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Pharmacogenomics is the study of how genes affect drug responses in patients. Because pharmacogenomics is still in its infancy and U.S. healthcare systems have not yet adopted it, the financial burden often falls to patients to pay for genomic testing. However, as pharmacogenomic testing becomes more common, it is unclear who should finance these tests. Should the burden of paying for pharmacogenomics fall to the patient, or is this the responsibility of the government and healthcare payers? 

Who Should Pay for Pharmacogenomics?

According to Kate Merton, Head of Commercial Development at Navigate BioPharma, we should consider which group will benefit most from pharmacogenomics. At first glance, the patient appears to be the primary beneficiary of pharmacogenomics because they receive customizable treatment plans that account for their genetic background. Kate adds that the healthcare payer may also see a monetary benefit. This is because pharmacogenomics allows the payer to get patients on the most appropriate treatment plan much quicker—decreasing the overall cost of covering that patient. 

The Role of Early Adopters

Despite the monetary benefits that healthcare providers may receive, patients will be the ones paying out-of-pocket for pharmacogenomics, at least initially. This is because pharmacogenomics is still in its early stages and the healthcare system has not yet integrated genomics testing. This is to be expected, says Kate Merton, and early adopters are typically willing to pay for genomics tests. The motivation behind this is often curiosity—whether that be knowing more about their ethnic background or for personal health reasons. As pharmacogenomics continues to develop, Medicare or large employers may cover the costs of genomic testing—taking some financial burden away from patients.

Why Hasn’t the U.S. Adopted Pharmacogenomics?

One barrier facing U.S. adoption of pharmacogenomics is the lack of genetic data available to patients, providers, and healthcare payers. Compared to countries with socialized medicine, the U.S. does not have access to as many genetics and disease datasets, and cannot easily determine the cost benefit analysis of adopting pharmacogenomics. This means that the U.S. may not experience the same pharmacogenomic development that countries that offer socialized medicine may. This may mean that U.S. pharmaceutical companies will have to be more imaginative and driven to make sure the speed of their uptake matches international companies.

Although patients and healthcare payers may finance genomic testing, this may end up changing further down the line as the U.S. healthcare system adopts pharmacogenomic testing. As pharmacogenomic testing becomes more common, the financial burden may end up falling on pharmaceutical companies and healthcare payers—lowering costs for patients.

If you have any other questions about how to communicate with the FDA or how your past and/or current FDA communications affect you and your business goals, reach out to me on Twitter, LinkedIn, or send me a message here.

I also host a podcast called DarshanTalks, a show that discusses newsworthy FDA issues and how they apply to bringing a product to market – and keeping it there. From patient centricity in clinical trials to the government shutdown to CRISPR and bioet...

4Pharmacogenomics: Changes in Drug Development

Pharmacogenomics is the study of how genes affect a person’s response to drugs.  As the field grows, it holds many implications for the future of drug development.  Pharmaceutical companies are now rethinking the drug development process and coming up with new approaches to the discovery, development, and marketing of drugs.  The growth of pharmacogenomics may also force the FDA to rethink its regulatory guidelines, particularly as companies change the way they develop new drugs.

Will Pharmacogenomics Impact FDA Guidelines?

One of the primary issues facing pharmacogenomics in drug development is the outdated drug template being used by pharmaceutical companies.  The traditional way of thinking about drug development is by using three separate phases.  Each of these phases shows different regulations that a product must meet before it can move onto the next phase.  The problem with this template is that it does not account for the genetic variability of subjects who are receiving medication.  With new gains in pharmacogenomics, however, this variability will need to factor into the development process. Introducing pharmacogenomics will increase the number of viable drug targets and decrease risks associated with development.  Because there are new factors involved in drug development, the old way of conceptualizing drug development may no longer apply.  

According to Kate Merton, Head of Commercial Development at Navigate BioPharma, the FDA will need to change its regulatory guidelines to reflect the changing landscape in drug development.  This may mean weighing in on the early research process and identifying targets and patient populations. The FDA will also have to account for new genomic technologies being used by companies to develop their drugs.  

Finding the Target Population

As information pertaining to population and genetics becomes more readily available, companies will more easily identify the causes of certain diseases.  It will also be much easier for pharmaceutical companies to find target populations that will be receptive to the drug they are developing. For example, a company may determine that its drug will not work in 45% of the target population.  This will allow the company to focus instead on the group that the drug will work in. Narrowing down the target population through genomic technologies will allow pharmaceutical companies to save their resources and develop the right drug for the right group at the very beginning.

How Is Pharmacogenomics Incorporated?

Incorporating pharmacogenomics into the drug development process begins with looking at population and disease area.  Disease areas including oncology, cardiovascular, and diabetes have been well-developed and researched. This makes it a good starting place for companies to look for correlations between genes and drug response, and may point them in the right direction.

As pharmacogenomics continues to change the shape of drug development, the FDA may reconsider its regulatory guidelines surrounding drug development.  Pharmaceutical companies should stay informed about changing regulations and consult with a lawyer with a background in FDA regulatory law to ensure compliance.

Pharmacogenomics has the potential to allow physicians to personalize treatment plans for their patients and make more informed decisions about patient health.  Despite the many benefits offered by pharmacogenomics, there are several concerns being raised within the healthcare industry. Among these concerns is the actual value pharmacogenomics provides patients and the likelihood that genetic testing will raise healthcare costs.  This post will examine the impact of pharmacogenomics on physicians and the patients they treat.

Does Pharmacogenomics Help Physicians?

The study of pharmacogenomics reveals how an individual’s genes influence his or her response to drugs.  According to Kate Merton, Head of Commercial Development at Navigate BioPharma, genetic information is valuable to physicians because it allows them to make a better initial decisions about treatment plans.  For instance, if a certain patient is a high metabolizer, genetic testing would alert physicians. This would allow physicians to change the dosage that the high metabolizing patient is receiving so it meets their individual needs.  This approach offers an alternative to the traditional method of treating a patient, which is to prescribe a standard dosage and then either escalate or de-escalate the drug dose depending on the patient’s response. Pharmacogenomics instead allows the physician to take information about the patient’s genetic composition and tailor treatments accordingly.

How Will Patients Benefit?

Although pharmacogenomics may not apply in all treatment decisions, it is highly important when testing for genetic abnormalities and in oncology.  When prescribing medication, it is important to ensure that genetic abnormalities are not present, which may interfere with the medication being prescribed.  This information helps rule out patients who may have adverse reactions to treatments, which may not be compatible with their genetic background.  Pharmacogenomics also proves highly important in oncology, where time is often limited and getting patients on the right drug is critical for recovery.  In these cases, genetic testing allows physicians to make better decisions regarding treatment plans for patients.

Is Pharmacogenomics Cost-Effective?

Despite the common belief that pharmacogenomics may lead to higher healthcare costs, this does not appear to be the case according to several economic studies conducted on the subject.  A recently published article by M. Verbelen in The Pharmacogenomics Journal reveals that pharmacogenomics-guided treatment is not only cost-effective, but proves dominant over alternative strategies.  The study examined economic evaluations and determined that if genetic information was freely available, as many as “75% of economic evaluations would support pharmacogenomics-guided treatment.”  This finding shows that the benefits of pharmacogenomics may outweigh the cons, particularly if the cost of healthcare remains unchanged. There may even be economic benefits as pharmacogenomics allows physicians to make better decisions when choosing pharmaceutical treatments.

As pharmacogenomics continues to be integrated into healthcare, it allows both physicians to make more informed decisions about treatment plans for their patients.  This means that pharmaceutical treatments are becoming increasingly personalized--representing a drastic departure from the standardized ...

Venture capital has occupied an important role in the US healthcare system, particularly as hospitals rely upon the capital to develop and test new technologies. The growth of venture capital-backed companies has allowed for hundreds of startups developing health technologies to thrive. As venture capital businesses become integrated into hospitals and the healthcare system at large, it becomes important to understand the critical role venture capital plays in health systems.

Creating Revenue Streams

Venture capital businesses allow hospitals to “create new revenue streams,” according to Neil Wyant, Managing Director of UH Ventures. What this means is that hospitals can invest in “internal ventures,” or internal ideas and resources, to establish new businesses. These business opportunities can range anywhere from specialty pharmacy to home care. Depending on the estimated value of the business, venture capital may bring outside resources to help with the development process. 

Venture capital businesses do not always rely on internal ventures; however—in certain cases, they may instead seek outside ventures. Neil, an early stage investor, added that he looks for outside opportunities to invest which he can add “strategic value” to. This may mean helping an outside company as a customer, clinical trial partner, or pilot site. This allows hospital investors to speed up the growth of the company because they have unique insights as customers.

Finding your Hospital Venture Capital

One of the primary challenges that investors must deal with is finding an internal champion that will allow them to develop strong connections with clinicians and hospital executives. The most efficient way to find a hospital venture capital is to begin by finding an internal champion. An internal champion is a person within the organization who will vouch for the company or business venture in question. Internal champions can coordinate meetings with busy executives or physicians who may otherwise not take an interest in the opportunity or company being promoted. 

Investors will often turn to an outside company for help if they cannot find an internal champion. Because an outside company is not likely to have internal connections that prove useful, this is not ideal. It is preferable for investors to find their own internal champion within the hospital. To do so, they must often sift through and qualify opportunities that internal champions propose to them.

How Should Physicians Gain Venture Capital?

Gaining venture capital for a product or technology can be broken down into three main components. Physicians should first get in contact with a venture capital firm and ensure that their intellectual property is protected. Once they have done this, the investor will then obtain venture disclosure for the idea and figure out how best to protect it, according to one investor. The final steps involve figuring out the potential market value, determining the commercialization staff, and qualifying the idea. 

The entire process of getting venture capital and developing a business around a new idea can be aided by turning to an early stage investor such as UH Ventures. Consulting with a lawyer may also be a good idea, especially with negotiating a deal right for your company.

The emergence in genetic research has generated considerable interest in the role that genetics plays on individual drug responses. Genetic research gathered by companies such as 23andMe has allowed scientists to map out the genetic variation among individuals, and use this information to identify potential biomarkers for disease, drug response, and adverse reactions. This link between genomics and pharmacy is called “pharmacogenomics,” and it has allowed clinicians to prescribe medications which are safer and more efficacious for patients based on their genetic make-up. Although pharmacogenomics is very promising for the clinical world, it is arguably still in its infancy and cannot be relied upon to make a diagnosis or prescription. 

What Benefits Does Pharmacogenomics Offer?

According to one clinician, pharmacogenomics has allowed for new ways of prescribing drugs to patients. Often, certain genetic mutations or deletions will harmfully interact with medication to produce toxicities. By testing the patient’s genotype, however, the clinician can avoid prescribing a medication that could prove dangerous to patients with certain genotypes. One example of this is when testing for the CYP2D6 gene, which allows people to metabolize drugs more rapidly. Clinicians can adjust their drug dose accordingly for people who have few CYP2D6 genes and who may not be capable of metabolizing the drug as quickly as other individuals. Pharmacogenomic testing allows clinicians to tailor their prescriptions to ensure that their treatments meet the patient’s needs on a personalized level. The FDA has recommended genotyping before prescription, particularly because it allows clinicians to choose the safest, most efficacious treatment option for their patients. Although pharmacogenomics allow for more personalized medicine, it may be too early to rely on genotype testing to diagnose diseases or choose medications.

Is Pharmacogenomics Viable in Clinical Practice?

There are many complicating factors that make pharmacogenomics unreliable in a clinical setting. Although pharmacogenomics offers insight into a patient’s predisposition for certain reactions to drugs, there are many environmental factors which make it difficult to establish a clear relationship between genetics and drug response. These factors can include the interaction of other disease states, drug interactions, and gene-environment interactions. This represents a complex interplay of different variables and makes it difficult to pinpoint exactly how a patient might react to a drug based on their genetics. In fact, one study has shown that only 10% to 15% of genetic biomarkers have a direct impact on drug response. This means that pharmacogenomics does not offer clinicians a way to choose treatment plans based on a patient’s genotype. 

What Is the Overall Value of Pharmacogenomics?

Despite the overall promise that pharmacogenomics holds in areas of drug development, it remains less viable in a clinical practice. This is because there are many factors influencing the relationship between genetics and a patient’s reaction to drugs—making pharmacogenomics difficult to rely upon in a clinical setting. 

At the moment, it does not appear pharmacogenomics can translate smoothly into clinical practice because of the scientific barriers it faces. Despite these barriers,

Although developments in pharmacogenomics have arguably led to an improvement in the efficacy and safety of medicine, there are several ethical and social issues that are being raised.  Pharmacogenomics is defined as the study of how genetic variation affects the way an individual responds to medications. This allows for doctors and pharmacists to select for medicines that are well-suited to patients depending on their genetic composition. Despite the benefits of pharmacogenomics, genetic research suffers from racial inequity and lack of informed consent. Clinicians and drug companies must consider these problems when using genetic data and taking part in pharmacogenomics.

How Does Genetic Testing Affect Data Collection?

The data collection being performed through genetic testing by companies like 23andMe has been proven to be skewed, particularly as the people who undergo the testing are predominantly Caucasian. In fact, as reported by 23andMe, Africans, African-Americans, and Latinos make up less than four percent of individuals included in genome-wide association studies. This represents a racial bias in the data being collected by 23andMe and other DNA-testing companies, which may affect the future of drug development. As drug companies use the data collected by DNA-testing companies to develop new drugs, there is the danger that these drugs will only treat the group that is most largely represented in the genetic data (in this case, the Caucasian population). This means that under-represented groups could be neglected, as the new drugs being developed rely on genetic data which excludes them. Clinicians should be wary when tailoring drug prescriptions to individual patients and keep in mind that the genetic data being collected does not represent all racial populations equally. 

Is Informed Consent Being Obtained?

Another ethical concern surrounding pharmacogenomics is informed consent. Informed consent is getting an individual’s permission before using their personal information. With genetic testing, this means that DNA companies disclose what they will do with their customers’ data, and must provide forms for their customers to sign stating how their genetic data will be used. Unfortunately, these forms are often not readily understandable for the average consumer—they are often lengthy and not straightforward. Consumers also rarely read through the terms and conditions provided on these forms, suggesting that genetic research companies often have not received the full consent from their testing population.

How Should Companies Address These Ethical Problems?

The responsibility of addressing these ethical problems falls to the companies conducting genetic research or developing drugs. To ensure that minority populations are being included in genetic data collection, companies should consider reaching out to these groups themselves to diversify their data. Perhaps the genetic data these companies are collecting is not completely validated. As a result, perhaps this data should not be collected because patients may end up making treatment decisions based on that information. 

Yet another consideration that companies should take into account is how transparent they are being with patient disclosure. Companies should investigate how their terms and conditions are when conveying to their customers how their genetic data will be used. Improving these forms would mean that a higher percentage of ...

Biohacking is often described as do-it-yourself biology. People in the community have begun injecting themselves with untested gene therapies. What are the risks?

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Disclaimers:

1. This discussion is merely an oral discussion and should not be relied upon solely on its own to support any conclusion of law or fact.

2. The discussion does not and should not reflect any individual products status as safe, efficacious, adulterated or misbranded or meeting or not meeting expectations at a local, state, federal, or international agency or organization.

3. The discussion should not be construed to be complete advice that is right for you and may not necessarily represent a specific product.

4. This discussion is provided for general educational purposes and should not be construed as legal advice, regulatory advice or medical advice.

5. This does not create an attorney-client relationship

​Biohacking is described as citizen or do-it-yourself biology. This can mean individuals changing their bodies using technology, taking dietary supplements, or increasing their amount of exercise. However, some have taken it to the next level and are incorporating technology or cellular modification based approaches to health. This was the case for Josiah Zayner, a “biohacker” who livestreamed himself injecting DNA for the genetic engineering tool CRISPR directly into his arm. Biohacking is an exciting field because it is not heavily regulated by the FDA, but this also means more risks involved for individuals who take part in it.

The Values of Biohacking

Biohacking offers people the chance to explore cybernetics and play with the intersections between technology and physiology. Biohacking also draws many to the field by the control it gives them over their personal data. Amid privacy disputes between big corporations and the public, biohacking offers an alternative way for the public to monitor and control their personal data. This method of control is conducted by the individual–making it ideal for people concerned about their privacy.

Biohacking also allows for open source medicine, in which anybody can contribute to the field of medicine not just scientists and researchers who have received adequate funding. This allows for people to develop their own medicines without having to rely solely on the pharmaceutical industry.

Who is Doing it?

Biohacking experiments have ranged from the practical to the absurd. Biohackers have modified insulin pumps so the pump will deliver a specific amount of insulin to the body, are attempting to identify negligent pet owners who don’t pick up after their pet by scanning the pet’s feces, and injecting untested experimental gene therapies into their bodies. Well-educated professionals appear to be the largest demographic to perform these experiments, and most biohackers have received college degrees. Most people performing these experiments are professional scientists, meaning that they are more likely to experiment on their bodies in a safe and controlled environment.

The Bayh-Dole Act

It is important to consider the various implications of biohacking before beginning to experiment. If you have an employment agreement, for instance, your scientific findings may end up being the property of your employer. Government-funded professionals should also be wary. The Bayh-Dole Act permits the government to use “march-in rights” to use patented products in a way that they deem appropriate.

If you have any other questions about how to communicate with the FDA or how your past and/or current FDA communications affect you and your business goals, reach out to me on Twitter, LinkedIn, or send me a message here.

I also host a podcast called DarshanTalks, a show that discusses newsworthy FDA issues and how they apply to bringing a product to market – and keeping it there. From patient centricity in clinical trials to the government shutdown to CRISPR and bioethics to why big data is doomed to fail in healthcare, we’ve got quite the list of topics to review! Listen to the podcast on

Registering with the FDA is necessary for facilities distributing or manufacturing dietary supplements, but often the process surrounding registration can seem unclear. What are some key factors that companies should keep in mind? In registering your dietary supplement with the FDA, companies must know: 

* Is a notification even necessary?* If so, what information must they submit to the FDA* What process is in place to facilitate registration?

When registering with the FDA is necessary

Registering with the FDA becomes necessary when companies introduce new dietary ingredients. If a company has a new dietary ingredient, they must submit a notification to the FDA informing them of the ingredient’s presence. The distributor or manufacturer using the new ingredient in their dietary supplement remains responsible for testing the safety of the dietary ingredient for potential users. 

What to submit

When submitting a report about a new dietary ingredient, the manufacturer or distributor must include information supporting the safety of the ingredient for users. This report needs to be provided at least 75 days before the dietary ingredient is introduced or delivered in interstate commerce before the marketing process begins. 

In the report, there must be mention of the company’s name and address and the name of the new dietary ingredient. It must also describe the dietary supplement containing the new ingredient. Additional details like conditions for use, history of use, and the company held responsible for determining its safety must be given. The FDA also requires a signature of the person overseeing the submission process.

Generally Recognized As Safe (GRAS)

Companies may come across what is known by the FDA as “GRAS,” or Generally Recognized As Safe. This is a substance that is regarded as being safe by qualified experts based on its similarities to pre-existing, FDA approved ingredients. The FDA has created a list including the consideration of chemically similar ingredients that are approved and already being used in foods. Companies can provide voluntary self-affirmations showing that the use of their substance is GRAS by meeting specific conditions. Under the law, this process of self-affirmed GRAS is considered being at the same level as a GRAS notification to the FDA.

Even if companies do not end up submitting reports to the FDA, there is still a need for them to understand the FDA regulatory process. This means notifying the FDA after introducing a new product to market and reviewing manufacturing requirements to ensure compliance. This may also require reaching out to compliance experts to develop a quality or hazard plan. Consult with an attorney to ensure that regulations are being properly followed.

If you have any other questions about how the opioid crisis and how you can help, reach out to me on Twitter, LinkedIn, or send me a message here.