There's no question that for patients coping with a rare or complex disease or a compromised immune system, COVID-19 resulted in multiple new challenges, as we detailed in the blog, “5 Ways COVID-19 Impacts Immunocompromised & Rare Disease Patients.” However, as Albert Einstein famously noted: "In the middle of difficulty lies opportunity." The following are some promising developments that have emerged during the pandemic, which are likely to positively impact pharmaceutical companies, physicians, and their patients in the years ahead.1) Better approaches to clinical trials: Rare disease research, already challenging due to the small numbers of patients eligible for clinical trials, became almost impossible to accomplish in 2020, resulting in the cancellation of the majority of non-COVID trials. An innovative new way was desperately needed in decentralized clinical trials and arrived in 2021. As presented at the Clinical Trials in Rare Diseases Conference 2021, the pandemic offers an ideal opportunity to accelerate the push toward this concept, providing a long-term solution to several formerly intractable problems encountered in rare disease research.
Decentralizing clinical trial components using telehealth, home nursing, remote monitoring, and direct delivery of products to participants has proven successful, and these are now increasingly incorporated into protocols. In a recent report from TMF Futures, 65% of companies in the life sciences sector say they will continue to run clinical trials remotely after the pandemic. Strategies that enhance convenience and comfort for trial participants result in a raft of benefits: faster, more robust trial enrollments, reduced costs, improved retention, shortened timelines, improved data interoperability, and greater diversity, according to Michele Rhee, a rare disease-focused drug development company executive. "Covid-19 just accelerated a lot of things that were in process and brought us closer to that decentralized model. Every barrier we can knock down can improve our chances of success. Every participant, every patient is precious, so understanding what can make this an easier, better process for them is something that we all strive for," she said at the Clinical Trials in Rare Diseases Conference 2021.2) New focus on the immunocompromised in COVID-19 research: Much remains unknown regarding the effects of COVID-19 vaccines, boosters, treatments, and the virus itself for those with complex conditions and compromised immune systems. A host of new studies aims to fill in the gaps with data that will help physicians and their patients better understand: response to COVID-19 vaccines in people with immune deficiencies; third and fourth vaccine doses; antibody response after the third dose; vaccine efficacy in multiple sclerosis patients treated with immunosuppressive therapies; the impact on immunity from boosters in transplant patients when immune-suppressing medications are reduced; and many others.
"The information we gather on how well COVID-19 vaccines protect these specific populations and about any adverse events experienced by those with immune dysregulation or other disorders will aid decision-making about vaccination," said Steven Holland, M.D., director of the National Institute of Allergy and Infectious Diseases Division of Intramural Research.3) Substantial rise in governmental support: Some promising advances on the federal level indicate increased recognition of the need to expand focus on rare disease treatment options as the pandemic wore on.
Over the next five years, a total of $76 million will fund the National Institute of Health's Bespoke Gene Therapy Consortium (BGTC). The public-private partnership will establish platforms and standards to speed developing and delivering customized gene therapies for rare diseases. Noting that "the limiting factor for developing a gene therapy is not scientific knowledge, but rather operational and financial hurdles," the BGTC is launching a multi-pronged strategy to overcome obstacles that can occur in the areas of clinical trials, manufacturing and production, and regulatory requirements.
The U.S. Food and Drug Administration (FDA) also boosted its Orphan Products Grants Program at the end of 2021 with 11 new research grants equaling more than $25 million. The FDA stated, "As challenges and increased costs continued this year for clinical trials due to the COVID-19 pandemic, the FDA remains committed to supporting rare disease research by providing existing grantees with additional funding." The resources will be used to implement necessary measures to assure the safety of the study participants, maintain compliance with good clinical practice and minimize risks to trial integrity.
Other encouraging news: in 2021, 52% of novel drugs approved by the FDA will help patients with rare diseases, including Duchenne muscular dystrophy, generalized myasthenia gravis, Pompe disease, and rare cancers, including cholangiocarcinoma and perivascular epithelioid cell tumor. The pipeline for new drugs continues to be robust, especially for those based on mRNA technology so successfully used for COVID-19 vaccines. For example, Moderna has several research projects using its mRNA technology to develop treatments for cystic fibrosis and rare diseases.
Providing patients with comprehensive and compassionate care since 1987, Orsini Specialty Pharmacy is the leading independent specialty pharmacy focused on rare diseases, gene therapies, and complex conditions. Orsini's high-touch care model centers around experienced, therapy-specific teams that provide personalized care to patients. Learn more about Orsini's essential services for rare and complex disease therapies and our dedicated support for physicians, manufacturers, payors, and patients.
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