Statement on Patient Access to Therapies
While we seek cures, we stand committed to people living with ALS today having a chance to access treatments that could slow disease progression and give them more time. We firmly believe that even one more person with access to an ALS treatment that might work for them is progress.
Regarding the current situation with Biogen’s Toferson, we feel the need to weigh in, particularly given Biogen’s recent statement regarding an Expanded Access (EA) program. We agree that people who volunteer to participate in a clinical trial are, indeed, brave and they and their families make countless sacrifices to make their participation possible; however, they are also considered by many in the ALS community to be the lucky ones. That is because this disease is 100% fatal, often rapidly debilitating, with most people living no more than 2-5 years from diagnosis.
We vehemently refute the notion that an expanded access program cannot be conducted in parallel with a clinical trial, and reject the notion that doing so poses an ethical issue. In fact, keeping a therapy that can only reach someone who doesn’t qualify for a trial through the provision of expanded access from them poses a far greater ethical concern.
As described by the FDA – expanded access is a “pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.” ALS clearly fits the definition.
Further, expanded access must meet a set of criteria, including that “patient enrollment in a clinical trial is not possible.” This means there is no conflict between those in the trial and those who would qualify for expanded access; by definition, you can’t fit both categories. For most ALS trials, people whose disease has progressed to a certain level or who were diagnosed a certain number of years ago, do not qualify for a single trial. Expanded access is their only hope for a chance at an experimental, disease-altering treatment.
We recognize that until there are uniformly available cures for ALS no approach to providing treatments, especially investigational therapies, will be wholly satisfying as expanded access cannot be provided to every qualifying patient. However, we expect that as a leading biotechnology company, Biogen should not undo the tireless efforts of people living with ALS who have made so much progress on setting the expectation that expanded access is the norm for ALS clinical trials. We have started to change the conversation and meet the expectations of those directly impacted by ALS. We can’t go back. We expect that all ALS trials provide some level of expanded access. We look forward to Biogen announcing this opportunity for Toferson as expeditiously as possible.
In the meantime, we will be relentless until safe, effective therapies reach everyone for whom they will provide benefit, until we realize the promise laid out in the FDA Clinical Trial guidance for ALS, and until we enact policy, regulatory, and programmatic solutions that make the system work better for ALS therapies to-come.
We will find cures for ALS. The only question is when. That answer depends on you.
Stay informed on I AM ALS’ progress and what you can do to help us make hope real.
Lou Gehrig Day for MLB
After a group of advocates impacted by ALS banded together to honor Lou Gehrig and the ALS community and raise awareness for ALS, Major League Baseball announced a league-wide Lou Gehrig Day that will be held yearly on June 2nd.
The Road to Accessing Promising Treatments
As we said last week, I AM ALS will not stand for the status quo when that means tens of thousands of people are diagnosed with ALS and told there is nothing medicine can do. We also recognize that leadership doesn’t mean we have all of the answers. That’s why we spent the last week listening to the ALS community. Here is our proposed response, not to the outcome of a single trial but to everything we see happening in ALS research.