Promising Pathway Act: Speeding Up Cancer Drug Access & Clinical Trials

•There may be many promising new therapies, but you can’t access a lot of them in a combination. You have to pick and choose which ones you're going to pursue.

•If a treatment doesn't work and your disease is very aggressive, you have to pivot quickly to the next one.

•The current drug development process is too slow and costly for rare diseases, yielding few new treatments.

•Only a very select few, about 5% of the population, can access clinical trials, which can offer many advantages over standard treatments. One of the ways to address the regulatory hurdles to accessing treatments and accelerate treatment development is through policy and regulatory changes. A key initiative to drive these changes is “The Promising Pathway Act”, pending legislation which will create a conditional approval pathway for the FDA. After you see that a drug is safe, and it has the effect you want it to have (Phases 1 and 2 of the three-phase regulatory approval process), it would get a conditional approval. Any doctor could prescribe it, and insurance would handle it like any other approved drug. Any patients who use these drugs would be followed as if they're in a clinical trial. We would learn from every patient. We would continue the research. The only difference from the standard pathway is that it's more flexible, where you can do combinations if you like, and any patient could get access. Al Musella is uniquely qualified to describe the issues behind the need for "The Promising Pathway Act" and what the changes might mean to patients and caregivers. He founded the Musella Foundation, which offers comprehensive resources including videos, written information, discussion forums, patient navigation services, and advocating for change in the system. They have been conducting a study on brain tumor patients since 1993, focusing on the treatments received and the outcomes, with the aim of identifying more effective treatment combinations. Al was personally motivated by his family's experience with brain cancer and has Musella) system. They have been conducting a study on brain tumor patients since 1993, focusing on the treatments received and the outcomes, with the aim of identifying more effective treatment combinations. Al was personally motivated by his family's experience with brain cancer and has been instrumental in creating an online database of clinical trials. The foundation also encourages participation in their brain tumor Virtual Trial , which tracks patient outcomes, and seeks support through updates on treatment options, spreading the word, advocating for change and donations to sustain these vital projects. What are the problems with the current FDA approval process, especially phase 3 trials?

•Access: Access to the drugs is limited to the small population able to qualify and get to a site, and getting access is expensive. It's very hard to get into the best trials right now. You may not get access because you don’t meet the eligibility criteria which favor relatively healthy patients.

•Unrepresentative: The patients selected for clinical trials are not representative of the patient population. They may publish some research on a select group of patients, but you won’t get published results on a typical patient.

•Slow: Clinical trials take a long time (typically five to ten years).

•Expensive: Clinical trials are expensive to run, and those costs are incorporated into drug prices to repay the research and development costs. A drug company can pay $1 million per patient to a hospital for a phase 3 clinical trial.

•Unreliable: The results are often not trusted by the research or provider communities. What about getting access through newer alternative pathways (“Right to Try” and “Expanded Access”)? These two pathways help, but they are not enough. The average patient is not going to get a drug through these alternative pathways. They are also not going to increase the number of drugs available to patients.

•Drugs are expensive. Insurance can't pay for it, and nobody's going to give it for free. Only the rich people get it.

•Drug companies are a little bit afraid to go outside of the FDA. They are also afraid to publish their actual costs.

•It takes too much time for the doctor. How does the Promising Pathway Act solve the problems with the current process? The difference is, instead of doing a phase 3 trial, you're letting anybody participate in this virtual trial. It's just a different way of doing the phase 3 trial. The benefits include:

•Access: Improves patients’ access to treatments, especially experimental treatments for rare and aggressive cancers.

•Information: Improves transparency and enables data analysis through a database of medical records, yielding much more data on each drug and on how to use it, including exploration of drug repurposing using real-world evidence and how drug combinations work.

•Engagement: Engages patients and caregivers in accelerating cancer research through clear consents.

•Cost reduction: Reduces drug costs, passing on the reduced research and development costs. After approval in the relatively small phase 2 trial, the drug company can start making money. Phase 3 trials, the largest and most expensive phase, will be minimized, and cut out the phase where the most time and money is spent.

•Drug discovery: Increases new drugs in the research pipeline.

•Learning: Speeds up the pace of learning from visibility into more real-world experiences – if something is working or isn't working everybody can find out about it, opening up treatment options and avoiding repeating the same mistakes.

•Incentives: Releases the liability of the doctor and the drug company if things go badly. What can you do to promote the Promising Pathway Act? Patients and caregivers need to be the cheerleaders for this. Pharmaceutical companies will be able to discover more drugs, but it will lower the bar to competition. You should contact your senators and representatives. An easy way is to send an email using the form on the Musella Foundation website . You put in your name and address, and it only takes a couple of clicks to send a message to your senators and representatives. The information and opinions expressed on this website or platform, or during discussions and presentations (both verbal and written) are not intended as health care recommendations or medical advice by Cancer Patient Lab, its principals, presenters, participants, or representatives for any medical treatment, product, or course of action. You should always consult a doctor about your specific situation before pursuing any health care program, treatment, product or other course of action that might affect your health. Meeting Notes KEYWORDS drug, patients, people, trial, brain tumor, treatment, clinical trials, doctor, approval, phase, good, pathway, years, fda, glioblastoma, data, access, tumor, progression free survival, give SPEAKERS Al Musella (82%), Chris Apfel (5%), Richard Anders (4%), Roger Royse (4%), Amit Gattani (2%), Brad Power (1%), Vanessa Hugo (1%), Jeff Waldron (0%), Dan Moynehan (0%) OUTLINE 1.Promising new legislation to transform the FDA drug approval process. (0:02) 2.Glioblastoma diagnosis and treatment in 1992. (1:03) 3.Cancer treatment and advocacy efforts. (2:56) 4.A drug that helps brain cancer patients with limited access. (8:03) 5.Drug development and access restrictions. (13:31) 6.Drug approval pathways and their challenges. (16:39) 7. trials and FDA approval for cancer treatments. (22:45) 8.Clinical trials for glioblastoma treatment. (27:27) 9.Accelerating drug development for glioblastoma. (29:34) 10.Improving cancer treatment through data analysis and patient consent. (35:48) 11.Accelerating drug development for rare and aggressive cancers. (41:28) 12.Brain tumor advocacy and legislation. (46:47) 13.Using a database to improve cancer treatment outcomes. (51:55) 14.Using real-world evidence for drug repurposing. (57:58) SUMMARY Promising new legislation to transform the FDA drug approval process.

•Roger Royce introduces Al Musella, president of the Musella Foundation for Brain Tumor Research and Information, to discuss the Promising Pathway Bill. Glioblastoma diagnosis and treatment in 1992.

•Al Musella shares his personal story of losing his sister-in-law to glioblastoma in 1992, despite doctors telling them there were no more treatment options available. Cancer treatment and advocacy efforts.

•Al Musella shares his personal experience with cancer treatment and the challenges he faced in finding clinical trials and affordable treatments.

•He created the first online support group and database of clinical trials, which helped revolutionize the way clinical trials are reported and managed.

•He has dedicated his career to advocating for brain tumor patients, including creating the first online registry of brain tumors and navigating over 25,000 patients through the healthcare system.

•Despite his efforts, Musella believes that most brain tumor patients are not receiving adequate care and are dying or suffering due to a lack of progress in the field. A drug that helps brain cancer patients with limited access.

•Dr. Musella suggests an experimental drug for a patient with glioblastoma, despite the patient's poor prognosis and lack of access to clinical trials.

•The patient experiences improvement in symptoms after receiving the drug through an expanded access program.

•Al Musella shares his experience with a drug that has helped a child with a rare disease, but the FDA has denied expanded access and requires a randomized trial to prove its effectiveness.

•The FDA's denial has left hundreds of other people without access to the drug, despite published reports showing it more than doubles average survival for this disease. Drug development and access restrictions.

•Al Musella discusses the challenges of accessing experimental treatments, including high costs and restrictions on expanded access programs.

•He believes the Privacy Pathway Act could provide a solution by allowing nonprofits to raise money for drug development and providing more affordable access to treatments. Drug approval pathways and their challenges.

•Al Musella discusses the limitations of the accelerated approval pathway for drugs, including the lack of impact on overall survival despite positive progression-free survival and response rates.

•The FDA has been critical of using progression-free survival and response rate as endpoints in drug trials, as they do not directly impact overall survival.

•He discusses challenges with accelerated approval for brain tumor drugs, including lack of good data and limitations in expanded access.

•He explains the Conditional Approval Pathway for drugs, where the FDA grants provisional approval for up to 8 years, with the possibility of full approval after presenting additional data.

•The drug must demonstrate substantial evidence of safety and early evidence of a positive therapeutic outcome in at least a phase 1 trial, with the level of risk being the same as a phase 3 trial. Clinical trials and FDA approval for cancer treatments.

•Al Musella argues that pathway X is more effective than traditional phase 3 trials.

•He discusses the challenges of getting FDA approval for a new cancer treatment, including the rejection of a phase 3 trial due to issues with the blinded design.

•He highlights the case of Optune, a treatment that was approved despite similar concerns about the blinded design, demonstrating the inconsistency in FDA decision- making. Clinical trials for glioblastoma treatment.

•Al Musella discusses issues with clinical trials for DCVax and Optune, including problems with endpoint analysis and patient response. Accelerating drug development for glioblastoma.

•Al Musella argues that phase 3 clinical trials for glioblastoma are useless due to selection bias and inadequate representation of the target population.

•He proposes a virtual trial as a better alternative, citing potential benefits of increased sample size and more accurate data representation.

•He believes the 21st Century Cures Act will improve cancer treatment by allowing for more clinical trials and access to new drugs.

•He expects the act to lead to better drug combinations and lower prices, as well as more data on how to use drugs effectively.

•He also notes that the current system makes it difficult for patients to access the best clinical trials, and that the act will provide more options for patients and doctors to make informed decisions. Improving cancer treatment through data analysis and patient consent.

•Al Musella emphasizes the need for transparency in tracking patient outcomes to speed up the pace of cancer research and find a cure.

•Roger Royse agrees and adds that there is resistance from doctors themselves, and the FDA and insurance companies are also significant barriers.

•Al Musella explains that the biggest obstacle to using new cancer drugs is the risk aversion of doctors, who are hesitant to prescribe them due to liability concerns.

•He also notes that the current system is not designed to be a moneymaker for hospitals, and that the promising pathway act could potentially incentivize doctors to prescribe new drugs by reducing liability concerns. Accelerating drug development for rare and aggressive cancers.

•Richard Anders and Al Musella discuss the complexity of excluding patients in clinical trials, with Al Musella suggesting that drug companies may propose specific criteria for inclusion and exclusion in their trials.

•Richard Anders raises concerns about the potential for subjective exclusions based on non-scientific factors, while Al Musella emphasizes the importance of scientifically-driven exclusions.

•Al Musella proposes a new law to reduce the cost of drug development by eliminating phase 3 trials for serious diseases with no effective treatment, such as ALS, brain tumors, and pancreatic cancer.

•Amit Gattani asks about the number of drugs that have gone through this process so far, and if it's focused primarily on brain cancer or open to other types of cancers. Brain tumor advocacy and legislation.

•Al Musella and Amit Gattani discuss the importance of contacting congresspeople to support a bill that would provide access to experimental treatments for brain tumor patients.

•Al Musella emphasizes the need for people to contact their representatives and push for support, as congressmen rely on public pressure to make decisions.

•Al Musella and Vanessa Hugo discuss the Promising Pathways Act, a bill that aims to provide funding for cancer research and treatment, particularly for rare and aggressive forms of cancer.

•They mention the Cancer Moonshot program and how it could be a catalyst for the bill's passage, and they plan to rally support in the House of Representatives. Using a database to improve cancer treatment outcomes.

•Al Musella discusses a bipartisan effort to create a database of medical records, with patients having access to their own data and the ability to verify its accuracy.