Common challenges when seeking market access for rare disease therapies

insights from industryDonovan QuillExecutive Vice President of Growth and StrategyAscellaHealth

In this interview, News Medical speaks to Donovan Quill, Executive Vice President of Growth and Strategy at AscellaHealth, about the recurring challenges of seeking market access for rare disease therapies.

How would you describe the unique challenges in bringing rare disease pharmaceutical products to the market compared to more common treatments?

Rare diseases, by their very nature, have low prevalence, which brings a unique set of challenges in gaining market access.

While as much as 10 percent of the world’s population is affected by a ‘rare’ condition, there are over 6,000 rare diseases globally, so the number of people affected by each condition is low.

This makes the process of recruiting for clinical trials, diagnosing within the disease state, and gaining an understanding of the patient journey challenging. As a result, low prevalence can impact the ability to demonstrate a new therapy's safety, efficacy, and cost-effectiveness.

Can you elaborate on how an effective market access strategy contributes to the commercialization success of a product?

Due to the small patient populations, rare disease therapies are often not recognized as much as they should be. This makes it increasingly important to understand how value is perceived by the specific audiences, such as the NHS or private payers involved, and ensure that all important elements are covered upfront to demonstrate value.

Key value indicators vary across different markets. However, typical areas to consider in a market access strategy include the cost of the drug versus lifestyle changes, real-world evidence, and health economics.

All key data elements and metrics must be gathered early in clinical trials to improve the chance of a treatment being proven, recognized, and paid for.

Considering the diverse needs of stakeholders, could you share your approach to tailoring messages to address each group's concerns during the market access process?

Priority concerns for different stakeholders share similarities and differences. First and foremost, patients will want to know that a drug is safe and effective. Physicians will also want to know that the drug is effective clinically, but they will also need to understand the route of administration and how compliance and patient engagement support enhance outcomes.  

The NHS or private payers will focus on affordability and value alongside efficacy. Patients may also want to understand the affordability aspect, but most likely solely from the perspective of the likelihood of a treatment being covered.  

It is helpful for patients to understand how they can help achieve the best possible outcomes resulting from compliance with treatment. When bringing a product to market, it is always helpful to consider what is important to each stakeholder and how they can positively impact the outcome.

rare disease therapiesImage Credit: AscellaHealth

How can proactive timing and coordination significantly impact the success of gaining market access for a rare disease product, and what steps can be taken to achieve this?

If pharmaceutical manufacturers partner with a single-source vendor for distribution and fulfillment, they can obtain the comprehensive, rich data necessary to help increase patient compliance and adherence to therapy. However, data from many sources can be messy, and pieces could be missing.  

Actionable data is imperative to optimize the program, patient engagement, and outcomes. Therefore, partnering is the key here – ideally with a single-source vendor throughout for distribution, fulfillment, and hub services so that you can obtain actionable clean data.  

To ensure activity is coordinated, it also helps to map out key program and product launch milestones in terms of an operational blueprint. It is imperative to identify critical touchpoints with all stakeholders regarding data gathering and key 'communication and intervention points.'

Could you briefly outline the key components of a compelling value story for a rare disease product and how it differs from other types of treatments?

The priority focus is that a product needs to demonstrate clinical value. A tight market access strategy with key clinical research endpoints is needed to achieve this. A partner with expertise in rare diseases that is patient-centric and can enhance outcomes through unique, innovative programs and commercialization expertise will be invaluable.  

A compelling value story, based on clinical value, also often centers around factors such as health economic scores and societal factors – determined by the expectation of the effectiveness of the treatment, including whether it will cure a disease, drastically change lives, or increase the patient's quality of life.

While these value indicators might be applied to various treatments, the difference with rare disease products is the small patient population. For example, if there is a population of just 100, the margin will be much higher than for a product with potentially higher volume.

Some treatments might not work after the patient has passed a certain threshold. Therefore, this is an added limitation because diagnosis, awareness, and detection can take much longer with rare diseases.

Can you explain the steps involved in accurately identifying the patient population size for a rare disease product, and how does this contribute to market access success?

As with any clinical trial, manufacturers must demonstrate efficacy with a pool of patients. In the case of rare diseases, this pool is significantly smaller, making it challenging to ensure that enough patients meet the specific criteria for participation in the trial.  

The number of eligible patients may be in single digits each year, so identifying (and maximizing) this number is paramount when dealing with such small numbers.  

It is a complex and challenging process, so it is essential to collaborate with the right partner with expertise in rare diseases to assist with clinical trial recruitment and manage this process. Partnering with an exclusive provider for distribution also allows patients in clinical trials to transition easily to therapy, increasing outcomes.

In the context of pricing and reimbursement for rare disease products, how do you balance reflecting the product's value and addressing budget constraints?

There is a cost to bringing therapies to market and gaining approval, which needs to be recognized; otherwise, we will never see innovation. However, we all need to ensure patients have access to innovative financial solutions to help offset out-of-pocket medication costs and demonstrate clinical effectiveness and key data metrics for payers.

AscellaHealth's patient-centric solutions help ensure specialty and rare disease patients have access to life-saving therapies via our dedicated programs to improve access to and affordability of therapies.  

rare disease therapiesImage Credit: AscellaHealth

How do you envision patients interacting with and adhering to the treatment plan of a rare disease product in real-world scenarios?

Compliance is over 90% on our dedicated, branded programs because we can do all it takes to service each patient, ensuring the patient has access to the therapy and disseminating educational, therapy-specific information. Our patient care coordinators develop relationships with patients and caregivers, delivering interventions at key points in therapy to keep them on track and alleviate concerns.

Hub and patient support services vendors that are patient-centric and aim to develop relationships with patients and caregivers will be invaluable here, providing individualized care plans, custom interventions at critical points in therapy, and multiple platforms to communicate, such as email, text, phone, etc. to increase patient response.

Can you discuss the significance of therapy discontinuation metrics and how they relate to supporting interventions that minimize the discontinuation of a rare disease therapy?

With most diseases, there are typical key points where it is important to reach and obtain data. Identifying these enables the identification of effective intervention points.

These touchpoints are likely to vary and different disease states will require different metrics. A patient-centric vendor will offer care coordinators who build relationships with patients, and patients will receive the same contact for welcome calls, refill reminders, etc. Continuity is essential to help identify patterns.  

A consistent supply of a specific dataset and baseline may be required. Then, armed with the improved insight, drop-offs are no longer a surprise and can be reduced, improving efficacy and reducing costs.

How early in the product development process do you recommend considering market access strategy for rare diseases, and how can this early planning positively influence market entry?

When identifying primary endpoints, by Phase 2, it is time to start considering market access, if not sooner. Alongside clinical trial protocols, begin gaining real-world evidence and tracking health economics. Obtain as much data as possible to help demonstrate value.

This data will enable earlier, improved conversations with payers regarding what they would like to see from the patient cohort and the health economics they need – ultimately, what it takes to cover the patient.

I call it the 'pre-booking' phase, and if product manufacturers can use insight to work with payers better in these early stages, the chances of success are increased.

Could you elaborate on the advantages of collaborating with external partners, particularly market access experts, and how their insights can enhance the market access journey?

Collaborating with market access experts improves the chance of a particular treatment being approved, paid for, and made available to the patient.

In a sector that faces significant challenges, it is important to collaborate with a single-source partner capable of providing end-to-end solutions, including pre-commercialization, market access, distribution, fulfillment, hub and patient services, outcomes tracking, and comprehensive data and analytics. This ensures seamless product commercialization, increased medication affordability, and optimal access.  

With the many specialty and rare disease products AscellaHealth has helped bring to market, an in-depth understanding of each specific therapeutic condition and the specialty and rare disease healthcare ecosystem has been the most impactful. This understanding encompasses challenges and opportunities, as well as the wide-ranging expertise necessary to streamline product commercialization and ensure success throughout each stage of the product lifecycle.

When addressing market access for rare disease treatment, what aspects should be taken into account that might differ from other pharmaceutical products, and how can this tailored approach contribute to success?

Having the correct understanding and then communicating that effectively is necessary to succeed. Firstly, understanding the disease state, what the product is doing, and its positive attributes. Then, it is about accessing the right people and knowing what to share with them and how.

It is essential to consider who is most influential, what information is meaningful to them, and how it needs to be presented to demonstrate value. Considering what is important to the patient, payer, physician, or hospital is essential and will vary from coverage and cost to patient longevity and economic benefits.  

It is also important that your market access team has a deep understanding of the payer landscape and the clinical value the therapy represents. If resources are limited, it is best to partner with an expert who can function as or work alongside your market access team to develop and implement the right strategy to ensure access.  

It takes a talented group of people to truly understand the landscape and do all these things. However, when done correctly, accessibility and outcomes are improved for those that matter most – patients.

About the Speaker

Donovan Quill, the founder of Optime Care and former CEO, plays a pivotal role as the Executive Vice President of Growth & Strategy at AscellaHealth. With a distinguished background in the pharmaceutical industry, Donovan has a proven track record of success. As co-founder and previous CEO of Optime Care, he successfully led the organization in delivering over 40 branded pharmaceutical programs tailored to rare disease manufacturers. Leveraging his extensive experience in rare and orphan diseases and profound industry knowledge, Donovan continues to spearhead the business development and strategic growth efforts at AscellaHealth by developing customized, strategic programs for pharmaceutical manufacturers. Through the development and implementation of custom programs for clients, Donovan contributes to the overall growth and success of AscellaHealth, solidifying its position as an industry-leading, global Healthcare and Specialty Pharmacy solutions organization.  

Furthermore, Donovan's multi-faceted career encompasses working with nonprofit organizations, event planning, customer service, sales and nonprofit management. Donovan entered the world of healthcare after a successful coaching career and teaching at the collegiate level. His personal mission was to help patients who suffer from an orphan disorder that has affected his entire family (Alpha-1 Antitrypsin Deficiency). Donovan became a patient advocate for Centric Health Resources and traveled the country raising awareness, improving detection and providing education to patients and healthcare providers.

A strong business development professional, Donovan holds a Bachelor of Arts (B.A.) focused in Criminal Justice/Law Enforcement Administration from DeSales University.

About AscellaHealth

AscellaHealth, a global Healthcare & Specialty Pharmacy solutions company, serving patients, life sciences manufacturers, payers, and providers, offers a comprehensive portfolio of uniquely tailored, tech-enabled services supporting complex, chronic conditions or rare diseases that require specialty medications and/or cell and gene therapies.  

A recipient of numerous industry awards for innovation and a Best Global Specialty Pharmacy & Healthcare Services Organization 2023 award winner in the annual Healthcare and Pharmaceutical Awards, AscellaHealth's best-in-class, patient-centric approach is built upon proprietary technology processes for novel programs and services to support the launch of specialty medications and proactively address multiple challenges, optimize clinical health outcomes, and improve the quality of life for this patient population.  

AscellaHealth brings a rare and special perspective to all stakeholders. Visit or email [email protected] to discuss the company’s custom solutions for specialty and rare disease pharmaceuticals.


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