The Key to Successful Licensing and Reimbursement of New Treatments in Clinical Trials: R-S-S Experts views

Bringing a new treatment to market is a journey that requires careful planning, detailed data analysis, and strategic execution. Successfully navigating the regulatory and reimbursement landscapes is essential for ensuring that new treatments not only gain approval but also reach the patients who need them. As experts who have worked in agencies such as the MHRA, EMA, FDA and others, we really do know how to navigate and guide biopharmaceutical companies through the critical stages, ensuring a smoother path to market.

For example, a client recently came to R-S-S for advice on a biosimilar drug: they had one failed trial, and another passed trial. The bioequivalence guidelines only ask for pooling data when such circumstances arise – what were they to do? R-S-S was able to advise and guide them to a positive opinion. These and many more case studies with strong experience lie within R-S-S. Not only that, but R-S-S also performed all the necessary simulations and statistical analyses along with PK/PD modelling. So not only do R-S-S advise well but we execute the functions well… and we do not charge big CRO prices. However, we do give the advice big CROs can’t always provide.

Navigating the Regulatory Maze

Understanding the regulatory landscape is the first critical step. Agencies like the FDA in the U.S. and EMA in Europe have stringent requirements to ensure the safety and efficacy of new treatments. These regulations can vary by region, adding complexity to global trials.

R-S-S’s team includes former regulatory assessors and experts who bring invaluable insights from their time working within these agencies. This expertise enables R-S-S to help clients prepare comprehensive briefing books, design regulatory-compliant protocols, and standardize databases for submissions. Their first-hand experience helps clients avoid common pitfalls and accelerates the approval process. Remember, many people who come from regulatory agencies have spent years reviewing, but when it comes to actually designing, for example a Bayesian or Adaptive design, they won’t always know. That is a key difference! We design the briefing documents, protocols and analysis plans for success.

Crafting a Market Access Strategy

Securing reimbursement is just as crucial as gaining regulatory approval. Health Technology Assessment (HTA) bodies evaluate the clinical and economic value of new treatments to decide their reimbursement status. This evaluation involves assessing the treatment’s cost-effectiveness and overall value compared to existing therapies.

R-S-S supports clients in creating robust payer value dossiers and crafting compelling value messages tailored to different markets. Our expert health economics have been former members of NICE advisory panels, or academics, with solid experience of taking drugs to reimbursement.

R-S-S conduct thorough health economics and outcomes research (HEOR), including cost-effectiveness modelling and systematic literature reviews, to build a strong case for the value the HTA has to offer. We want to build a collaboration to help bring new important innovative health technologies to patients, whether the disease is rare or more common. Our collaborative strategic approach helps secure favourable reimbursement decisions, making new treatments accessible to patients as we craft together our market access strategy.

Leveraging Real-World Evidence

Real-world evidence (RWE) is increasingly important in both regulatory submissions and reimbursement decisions. RWE provides insights into how treatments perform in the “real world” - everyday clinical settings, complementing clinical trial data. It can be especially useful for demonstrating long-term outcomes and cost-effectiveness. Using registries can be more challenging than it seems: companies can rush into believing the RWE will be impactful – but it requires careful design and thought.

For example, several clients bemoaned the situation where the registry they used with prior advice did not have the data – “it was all missing…”: in reality, the data was there, but it depended on the design of the registry study: if your protocol asks whether the outcome is in the database, but not over what period it is there, then you may miss the mark!

R-S-S integrates RWE into their regulatory and reimbursement strategies. By utilizing large healthcare databases and conducting detailed real-world data analyses, they provide comprehensive evidence that meets regulatory and HTA standards. We have strong experience with Audit data, Hospital Episode Statistics (HES), Central Practice Research Datalink (CRPRD), systematic Anti Cancer Therapies (SACT), linkage and much more. This approach supports initial approval and ongoing value demonstration post-market. See our publication on “Economic Evaluation of Cancer Drugs Using Clinical Trial and Real World Data”

Overcoming Challenges

The path to successful licensing and reimbursement is not without challenges. These include navigating different regulatory requirements across regions, meeting evolving HTA criteria, and the need for continuous evidence generation. Addressing these challenges requires adaptability, continuous stakeholder engagement, and a commitment to high standards of data integrity.

R-S-S’s multidisciplinary team (comprising of statisticians, health economists, medical writers, and regulatory experts) works collaboratively to tackle these challenges. They offer services such as optimizing study designs, employing adaptive trial methodologies and providing strategic regulatory advice. This teamwork ensures that new treatments meet rigorous safety, efficacy, and value standards.

Conclusion

Successfully bringing new treatments to market involves navigating a complex landscape of regulatory and reimbursement hurdles. With the right expertise and strategic planning, this journey becomes more manageable. Regulatory Scientific and Health Solutions (R-S-S) is a trusted partner in this process, offering unparalleled support and guidance. By combining regulatory expertise with innovative health solutions and robust data analytics, R-S-S ensures that new treatments not only gain approval but also reach the patients who need them.