OffTopic: The FDA Monitoring and Whistleblowing Scandal

Have you been following this issue?  It gets more and more fascinating the further you dive into it.  A excellent and definitive analysis can be found here, but for those of you too busy to read six small print pages, allow me to summarize:

The Setting:

Late in the 2^nd Bush Admin, at the Center for Devices and Radiological Health (CDRH)

The Baseline Concern:

Some first-line reviewers felt that computer-aided cancer screening diagnostics should not have been approved for use based on their interpretation of the risks associated with by-product radiation and their lack of confidence in the data within the application

The Baseline Grievance:

 The first-line reviews saw their recommendations overturned by their senior management, who granted approval.

The Work to Resolve the Grievance:

 The first-line reviews complained to Office of Special Counsel (OSC), OIG,  members of Congress and eventually to the New York Times about the situation.

OK, already a nightmare but at least fairly clear.  Government employees are permitted under the law to ignore certain confidentiality requirements if the safety of the public is at risk.  They are not, however, allowed to disclose non-safety related confidential information publically.  This is where the first line may have been crossed.

The NY Times Gets Involved:

The NY Times publishes an article describing the situation and including certain confidential information contained in the application and that may not have been directly related to safety concerns.

The Companies React:

The companies whose confidential information is now plastered over the front page of the NY Times demands that this stop.

 FDA Senior Management Reacts:

FDA senior management arranges to have the FDA-issued computers of a number of potential leakers secretly monitored – every website visit, every key stroke, every file downloaded.

Quick aside: to be fair to the FDA, every time an employee logs onto their FDA computer they are forced to read the following “You have no reasonable expectation of privacy regarding any communications or data transiting or stored on this information system….”  Seems to me its hard to argue that they did not have fair warning.

FDA Senior Management Finds the Leakers/Whistleblowers:

Some of those first-line responders are clearly found to be communicating with the NY Times and other media outlets.  They also find correspondence with Congress, OSC and OIG.

FDA Senior Management Terminates the Leakers/Whistleblowers:

Over the next couple of years the contracts of the leakers were not renewed.

Leakers/Whistleblowers Sue FDA for Wrongful Termination:

A lawsuit is, of course, already pending on this.

So far this is a really sad situation, but at least understandable.  Now, however, its all about to blow up.

FDA Contractor Screws Up Big Time:

An FDA document management vendor makes a world-class screw up – posting thousand of pages of information that was secretly captured from the computer monitoring on an unsecured website.  A terminated leaker/whistleblower discovers this accidentally while Googling her own name.

Everyone Blows a Gasket:

With the documents revealed, all sorts of groups blow a gasket.  The leakers/whistleblowers claim their rights were infringed.  Congress is furious because some of the communication that was monitored included communication to Congress.  The OSC and the OIG are very upset because some of the communication was to them.  Neither Congress nor OSC/OIG communications are supposed to be monitored.

And this is where we are today.

Frankly, this whole situation is a major fiasco.  It makes the FDA look like the most dysfunctional organization in the government, and that is saying something.  Clearly CDRH needs to be stripped down to the floorboards and rebuilt with a functional culture.

Why am I taking a “blame everyone” perspective instead of supporting the leakers/whistleblowers.  Normally that would be where my heart was at, but it was just released that those same leakers/whistleblowers had filed a secret lawsuit in court against the manufacturers whose products they felt were unsafe.  And this lawsuit was filed while those leakers/whistleblowers were still employed by the FDA reviewing exactly these products.  And those same leakers/whistleblowers demanded at least 15% of what would be a multi-billion dollar settlement.  Not what you would consider an impartial set of reviewers.

So, this is a pox on both houses situation.  We deserve and should expect better from the FDA.

What do you think?  Anything I missed?  Leave your comments below.

Topic 6: Measuring the Value of Medical Affairs

Introduction

A reader requested a discussion of different approaches for measuring the value of medical affairs.  This is a critical question and one that I touched on briefly when I discussed strategic planning in MA as it relates to launch.  But it is equally important overall for MA.

Like any function in pharma, MA must be able to communicate its value.  Value can be a tricky concept, however, for a non-commercial function like MA.  Value is ultimately a company-specific question.  It stems from the company achieving its strategic goals, which is why defining goals is so important.  Having said this, there are some general categories of value measures that we can discuss as well as some best practices around measuring value.

Value measures in MA typically fall into two types:

1. Objective measures


2. Subjective measures

Objective Measures

In MA, objective measures tend to focus around activity-based measurements.  Since MA cannot be seen as promotional, the other available objective measures like prescriptions written, sales numbers, profitability, etc. are not relevant.

Activity-based measures have both strengths and weaknesses.  On the strength side activity based measures are:

• Easy and cost efficient to gather


• Easy to explain to non-MA colleagues


• Non-controversial

Unfortunately, activity based measures also are:

• Not outcomes focused


• Not usually tied to the direct concerns of the organization


• Potentially misleading

While it may be interesting to know that the MSL group conducted 150 meetings with KOLs in the last quarter, it tends to beg the question – So What?  Activity-based measures tend to focus on the tactics (e.g. reach out to KOLs) rather than the goals (e.g. ensure broad awareness of the latest disease state information).

Activity-based measurement is very appropriate for certain MA functions.  For example, on CME funding, which by definition must be a hands off process, the only important measure is activity-based: Did the organization fund the number of CME programs it had intended to fund?

Or, in the Medical Information function, the key measures relate to answering inquiries and responding.  Activity-based measures (e.g. total number of inquiries managed, average turn-around on inquiries, number of inquiries requiring a second contact) are very appropriate for this type of service.

Finally, activities with multi-year goals like post-approval clinical trials, use activity-based measures to ensure that the overall program is on track.  So activity-based measures like patient recruitment and data collection provides valuable insight.

Activity-based measures are a necessary for MA’s ability to track progress against its goals, but they not sufficient to account for all of MA’s goals.

Subjective Measures

Most MA organizations have goals that require a more subjective type of measure.  Some of these goals relate to the degree of awareness or understanding in the healthcare community.  Measuring understanding or awareness is not as simple as measuring the number of discussions about a certain topic.  Instead, to measure progress on these types of goals, MA needs to gather more subjective data.  Simply put – it needs to ask.

For goals related to subjective measures, the only practical way to measure success is through research.  Unless a company is very lucky and finds that one of its objectives just happens to align with a topic that is already the subject of someone else’s research, this almost always means conduct primary research.

Research-based measures have their own strengths and weaknesses.  The strength of research-based measures include:

• Direct connection to goals related to healthcare community awareness and understanding


• Insight into the knowledge and beliefs of the healthcare community


• May provide insight into other, unmet needs

Research-based measures have some significant drawbacks, including:

• Expensive to conduct, leading to limited number of data points


• Subject to research bias


• May be taxing to the community that the organization is trying to serve

For research-based measures to be understandable, they usually need to be measured against a baseline.  For example, the measure of awareness of new disease-state information in the healthcare community after the efforts of the MA organization is only relevant if the measure of awareness before the efforts were known.  This requires the development of a baseline which both adds to the cost and requires good up-front planning.

But by far the biggest challenge of research-based metrics is the lack of experience and budget to conduct such research.  Many MA organizations do not have experience conducting this type of “market research”.  Without such experience, it can be daunting to initiate the research and structure it in the best manner.  Additionally, many MA organizations do not have money in the budget to conduct this research in a high quality manner.  This is both a product of the lack of experience and the view that this type of research is outside of the mission of medical affairs.

Best Practices

Clearly most MA organizations can benefit from a set of mixed objective and subjective measures.  Developing such a set of measures starts analyzing the strategy and goals outlined in the MA strategic plan.   From there a set of potential measures can be defined and prioritized.  Overall, MA should have between 5 and 10 metrics.  Specific functions within MA may have from 3 to 5 additional detailed metrics.  Once these metrics are defined, the approach for gathering each metric can be identified.  The activity-based metrics are typically gathered from existing tracking systems while the research-based metrics require more active management.

Once the research-based metrics are defined, a draft of a research outline can be developed, specifying the key questions, the research targets, the number of responses expected and the number of research samples to be taken.  This outline can be used as the basis for developing a request for proposal from a research company.

One caution – many organizations are tempted to combine this research with research already being conducted by the commercial market research function.  This may be a problem if the marketing questions are, in themselves, seen as promotional.  Most of my MA clients have been unwilling to risk this crossing of the line but some organizations do follow this practice.  Please work with your compliance function to discuss this approach.  Many organizations are not even comfortable with using research companies that are primarily commercially oriented but, frankly, there is little reason to avoid these companies.

After the approach to both activity- and research-based metrics is in place the next step is to determine how the results will be communicated and to whom.  This should become an ongoing process so it should be automated and standardized as much as possible.

Closing Thoughts

Explaining the value of MA remains one of the primary responsibilities of MA management.  For MA to be perceived as valuable to the organization, its progress against its strategies and goals has to be proven.

What has your experience been with value and measures in MA?  Leave your comments below.

Click on the email link to the right to suggest a topic for future posts.

Topic5: Relationship between MA and Managed Markets

Introduction

I received a question about what an optimal relationship should be between MA and Managed Markets in the US.  Managed Markets (MM) is the function in pharma that is primarily responsible for negotiating the relationship between the company and the major payers.  In some organization this group is known as Reimbursement, Access, Market Access, Payer Relationship, or Pricing.  The primary challenge is ensuring that the company’s drugs are listed as advantageously as possible on the formulary of the payer.  In the US, the MM function has had to deal with a range of both government and private payers, each with their own formularies.

The MM function in the US has grown in importance as the payers have worked to limit their exposure to potentially expensive treatments through formulary placements that drive limitations and co-payments for the patients.  Even specialty areas like oncology, which used to have very few restrictions, are now seeing greater control exerted by the payers.

Defining a New Relationship

Just as the pressures on MM are forcing changes in the way they work with payers, those same pressures are changing the relationship between MM and MA.  In the past, MA had a limited role to play in MM.  For example, MM might occasionally ask an MA resource, typically an MSL, to provide some scientific support for a formulary presentation.  But, in general, these situations were ad hoc and limited.

However, now that MM’s success more directly drives the success of the pharma company and thus their importance has grown, the relationship between MM and MA is changing.

For a formulary committee to control costs, they must be able to differentiate between more and less effective treatments, including the total cost of treatment.  This drives two major needs:

1. The formulary committee needs a more robust scientific understanding of the drug’s properties, its known efficacy, its known risks and its place in the overall therapeutic area’s treatment options


2. The formulary committee is demanding more specialized data, specifically health economics and outcomes research (HEOR) data, to allow them to understand the full impact of the drug’s use

Both of these ramped up requirements have direct impact on MA’s relationship with MM.

More Robust Scientific Understanding

MA’s role in terms of providing scientific support for formulary presentations is growing from a part of the presentation to the core of the presentation.  And with that growth comes the need for greater specialization by the presenters.

As the importance of the scientific presentations to formularies grows, the developer of those presentations and the presenters need to have a much more robust understanding of their audiences and how to effectively meet their needs.  This is leading to two trends in MA:

• Much greater degree of training for MSLs on the role of MM and formularies  

OR

• The identification and hiring of full time MSL-type roles specifically targeted at supporting MM                                                                 

Given the importance of MM, supporting their needs can no longer be seen by MA as a side responsibility.  Instead, it needs to be a core responsibility and an investment in training or personnel is needed to ensure that that the field force of MSLs is prepared to adequately support this need.

More Specialized Data

MA has typically taken the lead in developing data post-approval.  And while HEOR has always been an part of generating that post-approval data, its importance has grown significantly.  The increased demand for HEOR data has a number of implications:

• Data Generation Plans, which should be developed by MA to help drive the post-approval data efforts, must give greater consideration to the HEOR needs


• The priority given to Investigator Initiated Studies that cover HEO subjects may need to increase


• The need for specialized MA resources dedicated to developing and managing HEOR may need to increase, with new dedicated positions developed


• Processes for ensuring that the input of MM is gathered in the development of HEOR protocols should be re-examined to ensure that the results will meet the demands of the key formulary committees

In some companies have decided that HEOR is so important to MM that they have shifted the leadership of this research to the MM function itself.  Whether the HEOR function reports to MM or is developed within MA, the need to ensure that the needs of MM are addressed has become a major priority for post-approval research.

Closing Thoughts

MA’s role as the owner of scientific education and communication for post-approval drugs is a critical element in today’s formulary-driven environment.  MA needs to be an active partner to MM as it works to ensure patient access to the company’s drugs.

In your experience what has been the key to effective MA / MM partnership?  Leave your comments below.

If you have a topic you would like me to cover, please email me from the link to the right.

Authors Note:

Since I started this blog I have been focusing on major topics and posting three or more posts on those topics for your consideration.  I have received a couple of emails asking me to address more specific topics and as a result I will be adding new posts that address a single topic.

Topic4: Key Opinion Leaders – Managing KOL Interactions Efficiently

Identifying the right KOLs to focus on with the limited resources available is the critical first step and in the last post we discussed some alternate approaches.  Once those KOLs are identified, the next key to success is ensuring that the interactions with the KOLs produce the greatest value for both the KOLs and the pharmaceutical company.

Many organizations that invest in identifying KOLs, often fail to ensure that those KOLs are actually being well served by MA’s outreach efforts.  They provide their field force of MSLs with the names of the KOLs but very little direct direction.  The theory for this hands off approach is that the MSLs are expected to have a peer to peer interaction with the KOL and thus they want that interaction to guide the discussions.  While this is certainly the only way to discuss topics that are not suitable for proactive engagements (see this for further discussion on proactivity), for those topics that are suitable for proactive discussion it places an undue burden on the KOL to know what questions to ask.  In other words, KOLs don’t know what they don’t know and thus, when appropriate, benefit from the direct, proactive engagement of the MSL.

The only way to know how to provide the greatest value from direct engagement is to start by identifying the goals.  What need does the pharma company hope to support through its informational and educational outreach efforts?  The needs of the KOLs may be self-evident to the MA scientific interactions with the KOLs, but if it is not they will need to conduct some research to find out, which may be formal or informal.  Regardless of the approach, however, the needs should be defined by the KOLs and not by MA’s commercial colleagues in order to avoid any promotional bias.  The needs can then be expressed in terms of goals for MA’s outreach efforts.

With the goals in hand, a structured approach can be developed for these proactive outreach effort.  Plans can be developed on the type of outreach to be done, and materials developed to support that outreach.  And, once the plan is in place, progress against that plan can be tracked, measured and reported.

It is truly amazing to me how many MSLs lack a clear plan for their KOL engagement.  Operating without a plan denies the MSL the ability to measure their progress against a standard.  Operating without a plan denies their managers metrics they need to evaluate their performance.  And operating without plan makes justifying the investment in MSLs more difficult for MA leadership.

Building a detailed plan for each MSL that not only defines their KOLs but also details on a time-boxed basis the expected activities is a critical part of effective KOL engagement.  These plans can be in almost any form as long as they can be translated into direct measurement.

These measures should be as results oriented as possible.  Activity driven metrics may be easier but they rarely align directly with goals.  If the results sought relate to growth in understanding of a disease state, for example, then the measure may require some primary research.

Finally, those measurements need to be analyzed and fed back to the MSLs as well as their managers so that everyone can learn from the results of the outreach efforts.

Some of the vendors I mentioned in this post also support MSL plan management and tracking.  More about that in future posts.

Please share your experiences in the comments.

Legal Note:  All information and interpretations presented are only the opinion of the author(s) who are not lawyers. And, even if we were lawyers, given the wide range of interpretations of the current regulations you would still need to get the input from your own compliance organization. 

Topic 4: Key Opinion Leaders – Identifying the Right KOLs

The importance of KOLs in achieving medical affair’s goals was discussed in the Introduction.  Left open was the question of how are these KOLs identified.  There are a number of different approaches to identifying KOLs and in this post I will review those approaches and discuss their strengths and weaknesses.  I will also look into the vendors that can support KOL identification.Per

PERSONAL NETWORK

In the past, the standard approach for KOL identification was to hire good Medical Science Liaisons (MSLs) from various regions and rely on their Personal Networks to identify the right KOLs in their region.

This Personal Network approach has some strengths:

• It is very fast, since the MSLs typically already know the KOLs in their personal networks


• Good chance for strong access, since the MSLs typically target KOLs they have a existing relationship, they can often rapidly gain access to those KOLs for educational discussions


• It is inexpensive, since there are no additional costs from outside vendors

The Personal Network has some significant weaknesses:

• MSLs don’t know who they don’t know, some important KOLs may simply not show up on their radar because their personal networks do not cross


• MSLs are biased toward KOLs they have strong relationships who may or may not be the KOLs that are the most effective “hubs” of their formal and informal networks


• MSLs may specifically avoid “difficult” KOLs simply because they do not want to interact with them

The Personal Network approach is most effective in a very small, highly technical specialty medical community setting.  In these types of communities, since there are so few people involved, everyone knows everyone in the community and the risks of the Personal Network approach are mitigated.

SECONDARY RESEARCH

The next most common approach is the use of Secondary Research networks.  Secondary Research networks are developed by gathering information that is publically available about potential KOLs for secondary sources.  Information that is publically available may include:

• Name


• Degrees and institutions


• Specialties


• Institutional affiliation


• Authorship


• Conference presentations


• Principle investigator


• Committee involvement


• Board membership

The list can go on.  This information is collected; each piece is given a weight based on the what is perceived to be likely to contribute to their being a KOL.  The weighting produces a combined value which is then used to identify an individual as a KOL.

The Secondary Research approach has strengths:

• Fact based, eliminating personal bias found in the Personal Network approach


• Broad view, looking at everyone in the therapeutic area, not limited to individuals in a specific institution or with a specific background


• Less expensive than primary research, since the information in question is available in public databases

The Secondary Research approach has some weaknesses:

• Backward looking, since it relies on a preponderance of publically available information, it tends to favor those people who have contributed for the longest period of time


• Academic oriented, since it relies on public databases of information it tends to value those who publish and speak the most and those people tend to be academics


• Subject to weighting bias, if the weighting of the information does not correspond to what is truly makes someone a KOL, the exercise can produce seemingly valid data that misses actual KOLs

The Secondary Research approach works best in therapeutic areas that are fairly static and dominated by academicians.  In these types of environments, there is a strong correlation between age and importance and between academia and practice.

PRIMARY RESEARCH

The third approach to KOL identification is Primary Research.  The Primary Research approach identifies who is a KOL by polling the physicians treating a disease state and directly asking them who influences them.  These responses are then tabulated and the names of those individuals cited the most are given the most weight and are thus considered KOLs.

The Primary Research approach as its strengths:

• Accuracy, this is the only approach that actually asks physicians who influences them and thus is free of any bias or miscalculation


• Broad view, this approach does not limit itself to only KOLs already known by the organization


• Additional insight, at the same time that physicians are polled about influence, there is an opportunity to gain additional insight into their educational needs

The Primary Research approach also has weaknesses:

• Expensive, all direct research is expensive requiring a direct interaction with many physicians and potentially the cost for honoraria


• Sample bias, the sample of physicians polled may be biased, especially if the sample size is kept down by budgetary concerns


• Time consuming, it can take a number of weeks to conduct this type of research

The Primary Research approach is the best approach for less established disease states, less academically driven disease states and for any groups looking for less well established KOLs.

REAL LIFE EXAMPLES

Any of these three approaches may work, and they are often used in combination.

For example, I had a client who was entering a new disease state and thus needed to establish their MSL group from ground up.  During the initial six months, as the MSLs were coming on board and being trained, the company allowed each MSL to use their Personal Network.  Once the group was fully formed, they worked with a vendor to conduct a Secondary Research analysis of the community and create the initial list of KOLs to be targeted.  After the MSLs had been in the field for six more months, they identified a related set of potential KOLs that the Secondary Research had not targeted, and they commissioned a targeted Primary Research effort to identify all the KOLs in this new group.

I have had other clients that used the Primary Research approach to develop their initial list of KOLs then used the Secondary Research approach to continue to track those KOLs and ensure that their staff was fully up to speed on their activities.

VENDORS

Some vendors that support these types of research include:

Secondary Research: Heartbeat Experts, SteepRock, OpenQ, ThoughtLeader Select

Primary Research: AlphaDetail, Encuity Research (full disclosure - I used to be a part of Campbell Alliance, the parent company to Encuity)

In my next post, I will discuss how these KOL interactions should be managed.

As always, I am curious to know your experience with KOL identification.  Please leave comments below.

Topic4: Key Opinion Leaders – Introduction

Educating the healthcare community on new information is both an obligation of and an important benefit for a pharmaceutical company.  Impacting the knowledge of the healthcare community may be the single most valuable service that a medical affairs organization offers.

As much as a medical affairs organization may wish to educate all healthcare providers, the reality is that resources are always limited.  Given limited resources, most organizations look to educate people who will go on to share that education with others.

The fact is that healthcare providers, like almost all specialized professions, have strong formal and informal networks.  These networks may be directly associated with their institution, like a hospital, or indirectly formed by friends and mentors developed over many years.  These formal and informal networks exist because they are valuable to the members of the network.  That value takes many forms – from help with personal job searches, to patient referrals across geographies.  One value that these networks provide is supporting the group’s knowledge on their specific specialty.  If one member of the network discovers something of value concerning their specialty, they will very often share it with others within the network.   These formal and informal networks provide a multiplier effect for education – educating the right person in a network can result in multiple members of the network benefiting from the same information.

Medical affairs organizations tap into this network capability to multiply the impact of their limited resources.  Medical affairs organization look for individuals who serve as information “hubs” in their formal and informal network and apply their education efforts to those individuals, expecting that the information will then be conveyed out to the “spokes”.  As a short hand, many organizations use the term Key Opinion Leaders (KOLs) for those individuals who serve as information “hubs” of their formal and informal networks.  The term stems from the fact that others in their network value the KOLs opinion that a piece of new information is worth knowing.  Other terms for these individuals include Thought Leaders, Subject Matter Experts, Scientific Community Leaders, etc.  For simplicity sake, I will use the term KOLs.

Like everything in medical affairs, educating KOLs is rife with compliance concerns.  These concerns primarily hinge on the difference between education and promotion.  The way I like to describe it is:

Education (or scientific communication) is focused on presenting valuable information to healthcare providers to ensure that they are aware of the latest scientific information.

Promotion is encouraging a healthcare provider to use a given treatment.

Most organizations would cite disease state education as a clear example of the difference.  Disease state education is focused on the disease state in general and not on any particular treatment.  Therefore it is education focused not promotional focused.

Where things start getting “trickier” is around product-specific education.  Some organizations allow it and some do not.  For more in depth discussion on this topic, see this post.

Regardless of where your organization lands on the education question, there are a number of best practices that any organization that educates KOLs should be using.  I will discuss them further in the following posts:

• Identifying the Right KOLs


• Managing KOL Interactions Effectively

As always, I am curious to know your opinion.  How would you describe pharma’s efforts to educate KOLs?  Leave your comments below.

Legal Note:  All information and interpretations presented are only the opinion of the author(s) who are not lawyers. And, even if we were lawyers, given the wide range of interpretations of the current regulations you would still need to get the input from your own compliance organization.  Simply put – your mileage may vary.