As DIPG/DMG Collaborative members, we are helping facilitate the passage of the Promising Pathways Act through Congress. This act intends to make potentially life-saving treatments more accessible to entire classes of pediatric patients facing rare, terminal diseases.
The current accepted standard of care for treating DIPG needs to be improved. Radiation and chemotherapy are not cures but rather opportunities to lengthen a child's life expectancy by a few months or slightly improve a child's quality of life. Families facing a DIPG diagnosis deserve more. They deserve the ability to hope.
What does the Promising Pathways Act seek to accomplish?
The Promising Pathways Act, concerning DIPG, has the potential to significantly improve the lives of individuals facing rare, terminal, and pediatric diseases. It aims to make provisionally approved medicine more accessible and to mandate the usage of observational registries, enhancing transparency and our collective ability to study provisionally approved medicine.
Why not continue "Expanded Access" or "Right to Try?"
Expanded Access and Right to Try are current mechanisms we have in place intended to allow families to access provisionally approved treatments. However, these policies can be described as a "Wild West" when examining their functions.
Both Expanded Access and Right to Try require individual patients and families to apply for approval individually. This approval process does not serve the DIPG community. DIPG patients often have mere months to live post-diagnosis and cannot wait for the bureaucratic process to grant them conditional approval as individuals to try an experimental drug. The Promising Pathways Act would allow DIPG patients to access a specific treatment as an entire class. This results in the preservation of time, which is crucial when dealing with DIPG, while minimizing the effect of your own hospital's familiarity with DIPG and corresponding speed in making recommendations on your treatment experience.
Additionally, both Expanded Access and Right to Try lack the data transparency required when dealing with a terminal illness. Due to the "wild west" nature of their approval process and the lack of data reporting to researchers, other drug providers, and prospective patients, individuals seeking provisional approval under Expanded Access and Right to Try have no insight into the successes or failures of the drug they seek to use. Thus, if a specific treatment has had a 0% success rate, families and children will continue to enroll in this treatment because of the complete lack of transparency. Conversely, the Promising Pathways Act will require the usage of observational registries, meaning researchers, providers, and families will be appraised of the successes or failures of a treatment before enrolling. This will function to save lives and allow us as a research community to spotlight potentially game-changing therapies faster.
Improved clinical trial design for DIPG
Our current regulatory process concerning experimental treatment is not built for an illness like DIPG. Currently, to bring a potentially beneficial therapy to DIPG patients in a clinical trial, that treatment must undergo three phases of study on adult populations: safety, dosage, and efficacy. This becomes an issue when dealing with a rare disease that is almost entirely pediatric. There are not enough adult DIPG patients to conduct three-phase trials on adults before bringing these treatments to pediatric patients.
As a result, DIPG trials have adjusted by trying to take treatments that affect alternative cancers and translate those to DIPG. This means our researchers are often unable to start their work addressing DIPG at the baseline but have to mix and match puzzle pieces to bring drugs to DIPG patients.
The Promising Pathways Act would address this by providing a mechanism through which DIPG patients could receive treatment explicitly designed for DIPG without sacrificing safety. The PPA would still require prospective treatments to undergo phase one studies with adult communities to ensure the drug is safe to administer. However, once satisfied, the treatments would be permissible in trials with DIPG patients, provided researchers have preclinical evidence indicating the drug would be beneficial. This would result in access to provisionally approved therapies that can give hope to families and children, therapies currently being developed or already available that have been unable to clear the bureaucratic hurdles necessary to reach these children.
Conclusion
This is why we, as the Collaborative, are working with our Senate representatives to advance the Promising Pathways Act through Congress. This act would overhaul the current practice of approving individual patients to try a drug and would instead conditionally approve therapies for entire classes of patients. It is worth emphasizing that this act would not mean unsafe experimental therapies, as the medications and treatments would still be required to pass through typical FDA safety testing with adult patient groups and show potential lab testing benefits.
Moving legislation through Congress is long and has many potential obstacles. However, we will continue to push because we believe that families currently in the fight deserve the right to hope.