When Time Matters Most
Sarah sat in her oncologist's office in Pittsburgh, holding a printout about a promising new treatment for her rare blood cancer. The drug was still in clinical trials. Her doctor said it showed real promise, but it hadn't received FDA approval yet. Five years ago, Sarah's story might have ended there. But thanks to the 21st Century Cures Act, passed in 2016, she had options her doctor needed to discuss with her.
If you or someone you love is facing a serious illness, understanding this law could open doors to treatment options you might not know exist. Let's break down what changed and what it means for patients today.
What the Law Changed
The 21st Century Cures Act rewrote several rules about how patients can access experimental treatments. The law did three big things for patients: it sped up the approval process for certain treatments, especially cancer drugs; it gave patients' experiences more weight in treatment decisions; and it clarified the rules for accessing unapproved treatments.
Faster Access to Cancer Treatments
Through a program called Real-Time Oncology Review, the FDA can now evaluate certain cancer drugs faster than ever before. Instead of waiting until all trial data is complete, they can review results as they come in. For some cancer patients, this has cut months or even years off the wait for new treatments.
Your Voice Matters More
Before this law, a treatment's success was measured mainly by clinical data. Now, the FDA must consider 'patient experience data' when evaluating new treatments. This means they look at things like: How does the treatment affect daily life? What side effects do patients find acceptable? What benefits matter most to patients?
Clear Rules for Accessing Unapproved Treatments
The law created clearer guidelines for expanded access (sometimes called compassionate use). Drug companies must now:
- Make their expanded access policies public
- Respond to requests within 15 days
- Explain their decisions if they say no
The Right to Try Act of 2018 built on these changes, creating another path for terminally ill patients to access experimental treatments.
What This Means for You Today
If you're facing a serious illness, these changes give you more options to consider with your healthcare team:
- You can ask about treatments still in development
- You can request expanded access to treatments not yet approved
- Your experiences and preferences should be part of treatment discussions
But there are limits. Not every company will grant access to experimental treatments. Insurance rarely covers unapproved treatments. And even with faster reviews, new treatments still must prove they're safe and effective.
Getting Help Understanding Your Options
The rules around accessing experimental treatments can be complex. Patient navigators at Wondrlink Foundation can help you understand what's available and how these laws apply to your situation. We won't make decisions for you, but we'll help you ask the right questions when talking with your healthcare team.
Your doctor remains your best source for medical advice. Our role is to help you understand your options so you can have informed conversations about your care.
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