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Optimizing commercial launch for orphan drugs with Expanded Access Programs

29 Jan 2024 | By Uniphar Access

Expanded access programs (EAPs) can be instrumental in both ensuring patient access to treatment, and as a key step on the path to commercialization.

How Expanded Access can complement rare disease drug development

The National Institute of Health estimates that there are between 25 and 30 million people in the U.S. alone living with rare conditions. While some rare diseases are more frequent, affecting up to 200,000 individuals, others are ultra-rare, found in only a few people. Along that spectrum, the impact of rare diseases on patients and caregivers is significant, with access to treatment often presenting a serious challenge. Enhanced regulatory frameworks and increased collaboration between industry and patient groups, have in the recent years enabled the advancement of orphan drug development and patients’ access to treatments like never before.

The continuing COVID-19 pandemic has, without a doubt, had a negative impact of rare disease patient communities, making access to treatments or clinical trials even more challenging. The pharmaceutical industry has been swift to respond, with the implementation of remote practices. Expanded Access Programs (EAPs), also known as Managed or Early Access, or Compassionate Use Programs (CUPs) are often instrumental in both ensuring patient access to treatment, and as a key step on the path to commercialization.

Advancements have been made in the EAP space to improve collaboration between caregivers, pharmaceutical manufacturers, healthcare providers (HCPs) and patient groups. However, there is still a strong case and apparent need for better communication and connectivity between pharmaceutical organizations and non-industry stakeholders in the rare disease space, too.

The discussions throughout World Orphan Drug Congress USA 2020 demonstrated this well. One of the notable events on this topic was the virtual roundtable discussion hosted by Uniphar that focused on how EAPs can complement rare disease drug development and optimize their commercial launch and drew valuable insights from members of the industry.

Expanded Access Programs for rare diseases

Regulators recognize the existing high unmet medical needs in the treatment of rare diseases. There have been significant regulatory advances aimed at expediting the development, review and approval of medicines intended to treat rare diseases, such as the FDA’s Fast Track and Breakthrough Therapy designations to move investigational new drug (IND) applications through the system, and EMA’s PRIME Priority Medicines scheme. However, the lag between the end of the pivotal clinical trial and the commercial availability of the drug can still range between 12 months and two years, and rare disease patients require access to potentially life-saving medicine during this time.

Expanded Access programs are instrumental in ensuring that patients’ unmet medical needs are sustainably addressed. With keeping the patients’ needs and safety as a top priority throughout this stage of drug development, pharmaceutical manufacturers can also gain value from offering their investigational therapy via an EAP as a vital step in the commercial launch. Early access can increase awareness of a therapy, expand and strengthen relationships with healthcare providers (HCPs) and provide an opportunity to collect Real-World Data (RWD) to support payor and regulator communication in the future. If implemented correctly, an EAP can be a significant asset to both the rare disease patients and pharmaceutical manufacturers.

Real-world data collection and use

While the purpose of Expanded Access Programs is to provide treatment to patients with unmet medical needs, regulators have increasingly been recognizing the value that can be derived from collecting and analysing real-world data (RWD) as part of an EAP. The 21st Century Cures Act calls for the exploration of ways in which RWD (and real-world evidence) can support the approval of new drug indications or post-approval study requirements. The agency has recently awarded four grants for research programs, which continue to explore innovative approaches to analysing and using RWD in regulatory decision-making. For the industry, this is a clear indicator that moving forward, U.S. domestic and foreign regulators will be increasingly supporting the use of RWD in marketing authorizations.

Expanded Access offers an avenue to compliantly collect RWD, which can later be analysed and used to illustrate real-world benefits of treatment to the patient community, to support wider reimbursement claims with the payors, to assist HCPs make more informed decisions on treatment, to support regulatory approval of a broader indication, to collect additional safety data or to inform future randomized clinical trial design.

A challenge, however, exists in the collection of Real-World Data, partially owing to the voluntary nature of RWD collection by the HCPs. Furthermore, there may be certain regulatory limitations on what types of data can be collected during an Expanded Access Program. In contrast to clinical trials, EAPs do not allow for payments to HCPs for their services in administering the therapy to patients, as by their nature EAPs are intended for treatment rather than research purposes, which can translate into HCP time constraints for RWD collection. Robust design and well-executed implementation of the collection of Real-World Data thus becomes one of the key aspects of successful Expanded Access Programs. A combination of centralized powerful RWD collection technology and careful dataset design becomes a necessity for companies wishing to take advantage of RWD.

To address this challenge, Uniphar has developed a proprietary robust data platform designed to increase the uptake of RWD collection during expanded access programs. Dubbed “uniphi,” this powerful cloud-based solution makes the data inputting process streamlined and convenient for HCPs and patients alike. Innovative workflows decrease the HCP time burden and increase HCP compliance with the requirement to collect RWD, while the pharmaceutical companies can access metrics, key program indicators, automated process flows and fully customizable real-time reporting and analytics dashboards. With the RWD continuing to play an increasingly important role in the commercial launch, payor negotiations and in support of regulatory reviews and potential approvals of broader indications, technologies intended for compliant, secure and effective collection of RWD will continue to play an instrumental role in the success of expanded access programs, as well as overall commercial drug launches.