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by myTomorrows myTomorrows | 30 Apr 2020

Accessing Investigational New Drugs: Clinical Trials and Expanded Access

Whether patients are newly diagnosed, or have not been helped by standard treatments, there is growing interest in learning whether participating in a Clinical Trial could be an option. Clinical Trials are research studies that evaluate investigational drugs. Researchers, typically sponsored by pharmaceutical companies, use these studies to evaluate the safety and effectiveness of an investigational drug in a pre-selected group of patients. 

 

Patients with an unmet medical need often ask, “Are there clinical trials near me?” This question is increasingly a topic of conversation among patients who are now more interconnected and empowered than ever before. While Clinical Trials are getting more attention as a possible option for patients at various stages in their disease, there is still a need for more awareness and education. Many patients do not participate in Clinical Trials simply because they do not know about them.

 

 

What determines Clinical Trial participation?

Each Clinical Trial has very specific criteria to determine eligibility i.e. who can and cannot participate. For example, the trial may seek patients with specific symptoms or a certain duration of illness, while excluding patients who also have other unrelated health conditions - such as diabetes or heart disease. 

 

A recent study by the Fred Hutchinson Cancer Research Center published in the Journal of the National Cancer Institute found that almost a quarter of cancer patients do not participate in Clinical Trials not because of these eligibility criteria but due to patient- or physician-related barriers. For example, many patients are never asked to participate or not informed about the possibility of participating in a Clinical Trial.

 

According to the same study, more than 20 percent of cancer patients were ineligible for available trials due to these criteria that pharmaceutical companies determine are needed to successfully develop their drugs. Recently, there has been discussion about widening Clinical Trial eligibility criteria to increase patient recruitment in Clinical Trials. Apart from implications for the quality of research produced, such a move may also have limited feasibility from a resource and execution perspective.

 

Side effects or the fear of side effects of Clinical Trials may also have an impact on participation and are always something that patients need to consider. It is important for patients to understand that much may not yet be known about the treatment being tested, including details about what side effects may result in the short or the long term.  

 

More than half of the patients (55.6 percent) in the Fred Hutchinson Cancer Research Center study were found to have not participated in a trial simply because no trial was available for their type and/or stage of cancer at their treatment center. The realities identified by this pivotal research highlights how often patients are left wondering if they have any other options. 

 

Addressing Clinical Trial eligibility and other factors that make fewer Clinical Trials available 

Patients suffering from a serious or life-threatening illness for whom there may be no other standard treatment options look to Clinical Trials as their next best option. As described above, due to Clinical Trial eligibility criteria, quite a number of these patients may not be allowed to enroll. However, they may still be able to access a drug in development through other pathways. The pathway chosen is determined by the treating physician’s clinical judgement. They may sometimes opt for repurposed or off-label drugs, but they could also utilize an Expanded Access Program (EAP). These EAPs (also known as Compassionate Use Programs, Early Access Programmes or Managed Access Programs) at times allow patients excluded from Clinical Trials to still access investigational drugs.

 

An Expanded Access Program may offer a solution to some of the barriers to access that patients face. For example, a patient who met almost all inclusion criteria — but was excluded because of a factor such as age — might be able to participate in an Expanded Access Program. In addition, patients who cannot (due to lack of time and/or funds) travel to a trial site or who have missed the window to enroll, may be able to participate in an Expanded Access Program. These are managed by a patient’s treating physician at their local hospital. Clinical Trials, on the other hand, are primarily run from major academic medical centers in larger cities. Again, this is less than ideal for the great number of patients who often first ask: “Are there Clinical Trials near me?” 

 

Although Clinical Trials and Expanded Access Programs have many similarities, their objectives are different. The goal of the former is to conduct research, while the goal of the latter is to extend treatment to an additional group of patients. This may be with or without the intention to collect data as well. Expanded Access programs do also have rules and specific criteria about who is eligible to participate. The criteria for participation typically differ from a research study in such as manner as to allow the inclusion of a broader group of patients. Those excluded from Clinical Trials on the basis of more advanced disease or significant comorbidities, for example, may be able to access investigational drugs via Expanded Access Programs. 

 

Promoting better-informed shared decision making and enhancing Clinical Trial recruitment

With both Clinical Trials and Expanded Access Programs, lack of information is a barrier to participation that, for the sake present and future patients, must be tackled head on. Clinical Trials are extremely important to advancing medicine. Greater participation in them benefits future patients, as well as other stakeholders. While Clinical Trials are the preferred avenue for research, there is a growing appreciation of understanding how insights from treating patients in clinical care (or Real-World) settings can also materially benefit research.

 

The interconnectedness of pathways to access pre-approval drugs, combined with patients being more empowered and involved in their own care, mean that research and treatment will become increasingly complementary. Patients want to know about all their possible treatment options and how to access them. This is where myTomorrows operates and can help. The medical team at myTomorrows analyzes each patient’s medical history and then searches our internal Clinical Trial database and other medical databases around the world to identify all Clinical Trials or Expanded Access Programs that may be a possible match. 

 

We provide each patient with a free, personalized report listing possible treatment options in a patient-friendly format. It is called a “Treatment Search Report.” The Treatment Search Report is a tool to help patients and their physicians to have a discussion about all possible treatment options. If a patient and their physician decide to pursue a treatment, myTomorrows can help them access it. The patient may enroll in a trial or may opt for another one of the treatment options available to them. We believe the Treatment Search Report empowers patients to engage in a shared decision-making process with their physician and in this way contributes to better understanding and increases patient recruitment in Clinical Trials.

 

Recognizing that a fundamentally different strategy will be required to address the different barriers to access, myTomorrows helps by using a, perhaps unconventional, integrated approach. By taking this approach to addressing the many barriers that patients face, together we can help overcome the failure to treat and failure to learn dilemma that exists in the current development model. The more patients who participate in a reimagined and redesigned treatment paradigm, the more all stakeholders can learn from and improve on it.