The Value of Real-World Data from Global Expanded Access Programs for Rare Diseases

John Lagus, Tanners EVP of Business Development focusing on managed access programs, recently spoke at MarketandMarkets Digital Event on Orphan Drugs and Rare Diseases about the value of real-world data (RWD) from expanded access programs (EAPs) for rare diseases. Here are a few highlights from his talk. 

Years ago, while in Botswana, John Lagus met a woman who offered her life savings to purchase critical medicine for her daughter. This conversation stayed with him and showed him how important it is to provide therapeutics to people who need them but may not have access to them because of geography, affordability, regulations or other reasons.  

It also made him realize that the information generated through expanded access programs (EAPs) could provide valuable data from diverse groups of people who aren’t included in clinical trials. Clinical trials have well-known diversity and inclusivity gaps because they cant include people from every demographic affected by a disease. Therefore, clinical trial data may not reflect what happens to people in the real world, especially those with rare diseases 

What are EAPs? 

EAPs allow physicians and patients access to pre-approval, investigational drugs outside of the clinical trial setting. Because there is some risk involved, EAPs have strict participation requirements: 

  • The patient must suffer from a life-threatening, long-lasting or seriously debilitating illness 
  • There cannocurrently be authorized therapies available to treat the disease or condition 
  • The patient cannot enroll in an ongoing clinical trial 
  • In most cases, the medicine or therapy must be undergoing clinical trials or have a marketing authorization somewhere in the world 

Regulators, physicians and patients now appreciate that certain investigational or unlicensed medicines should be available outside of controlled clinical trials to help patients while they live out their daily lives.  

Benefits of capturing EAP data
This recognition is spurring a new perspective on the role of EAPs in providing valuable, real-world data (RWD) for patients and the pharma industryEAPs can produce RWD that may open new doors for treatment that wouldn’t be available otherwise. 

Historically, global EAPs were exclusively used to address unmet medical needs for patients around the world. But many companies have more patients enrolled in EAPs than in their formal clinical trials.  

EAPs already capture patient data on demographics, safety events, efficacy, treatment dosage, duration, adherence, and discontinuation, as well as how the therapeutic is performing in the real world. The inability to incorporate RWD, especially on rare diseases, from EAPs alongside clinical trial data is regrettable. This data potentially contains valuable information that could help patients live better, healthier or longer lives. 

Using RWD in regulatory submissions
Today, patients are demanding faster access to investigational medicines. People with rare diseases are usually well-informed and interested in contributing to knowledge about their medical condition, but many don’t qualify or have access to formal clinical trials. As companies develop medicines that address small patient populations, every data point captured can be critical to enhance knowledge and uncover opportunitiesEven simple data points may be a valuable source of information on real-world product use when the constraints imposed by a clinical trial protocol are lifted. 

Therefore, industry and regulators are increasingly incorporating data collected outside clinical trials and using it in their decisionmakingIn fact, RWD from global EAPs is becoming accepted by regulators as supportive evidence for safety and effectiveness, especially with rare diseases and small patient populations. It is also beginning to be used in support of US FDA and UK Marketing Authorization Approval (MAA) applications and can play an important role in corroborating clinical trial results, informing publications, setting initial pricing, and more.

What pharma companies can do 

John advises pharma companies that want to use RWD for regulatory submissions to think about and plan for how they will validate the data. The quality and usefulness of RWD depend on how well patients, physicians and companies can capture the data.  

Therefore, he encourages companies and sponsors to speak with current investigators, patients and patient advocates about the best ways of collecting RWD and provide them an easy-to-use mechanism to capture their experiences. He also recommends keeping things simple, providing compensation, and keeping data collection to a few key measures. He also advises using the same system for enrollment and data capture, if possible, for simplicity’s sake.  

Opportunities abound 

Global expanded access programs offer opportunities for patients with rare diseases to access to lifesaving or lifechanging medicines. They also help companies developing therapies for rare diseases to derive even more value from these programs by including RWD in their body of knowledge. Furthermore, as global EAPs become more common as new treatments come into play, well have access to more and more potentially valuable data from patients real-life experiences.  

Tanner Pharma appreciates the key role our company plays in facilitating and supporting EAPs. We are hopeful that this inclusive, diverse way of incorporating real-world data into the body of knowledge will play a more significant role in evaluating how medicines work in the real world, especially for people with rare diseases 

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