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

The challenges of clinical trials for patients with rare and orphan diseases.
· 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, Duchenne's Muscular Dystrophy and even diabetes -- 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 companies that may be agile but are lean in terms of breadth of expertise, limited capabilities, influence and experience. These sponsors are seeking guidance on ways to tackle the issues, given the stunningly complex commercialization landscape and the constantly evolving payer, regulatory 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.

At AscellaHealth, we take this opportunity to help the industry better understand and tackle some of the unique challenges facing research and development teams, scientists and clinical trials stakeholders as they strive to speed up recruiting and maintain trial participants, strategize drug warehousing and delivery services and support launch and market access activities.

Even though clinical trial teams may view product launch dates very far off in the future, these processes cannot start early enough -- as early as four to six years into development since it takes several years to develop a potential new medicine for treating a complex condition or rare or orphan disease.

This is a significant departure from legacy launch plans given the fallout of risks that occur by delaying the planning for a CGT. These events could be disastrous and result in a significant waste of time and money when the product fails to gain the acceptance of healthcare professionals or achieve optimal reimbursement.

Defining the Challenges

Introducing new pharmaceutical products can be an ambitious, risky and costly process. These issues are magnified for specialty drugs and CGTs designed for this population.

Low Prevalence of Rare and Orphan Diseases

When there is a low number of people affected by a condition, conducting targeted clinical trials is difficult. This results not only because of the paucity of subjects, but also the lack of a suitable infrastructure to make it happen or to demonstrate safety, efficacy and cost-effectiveness. For controlled drugs that need to be administered in a hospital or infusion center, this may be even more problematic as there may fewer suitable sites available.

Furthermore, low numbers of people to be treated makes the relative cost of therapies incredibly high, particularly if the drug is difficult to manufacture, or needs to be administered within a controlled environment due to instability or time limitations.

These challenges are most acute in the case of CGTs, because they can be difficult to develop and there is inadequate understanding around effectiveness. This is especially relevant if less expensive alternative therapies are available or in the absence of credibility it becomes difficult to justify high costs.

Capture Critical Data Supporting Payer Negotiations

Gathering the right clinical data and developing a safety profile during the drug development process not only helps to optimize pricing on the day of launch, but also impacts payer negotiations. This is a highly valuable exercise because the disease burden is often not quantified for rare subpopulations indicated for CGT or certain orphan drugs. Data are also needed to engage in outcomes-based contracts and establish the causal relationships between the disease and outcomes. To establish product safety, manufacturers must create a holistic projection of the product’s durable pipeline success and assess the direct financial impact on the payer through analyses of data that address:

  • Dosing regimens
  • Treatment durations
  • Route(s) of administration
  • Patient population size
  • Specificity on the eligible patient population
  • Benchmark prices
  • Number of patient lives affected
  • Long-term outcomes and real-world evidence supporting payers with early visibility into the total health system costs related to a particular disease

An experienced payer negotiation team with expertise, industry contacts, and a track record of success for negotiating pricing strategies is essential in market preparation: ensure patient access to therapy and determine pricing and reimbursement at the time of launch.

Of special note regarding payer coverage of gene therapies: a 6-year-old Duchenne Muscular Dystrophy patient recently received a gene therapy. His treatment is notable because Highmark Delaware initially denied him access to the $3.2 million drug, deeming it “experimental.” It was an early test case in how insurers would approach a medicine that might alter the lives of patients with the fatal muscle-wasting disease but has only limited data and a sky-high price tag.

But after a public campaign from his family and advocates, Highmark reversed its decision, putting it in line with other payers, which have largely chosen to cover the treatment.

Optimizing the Supply Chain

Demand forecasting should begin as early as three years before the actual launch to ensure that patient-specific doses are delivered just in time to sites of care. Many manufacturers look for clean, actionable, real-time data from one source for integrated support and collaboration. They also contract for exclusive drug distribution services with entities that specialize in rare diseases, since specialty medications and CGTs often require special handling, such as refrigeration, overnight delivery, and shipment tracking.

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 therapy and access to 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 since there is little room for disappointments, holdups or delays throughout the supply chain.

Streamlining the Care Continuum

Anticipating the requirements and barriers for healthcare professionals to communicate with insurance providers and external pharmacies is critically important. With a goal to streamline communications between clinicians, patients, and pharmacy, clinical trial teams should have methodologies and technologies in place that notify doctors if a patient is non-compliant with taking medication on schedule or as prescribed.

Clinician Education

Clinicians typically deal with fewer specialty or rare disease patients and require 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 or treatment lapses. Manufacturers need to provide full support for the therapy itself by offering evidence-based information and frequent updates.

Anticipated Growth of Rare Disease Space

Life sciences companies are increasingly entering the rare disease space, with the market expected to grow at a compound annual growth rate 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 drug 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 maximize 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.

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-commercialization/clinical trials through launch and market access—prompting many to work with a single-source solutions partner that provides customized programs to meet these challenges.

Through this approach, a company eliminates the need to engage with multiple vendors, eases administrative burdens, and collects purely actionable data from one 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 customized program for a specialty or rare disease product, click here.

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