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by Administrator | 8 Nov 2016

No more options?

How drugs are  approved 
 

To explain how this process works, it’s important to understand how drugs are approved.

When a potential new drug is identified, it needs to be thoroughly tested.
First, data about safety must be gathered and the  optimal and/or maximum tolerated dose  must be identified. This is typically studied in a phase I clinical trial.
When results are positive, the drug moves on to phase II to find out if the  drug is effective  (and safe) enough to move on to phase III.
This is where the effectiveness and the  safety profile  of the drug is reconfirmed in a large group of patients. If the results of a phase III trial are positive, the manufacturer of the drug can apply for a marketing authorization with a competent authority (e.g. FDA or EMA). 

 

The downside of this process is that it can take approximately  12–15 years  to complete while patients who don’t have the time to wait this long, do not have access to the drug. 


 

What are the  options? 

 

Clinical trials 

As a consequence of the procedure described above, there is a need for patients to participate in clinical trials.
Entering a clinical trial might thus provide an opportunity for a patient to  get access to a drug before it is approved. Especially phase III trials can be an opportunity for patients because then there is a good indication of safety and efficacy of the drug.

A downside of participating in clinical trials is that there is a chance that a patient is allocated to the  control group  where you don’t receive the drug that is being tested. Also, not many patients qualify for clinical trials as their  inclusion criteria  are often stringent. 

myTomorrows has developed a Knowledge Base that combines large amounts of medical and clinical data to help patients find a clinical trial they might be eligible for. 


 

Compassionate use and Early Access 

At  myTomorrows  we believe that patients who have no approved treatment options left and also do not qualify for clinical trials, should be able to get access to an  unapproved drug when it already passed phase II Clinical Trials.

These drugs already have safety and effectiveness data and most governments have compassionate use laws  in place that enable physicians to use drugs in development for their patients, after receiving permission from the authorities if needed.
In practice, these processes are  complex and time consuming. myTomorrows can help patients and their doctors to identify possible compassionate use and Early Access Programs and offer assistance with the often complex regulations and procedures.


 

Off label  use 

There is a legal opportunity for doctors to prescribe drugs that are registered for  another indication  than the indication that they want to prescribe the drugs for. This is called off-label drug use and also comprises prescription of other dosages, other administration routes and prescription to other age groups than those where the drug was initially registered for. 
For example, if a drug is approved for lung cancer and the drug is now also being tested in ovarian cancer, there might be a good rationale to prescribe the drug on an off-label basis to a patient that suffers from ovarian cancer and does not qualify for the clinical trial. 

Off-label drug use is  in general possible subject to strict conditions  unless there are serious ethical or safety objections.


 

Expert centers 

A last opportunity often represents the close contact with expert centers. 
One way to identify expert centers is by analyzing how many clinical trials these centers run for a certain condition. The expert centers can be ranked by the  number of clinical trials for a certain disease.
Chances are that the doctors of the highest ranked expert center for prostate cancer have a lot of experience, not only with treating the disease, but are also very familiar with clinical trials that are upcoming and possibly even other options. 

 


At myTomorrows we believe  treatment options don’t end with approved medicines  and that it’s important that all physicians and their patients with high unmet medical needs are informed about these options. If you have any questions, please get in contact with our medical team