Tag Archives: Focus Group

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

 

Advertisements

Consensus building: The Delphi Method and the Nominal Group Technique

By Aina Pi, M.A., Consultant at Insights in Life Sciences (ilS).

p4_newsletterjpg

Two of the best known methodologies to achieve consensus of opinion in expert panels or workshops are the Delphi Method and the Nominal Group Technique.

Both consensus techniques, widely used in the field of health, are a mean of collecting experts’ opinions where limited or conflicting evidence exists, and an approach of guiding health decision-making.

In practice, the Delphi method and the nominal group technique are frequently implemented to reach agreement on the classification of diagnostic criteria, the development of clinical guidelines, the identification of health professionals’ needs, as well as to orientate research.

The methodological manual for the development of Clinical Practices guidelines of the Spanish Ministry of Health1 states that these methods are particularly useful specifically if a general agreement on the formulation of recommendations is required: when there is a lack of scientific evidence, existing evidence is disputed, or when there is a little risk-benefit balance.

The Delphi method and the nominal group technique are described below:

Delphi method:

  • A Delphi study works as follow: An open-ended questionnaire about a particular topic is shared for an initial round of independent opinions from isolated respondents. The full range of opinions are summarized and distributed for several rounds of independent opinion and ranking, until a high degree of consensus is reached. Delphi studies usually consist of three to five rounds.
  • The Delphi method minimizes the influence of individuals and maximizes the reliability of results
  • The anonymity of the procedure allows to easily raise controversial issues
  • Since the exchange of information takes place via written documentation, no interactive discussion occurs. Nevertheless, the Delphi method can be adjusted for face-to-face meetings. In this regard, we have developed a “Delphi-like” methodology at ilS that counts with previous Interviews or Surveys, followed by Expert panels, known as ilS Knowledge(SM).
  • The Delphi method is very useful for international research since there is no need to gather all experts in the same location
  • The Delphi method requires an exhaustive prepared questionnaire, adequate time and high participant motivation since it involves a considerable workload for participants

The following case study, allows to gain a better understanding of how the Delphi method works:

Case study: Rationales behind the choice of administration form with fentanyl: Delphi survey among Danish general practitioners (GPs)2

  • The objective was to describe the rationale behind the choice of fentanyl (often used in the management of chronic pain) administration forms among Danish GPs.
  • An expert panel of 33 GPs collaborated in the study. In the first round, they wrote the main reasons for prescribing and not prescribing fentanyl patches, oral transmucosal systems, and nasal sprays. Their answers were summarized, and in two additional rounds of consultation, they were required to rate the importance of each reason.
  • As a result, a ranking of the most important rationales behind the choice of fentanyl administration form was obtained.

Nominal Group Technique:

  • The nominal group technique methodology works as follow: Participants meet in a session and complete a first round of independent opinions in writing about a particular topic along with its rationale. The results are summarized and distributed to participants. With the support of a moderator, participants expose and discuss their opinions for clarification and evaluation. Several rounds of independent opinions and ranking may be conducted. When the final round of ratings is carried out, the highest total positions are selected as the final decisions. Nominal Groups studies usually consist of four to five rounds
  • The Nominal Groups technique maximizes the compilation of information since all individuals inputs are considered, preventing people from dominating the discussion and encouraging minority views to be shared
  • It is a particularly useful technique since it gathers all experts at the same time and location, being a cost-effective and time efficient method
  • The Nominal Groups method requires an accurate pre-planning from the moderator and experts, and availability from experts since they are assembled together in a single session.

 

The following case study, allows to gain a better understanding of how the Nominal Group Technique works:

Case study: Identifying areas to improve pain management in hospitalized patients3

  • The objective was to identify the areas to prioritize interventions in the pain management for inpatients in a hospital from the USA
  • A multidisciplinary group of 27 health professionals participated in study.
  • In the first round, 94 ideas were generated. General and context-specific priority ideas were summarized and grouped into several categories. In the following rounds, participants were required to rate the importance of each idea.
  • Consequently, a ranking of the main priorities to improve pain control among hospitalized patients was set.

If you would like to gain further knowledge, you can explore the following case studies:

  • Centre for Disease Control and Prevention. “Gaining Consensus Among Stakeholders Through the Nominal Group Technique” Department of Health and Human Services 7 (2006): 1-3.
  • Davies et al., 2011S. Davies, P.S. Romano, E.M. Schmidt, E. Schultz, J.J. Geppert, K.M. McDonald. “Assessment of a Novel Hybrid Delphi and Nominal Groups Technique to Evaluate Quality Indicators. Health Services Research”, 46 (6pt1) (2011). 2005–2018.
  • Van E, Pitchforth T, Bishop C, Russell E. “Delphi method and nominal group techniques in family planning and reproductive health research”. J Family Planning Reprod Health Care (2006);32:4:249-252.

At ilS we are specialized in Primary Research in the Life Sciences field and we use both techniques for consensus building: the Delphi Method and the Nominal Group Technique while moderating Expert Panels. Based on our own experience accumulated during over 5 years, we have adapted those methodologies in order to make sure consensus is achieved in the most efficient manner for our Clients.

____

REFERENCES

1Manual metodológico de elaboración de guías de práctica clínica en el Sistema Nacional de Salud. MSPS, 2007. Link

2Jacobsen R, Møldrup C, Christrup L. “Rationales behind the choice of administration form with fentanyl: Delphi survey among Danish general practitioners” Journal Opioid Management (2010);6(4):259-68.

3Peña A, Estrada CA, Soniat D, Taylor B, Burton M. “Nominal group technique: a brainstorming tool for identifying areas to improve pain management in hospitalized patients” J Hosp Med. 2012;7(5):416-20.