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How FDA is preparing for wave of change to personalized medicine?
The cost of DNA sequencing is dropping continuously. In very future, majority of the population will have access to their genetic background and they will be able to take it their doctor and will expect to receive personalized treatment rather than a drug that has worked for millions of people but not guaranteed to work for them. Pharmaceutical industry will eventually have to change their business model and transition to personalized medicine. I am wondering how this impacts the regulatory affairs in pharmaceutical industry. How the FDA is preparing itself and evolving to address this trend of change to personalized medicine?
Some one from the FDA can no doubt answer this question.
Let's step back and verbalize what we anticipate the "personslized" medicine will actually look like in practice. I anticipate that the main change will be in terms of disease diagnosis, for example based on gene analysis determining what drug would be most effective for an individual patient. No drugs will specifically be made for individual patients. The current Precision Medicine Initiative does not include developing personalized precision drugs, hence the treatment will still use the existing therapies.
First of all we have to define the needs of personalized medicine in specific and only then it can be applied efficiantly.second, it should include building a personal profile of ones needs as for the all taken drugs inpot and address also the interaction infuance between all the drugs input because now it is very much can cause more harm then help if not taking this issue into consideration . - ORIT
I think that FDA will have time to decide in early stages of personalizing medicine, because the main thing that needs adjustment is the quantity of drug a person should take to be effective.
If some existing drug is not effective, the second approach should be changing the drug, combining two drugs or using enhancers.
In the future, and taking on account the evolution of CRISP-like methods and gene therapies, the framework should change to include this as a new genome driven medicine.
Juan Pablo Allocati
Reading this informative comment it occurred to me that the term "personalized medicine" is somewhat of an exageration. To some extent it matches the definition that designation as "personalized" "meets someone's individual requirements".
However, to be more exact, the "personalized" medicines in the current context simply add more definition, specification about what group of patients the drug should be used.
Every FDA-approved drug comes with a package insert that clearly describes its INDICATIONS AND USAGE. Adding to this information more specific description relating to the mode of drug action does not make it "personalized". It simply narrows the size of the population for which the drug may be effective...
The Personalized Medicine is just an ongoing practice fro FDA. I'm describing a briefly report to reply to the main question.
In 2016, for the third year in a row, personalized medicines accounted for more than 20 percent of all new
molecular entities (NMEs) approved by the U.S. Food and Drug Administration (FDA). FDA’s Center for Drug
Evaluation and Research (CDER) approved 22 NMEs, new drugs, agents or
therapeutic biologics, in 2016. Of the 22, the Personalized Medicine Coalition (PMC) classified six of them — more
than 25 percent — as personalized medicines, continuing a trend that PMC first documented in 2014 when it pointed out that nine of 41 NMEs approved that year are personalized medicines. The analysis underlines that nearly one of every four drugs the agency approved from 2014 to 2016 is a personalized medicine. That ratio is a sharp increase from 2005, when personalized medicines accounted for just 5 percent of NME approvals. When evaluating NMEs, PMC categorizes personalized medicines as those therapeutic products for which the label includes reference to specific biological markers, identified by diagnostic tools, that help guide decisions and/or procedures for their use in individual patients.
Newly Approved Medicines
The six personalized medicines approved in 2016 include:
- Rubraca (rucaparib) for the treatment of advanced ovarian cancer. The decision to use this product
is informed by the BRCA1/2 biomarker status in patients.
- Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy. The decision to use
this product is informed by the DMD mutation biomarker status in patients.
- Epclusa (sofosbuvir and velpatasvir) for the treatment of chronic hepatitis C infection. The decision
to use this product is informed by the HCV genotype status of the viral infection in patients.
- Tecentriq (atezolizumab) for the treatment of advanced or metastatic urothelial cancer and
metastatic non-small cell lung cancer. The decision to use this product is informed by PD-L1
expression levels in the tumors of patients.
- Venclexta (venetoclax) for the treatment of chronic lymphocytic leukemia. The decision to use this
product is informed by the chromosome 17p deletion biomarker status in patients.
- Zepatier (elbasvir and grazoprevir) for the treatment of chronic hepatitis C infection. The decision
to use this product is informed by the HCV genotype 1 and 4 biomarker status of the viral infection