Tag Archives: Health

Guess who invented the Target Product Profiles…

By Itziar Escudero, Ph.D., M.B.A. Partner at Insights in Life Sciences (ilS).

A Target Product Profile (TPP) is a planning tool for therapeutic candidates based on FDA guidelines.

The TPP defines the minimal and ideal profile of the final marketed product and shows the ultimate goals of the proposed therapy development effort such as disease indication, patient population, delivery mode, treatment duration, treatment regimen, and standards for clinical efficacy.

Target-product-profile-template (1)

Fig1. Standard TPP (you can download a template here)

The TPP concept was used for the first time more than 20 years ago (in 1997), when FDA representatives and pharmaceutical sponsors were looking for ways to improve sponsor and FDA interactions during the drug development process.

The purpose of a TPP is to provide a format for discussions between a sponsor and the FDA that can be used throughout the drug development process, from pre-investigational new drug application (pre-IND) or investigational new drug application (IND) phases of drug development through postmarketing programs to pursue new indications.

The TPP embodies the notion of beginning with the goal in mind. That is, the sponsor specifies the labeling concepts that are the goals of the drug development program, documents the specific studies intended to support the labeling concepts, and then uses the TPP to assist in a constructive dialogue with the FDA.

The use of the TPP has evolved and nowadays does not only facilitate the dialogue between the sponsor and the FDA, but with other stakeholders (such as physicians and payers) as well.

In fact, at ilS we help Companies assess how those stakeholders (physicians and payers) “react” to the different TPP presented, including the minimally acceptable and the desired scenarios. For this purpose we count with a network of life sciences experts (ilS NetworkSM) from all over the world, including physicians and payers, with whom we conduct in-depth phone interviews and collect their perspective around the TPP.

Ideally, the TPP provides a statement of the overall intent of the drug development program, and gives information about the drug at a particular time in development. Usually, the TPP is organized according to the key sections in the drug labeling and links drug development activities to specific concepts intended for inclusion in the drug labeling.

The TPP is a dynamic summary that changes as knowledge of the drug increases. For optimal use, it is recommended to update regularly the TPP to reflect new information about the product.

Early TPPs can be brief depending on the status of the sponsor’s development process.

A well-organized TPP is also useful for updating quickly sponsor personnel who join the program for the first time.

The is part of the proprietary IND file. Nevertheless, a TPP does not represent an implicit or explicit obligation on the sponsor’s part to pursue all stated goals.

According to the FDA, sponsors have seen advantages of using a TPP at meetings early in the drug development process. Use of a TPP can facilitate the efficiency of sponsor-FDA interactions and communications. A TPP can help address issues early on in the drug development process thereby preventing late stage drug development failures and decreasing the total time involved with drug development.

In the same way, we at ilS, have seen advantages of using TPPs for Companies (sponsors) that seek stakeholder input. This tool allows physicians and payers to comment on the product profile in an organized manner and to evaluate different scenarios. We have observed that this tool is useful not only in later stages of drug development, but also in early stages where there is still so much room for change for the sponsors.

Interestingly enough, we have also found this tool useful for evaluating in vitro diagnostics (IVD) devices, adjusting the TPP template.

References:

https://www.ninds.nih.gov/Funding/Apply-Funding/Application-Support-Library/CREATE-Bio-Example-Target-Product-Profile-TPP

https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm080593.pdf.

The CBER Office of Cellular, Tissue and Gene Therapies (OCTGT) web page for industry education also has a Webinar on TPP

 

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Personalized Medicine perspectives in the United States

By Alexandria Kyle-Hammer

p3The use of one’s genetic profile to inform and guide the diagnosis, treatment and even prevention of disease in individuals (i.e. personalized medicine),1  has made great strides over the last decade and is becoming increasingly popular and common as a treatment tool around the world. Although its use applies to the treatment of many diseases in various fields of medicine, it is worth noting that particularly in oncology, the role of personalized medicine has been revolutionary.

A recent Nature Reviews article, Defining and Quantifying the Use of Personalized Medicines2 , where the authors use the following definition for personalized medicine, 1) “A medicine that has a US Food and Drug Administration (FDA) or a European Medicines Agency (EMA) label stating that its choice as a treatment must be governed by results from a companion diagnostic test” 2) “A medicine that has a label that recommends (but does not require) the companion diagnostic, and at least one authoritative professional organization also recommends use of the test to guide treatment”2 , analyzes the growth in the use of personalized medicine from 1998 to 2009.

When personalized medicine first began to be used in the 1990´s the United States quickly became the world leader in the per capita use of biological personalized medicine. However by 2007 the US was overtaken by the EU, specifically the 5 major markets of France, Germany, Italy, Spain and the United Kingdom (the EU5), and by 2009 the United States had also been passed by Japan. By the end of 2009 the U.S. market was 25% smaller than that of the EU5. In 2011 and 2012 only 8 out of the 69 new molecular entities (NME) approved by the FDA were personalized medicines (i.e. 11.5% of the NMEs).

With the great potential and many befits of personalized medicine it is important for the U.S. not to fall farther behind in this field. The article proposes that by studying over time the various geographical regions where the use of personalized medicine has been the most successful we may be able to draw conclusions about the policies and healthcare systems used that might be most useful in supporting the further adaptation of personalized medicine in the United States.

The article also offers several other interesting findings. One being that the majority of the usage and sales of personalized medicine has been focused in the area of Oncology, suggesting a great unmet need in the field. Many older drugs that were not previously used as personalized medicine have since been converted into personalized medicines, as it has become clear that they are more effective when used as such. An example of this is the drug Tamoxifen, which was commonly given to women with ER+ breast cancer. It became a personalized medicine when it was discovered that 65% of women taking the drug developed resistance due to a mutation in their CYP2D6 gene. Now women are genotyped for that specific mutation so that the right treatment is given to each breast cancer patient. This conversion of medicines already on the market to personalized medicines has impacted the growth of the field.

Although the overall use of personalized medicine is growing at a 22% Average Annual Growth Rate (AAGR) globally between 1998 and 2009, the United States is lagging behind when compared to Japan and the EU5.

The FDA and the US National Institutes of Health (NIH) have promised to invest heavily in the field to support the growth of personalized medicine and the effort to make it a reality in the USA. As part of this effort the FDA has released a set of guidelines to regulate the field of personalized medicine. Within the Agency they have also created a Personalized Medicine Staff (http://goo.gl/7FCkcr) dedicated to “addressing the opportunities and challenges associated with diagnostics used in personalized medicine”3.

While these efforts, along with others, are a step in the right direction the progress of personalized medicine in the US is still slow. Further investigation is needed to identify the essential driving factors of the growth of personalized medicine in order for it to reach its full potential as a treatment method. It is also possible that more “public funding for translational research, greater use of electronic medical records to better access patients’ test information, and incentives for developers to personalize both approved and investigational therapeutics”4 are needed to further drive the advancement of personalize medicine.

1 http://ghr.nlm.nih.gov/glossary=personalizedmedicine
2 Hu, Sean X., Murray L. Aitken, Arnold L. Epstein, Mark R. Trusheim, and Ernest R. Berndt. “Defining and Quantifying the Use of Personalized Medicines.” Nature Reviews Volume 12 (2013): 896-97. 1 Dec. 2013. Web.
3 http://www.fda.gov/downloads/scienceresearch/specialtopics/personalizedmedicine/
ucm372421.pdf
4 See footnote 2