No participant left behind
When a clinical trial ends, it can take months or even years before a successful investigational drug reaches the market. During that time, patients who have participated in a trial can potentially lose access to the treatments that have been benefiting them, or even keeping them alive. International guidelines and regulations in a few countries have been pushing for an industry-wide shift to ensure that these patients receive continued treatment access, even after the trials end. Novartis has ranked as one of the leaders in this movement by committing to policies and practices that ensure patients who have benefited from treatment while in the study continue to receive uninterrupted access for as long as they need it until the product becomes locally available and accessible to them.
Text by K.E.D Coan, illustration by Lehel Kovács, photos by Laurids Jensen
What happens to clinical trial participants after trials end? If there is no evidence of benefit, the trial is discontinued and there is little more to do. But if the results are positive enough, the new drug will eventually be approved and, in many countries, the costs will be covered by local healthcare systems.
But these steps can take months to years. During this gap between the end of a patient’s participation in a trial and the launch of the drug on the market, clinical trial participants may lose access to the drug. Novartis has been among the first companies to guarantee continued access – called post-trial access – for participants of any clinical trial from the time each patient finishes the trial, if the patient has derived benefit. If the results of the trial show superior efficacy, this access is continued until the drug is on the market and reimbursed (where applicable) in the patient’s country.
“Post-trial access is particularly important for rare diseases because these patients usually have very limited treatment options,” said Marion Dahlke, a Global Program Clinical Head at Novartis. “These are chronic, lifelong diseases and the patients may suffer or even die if they lose access to a treatment that has been benefiting them.”
Novartis has several types of post-trial access programs that can be tailored to fit the trial strategy, different country regulations and patient needs. Over the last five years, the entire pharmaceutical industry has been incorporating similar practices. But Novartis is unique in pledging to provide all of their trial participants uninterrupted access to the company’s investigational drugs, regardless of disease severity or geographical location until the drugs’ launch and reimbursement (where possible).
Partners in development
“Clinical trial participants are really like our partners in our clinical development programs,” said Michael Steel of the Chief Medical Office at Novartis. “They are volunteering their time and potentially their health and wellbeing to help us advance the development of new medicines – it would be unethical to leave them hanging without access after a trial ends.”
Paul Aliu: Patients are always on my mind.
Michael Steel: Patients are partners.
Steel joined the Chief Medical Office team shortly after the company wrote its first post-trial access guidelines in 2018. But before that year was out, it was apparent that clearer directions were needed. Steel oversaw the complete revision of the guidelines and position statement, as well as the development of resources to help teams put those policies into practice.
The guidelines and company position statements are based on several international ethical frameworks such as the Declaration of Helsinki and the International Council for Harmonisation of Good Clinical Practices. But these only provide a starting point, and they are generally focused on the regulatory requirements for severe and life-threatening diseases.
“Oncology clinical trial teams are very used to providing post-trial access because if you have a patient in an oncology trial and they’re responding to the drug, you can’t just take it away because they might die,” Steel explained. “But the general medicines part of the business was not familiar with post-trial access when we got started.”
To make sure that the guidelines were useful for the entire business, Steel helped to coordinate a team of subject matter experts from across the company to draft the second set of guidelines. The result was an enterprise-wide set of guidelines that was posted on the company website in 2019. This was accompanied by manuals, training materials, roadshows and a Post-Access Trial Access Consultation Board that would be a contact point for any future questions.
“It was the sort of program that touched people’s hearts and so every single division was on board to be a part of developing these guidelines,” said Steel.
“Of course, one of the first steps was also putting together a budget that would allow us to provide this access at no cost to trial participants,” said Paul Aliu, Head of the Global Governance Office within the Chief Medical Office. “From the start, when we brought this proposal to our CEO Vas Narasimhan, he affirmed the company’s commitment because we all believe that this is the right thing to do.”
Leading the industry
Since 2019, Novartis has received international recognition for its post-trial access policies. In 2021, the independent non-profit Access to Medicine Foundation ranked Novartis as a standout among pharmaceutical companies. This was particularly in recognition of the company’s commitment to post-trial access for participants in all trials designed to demonstrate superior efficacy, even those outside of severe and non-threatening diseases.
A related aspect of these policies is that Novartis also ensures that its clinical trials will only be performed in countries where the company intends to register that drug, should the outcome be successful. This is important because it avoids the scenario where clinical trials are performed in countries where there is no chance of the drug ever becoming available, even if it is approved.
Another group, the Multi-Regional Clinical Trials (MRCT) Center of Brigham and Women’s Hospital and Harvard, has also contacted Novartis in order to learn best practices from the internal guidelines and frameworks in place at the company. The MRCT Center acts as an independent convener bringing together industry, academia, patients and patient advocacy groups, non-profit organizations and national regulatory agencies. The Center employs a defined and deliberative process to achieve ethical, actionable and practical solutions, and is dedicated to improving the quality and efficiency of clinical research worldwide. This collaboration between the MRCT and Novartis may help share the practices at Novartis with other companies and institutions conducting clinical trials.
Commitment to access
“I think one of the big wins with these guidelines and the policy is that our teams have the mindset and the resources they need to plan upfront about the possible outcomes and what the implications will be for the patients in the trials,” said Aliu. “And we have a suite of different options for post-trial access to make sure that no trial participant is left behind.”
In some locations, post-trial access can be as straightforward as continuing to supply treatment to the participants in a non-clinical trial setting. But this model – called post-study drug supply – is not permitted in all countries.
“The rollover extension program option has been particularly useful with the investigational drug we’re working on because we could provide uninterrupted access,” said Dahlke.
This version was adapted on April 4, 2024.
