether the candidate shows significant or improved efficacy over existing treatments. The comparative analysis of efficacy not only informs the potential impact of the repurposed drug but also plays a critical role in its regulatory approval and market acceptance.

Safety and Toxicity Risks: Addressing potential safety and toxicity risks is paramount in the repurposing process. Researchers must predict side effects and adverse events, considering the drug’s mechanism of action in the context of its new use. This involves an intricate assessment of pharmacology data, drug interactions, and adverse event reports to ensure patient safety.

Commercial Viability: The article emphasizes the importance of assessing the commercial viability of repurposing candidates. This includes evaluating the patient population size, market demand, intellectual property protection, and the regulatory pathway for the repurposed drug. The economic analysis extends to manufacturing and distribution costs, highlighting the need for cost-effective solutions to ensure the sustainability of repurposed drugs in the market.

Advanced Methodologies for Drug Repurposing: The role of advanced data science and computational research methodologies in answering these critical questions. Techniques such as disease modeling, knowledge graphs, and graph neural networks are instrumental in dissecting disease mechanisms, identifying repurposing targets, and predicting drug-disease interactions.

By systematically addressing the outlined questions, researchers and pharmaceutical companies can enhance the efficiency and efficacy of drug repurposing projects, ultimately accelerating the development of innovative treatments for patients in need.

Elsevier

Here we highlight the pragmatic aspects of drug repurposing but also delves into the challenges and barriers that hinder its wider adoption, particularly focusing on profitability and the commercialization landscape.

Sexton, an Assistant Professor of Internal Medicine and Medicinal Chemistry at the University of Michigan, begins by elucidating how drug repurposing can occur both serendipitously and through deliberate scientific inquiry. He brings to light successful instances of drug repurposing, such as sildenafil’s transition from a potential angina medication to a treatment for erectile dysfunction.

Central to Sexton’s discussion is the story of thalidomide, a drug that encapsulates the transformative power of repurposing. Originally marketed as a sedative and morning sickness treatment, thalidomide’s tragic legacy due to its teratogenic effects led to its withdrawal. However, its later discovery as an angiogenesis inhibitor opened new therapeutic avenues, particularly in treating multiple myeloma and other cancers.

Sexton uses thalidomide’s journey as a prime example of how drugs with a controversial or failed past can emerge as lifesaving treatments, underscoring the dynamic and often unpredictable nature of drug repurposing.

Turning the focus towards the COVID-19 pandemic, Sexton showcases drug repurposing as a critical strategy in the global fight against the virus. He recounts the extensive efforts to identify existing drugs that could be redirected to treat COVID-19, illustrating the agility and potential of repurposing in addressing emergent health crises.

Despite the successes, Sexton points out the challenges faced in bringing repurposed drugs to market, particularly those related to profitability and intellectual property.

The commercialization of repurposed drugs, especially generics, confronts significant barriers due to the intellectual property landscape and reduced profit margins.

Sexton critically analyzes the impact of these barriers on the pharmaceutical industry’s motivation to invest in repurposing efforts. He highlights the stark contrast in the commercial potential between novel drugs and repurposed generics, suggesting that the lack of exclusivity and competitive market dynamics disincentivize investment in repurposing projects.

The Conversation

It’s evident that this innovative approach in pharmaceutical development is not merely a scientific endeavor but a significant investment opportunity that promises to reshape the healthcare industry.

The allure of drug repurposing is twofold.

Firstly, it offers a more efficient and cost-effective route to drug development, leveraging existing knowledge and data to mitigate the traditional risks associated with bringing new therapeutic options to market. The lower developmental costs, combined with shortened timelines due to the bypassing of initial safety trials, present a compelling value proposition.

Secondly, drug repurposing holds the promise of generating significant social impact by addressing unmet medical needs, particularly in areas such as rare diseases, oncology, and emergent health crises like the COVID-19 pandemic.

The role of advanced technologies and data analytics cannot be overstated. Artificial intelligence and machine learning are proving to be invaluable tools in identifying repurposing candidates and elucidating mechanisms of action, thereby de-risking investment decisions. As we look towards the future, the integration of these technologies with biopharmaceutical research will undoubtedly unlock new opportunities and efficiencies in drug repurposing.

Until next time,

The Longr Reads Team

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