Regulatory Affairs Consulting
Alacrita’s regulatory affairs specialists help our pharmaceutical, biotech and medtech clients overcome critical regulatory hurdles, devise effective regulatory strategies and provide ongoing, hands-on regulatory support, including assistance with regulatory applications. The team also supports companies and investors conducting regulatory due diligence.
All members of the practice are seasoned industry executives who have spent their careers either working within regulatory agencies or shepherding life science products through regulatory processes. Alacrita’s consultants have particular expertise in EU and US regulations and have many years’ experience of interacting with competent authorities in these regions.
The group has deep preclinical and clinical technological experience covering biologics, pharmaceuticals, API and generics. By pooling our collective experiences and expertise, the team represents a formidable resource for any life science company needing to supplement its in-house regulatory capability.
Types of activities we typically support clients with:
- Assessing regulatory feasibility and the best path to approval for novel medicines
- Developing, or critiquing as part of due diligence, a regulatory strategy alongside clinical and commercial planning
- Supporting scientific advice and pre-IND meetings with EU and US regulators
- Managing the regulatory process for obtaining designations like Breakthrough, Fast Track, PRIME, EAMS or Orphan Drug Designation
- Managing, preparing and submitting CTAs/INDs
The below selection of case studies and recent projects can help illustrate the type of consulting support we typically provide.
Regulatory due diligence for a clinical-stage biopharmaceutical: A clinical stage biopharmaceutical specialist needed support with its regulatory due diligence activities. We assembled an expert team comprising an ex-quality assessor who had previously worked in the biologicals unit at the MHRA (primarily on EMA-centralized procedures), and an ex-Senior Director and ex-Medical Advisor for a biotechnology company. The team reviewed all correspondence with regulatory authorities to identify any red flags that needed to be addressed.
Successfully appealing FDA refusal of an orphan drug designation application: A biopharmaceutical company was seeking assistance to prepare a response to the FDA’s initial refusal of its orphan drug designation application for its novel CAR-T therapy intended for use in an orphan subset of a non-orphan condition.
Supporting medical device 510(k) submissions: A company had successfully CE marked a novel lung function device, and the next step in the commercial strategy was to obtain 510(k) clearance to market in the USA. This was a complex submission that required careful planning in order to expedite the approval process while at the same time maximizing its use over a number of potential measurement parameters
Supporting a CTA submission for the oral form of an approved biologic drug: Our client was developing an oral form of an approved biologic drug which appeared to have excellent properties in terms of a rapid time to maximum concentration in the blood. A Phase II trial in an orphan indication was underway in the client's home country and the company intended to open a UK trial site as soon as practicable.
BLA submission support: Our client was nearing the completion of a pivotal Phase III trial with a novel antibody-based therapeutic. The product was designed to delay recurrence in cancer patients with minimum residual disease following debulking surgery and standard chemotherapy. The Phase III trial design had been agreed with the FDA under the SPA procedure and the client had gained fast-track status. In addition, the client had received EMA scientific advice and intended to file for conditional MAA approval, subject to satisfactory Phase III results. Although our client was a fully integrated pharma company, and had significant regulatory experience with small molecules, this was the company’s first biological product candidate to reach the registration stage. The company was therefore seeking to supplement its existing in-house capabilities with the appropriate external biologics regulatory expertise. Specifically, the company needed to gain a better understanding of the BLA process, clarify the level of detail needed for the various sections of the license application and obtain high-level review and sign-off of the regulatory dossier.
Pre-IND meeting support with the FDA: Our client was developing an oral form of an approved biologic drug for endocrine disorders which appeared to have excellent properties in terms of a rapid time to maximum concentration in the blood. A Phase II trial in an orphan indication was underway in the client's home country. The client also intended to submit an IND in the US to develop the drug there, and engaged Alacrita for support with a preparation and execution of a pre-IND meeting with the FDA.
Cell and gene therapy regulatory support
Challenge: Our client is an innovative, publicly-traded, lean biotechnology company focused on developing potential cures for infectious diseases and cancer utilizing cell and gene therapies and proprietary platform technologies. The primary infectious disease target is Human Immunodeficiency Virus (HIV). There are numerous cancer targets, focused on hard-to-treat solid tumor indications where, currently, Chimeric Antigen Receptor (CAR) technologies have faced numerous challenges.
Alacrita was engaged to provide cell and gene therapy regulatory affairs consultant support for Chemistry, Manufacturing & Controls (CMC), and Pharmacology & Toxicology to help support and accelerate client Research & Development (R&D) activities.
Solution: Alacrita's cell and gene therapy regulatory affairs lead consultant has over fifteen years' experience working in this field, both with the FDA as a senior regulatory specialist and subsequently within the pharmaceutical industry developing novel cell therapies. Our regulatory affairs team worked with the client to develop a regulatory strategy for its lead cell therapy product, as well as additional products in the pipeline.
Other members of our consulting team were then integrated into the client R&D Team and their external contract organizations to ensure a smooth implementation and oversight of the strategy. For the lead drug, Alacrita's regulatory affairs expert supported the client with the management of contract manufacturing organization relationships, strategic support and implementation of manufacturing process and analytical method development and optimization, strategic support and implementation of Investigational New Drug (IND)-enabling in vitro and animal studies, as a regulatory liaison for first-in-human clinical trial design and associated information client education on FDA requirements, meetings/interactions and submissions for cell and gene therapies.
Case study: Providing regulatory strategy for T-cell therapy
Challenge: Our client is a leading T Cell Receptor (TCR) biotechnology company, focused on delivering first-in-class biological therapies for serious diseases. The client wanted to understand whether it had the capabilities to launch its lead product if it were to receive accelerated approval without partnering with larger companies and asked Alacrita for commercialization assistance. The overall objective of the project was to ensure the client was adequately prepared for the eventuality of an early commercialization event for the product. This included an assessment of the regulatory pathway for the product and any obstacles that had to be considered.
Our assessment comprised three umbrella categories:
- Marketing approval pathway and data requirements: As a prerequisite for commercialization, the marketing approval pathway and data requirements were evaluated based on the sponsor’s interactions with relevant regulatory authorities and development plans. Having a preferred marketing approval pathway agreed with the agency(ies) and well-defined data requirements and desired labelling for the application reduces risk to achieving a timely approval and, in turn, market launch, for a commercially viable product.
- Regulatory strategy plans and execution: The second group of criteria evaluated organizational capabilities to analyze and mitigate development risks and to adequately resource, plan and execute a regulatory strategy leading to marketing approval.
- Processes for post approval compliance: Lastly, the regulatory assessment examined the organization’s preparedness to support post-approval compliance requirements to maintain a product license.
A written report was provided to the client summarizing the findings from each analyses and highlighting areas for focus as they work towards.
Case study: FDA cell therapy regulatory affairs consulting
Challenge: A company developing a first in class allogeneic Natural Killer (NK) cell therapy needed regulatory support for its FDA interactions. The product was in early clinical trials in an ex-US territory and had shown preliminary evidence of efficacy. The company was looking at establishing an IND to enable further clinical development in the United States. Alacrita was engaged to help with this process.
Solution: Alacrita's cell therapy regulatory consultant, who had worked in the FDA and industry, provided strategic advice to the company as it planned for the IND submission. Deliverables from the work included the development and implementation of an overarching FDA strategy, advice on the CMC regulatory strategy (e.g. comparability exercises) and support with the pre-IND meetings, IND submissions and expedited pathway designation applications such as Fast Track, RMAT and Breakthrough.