Ensuring that the right patients get the right treatments at the right time

Integrated Scientific Advice

Early Regulatory and Health Technology Assessment (HTA) consultation is valuable in terms of reducing the overall go-to-market risks associated with new pharmaceutical products, medical devices, or diagnostic tools. Specifically, early Regulatory and HTA scientific advice supports manufacturers by optimizing their product development program to generate relevant evidence for health technology assessors, payers, and regulators.

Scientific advice has gained momentum globally over recent years, and many processes have evolved. There is much value to be gained from early engagement from regulatory, payer, HTA, and industry perspectives. Mapi has a deep experience in European, US, and global processes, including the NICE, G-BA, HAS, FDA, and EMA scientific advice processes, including parallel HTA-regulatory scientific advice.

Integrated Scientific Advice Processes and Trends

The objective of integrated scientific advice is: to support manufacturers in optimizing their product development program to generate evidence that is relevant to regulators, HTA bodies, and payers for timely patient access.

A shift in the regulatory/HTA paradigm is occurring and drivers of this include affordability concerns, efficacy-effectiveness gaps, patient centricity, and early access to important medicines. The value of early integrated scientific advice is to support navigating this paradigm shift, manage uncertainty and de-risk, manage evidence complexity in HTA submissions, and promote company collaboration to eliminate what Dr. Bending described as the “silo effect”. The most important reason for seeking integrated scientific advice is to provide support for timely access to innovative medicines.

Transformational medicines may be made available to patients up to 4 years earlier through the Early Access to Medicines Scheme (EAMS), an innovative new scheme just proposed in the UK. Obtaining this designation will require integrated early dialogue between both the regulatory and HTA agencies to support these new changes to align the regulatory and HTA bodies earlier.

Emerging overlap between Regulatory & HTA bodies scientific advice

Key Considerations for Engaging in Integrated Scientific Advice

There are many considerations from the regulatory and HTA perspectives with respect to the overlap and synergies in the consideration of the definition of population, study design and comparators, outcomes, and subgroups. Value proposition is important to the development of a robust briefing book to provide a framework to develop “value added” questions for the agencies.

Patient involvement in these early engagements is also important to support early alignment of all stakeholders to optimize the clinical development plan and identify potential gaps.

Integrated scientific advice and adaptive development pathways enable companies to identify and address both regulators and HTA bodies’ data requirements where both agencies are in agreement. More importantly, where there is disagreement, integrated scientific advice provides the opportunity to provide plans to address this misalignment in the evidence generation and access plans.

Emerging overlap between Regulatory & HTA bodies scientific advice

Factors Influencing Perspectives About Integrated Regulatory And HTA Scientific Advice

Integrated scientific advice focuses on guiding development of new transformational treatments to support timely patient access, rather than telling companies what to do or how to do it. Advice should be considered but there is a gradient in the value of advice depending on the development challenges and company experience.

While the number of joint MHRA/NICE scientific advice is small (4), given the paradigm shift that is occurring and the increased value of this type of advice, the number of these is expected to increase, fueled by regulatory and reimbursement process changes.

There is large potential for consolidation of processes given the variety of emerging integrated scientific options which provide the opportunity for alignment on some aspects of advice in the clinical development program. This is linked to some previous resource limitations for HTA bodies and combining this with consolidation was thought to alleviate this in the future.

Patient involvement in these processes has grown over the last few years. This can be very powerful in contextualizing new outcome measures, particularly in rare diseases and in positioning the importance of subgroups for focus.

There are strategic considerations for the reimbursed population versus the broad label considered from a regulatory perspective, and aligning this is important in seeking such scientific advice.

Regardless of previous experience, therapeutic area, or stage of development, manufacturers are encouraged to always consider seeking integrated scientific advice, especially for transformational medicines.

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