"While there has been talk of allowing natural history data for devastating indications like DMD, natural history is not as good as placebo."
Why are you so sure about that? If that's the case why FDA was keen to discuss the importance of patient engagement and natural history studies in rare disease product development....?
-- Janet Woodcock, acting Commissioner of the FDA and Director of the Center for Drug Evaluation and Research, explained why natural history data is important in research. The information collected “represents the enactment of strategies to harness the very type of knowledge so essential for the design and execution of clinical trials of potential new therapies, but which has been lacking in the rare disease space,” Woodcock wrote in “The Power of Patients: Informing Our Understanding of Rare Diseases.”
Duchenne Muscular Dystrophy Natural History Similar to Placebo Treatment "In the largest multinational analysis of data from clinical trials of potential treatment for Duchenne muscular dystrophy (DMD), results of placebo treatment suggest real-world (RWD) or natural history data (NHD) could be used instead. Substituting RWD or NHD for placebo treatment potentially aid efforts to modernize how new treatments for DMD are tested as well as increase enrollment in clinical trials."
"...This rigorous study establishes a strong foundation for using natural history data as a substitute for placebo control in clinical trials and as a comparator to determine the effectiveness of prescribed drug treatments versus standard of care treatment."
From our ATL1102 Phase 2 study: Statistically significant improvement in PUL2.0 with ATL1102 treatment in non-ambulant boys with DMD compared to a matched natural history control ATL1102 was recently assessed in an open label Phase II study in adolescent non-ambulant patients with DMD. In the Phase II study, nine patients, 12 to 18 years old [mean 14.9 (SD 2.1) years] were dosed with ATL1102 for 24 weeks (8 of 9 on corticosteroids) and assessed using PUL2.0. In a post study analysis, the mean PUL2.0 data from the ATL1102 treated patients was then compared with the 24-week PUL2.0 data of 39 assessments in 20 non-ambulant patients [mean age 15.61 (SD 2.02) years; 19 on corticosteroid] from a natural history database of DMD patients in Rome, Italy - the Rome cohort (RC). The RC were identified using the same inclusion criteria used to enrol patients in the ATL1102 Phase II study. The rare disease pharmaceuticals landscape is undergoing revolutionary change. The industry is making unprecedented investments in the development of innovative therapies and embracing novel trial designs in an effort to reduce time-to-market, in turn bringing much needed treatments to patients with rare disease who often have no existing treatment options. These investments are paying off. In 2019, just over half of new active substances received an orphan drug designation at time of approval.
Many of these new therapies benefit from fast track approval programs for breakthrough therapies, designed by regulators to speed new drugs to market. To achieve this accelerated approach, developers must first deepen their understanding of disease progression, population heterogeneity, the patient and caregiver perspective, and the current standard of care when one exists. These insights can be gained through natural history studies.
When developers incorporate natural history studies into their rare disease drug development plan, the insights can be used to optimize clinical planning, support regulatory alignment, and provide the longitudinal data needed to support single arm trials.
"Natural history studies collect information from patients to better understand diseases, especially rare ones." CSL Behring
Gathering information of how a disease affects a person over a lifetime. If the disease is rare, that collected data is especially valuable and can lead to new understandings, breakthroughs and treatments. According to the U.S. National Organization for Rare Disorders, natural history studies can include diagnosis and treatment, testing samples, reports from clinicians and information from patients about their quality of life.
Everyone has his/her point of view, but sometimes it's best to listen to the regulatory/scientists ....
ANP Price at posting:
19.0¢ Sentiment: Buy Disclosure: Held
(20min delay)