From Development to Access: Overcoming the Challenges of Clinical Trials for Specialty Drugs, Cell & Gene Therapies

Navigating the challenges of clinical trials for patients with rare genetic diseases.
AscellaHealth EU
· 10 min read

The promise of meaningful treatments and potentially a cure for some of the most devastating and rare genetic diseases -- such as hemophilia, cancer, sickle cell anemia, and Duchenne's Muscular Dystrophy -- is bringing hope to millions of people worldwide.

How to navigate the challenges of clinical trials and actually deliver these therapies to patients with rare and orphan disease or complex conditions strains the resources of many pharmaceutical and biotech manufacturers. This is especially relevant for small to mid-size biopharmaceutical manufacturers that may be agile but are lean in terms of breadth of expertise, go-to-market capabilities, influence and experience. These manufacturers are seeking guidance on ways to tackle the issues, given the increasingly complex commercialisation landscape and the constantly evolving regulatory, payer and marketing environment.

While the revolutionary science for developing cell and gene therapies (CGTs) may still be in its infancy stage, there are more than 900 Investigational New Drug applications to the U.S. Food and Drug Administration (FDA) for ongoing clinical studies of gene therapy products and the agency is predicting it will be approving from 10 to 20 gene therapies per year.

In Europe, the European Medicines Agency (EMA) has approved more than 19 cell and gene therapy drugs, and the new product pipeline has approximately 193 investigational therapies, with more than half of these in Phase 2 clinical trials. Oncology and rare diseases continue to be the top focus areas for CGT from pre-clinical through pre-registration. Approximately 1,564 clinical trials are under investigation for various CGTs in Europe.

At AscellaHealth, we take this opportunity to help the industry better understand and tackle some of the unique challenges facing industry stakeholders such as biopharmaceutical manufacturers as they strive to speed up recruiting and maintaining clinical trial participants, develop drug warehousing and delivery services and strategise launch and market access activities.

Keeping market access and commercialisation in mind at all times should deliver the best possible outcome for patients. Research teams may view product launch dates as being very far off in the future, as the average time to develop a potential new medicine is ten years. However, market access should be a focus from the product’s inception and the market access strategy should ideally be developed by the time of Phase 1 trials.

This approach is a significant departure from legacy launch plans and arises due to the potential risks associated with delayed planning for a CGT launch. The knock-on effect could be disastrous and result in a significant waste of time and money if the product fails to gain the acceptance of healthcare professionals, achieve optimal reimbursement, or even regulatory approval.

Defining the Challenges

Developing new pharmaceutical products is a risky and costly process, and can often be ambitious. These issues are magnified for advanced therapy medicinal products (ATMPs) and CGTs designed for rare and orphan diseases given the small patient populations and the complexity of the product development.

Low Prevalence of Rare and Orphan Diseases

When there is a small number of people affected by a condition, conducting targeted clinical trials is difficult. This is due to the low number of eligible participants, and also the lack of a suitable infrastructure to run the trial and generate robust data to demonstrate safety, long term efficacy and cost-effectiveness of the product. For drugs that need to be administered in a hospital or specialised infusion center, this may be even more problematic as trial sites may be limited to a small number of specialised institutions.

Furthermore, low numbers of people to be treated makes the price of CGTs relatively high, particularly if the drug is complex to manufacture, or needs to be administered within a controlled environment due to stability issues or special handling requirements.

Capture Critical Data to Support Payer Negotiations: National Healthcare and Private Healthcare Organisations

Gathering the right clinical data is critical to the regulatory approval and reimbursement of any new therapy. Product safety, efficacy, epidemiology, and clinical results are key to informing later-stage decisions that affect pricing, reimbursement, clinician prescribing, and other aspects of market access. Outcomes data is especially relevant to value-based contracts (VBCs) and performance-based reimbursement agreements between health care payers and manufacturers. These innovative pricing agreements are increasingly negotiated for specialty and CGTs given the relatively high cost of these products. They allow manufacturers to improve their product positioning and demonstrate that the therapy can actually reduce overall health expenditure by providing long term clinical benefits and meaningful impacts to patients, while at the same time, generating real world evidence.

An expert negotiation team with experience, industry contacts, and a proven record of developing pricing strategies and successfully negotiating reimbursement is essential to the commercialisation process.

Optimising the Supply Chain

Demand forecasting should begin as early as three years before the planned launch date to ensure that the infrastructure is in place to deliver patient-specific doses on time to sites of care. Many manufacturers. contract for exclusive drug distribution services with entities that specialise in rare diseases, since specialty medications and CGTs often have very specific handling requirements with regard to temperature control, speed of delivery and traceability. They also look for clean, actionable, real-time data from one source for integrated support and collaboration.

A distributor that focuses on this unique market sector has the resources to coordinate third-party logistics for warehousing or shipping, providing patients with uninterrupted access to the medications they need when they need them most. An exclusive distribution model not only results in significant cost savings, but also assures manufacturers that the therapy itself is readily available. Given the complexity of these products, there is little room for holdups or delays throughout the supply chain.

Clinical Education and Raising Awareness

Physicians typically deal with few rare and orphan disease patients. Specific education, training and experience are often required to help diagnose and treat rare and orphan diseases. Finding suitable trials and getting patients enrolled in them can prove challenging. Additionally, physicians require a clear understanding and awareness of medication costs, methods for communicating financial information with patients, and tools to help patients access necessary therapies and avoid missed doses. The current scarcity of dedicated resources to facilitate navigating this landscape can be frustrating for physicians, as well as for patients and their families.

Manufacturers can address these issues by working with a specialty pharmacy solutions partner that provides full support for the therapy itself and offers evidence-based clinical information to physicians. This partner can prioritise streamlined communications between the physician, patient, pharmacy and manufacturers, enhancing patient compliance with treatment. Manufacturers also need to anticipate requirements and potential barriers for physicians in navigating the funding process and ensuring payment for these therapies. A specialty pharmacy solutions partner can also support this process.

Anticipated Growth of Rare Disease Space

Life sciences manufacturers are increasingly entering the rare disease space. The European CGT market was valued at $2.17 billion in 2022 and is projected to reach at $15.15 billion in 2028, with a compound annual growth rate (CAGR) of 38.20%. The U.S. market is expected to grow at a CAGR of 12.8% between now and 2030. Despite this growth, more than 90% of the 7,000 known rare diseases lack a treatment option.

Launching a specialty or CGT product into the market not only requires additional planning and preparation time, but also expertise that differs from launching a drug used by a considerably larger population. One practical step that can be taken is to maximise focus on ensuring patient-centric services are in place. Effective hub services, for example, enable the identification of key performance indicators which can be built into how clinical trials are developed and implemented. They also provide support for early access programmes, right through to product launch.

Emerging and small to mid-size manufacturers do not typically have in-house capabilities for executing end-to-end solutions that span the entire product lifecycle—from pre-commercialisation/clinical trials through launch and market access—prompting many to work with a single-source solutions partner that provides customised programs to meet these challenges.

Through this approach, manufacturers eliminate the need to engage with multiple vendors, ease administrative burdens, and collect purely relevant data from one single source. An all-inclusive vendor like AscellaHealth that offers white-glove support and service will accelerate all processes and enable manufacturers to reach drug launch milestones.

To discuss a customised programme for a specialty or rare disease product, click here.

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