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Benefits of the Compassionate Use Programs for Developers
(Thursday, July 14, 2022) For patients with untreatable serious or life-threatening diseases, the benefit of compassionate use or expanded access programs is indisputable. However, companies providing drugs to patients under these programs can also use these as opportunities to generate valuable real-world data in support of their regulatory applications. A report by Novartis’ compassionate use program recommends three steps for companies to enhance their expanded access program for mutual beneficence. First, companies must be open and willing to offer their drugs for compassionate use. Such programs should be encouraged and supported to collect real world data from a “broader patient population beyond the clinical trial”. This would also help engage with patient communities that could be invaluable in rare diseases. Second, the compassionate use requests should be professionally managed. Companies should have clear policies, written processes, and management systems to handle compassionate use requests. Rather than handling such requests on a case-by-case basis, which leads to subjective decisions and bias, company policies must define the criteria for approving requests for compassionate use, timelines for decision making, clear message to requesters, and well managed process. Third, companies must have processes to collect, collate, analyze, and report patient experiences from the compassionate use. There should be robust follow-up with patients, likely led by subject matter experts at the company, to collect complete patient experience data. Company policies can dictate the responsibilities for the requesters in terms of transparency of data. Also, data from compassionate use should be shared with research teams and patient communities to create improved solutions, with considerations for regulatory compliance in terms of sharing information for unapproved products with patients. At the minimum, data from the real world experiences could guide improvements in the ongoing clinical trials and may provide additional supporting data for regulatory applications. The Novartis experience is certainly not limited to one company but more companies should share similar experiences with their programs for the ultimate mutual benefit for patients and developers. 
AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: mkumar@fdamap.com Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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