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Topic 41: The Implications of Amarin. Is It “Good” for MA?

A number of my friends in MA leadership have been debating the implications of the Amarin case for MA overall. Spoiler alert – I think it will prove to be another avenue for MA to add value and I will share my rationale below.

In case you have not been reading up on this case, you can see my brief discussion and some links to other sources of information on this blog post. When discussing the Amarin case it is critical to understand the context – this case was decided by a federal district judge in Manhattan for the Southern District of New York – so obviously this ruling does not set national standards. However, the Southern District court is one of the most influential and active courts in the US and it has a history of leading the nation.

Assuming that this ruling becomes the precedent for either other cases in other districts or even national cases, the question is if/when pharmaceutical companies have the flexibility to promote off-label data (with all the fair/balanced caveats) – what are the implications for MA?

So let’s go on a trip to a speculative future in which the ability to share off-label data with HCPs on proactive basis becomes accepted in the US. In this future, I would fully expect that the FDA decides to put guardrails around this freedom. Their rationale will be simple – there is high risk to patients if HCPs make decisions based on off-label information which have not run the full risk/benefit analysis of a product with an approved NDA. What would these regulations look like? We can get a good view from FDA’s response to Amarin (which we covered in detail here) the summary of which is that this education would need to be fair and balanced and, among many other things:

  • Discussions should be conducted by persons with the appropriate background or training to accurately communicate scientific information

That would clearly call for a role similar to the MSL or field force role of today’s MA. But would that role need to be in MA, or more provocatively, would MA need to be a separate entity from commercial.

Those you that have been around the industry long enough remember when MA was sometimes a function of commercial, often called Scientific Sales. Driven by increasing FDA and EMA scrutiny primarily concerning off-label promotion and a desire to be seen as a voice of science instead of promotion, MA as an independent, non-promoting entity became the standard.

In our speculative future the US has loosened the off-label promotion rules, but the EMA has not and the need for an independent voice for science has not decreased so I do not foresee an effort to move MA back under commercial. Frankly that ship has already sailed since so many other functions that MA serves also benefit from it being independent from commercial.

So if MA is likely to remain independent would the MSL role remain in MA? I think that they would, not only due to inertia (although you can never over-estimate the power of inertia in pharma) but also because keeping the MSL role in MA would improve the case that the information presented was fair/balanced and not tainted by promotional messages.

Following my logic stream, MSLs would gain a new capability (ability to pro-actively share off-label data) while remaining in an MA function independent from commercial – so a net improvement to the value that MSLs and MA overall brings to the company. That can only be a good thing for MA leaders.

That was quite a bit of speculation – what is your opinion? Please click here to leave a comment.

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Topic 37: Medical Affairs and the Integrated Payer / Provider Model

For a while we have been discussing the need for a specialized medical affairs function focused on Payers – we discussed it here.

But what about Accountable Care Organizations (ACOs) or other integrated payer / provider models, where the provider owns both the cost and the outcomes of their work. This type of model is becoming more and more prevalent, yet most MA organization have not flexed to directly engage with these types of organizations.

The needs for these organization are not a match for current MSL field organizations focused on HCPs and, while payer oriented organizations may be better suited, they are not a perfect match either.  Like payers, these groups are interested in population-level information. And, like payers, they care about total cost of care. But like HCPs they also place a greater emphasis on understanding treatments in the context of the overall disease progression and methodologies for approaches for ensuring improved outcomes with existing treatments.

I suggest that MA organizations are going to need to develop groups that directly target these ACOs. These will be teams that understand population health and quality metrics.And MA is going to need to collect this population data directly.  One thing that seems clear is that HEOR secondary endpoints gathered during P3 simply lack credibility with these audiences since they know the P3 had inclusion/exclusion criteria that did not model their patient population. Real world data and post marketing studies, already important for payers, is going to be equally important for these ACOs.

MA is going to need to come to them with models of costs and outcomes and budget impact, then partner with them to validate the model and gather relevant data about treatment approaches which produce the best results for the least costs.

What do you think? Leave a comment by clicking here.

Topic 34: New FDA Consumer Advertisement Guidance and Potential MA Impact

Have you seen the new FDA guidance about disclosing risk in consumer-directed print advertising that came out in February? (You can see it here) Unless you are a gluten for FDA guidance-reading punishment, my guess is that you skipped this one since it seems to be commercially focused.

BUT, there is actually something that MA should be aware of and perhaps an opportunity to add some value to our commercial brethren. The focus of the guidance is straight forward – under current law print advertising has to also disclose risks, and the safest approach for disclosing that risk is to publish the full package insert (PI) along with the print ad. As we know PIs are a tough read normally, but when shrunk down to fit in a magazine they are almost unreadable and certainly mostly incomprehensible to the very audience they are supposed to be protecting – consumers.

This has not been lost on the FDA and the guidance linked to above was entirely focused on resolving this issue.

In an FDA survey, few respondents reported reading half or more of the brief summary presented in the traditional format. Of those who read at least some of the brief summary, 55 percent described it as hard to read. Over 40 percent of respondents in the survey reported they do not usually read any of the brief summary in direct-to-consumer prescription drug print advertisements.

The FDA realizes that the full PI is aimed at medical professionals and full of details that the vast majority of consumers don’t care about like clinical pharmacology or chemistry. So the FDA is suggesting that manufactures should have the flexibility to replace the PI with something they are now calling “consumer brief summary.”

What is a consumer brief summary? Per the guidance it is an explanation written in consumer-friendly language (ie. drowsiness not somnolence) that includes:

  • Boxed warnings
  • All contraindications
  • Certain information regarding Warnings and Precautions:
    • The most clinically significant information from the Warnings and Precautions section(s) of the PI;
    • Information that would affect a decision to prescribe or take a drug;
    • Monitoring or laboratory tests that may be needed;
    • Special precautions not set forth in other parts of the PI;
    • Measures that can be taken to prevent or mitigate harm
  • Most frequently occurring Adverse Reaction, and those ARs that are serious or that lead to discontinuation of use, and the severity of the risk
  • Indications for use
  • Significant drug interactions

And this is where Medical Affairs comes into play. Now our commercial colleagues and their agencies are going to be needing to develop information that includes medical judgement, like:

  • What is the most clinical significant information?
  • Why is that information considered most clinically significant?
  • How do practitioners view what is most clinically significant?
  • What information should affect the decision to take or prescribe the drug?

These questions are great ones for Medical Affairs to either provide guidance, answer directly or gather information from practitioners during their interactions to answer these questions and others. Given the proper but strong firewalls between MA and commercial, this new guidance provides a value-added opportunity for MA to provide some guidance to commercial.

MA leaders may wish to discuss this topic with their commercial colleagues.

Do you have any thoughts on the new guidance? Leave them in the comments by clicking HERE and scrolling down to the comment box.

Topic 32: Big Data in MA – Revisited

Overview

A couple of years ago I wrote a post (check it out here) on the emergence of big data for Medical Affairs. Given the rapid evolution of big data, two years is a long time ago so it’s worth revisiting this topic.

Let’s recap what we mean by “big data.” It is a broad concept, but for our discussion today we will be using big data to refer to the new capability to pull together huge quantities of data that were not directly generated for the purpose they are now being applied. Biopharma has excelled at generating proprietary data sets for a specific purpose, but big data take advantage of non-proprietary data that was generated for a different purpose by applying it in a new way.
These external data sources range in structure, format and value. The real trick to big data is pulling the data from disparate sources, efficiently cleaning it and standardizing it to allow it to be cross-referenced, then finding novel ways to use it.

Example of Big Data in MA

In the last couple of years we have seen examples of companies set up to provide big data services to MA. I will single out one here as an example, but this is not intended as an endorsement. I have no relationship with this company or practical experience with their products.

The company, Med’meme, is a case study of big data in MA. Based on their website, Med’meme takes large, public data sets – in this case lists of scientific presentations from medical meetings and peer-reviewed journals and clinical trial information at least – and in their backroom they apparently standardize it to make all those data cross referenceable. How well they do this, how complete and how accurate the data is, I can’t say. But, when you think about that data source as an MA professional I am sure you are jumping to a bunch of potential uses – like the ability to rank KOLs, to identify new KOLs, to track TA trends in publishing, to identify potential investigators, to be alerted to new publication identification, etc.

And that is the beauty of big data – there does not appear to be anything in their data set that has not been available (with some costs) to biopharma for years. Their service is finding a way to scrape it all together, standardize it and allow it to be searched effectively.

Buy v Build in Big Data

When I first published the article about big data I had a number of “buy vs. build” questions. The reality of big data in its current form is about re-using publically available data in novel ways, so building it internally is unlikely to produce proprietary value. However, combining these data sets with proprietary data, or asking interesting and unique questions of the data is something that can remain proprietary – so some hybrid solutions may be valuable.

If big data is not a part of the MA information technology planning it should be.  This capability represents an opportunity for strategic advantage in the short-term until it is widely adopted.

Conclusion

Big data is a new reality. A huge new data set, the Sunshine Act database, has just come on-line, and other data sources are increasingly making their data available for these types of analysis. Expect to see major development in this area in the coming couple of years.

What has been your experience with big data in MA? Leave a comment.

Topic 30: Virtual MA Organizations – Leadership Implications

I have posted about outsourcing in MA in past posts here and here.

Today’s topic is focused on the virtual MA organization – an organization that outsources all or almost all of its key functions.  Given the capabilities of service providers, it is entirely feasible to outsource every sub-function within MA, including medical communications, grants management, medical information, standard and specialty field forces / MSL groups.

My goal today is not to discuss the pros and cons of a decision to form a virtual MA organization but instead to discuss some key aspects to making a virtual MA organization successful.  Although today’s post is looking at a fully virtual MA, the key points would be just as relevant for a mixed virtual and internal MA organization.

Reality of Virtual MA Organizations

Most organizations that decide to use virtual MA are organizations where very little MA infrastructure exists.  Either they are small organizations building their first MA function or they are mid-sized organizations that are going into a new TA.  Regardless of situation, the analysis that leads to a virtual MA organization is usually a buy vs. build decision.  When considering how to make virtual MA successful, we need to start at that point.

Keys to Success

There are three keys to success to managing a virtual MA organization:

  • Upfront Expectations
  • Sufficient Internal Management Resources
  • Structured System for Evaluations

Upfront Expectations
In order for the virtual MA organization to be successful, the vendors that provide the services need to have a clear understanding of what is expected of them and when so that they can develop the correct scope of the work for their pricing.  Defining what is expected of the vendor is easier in some cases, like for Medical Information defining expectations about call wait time and speed of fulfillment.  But, defining expectations is much harder in cases like an MSL group or a specialty education group.  This challenge is heightened by the fact that the reason some organization’s decided to go virtual is that they don’t have a lot of expertise in house.

Nevertheless, it is vital that a clear set of expectations and measures are agreed upon as a part of the vendor selection and contracting process.  There is no point in the process where the company has more control than at the point of contracting.  If clear expectations are set, both sides win because the vendor can appropriately staff and manage the group and the company can achieve their goals.  If not, the vendor may either need to increase the scope during the contract or simply fail to achieve some needed result and the company will face unexpected costs and missed expectations.

In order to set the right expectations a strategy for MA’s work for the next period must be developed in detail.  And this strategy should be developed before the vendor selection process occurs to ensure that the vendors are supporting the strategy not the strategy supporting the vendors.

WARNING: Some vendors in our industry will encourage buyers against doing this work in advance.  The vendors will tell the buyers that the vendor will develop these expectations after the contract is signed and/or after they are active.  Companies that take this approach usually end up spending much more than they expected and achieving less results than they expected.

Sufficient Internal Management Resources

Virtual does not mean management free.  While the vendors will definitely have their own managers, successful virtual MA organizations have learned that they need to closely manage the vendors to achieve expected results.

Given the situation that leads to a virtual MA organization as discussed above, it sometimes comes as a shock to those setting up one that they still need to hire.  And those hires need to be managerial-level staff.

A general rule of thumb is the greater the range of responsibilities and lack of clarity, the greater the need for management.  So, for example, outsourcing a MedInfo group, with clear metrics and expected volumes, might require only a ¼ Associate Director.  But managing an outsourced MSL group during launch, even if there is a clear expectations set up-front, would require a ½ time director to deal with the unexpected and new learnings from the healthcare community.

If the company is unwilling to invest in the resources needed to manage the virtual MA vendors, that is a sign that the value of MA is not really understood within the organization.  Unless that core issue is addressed it is unlikely that the virtual MA group will be successful.

Structured System for Evaluations

In addition to being actively managed, virtual MA vendors need to be on a half yearly or yearly structured evaluation process.  The structured process will force a review of the original goals and scope of the relationship and provide an opportunity to resolve ongoing issues.  Without a structured process for reviews, problems tend to fester and eventually result in a complete breakdown of the relationship.

Many companies entering into a virtual MA environment rely on the vendors to suggest the structures of these types of meeting.  I would recommend that the company own the process and set the agenda.  This will ensure that those issues most important to the company serve as the focus of the process as opposed to an add on.  It also avoids the “make the case for added scope” that many vendor-driven processes tend to become.

Do you have experience setting up or managing a wholly or partially virtual MA organization?  What would you recommend?  Please leave a comment or send me a message.

Topic 28: Big Data and Medical Affairs

“The era of Big Data is here!”  That may be true but what does that mean for Medical Affairs?  As in all of biopharma, MA is comfortable working with data.  So much of our work revolves around discussing data and the implications of data that many people may think that we were already living in the era of Big Data.

But for most MA organizations, the data sets we have focused on are purpose generated – either our own data or data from similarly-scaled studies conducted by others.  Big Data refers to something different.  I like the differentiation that SAS uses when comparing Big Data to the past data sets.  They break it down to four “V”s and a C:

  • Volume: Hugely increased data volume from the past
  • Variety: Since the data is produced in many different ways, it has many different formats and structures
  • Velocity: Both how fast the data is being produced and how fast it must be processed
  • Variability: Inconsistent data flows, with peaks and valleys
  • Complexity: Driving value out of these data sets is highly complex and difficult

This is not your grandfather’s data sets.  What are some examples of Big Data as relevant to biopharma and MA:

  • Electronic Health Records data from a variety of sources
  • Search engine data (see an example of analyzing search data to find safety signals here)
  • Sunshine Act Physician Spend Data (when it becomes available)
  • Social media data
  • Competitors clinical trial data as it is released

Contained within these and many Big Data sources are key tools for MA:

  • Valuable therapeutic information
  • Unique customer insights
  • KOL identification and information
  • Visibility of competitors drug development and support efforts
  • Important drug safety signals

But, none of these benefits can be achieved unless the question is asked and the data is analyzed.  I would suggest that effective MA organizations of the future will need to have the capacity to ask and answer these types of questions.

In order to do so, MA organizations will either need to build or have access to increased levels of biostatistical and epidemiological resources.  And these resources need to have skills directly related to Big Data.  The characteristics that differentiate Big Data from existing data sets also means that many existing biostats and epi staff do not have the expertise or confidence working with these large, external data sets.  MA organizations need to ensure that people with exactly these skills sets are available within their organizations or from outside vendors and that these resources have the capacity to support MA.

Then, MA needs to improve its overall level of confidence defining Big Data questions, conducting Big Data analysis, and discussing the results with others.  Given the difference in the source of this type of data, the way this data is presented and discussed must be different too.  Everyone in MA, but especially the MSLs, must become more comfortable understanding the nuance of this type of data analysis and discussing both the strengths and weaknesses of working with Big Data.

The era of Big Data is here.  MA has a long history of effectively using data and explaining data in support of its organization.  MA leaders must investigate and embrace Big Data to take advantage of all the tools available today.  The questions unasked are always the questions unanswered.

What is your experience with Big Data?  Please leave a comment.

Topic 27: Disruptive Change in CD and the Opportunity it Presents to MSLs

Disruptive change is a frequent topic on this blog because it represents both a threat and opportunity for MA Leaders.  For example, I have discussed the Caronia ruling at length because it could fundamentally alter the relationship between MA and Commercial.

Clinical Development is facing a disruptive change of its own that not only will alter the way it works but offers an interesting opportunity for some MSL groups.

As most of my readers know, one of the largest expenses in executing a clinical trial is monitoring costs.  The need to send a human being to each site every 4 to 8 weeks during a trial to manually review the paper source records and compare those results to the data entered in the electronic data capture (EDC) system accounts for roughly 30% of a major study’s budget.

However, this is about to change.  With the rise of tablet computers and ubiquitous internet, a range of technology platforms are now available to allow for the elimination of paper-based source records.  For example, the company Clinical Ink, provides a Windows tablet that completely eliminates the need for paper source records by recording not just that data required for the study sponsor (which is what is in the EDC) but also the data needed by the physician administering the study to manage their records.  Once the physicians can eliminate the need to keep separate paper records, the last stumbling block to all electronic source records will have been overcome.

All electronic source records is a highly disruptive change to clinical development.  All electronic source records means that source data verification no longer needs to occur on-site.  The majority of the monitoring visits could be eliminated and the role of the Clinical Research Associate (CRA)  versus role of the Clinical Data Manager (CDM) will change significantly.

Today CRA’s do more than just monitoring visits.  They also conduct site initiation visits, and other high-visibility meetings with the site to ensure that it is on track. However, when you eliminate the need for monitoring visits, it opens an opportunity to ask if the CRA is really the best representative to provide other support to the site.

I would suggest that this disruptive change offers and opportunity for the MSLs to step up and serve as the face of the company for key studies.

Already many MSL groups get involved in this manner along side CRAs for key studies.  In the future MSLs could replace the CRAs for the customer-facing aspects of key studies, just like CDMs will replace CRAs for the electronic source data verification processes.  It would require changes to SOPs and some additional work and training for the MSLs but the benefits for the organization would be significant:

  • MSLs already have relationships with many KOLs which serve as PIs and this is an opportunity to strengthen that relationship as well as build new relationships
  • MSLs work for the company and therefore understand the importance of building and maintaining long-term positive relationships with the PIs (as opposed to CRAs which are often outsourced and only focused on a single study)
  • MSLs have superior scientific training than most CRAs and thus can make a stronger case for the scientific importance and structure of the study, which is cited by PIs as one of the key factor’s driving their willingness to engage in the study
  • MSLs can have peer-to-peer discussions with the PIs to ensure that they understand the protocol
  • MSLs often glean key scientific insights from PIs when working with them on studies that can be brought back and shared with the organization’s scientific leadership
  • MSLs can offer their scientific opinion on aspects of the trial that may be limiting recruitment given their in-depth engagement

These are just a few of the benefits for engaging the MSLs in this new manner.  But, like all disruptive change, this is not going to come about naturally.  For MSLs to engage in this new manner, MA Leadership will need to reach out to their CD counterparts, discuss this disruptive change and push for a new role for MSLs.

What are your thoughts?  Leave a comment or send me an email.

Topic 26: Highlights of Day 3 of the Medical and Scientific Communications 2013 Forum

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The final half day for the MSC meeting had an excellent and well received discussion about MSL metrics.  I also attended an interesting talk on coordinating global publications.  My take on it below

DISCLAIMER:  My focus is on interesting information, I did not try to take verbatim notes and will not try to assign comments to a particular speaker.  Any mistakes are mine alone.

In no particular order, some of things are found interesting included:

–  Metrics

  • Must vary as focus of the organizations varies
    • Plays back to earlier comments about staying focused if you want to see improvement over time
    • Mix of Activity, Value and Compliance measures are needed
    • IIS Metrics
      • Good to track in terms of just a sense of the program
      • NO goals should be put in place
      • Additional metrics discussed
        • Engagement with ACOs or Cancer Networks or other new healthcare entities
        • Congress attendance valued by quality metric of the outputs not the activity
        • Dollars saved by asking MA to assist CD, especially when CD has a bid from a vendor to provide the same service
        • Internal stakeholder feedback
          • Important – best to solicit directly via email/conversation
          • BE CAREFUL – some organizations do not allow feedback from Commercial

–  Reports/Dashboards/Scorecards

  • Need to develop different outputs for different stakeholders
  • What is needed internally at MA is not what you want to focus on with other internal stakeholders
  • Always pair summarized numbers with text explaining value and highlighting accomplishments/learnings

–  MA IT Systems

  • As mentioned earlier, a number of companies seem to be moving toward the Veeva Platform which is built on top of Salesforce.com CRM.
    • Veeva seems to be driven by the commercial side, but it has MA capabilities
    • Some people mentioned that you need to be careful about Veeva training and documentation as it often defaults to commercial language
    • Some organizations use it to track not only KOL interactions but also time spent in the office doing various projects
    • Focus on time tracking seemed to be for internal management not for external publication

–  Global Publication Coordination

  • Increasingly important to have global plan
  • Much easier to manage when Pub Team gets involved earlier in the development cycle
  • MA on Pub Team should work as a facilitator for Pub Team’s interaction with other functions given their central role
  • Press release management globally remains a major challenge
    • General consensus that prior to first presentation at a meeting, press release should be simply binary – met endpoints, did not meet
    • Too much data in press release can tick off journals threatening publication

–  Odds and Ends Ideas

  • Heard from a number of different companies that MSL groups focused on payer support in the US need to be between 6 and 10 in size to cover all the needs – not scientific but an interesting convergence
  • MSLs at clinical study initiation visits:
    • Good idea BUT has GCP implications
    • Need to hammer out how MSLs can fit within CD’s SOPs with ClinOps before engaging
    • May ultimately require update to CD’s SOPs

Obviously, this is merely a few key concepts from the day but it should give you some flavor of the type of discussions and the focus of the meeting.

If you were at the meeting and have other thoughts please leave them in the comments.

Topic 25: Highlights of Day 2 of the Medical and Scientific Communications 2013 Forum

A great second day for the MSC meeting, with a range of interesting topics.  My take below.

DISCLAIMER:  My focus is on interesting information, I did not try to take verbatim notes and will not try to assign comments to a particular speaker.  Any mistakes are mine alone.

In no particular order, some of things are found interesting included:

–  MSL Value Proposition:

  • Questions of value are often linked with a lack of fully understanding what a modern MSL does, vs. MSLs of old
  • Many in group have had good success inviting key stakeholders, up to and including CEO, for ride-alongs with MSL
    • Proven track record at overcoming skeptics about MSL skills and value
    • One key to proving value is remain consistent in priorities to show value over time
      • When priorities are shifting its hard to build up track record
      • Remember that one of the greatest MSL values, relationships, is the least structured and always make that point clear

–  Other non-traditional areas where MSLs can show value:

  • Training CRAs on underlying science before trials
  • Conducting formal evaluations of CME vendors to ensure vendors are providing good value
  • Identifying authors for Pub group

–   Measure what matters to the people that matter

  • Develop measures that highlight accomplishments that only MSLs can achieve
  • Create monthly list of these unique accomplishments to address qualitative questions

–  External Surveys

  • Important to get qualitative measures
  • But expensive and difficult
  • Also, expect lots of push back from people who don’t think they are fair
  • Don’t let the complainers win, the data is critical to effectively target efforts

–  A number of MSL organizations use monthly internal newsletters

  • Highlight key activities and accomplishments
  • Provide some science training
  • Implicitly defines values and culture through examples chosen and emphasis

–  Challenge of “boom bust” in MSL team sizing

  • Number of speakers referred to the challenge of staffing for launch only to see the team significantly reduced 1 to 2 years after launch
  • This is especially challenging outside of the US where country-level resources are very limited
  • Need to consider this issue up-front during initial staffing and make the case for the long term from the start

–  Breakout session about MSL management

  • Almost everyone in group had turnover of less than 10% and most less than 5%
  • Most organizations had two non-managerial  levels of MSL – MSL and Sr. MSL
    • Some organizations had three with an Executive MSL above Sr.
    • Most organizations had at least two managerial levels but the titles varies
      • Some had three
      • Expectations about activity level varied but some examples from different companies:
        • 4 visits per week
        • X number of interactions per week (phone/email/visit)
        • MSLs will be out in the field 3 to 4 days per week
        • Key to expectations is flexibility
          • Some MSLs in small geogs some in large, their expectations should be different
          • Some TAs have large number of meetings, some few, so their expectations should be different

–  Mobile technology

  • IPADS is the de-facto mobile tech of today
  • Currently adjunct devices to laptops, NOT replacements
  • Hard to justify on “cost saving” but easy to justify on WOW factor
  • Be careful of SOPs that are linked into the old way of doing things, may need to be re-written to accommodate the tablet
  • User experience is more important on Ipad than on PCs
    • Users have very low tolerance for clunky Ipad interfaces
    • Some other interesting ideas:
      • KOLs are likely to grab the Ipad so make sure everything you don’t want them to see is locked down
      • Ipad syncing is a manual process and may people may avoid, so build in lock outs to programs to force them to update at least monthly
      • 4 Digit standard Ipad passwords stink – they are breakable in minutes, use a much longer password
      • Use  cloud-based apps like DropBox or Google Drive only after being approved by compliance
      • People want to use their Ipads for personal stuff (photos, music, games) and most companies let them use it for that as well
      • Allow people choice in covers and other accessories since it is very personal
      • Have an accessories swap program to allow people to turn in accessories they no longer want and others can use

–  Odds and Ends Ideas

  • Conduct “Office Hours” during major meetings when top CD resources are available in booth so MSLs can introduce their KOLs
  • Compliance ride alongs – if compliance is unsure of an approach, offer to pilot it with them on a ride along so they can see it in action then make a determination
  • Community MSL group focused on non-HCPs, like Advocacy Group

Obviously, this is merely a few key concepts from the day but it should give you some flavor of the type of discussions and the focus of the meeting.

The meeting remains a very positive experience.  One of the key takeaways from today, however, was the desire of the audience to have more time to interact and learn from each other.  A good problem to have!

Topic 19 – EU Considering Major Change in P4 Trials

A new position paper has been floated by the European Commission Health and Consumer Directorate-General that may have HUGE impacts on the way P4 trails are conducted in the EU (and the rest of the world).  My take on it is below:

Stemming from the new EU regulations around pharmacovigilance, the EU is also considering the role of post-marketing efficacy studies (PAES).  In a position paper seeking feedback, found here, the EU lays out its perspective that the new PV legislation also refers to the possibility of requiring the market authorization holder to develop PAES to complement the efficacy data used to grant marketing authority.  That power is granted where “concerns relating to some aspects of the efficacy of the medicinal product are identified and can be resolved only after the medicinal product has been marketed.”

Regulatory Purpose

The position paper goes on to lay out the regulatory purpose of PAES and suggest that after the results of a required PAES were provided to the regulatory agencies, they would be free revisit the authorization decision and determine if “…the marketing authorization should be maintained as granted, varied or even withdrawn on the basis of the new data resulting from the study.”

While regulatory agencies have always been free to revisit their decisions, this approach seems to imply that they are obligated to reconsider their decision after the required PAES.  This makes thoroughly changes the role of the non-PV focused P4 trial from today’s approach to a much more high-stake trial.  Under this approach, these types of P4 trials would have much higher risk and would likely need to be treated much more like we treat P3 studies.  This means a likely significant increase in cost for these trials.

Efficacy vs. Effectiveness

The next section of the position paper focuses on the question of efficacy vs. effectiveness.  The paper comes down clearly on the side of efficacy.  Again, this could be a major change for P4 trials, many of which are focused on developing real-world effectiveness data to provide support for reimbursement decision makers.  This change may also force a significant revisiting of the P4 approach followed today.  Additional P4 trials may be needed if the mandated trials focus on efficacy and cannot be tasked with also considering effectiveness.  This will have a significant impact on time and budget as well.

Situations Where PAES May Be Required

The paper then proposes a set of 7 situations where PAES may be required.  They are:

Situation Where PAES May Be Required Comment
1.   Studies aimed at determining clinical outcome following initial assessment based on surrogate endpoints This type seems very logical.  If surrogate end points were used to get authorization, then a study may be required investigating the underlying endpoint sought.  But, this may significantly add to the cost of using surrogate endpoints for approval, since surrogates are sometimes used because of the time (and cost) of gathering data on the underlying endpoints.
2.   Studies on combinations with other medicinal products Acknowledging that testing for authorization can only cover a limited range of combinations, P4 trials may be required to test additional combinations.
3.   Studies in sub-populations Given the limitations on the number of sub-populations, additional sub-populations may be required to be tested.
4.   Studies in the context of the European standard of care If the trial uses subjects primarily from outside of the EU, the EU may request additional information from patients treated within the EU.  This would negate any cost savings a company might hope to garner by working in other markets.
5.   Studies linked to a change in the understanding of the standard of care for the disease and/or the pharmacology of the medicinal product This is a scary one.  It proposes that if the standard of care for a disease had changed, the authorization holder may be required to develop new efficacy data addressing the new standard of care.  This could result in a never-ending need for new efficacy trials – a major change in way we think of authorized products.
6.   Studies aimed at determining the long-term efficacy of a medicinal product This is one of the “traditional” reasons for P4 is long-term analysis.  Difference is focus on efficacy instead of effectiveness.
7.   Studies in everyday medical practice The proposals suggests that these would be required when “…there is clear evidence that the benefits of the medicinal product underdiscussion as shown by randomised controlled clinical trials might be significantly affected by

the real-life conditions of use.”  This seems to open the door for any misgivings or concerns by the regulatory authorities to result in the requirements for a trial – and when they get results they will be obliged to revisit the approval.

Final Thoughts

While this is still in proposal stage, the direction is clear.  PAES is going to become a new tool for regulators who have efficacy concerns about both new approved and legacy products to address their concerns.  It is likely to have a significant impact on the cost, risk and structure of Medical Affairs if it comes into effect.

I urge you if you have comments to follow the process found in the document to express them.  I will be doing so.

What are your thoughts?  Am I overreacting? Please leave a comment.

h/t PharmExecBlog