Dr. Stephen M. Hahn Profile picture
Jul 29, 2018 22 tweets 8 min read Twitter logo Read on Twitter
For this weekend's #FDA #SUNDAYTWEETORIAL I’m going to talk about what FDA is doing to modernize clinical trials to reduce the cost of drug development, potentially lower drug costs and bring more safe, effective therapies to patients.
FDA’s mission is to both promote and protect public health. For medical products, this means ensuring that they are safe and effective for their intended use. Clinical trials are the primary vehicle for generating the evidence FDA needs to ensure that.
The traditional drug development paradigm calls for 3 clinical trial phases: A small phase 1 tests safety and dosing, phase 2 tests short-term side effects and effectiveness over a range of doses; and the large phase 3 assesses whether the product’s benefits outweigh its risks.
Trials are becoming more costly & complex to administer. It can make drug development more risky, uncertain, time consuming. Overly complex trials can also deter enrollment & delay completion studies so long that findings aren’t relevant for patient care bit.ly/2uQ6sjs
Adding to complexity is “endpoint creep”, with pharmaceutical companies spending $4-6 billion annually on non-core or exploratory endpoints that aren’t related to primary safety or efficacy endpoints. bit.ly/2LK73gb
The result: spiraling drug development costs. One estimate: Rising 145% to an inflation-adjusted $2.8 billion between 2003-2013, a pace that dwarfs other healthcare cost increases. Higher trial costs & complexity impacts market competition & drug pricing bit.ly/2uKax8W
A soon to be published #FDA study found that nearly 5% of novel drugs approved from 1991-2000 had a competitor within 2 years. But it took 5 more years to achieve the same level of competition for drugs approved from 2001-2010. And less competition can mean higher prices.
One reason for the longer development period: It can be difficult to run clinical trials with a second to market drug once therapy is available to treat an unmet need or rare disease. It’s becoming harder and harder to be second.
Modern approaches to designing and conducting clinical trials can address some of these challenges. #FDA is working across our medical product centers to facilitate innovative trial designs and patient-centered endpoints that can make clinical trials more rigorous and efficient.
We’ve been partnering with the nonprofit Clinical Trials Transformation Initiative on the design and conduct of clinical trials that can address these challenges while building on our gold standard. We will not compromise that gold standard one bit. bit.ly/2Ljkocc
And FDA has encouraged several pioneering advances in clinical trial design, including Master Clinical Trial Protocols (MAPs), first used in cancer and now used more widely across FDA, in other therapeutic areas. bit.ly/2JX0pyk.
One MAP, I-SPY 2, compares up to 12 experimental therapies in subgroups of patients with metastatic breast cancer with 10 distinct biomarker signatures. This hub and spoke design uses a common control, reducing the overall # of patients to be recruited and enrolled.
Seamless trials, used in oncology for drugs that might qualify for breakthrough therapy designation, are another novel approach bit.ly/2NQy52K. They compress the traditional 3 trial phases into one large, continuous trial that may be able to speed access for patients.
A lot of time and cost is spent between the starting and stopping of the traditional 3 phases. Continuous trials can save time, lower costs and reduce the number of patients. Look for new guidance soon from #FDA on how developers can conduct these “expansion” trials
Surrogate endpoints are another clinical trial modernization tool. These endpoints are often used when clinical outcomes take a long time to study or when the surrogate’s ability to predict clinical benefit, such as reducing the HIV viral load, is well known.
In cancer, innovative endpoints are facilitating the development of products for both accelerated and traditional approvals putting promising treatments into the hands of high-risk patient years before they would’ve been available using previous endpoints.
FDA just published a list of surrogate endpoints which were the basis of approval or licensure of a drug or biologic product under accelerated or traditional approval bit.ly/2uPQLJ3. It provides useful information for drug developers on endpoints that may be considered.
Although I’ve been focusing on modernizing drug clinical trials, we have efforts underway to modernize medical device clinical trials as well, in part through work we are doing with the Medical Device Innovation Consortium (MDIC) on “virtual patients.”
MDIC is using computer modeling and simulation to create virtual patients. If validated to be like real patients, future device trials may be able to rely on virtual patient information when appropriate, lessening the burden of enrolling real patients and improving on our rigor
FDA is also looking to real world data collected from device registries and electronic health records to answer important premarket and post market questions potentially allowing sponsors to meet evidence needs in less time, at lower cost.
Our aim is simple: innovative, advanced evidence generation to assure the timely availability of safe and effective therapies. Our aim is to improve patient care, and give people the assurance of #FDA's gold standard for safety and efficacy in the most efficient way possible
We need to reduce the burden and cost of advancing care. And we need to make it easier for competition to reach the market once we have a promising new therapy. Our clinical trial advances will ensure that more patients have more safe and effective treatment options available.

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More from @SteveFDA

Sep 30, 2018
It’s the start of flu season – time to protect yourself and your family by getting vaccinated! In today’s #FDA #SUNDAYTWEETORIAL I’m going to talk about #FDA’s role in ensuring the safety, effectiveness, and availability of the 2019 influenza vaccines. #VaccinesWork #FightFlu
I know there are questions about the effectiveness and benefits of the flu vaccine given the severity of last year’s flu season, so I want to update you on what we’ve learned, the effectiveness of this year’s vaccine and why vaccination is important go.usa.gov/xPWhS.
We've made important modifications to this year's #Flu vaccine to improve its effectiveness; changing two of the strains (for the H3N2 and Influenza B components) to better reflect the influenza strains that we believe are most likely to circulate this winter. More on this later.
Read 22 tweets
Sep 9, 2018
Compounded drugs serve an important medical need for patients who can’t use an FDA-approved medication. In today’s #FDA #SUNDAYTWEETORIAL, I’m going to talk about what FDA is doing to preserve access to appropriately compounded drugs while protecting patients from serious risks.
In drug compounding, ingredients are combined, mixed or altered to create a sterile or non-sterile medication tailored to a patient - such as those with an allergy who need a drug without a certain dye or a child who can’t swallow a tablet and needs medicine in a liquid form.
But compounded drugs, which aren’t FDA-approved, may pose serious risks if not produced properly, potentially resulting in contamination or medications that do not possess the strength, quality or purity they are supposed to have bit.ly/2G7IpjB
Read 23 tweets
Sep 2, 2018
As kids are heading back to school, for this #FDA #SUNDAYTWEETORIAL I’m going to talk about MEASLES and the vital importance of vaccination. You may’ve seen recent news stories reporting cases of measles in parts of the US and EU. Most cases have occurred in unvaccinated people
In 2018 there have been 124 individual cases of measles in 22 states and D.C. Measles can spread when it reaches a community where there are groups of unvaccinated people. And travelers with measles continue to bring the disease into the United States go.usa.gov/xUzwa
Measles is caused by the rubeola virus and is highly contagious. It is normally passed through direct contact or through the air. The virus infects the respiratory tract, then spreads throughout the body, causing a skin rash, fever, cough and runny nose.
Read 19 tweets
Aug 19, 2018
Consumers want and expect safe, wholesome and affordable food. They deserve nothing less. In today’s #FDA #SUNDAYTWEETORIAL I’m talking about what steps the FDA, the states and produce farmers are taking - in collaboration - to ensure the safety of fresh fruits and vegetables.
We hear too often about food safety concerns involving fresh fruit and vegetables -most recently the outbreak of E. coli O157:H7 infections associated with romaine lettuce from the Yuma growing region, the largest such outbreak in the U.S. in more than 10 years.
From 1998-2008 there were 455 produce-associated food outbreaks, with more than 16,000 people becoming ill, more than twice the number of produce-associated illnesses from 1973-1997. This is a significant public health burden that should be largely preventable.
Read 25 tweets
Jul 30, 2018
THREAD ON NEW ENFORCEMENT ACTION: We’re taking action against manufacturers marketing “vaginal rejuvenation” devices to women by deceptively claiming the procedures treat conditions and symptoms related to menopause, urinary incontinence, & sexual function fda.gov/NewsEvents/New…
These products have serious risks and don’t have adequate evidence to support their use for these purposes. We’re deeply concerned women are being harmed by these procedures
In some cases, the devices are marketed to women who’ve completed treatment for breast cancer and experience symptoms caused by early menopause. Deceptive marketing of a dangerous procedure with no proven benefit, including to women who’ve been treated for cancer, is egregious.
Read 7 tweets
Jul 15, 2018
The topic of this week’s #FDA #SUNDAYTWEETORIAL is #drugshortages. FDA does everything possible within our authority to help address shortages. While we’ve made progress to mitigate shortages; we must do even more to understand and address the root causes of persistent shortages
Manufacturing & quality issues are some of the proximate causes of drug shortages. Availability of raw materials; unexpected increases in demand, i.e. due to illness outbreaks or changes in clinical practice; or unavailability of other drugs in a class also can lead to a shortage
We’ve seen # of new drug shortages steadily decline due to work by FDA, industry and others. In 2017, we worked w/ manufacturers to prevent 145 shortages. There were still 35 new shortages last year, but this compared to peak of 251 new shortages in ‘11 bit.ly/2JiNGpf
Read 17 tweets

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