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Pre-launch drug access programmes can help patients access investigational therapies before they receive regulatory approval and are launched commercially. This option is generally used for therapies that demonstrate acceptable levels of proof of concept and safety. They can help expand treatment options for patients and can be especially important in indications that have limited or no approved therapies available, and are now widely seen to play a role in advancing drug development.

Early access schemes can also often be structured to provide additional safety and efficacy data to support regulatory requirements, and can build broader awareness of advances in research among patients and clinicians, highlighting and reinforcing a company’s commitment to patients.

In some cases, early access or import programmes can boost early revenue, helping companies gain a foothold in a market before approval while positioning therapies for broader uptake among both clinicians and patients after commercial launch. Based on these and other potential advantages, a growing number of pharmaceutical companies in countries around the world have recognised the potential benefits that pre-launch access programmes can offer, either as components of later-stage clinical development or to support access to therapies in countries that recognise US or EU product approvals.

With their advantages, pre-launch access programmes can also present some challenges and potential risks. In many cases, they require ongoing reporting of adverse events (AEs) and are used in patient populations that do not reflect clinical research populations. Consequently, efficacy results may differ from prior clinical data. There may also be higher than expected costs to implement and maintain a programme. As they consider development of a pre-launch access plan, companies must carefully assess the potential advantages and risks and work to develop a programme designed to optimise benefit. It can be helpful to review real-world examples to identify the best options related to timing, monitoring and other factors.

Identifying the challenges

While some risks associated with pre-launch access programmes might apply broadly, others can depend on factors such as the target indication and regulations in global markets. In general, the primary concern is the process and impact for reporting AEs among patients enrolled in the programme. Reports of AEs can affect patient and clinician opinions about a drug and potentially cause delays in a development programme, even in cases where reported AEs are in line with results from previous clinical research. Concern about AEs can also reach other stakeholders, including advocacy groups, regulators, and investors.

The experiences of enrolled patients in pre-launch access programmes can also create misperceptions about a drug among clinicians or future patients, and is an important factor to consider when participants in a programme have a different profile compared to patients who participate in clinical research. For example, if clinical programmes are structured to include patients with moderate forms of a disease, it is possible that a pre-launch access programme might be restricted to patients with a higher unmet need or in later stages of treatment. This could result in safety and efficacy data that does not reflect results from prior clinical research. The differences in treatment outcomes could influence clinicians’ perspectives about which patients are appropriate for treatment when it is available commercially. Such differences could also influence payer decisions regarding which patients should be eligible for reimbursement.

As more drug developers around the world advance programmes involving single-dose or other curative therapies, pre-launch access programmes could reduce the number of patients who are appropriate for treatment if that product becomes available commercially. This is an especially important consideration for companies developing single-dose treatments for rare or ultra-rare diseases and disorders. Delays in development or approval can also require drug developers to maintain pre-launch access programmes for longer than anticipated, driving up costs further. Companies must also carefully assess and follow guidelines related to their promotion in every country where they are established. In many countries, different forms of promotion are prohibited and could be seen as unethical in cases where available safety and efficacy data are limited.

Planning programmes

In many cases, companies now recognise that collaboration among a range of internal stakeholders is essential in planning pre-launch access programmes. Companies now regularly invite the participation of medical, commercial, regulatory, market access, legal and compliance, and production and supply chain teams. They also work closely with the regional managers and launch teams in every country where a plan is to be established. They look to production and supply team leaders as well as third-party manufacturers to plan for adequate product supply at every stage of programme execution based on anticipated timelines and requirements in expansion of manufacturing capacity. While commercial teams provide essential insights on a target market, medical teams provide essential insights on patient compliance and safety considerations.

In most cases, it will be advantageous and even essential for companies to begin planning access programmes early in the drug development process. It is now not uncommon for planning to begin in the earliest stages of clinical research. By starting early, they can be better positioned to align strategy to reflect the needs and interests of both internal and external stakeholders, and are less likely to face delays associated with regulatory requirements. Early planning can also help companies be better prepared to reallocate resources as necessary to avoid delays in clinical research and regulatory review.

To proactively address potential challenges regarding AEs reported in a pre-launch access programme, drug developers should work to provide clear guidance on data related to AEs and label restrictions to clinicians. Healthcare providers (HCPs) may be required to conduct pre-treatment screenings and in-treatment monitoring of patients. They must also have clear guidance on requirements, guidelines and procedures for reporting AEs to regulators and to the developer. Programmes must also have a turnkey process for communications to HCPs, patients, investors and other stakeholders about AEs.

Procedures for patient monitoring in pre-launch access programmes must be established early and properly maintained at every stage, including a tracking system to monitor patient screening and enrolment, records of the number of treatment centres and HCPs participating in the programme, generated revenue, and schedules for programme duration, expansion, or contraction. Companies must also outline the plan for termination or transition of the programme when a drug becomes available commercially, and should also establish clear lines of communication to HCPs and patients to be able to address any issues as quickly and effectively as possible.

A global focus

Assessments of the benefits of pre-launch access programmes and the opportunities to mitigate risk indicate that these programmes are likely to be considered for a wider range of investigational therapies in more countries around the world in the years ahead.

In every country they target, companies will have to identify the local expertise and services necessary to plan and execute these programmes successfully. Planning should address the impact of pre-launch access programmes on all stakeholders, as well as the commercial success of a therapy.

A focus on early and careful planning and efforts to fully customise programmes to reflect the unique needs of each country and indication can help mitigate risk and maximise benefit while effectively supporting access to treatment for appropriate patients.

By Dr Lorenzo D’Angelo

Source: Pharma Times