We have some exciting news to report! As you may recall, Hope NOW (HNFA) met with the FDA for the fourth time in late August and proposed a clinical study concept that incorporated predictive algorithms, more-sensitive and faster “adaptive trials” as an alternative to the current slow and less-sensitive “population mean comparison” trials, and crossover of patients from the placebo control arm to the active drug. The FDA expressed support and suggested we might want to work directly with the researchers – and we have! So far, two biopharmaceutical companies are on board. We connected them with a leading data analytics firm so that impending trials can be designed far more efficiently with faster and more meaningful results. This could pave the way for a drug or treatment to be given faster consideration for Accelerated Approval (AAP) – or even full approval (registration) – as the evidence of efficacy will be stronger. All the components of our proposed study concept have the potential to foster ALS trials that are smaller, faster, smarter and more ethical.
Hope NOW (HNFA) has also completed filing a 501(C)(3) nonprofit application and created a Board which recently held its first official meeting. Robert Hebron was elected Chair, Jehad Majed Vice Chair, and Linda Clark Secretary.
Other board members include Nick Grillo, Eric Valor, Jay Smith, and Stephen Finger. An advisory committee will also be created and officer positions filled as necessary. These will be announced and shown on our website so keep coming back!
Several members of the HNFA board are actively serving on committees for the ALS Association’s FDA Guidance Document. This involvement has been critically important. Our members have already played a significant role shaping the direction of this project.
Looking ahead to 2016 we expect to lead more public events to campaign for smarter trials and faster drug approvals. We still to tackle the FDA’s resistance to utilizing real clinical endpoints in ALS for Accelerated Approvals (e.g. FVC). The swift progression of ALS should not require the use of surrogate endpoints to measure the statistical effects of a therapy. The FDA has granted AAP for the treatment of cancers, HIV, and made ZMAPP available for Ebola patients – but rejects the same consideration for treatments aimed at ALS. Not many fatal diseases move as quickly as ALS, have no treatment, and affect so many people at any age. With your help we will keep up the pressure for swift and compassionate action.
Hope Now for ALS is focused on two objectives:
- Establishing a pathway for Accelerated Approval (AAP) for all safe and promising therapies for ALS,
- Modernizing the clinical trial process to bring it into the 21st century.