Last November, Janet Woodcock – the Director for the FDA’s Center for Drug Evaluation and Research – gave the keynote address at the Pulmonary Fibrosis Foundation‘s summit in Washington, D.C.
The speech focused on developing new therapies, not just for IPF, but for all under-treated conditions. As Woodcock sees it, polishing up three critical areas could lead to a significant rise in treatment developments.
So, let’s break ’em down:
1. Biomarkers
Developing new treatments is an expensive and time-consuming process. Consider how many different evaluations need to happen just for a potential therapy to progress through Phase I, II, and III trials. That commitment of time and money can scare some drug developers off.
Biomarkers, or specific biological indicators of a condition (that can be measured), can make things a little easier.
By identifying biomarkers for specific conditions (such as forced vital capacity for IPF), drug developers have more tools to assess their success or failure earlier in the development process. That way, time and money don’t need to be invested unless they already know they’re on the right track.
Biomarkers can also serve as surrogate endpoints for clinical trials.
If a company can prove their medication has a significant effect on something directly associated with the disease, then the FDA might approve the medication even without other endpoints being proven.
And, for a condition like IPF where multiple drugs may be needed to achieve maximum effect, biomarkers can also be used to assess how different treatments work together.
2. Clinical trials
As mentioned above, clinical trials take a long time. They also require a lot of people to help them run efficiently. Unfortunately, many clinical trials face numerous challenges just getting off the ground.
For example: recruiting prospective patients.
Recruiting patients can be a problem because trials are often designed with ridiculously specific inclusion/exclusion criteria. Additionally, once people are recruited, their experience in the trial might be less than ideal.
They’re living with a condition that affects what they’re able to do, and yet they’re being asked to make repeated visits to inconvenient locations for multiple evaluations.
Woodcock argues that patients, advocates, and support groups can help improve clinical trials by:
- working with the biopharma industry to establish realistic inclusion/exclusion criteria,
- structure the trials in a way more convenient to people living with various ailments,
- and to spread information so that potential trial participants hear about the trials from trustworthy sources they’re already familiar with.
In fact, the Clinical Trials Transformation Initiative is already working to make sure these things happen.
3. Patient-focused drug development
Before a potential new therapy is approved for use in the United States, the Food and Drug Administration needs to sign off on its safety and efficacy. Unfortunately, efficacy might be evaluated with aspects of the disease that aren’t that important to the people who are actually living with it.
In September of 2014, the FDA had a Patient Focused Drug Development meeting specifically for pulmonary fibrosis. During that meeting, people living with IPF and those close to them shared their experiences with the disease and using current therapies.
Among the key learnings was that, for many IPF patients, the cough is one of the most difficult parts of the disease to deal with.
Coughing hasn’t been examined in clinical trials because it might not help prove the drug’s efficacy. But now, since the FDA and biopharma companies know this is an important consideration for people with IPF, they can start observing/reporting on this even if it isn’t the primary endpoint for the study.
