The client faced a complex fourth re‑submission to the Scottish Medicines Consortium, with gaps in adverse event evidence, adherence‑related outcomes, and limited internal experience navigating SMC requirements. Kintiga partnered closely with the client to rebuild confidence, strengthen the value story, and deliver a fully refreshed submission under tight timelines. Through strategic coordination, expert economic modelling, and rapid turnaround on multiple clarification rounds, the medicine was ultimately accepted for use — restoring trust and demonstrating the impact of a unified, high‑performing team.
Background
- Organisation: A mid‑sized pharmaceutical company seeking approval for a treatment for nephropathic cystinosis.
- Setting: Rare disease therapy area; UK market access environment; high‑stakes SMC re‑submission with prior unsuccessful attempts.
- Context: The therapy addresses a chronic, progressive condition where adherence and long‑term complications are central to demonstrating value.
Client Challenge
- The client required support for a fourth SMC re‑submission, following previous attempts that did not sufficiently capture adverse events affecting quality of life or provide strong evidence linking improved adherence to delayed complications.
- The submission scope had been underestimated, and a PTL transition during submission week created additional uncertainty and pressure.
- Limited internal experience with SMC processes meant the client needed both strategic guidance and hands‑on economic modelling expertise.
- This challenge was critical because approval would directly impact access for patients with a rare, lifelong condition — and the client needed to rebuild confidence in the submission strategy.
Kintiga Approach
Kintiga conducted a full review of prior submissions and aligned with the client on a refreshed, evidence-driven strategy. The team coordinated closely with an external SMC expert contracted by the client to ensure strategic coherence. Clear and transparent communication was maintained throughout, enabling rapid decision-making under tight timelines. Kintiga secured a budget uplift and mobilised a cross-functional team across medical writing, health economics, and project leadership. The team delivered targeted literature review work, strengthened cost-effectiveness arguments, and developed new scenario analyses. Where needed, Kintiga provided strategic pushback to maintain a consistent and defensible submission narrative.
The Result
Kintiga delivered a fully refreshed submission with a stronger value story and robust economic model. The team rebuilt client confidence despite the PTL transition, with the PTL ultimately representing the company as the economic expert at the SMC meeting. Kintiga responded to five rounds of clarifications and NDC/DAD comments with fast turnaround. The medicine was accepted for use by the SMC, representing a significant milestone for patients with nephropathic cystinosis.
This added value for the client by strengthening strategic positioning, creating a clearer narrative linking adherence, complications, and quality of life, restoring confidence in the submission process, and demonstrating reliability under pressure.
