A political savant and leader in policy and legislation, Donald Gainsborough is at the helm of Government Curated. Today, he speaks about the latest pharmaceutical industry trends and drug development.
Can you discuss how the Drugs to Watch list for 2025 was created?
The list is created through the expertise of over 160 analysts and 11 integrated data sets spanning the R&D and commercialization lifecycle. This allows us to provide meaningful insights and identify key drugs that are likely to significantly impact the market.
What criteria were used to select the eleven drugs on this list?
We look at various factors such as innovation, clinical trial results, regulatory progress, and the potential market impact. The goal is to highlight drugs that address unmet medical needs and have a strong value proposition.
How has the process for deciding the list changed over the years?
We’ve incorporated more real-world data and advanced analytics to refine our predictions. This has improved our accuracy in forecasting the impact of these drugs. Additionally, the growing role of AI in analyzing large datasets has streamlined the process.
What are the biggest challenges in drug development today?
The key challenges remain proving that a drug is safe and effective while managing the high costs and long timelines. Regulatory hurdles and clinical trial recruitment difficulties also pose significant obstacles.
How do time and cost impact drug development?
Drug development is a lengthy and expensive process. It typically takes 10 to 15 years and costs around $2.5 billion to bring a new drug to market. The patent clock starts early, limiting the window to generate returns before generic competition.
Can you explain the impact of the patent clock on drug development and commercialization?
The patent clock starts ticking as soon as R&D begins, reducing the time to recoup costs once the drug is approved. Companies must balance speed and thoroughness to maximize their product’s commercial lifespan before generics and biosimilars erode the market share.
What strategies exist to manage clinical trial recruitment challenges?
One effective strategy is to collaborate with up-and-coming investigators who are not burdened with multiple trials. This ensures your study gets prioritized, improving recruitment and trial efficiency.
How can companies improve patient recruitment for clinical trials?
Companies should consider diversifying their pool of principal investigators, leveraging real-world data to identify the best trial sites, and using AI to match patients to suitable trials more efficiently.
What role do AI and real-world data play in drug development?
AI and real-world data are crucial in accelerating drug discovery, enhancing trial designs, and improving decision-making. They help predict drug efficacy and safety and streamline the development process.
How does AI help in accelerating drug discovery?
AI analyzes massive datasets to identify promising drug targets and predict outcomes, which speeds up the drug discovery phase. This can significantly shorten the time required to move from concept to clinical trials.
Can you provide an example of AI tools currently used by regulators?
The FDA uses AI-powered tools like OFF-X to assess potential risks in molecules. These tools enhance the review process by identifying safety concerns earlier.
How is real-world evidence improving clinical trial design?
Real-world evidence helps optimize trial designs by identifying the most suitable patient populations and reducing the required sample sizes. This increases the likelihood of trial success and minimizes costs.
How can real-world evidence contribute to go/no-go decisions during drug development?
It provides insights into the drug’s performance in a broader population, helping companies assess commercial viability and make informed decisions on whether to continue or halt development.
In what ways do AI and real-world data cut down dead time between trial phases?
They streamline various processes, from patient recruitment to data analysis, reducing downtime and accelerating progression through trial phases. This efficiency can significantly shorten development timelines.
How does real-world data continue to be important after a drug’s approval?
Real-world data is essential for post-market surveillance to monitor a drug’s performance and safety in the larger population. Regulators often require ongoing evaluation to ensure long-term safety.
What challenges exist in using AI and real-world data?
Challenges include ensuring data quality and standardization, addressing ethical concerns around privacy, and overcoming AI models’ potential to generate incorrect insights.
Can you talk about ethical concerns related to AI and data privacy in drug development?
Ethical concerns focus on the potential misuse of personal data and ensuring that AI-driven decisions do not perpetuate biases. Robust regulatory frameworks are needed to address these issues.
How have regulatory frameworks evolved to address AI-driven findings’ alignment with clinical outcomes?
Regulatory frameworks are increasingly incorporating AI and real-world data, driven by the need to align AI-generated findings with actual clinical outcomes, ensuring that these technologies support safe and effective drug development.
How did COVID-19 influence the use of real-world data in regulatory decision-making?
The pandemic led to greater acceptance of real-world data by regulators, which helped to accelerate the approval of targeted therapies. This pragmatism is likely to continue, benefiting future drug developments.
What makes mRESVIA, Moderna’s RSV vaccine, different from other vaccines?
mRESVIA uses mRNA technology and is the first mRNA vaccine targeting RSV, adding further momentum to RNA-based therapies which have shown significant progress in recent years.
Can you share insights on the progress being made in RNA-based therapies?
RNA-based therapies, including mRNA and siRNA, have seen considerable progress. Recent approvals of various RNA-based products demonstrate their potential in treating diseases beyond just symptom management.
How is gene editing evolving as a treatment modality?
Gene editing holds promise for potentially curative treatments. However, it is still in early stages with challenges such as safety, off-target effects, and efficiency needing to be addressed.
What are some of the challenges facing gene editing therapies?
The main challenges include ensuring safety, managing off-target effects, achieving high efficiency, and making these therapies accessible and affordable to all patient populations.
How can we ensure equity and access to gene editing treatments?
Strategies to ensure equity include developing scalable treatment modalities, reducing costs, and implementing policies to support access for underserved communities.
What strategies can help tackle the high costs associated with gene editing therapies?
Efficiency improvements in production, regulatory incentives, and innovative pricing models like value-based pricing can help manage and reduce the high costs of gene editing therapies.
How does the end of a drug’s patent protection typically impact the market?
Once patents expire, generic and biosimilar competition increases, leading to price reductions. However, original manufacturers may maintain market share due to brand loyalty and clinician familiarity.
Can you discuss how companies respond to competition from generics and biosimilars?
Companies often strategically lower prices and leverage their existing production advantages. They focus on maintaining market share, especially for existing patients, while navigating complex manufacturing challenges.
How are rising costs and supply chain disruptions affecting the pharmaceutical industry?
Rising costs and supply chain disruptions have led companies to shorten supply chains and consider local production, which increases costs. They must balance these increased costs with regulatory and pricing pressures.
What impact do legislative initiatives like the Inflation Reduction Act have on drug pricing?
Legislative initiatives like the Inflation Reduction Act allow for drug price negotiations, which will progressively impact drug pricing dynamics and market strategies over the coming years.
How are value-based pricing models changing the industry?
Value-based pricing models, which link drug prices to clinical effectiveness and patient outcomes, are shaping industry strategies by prioritizing real-world benefits and cost-effectiveness.
Why do biotech companies now need a solid market access strategy from the start?
Biotech companies must demonstrate a clear value proposition and robust market access strategy to attract partnerships and ensure successful commercialization. This shift demands a commercialization mindset from the early stages of development.
What factors should biotech companies consider for a successful market access strategy?
Factors include understanding payer expectations, ensuring cost management, demonstrating clinical differentiation, and developing strategies for pricing and reimbursement challenges.
How can pharma and biotech companies balance innovation with affordability?
Companies can balance innovation with affordability by focusing on strategic pricing, prioritizing high-impact projects, and using real-world evidence to support value-based pricing models and market access strategies.
Do you have any advice for our readers?
Stay informed about industry trends and be proactive in leveraging new technologies and data-driven approaches. Balancing innovation with practical market strategies will be crucial for success in the evolving pharmaceutical landscape.
