Tag Archives: research

The Five Types of Biotechnology Companies

Type One:  Big Biotech

Biotech companies with full product lines and significant revenues that rival those of Big Pharma.  They have sufficient cash from operations (profitable) to support healthy, well-funded research pipelines and to acquire technologies that fit their business strategy.

Type Two: One Trick Ponies

Companies with a single major marketed product and sufficient revenues to support some operations with or without profit.  These companies are often acquisition targets for Big Pharma.  With no follow-on products or pipeline, company executives and investors are counting on the company being acquired before they run out of money, lose investor interest, or their product becomes obsolete.  

Type Three:  Prolific Pipelines

Biotech companies with pipelines of high probability technologies.  Their compounds have a sound basic science premise supported by encouraging preclinical and perhaps even Phase II clinical data.  These biotechs focus on developing a strong regulatory package of clinically relevant “proof of concept” data to support optimism for FDA approval and commercial success.   Single product companies might be acquired outright while multiple product companies may sell individual compounds (or technologies) while leaving the discovery research and early clinical development infrastructure in place.  These companies have recently been the source of compounds for Big Pharma looking to replenish and bolster their depleted pipelines.

Type Four:  Too Little, Too Late

These companies have compounds that will probably work, deliver positive clinical results, and even achieve FDA approval.  Unfortunately, their product will provide no meaningful clinical benefit over currently marketed products (some of which might even be generic drugs).  The commercial potential for these products  is limited.   These companies, nevertheless, continue to persuade investors with slick presentations espousing large market size numbers and an insistence that with “a new treatment” even a small percentage of that large market will result in significant revenues for the company.

 Type Five:  “Promises, Promises”

Biotechs that are developing products based on extrapolation of flawed or misinterpreted laboratory-derived conclusions.  This is different from bonafide discovery research based on progressively revealing evidence of potential safety and efficacy.  Unfortunately, while their story may sound plausible, there is little or no evidence to support hope for clinical or commercial success.  These companies thrive on finding investors who are looking for lottery size payouts from picking a winner where nobody else seems to appreciate or understand the medical and scientific techno babble.  These companies require eloquent CEOs who can tell a story and preach the promise of a promising technology.  Unfortunately, the only winners for this type biotech company are the executive teams who continue to be paid Big Pharma salaries and bonuses for prolonging the unfulfillable promise.

So what’s the point?

There are probably in excess of 4000 companies (private or public) worldwide that consider themselves “biotech” companies. 

             Think about the large number of Type Four and Five companies masquerading as Type Three companies.

             Think about the venture capital, investment, and research grant money that Type Four and Five companies wastefully divert from Type One, Two, and Three Biotech companies.

             Think about investor confidence in Biotechnology when Type Four and Five companies fail to deliver.

 mike@pharmareform.com

drugs

Big Pharma R & D Failing

Research and development … innovative new drugs … “the life blood of the pharmaceutical industry.”

Tens of billions spent on research and what does Big Pharma have to show for their investment?  What do the multi-million dollar a year CEO’s and executive teams have to show for their brilliance in delivering the much needed new treatments they keep promising?   Not much, according to the FDA calendar for 2013 drug approval decisions chronicled in a recent article by Seeking Alpha (http://tinyurl.com/qjdyc3m).  Moreover, based on their pay and bonuses (http://tinyurl.com/p57yt5o) CEO performance reviews obviously do not put much importance or weight on drug discovery or approvals. 

Here’s my assessment.

From what I could determine, only five drugs (suvorexant, dabrafenib, tivozanib, dolutegravir, trametinib) discovered and developed by the sponsors are what I would consider “new”.  GlaxoSmithKline developed and sponsors three of those five drugs and one was developed and sponsored by Merck.  Two new drugs (efinaconazole and sofosbuvir) were acquired and then developed by the sponsors, Valeant and Gilead.

Six additional drugs scheduled for FDA decisions in 2013, are merely reformulations or new delivery systems for already approved drugs.

And finally, there are five,  already approved drugs,  awaiting new or expanded indications. 

Think about that for a second.  Tens of billions of dollars spent on R & D and technology acquisitions every year for decades and only seven new drugs.   Every year PhRMA touts the thousands of new compounds in development, several hundred by disease state.  How many Big Pharma company executives get up at the never-ending investor conferences to brag about their pipelines of new drugs coming?  With more than a thousand drug or biotech companies, spending tens of billions of dollars year after year and we only get seven new drugs?

I would also point out that, from my perspective, only Merck and Glaxo qualify as Big Pharma.  Ok, Pfizer is in the joint venture on dolutegravir, but still.  Even if you throw in Cubist and Gilead, where are the rest of the Big Pharmas? 

This is a sad commentary on disastrous pharmaceutical R & D productivity (and the lack thereof). More importantly, it’s a very scary picture for investors and worse for those with diseases for which there are few or no therapeutic options.

Perhaps Pharma will figure out that it is not more money that delivers breakthrough new treatments.  Focused, high-level basic science expertise (not just academic degrees) and a deep multi-disciplinary understanding of the target disease are required to crack the drug discovery code.  Unfortunately, for the past couple of decades, Big Pharma hasn’t valued discovery research. They would rather just write a check than do the hard work.  And the results show.

What did Big Pharma contribute to 2012 New Drug Approvals?

I’ve read several press releases over the past couple of week announcing and interpreting the significance of FDA drug approvals over the past year.  Most give you the impression that the results are encouraging and suggest Pharma R & D is in recovery mode.  Traditional PhRMA bragging points of spending more than $60 billion per year on R & D, the thousands of compounds in development, and hundreds of innovative new medicines approved over the past couple of decades are, however, somewhat tempered by the reality of the recently reported drug approvals, especially for Big Pharma.  I don’t mean to downplay the challenges of drug discovery and development but it’s important to look beyond the total number of approvals to determine what that number really represents.

A closer look at the list of 35 FDA approved drugs for fiscal 2012 (October 1, 2011 to September 30, 2012) reveals rather dismal R & D productivity and lack of innovation that has haunted Big Pharma for the past several decades.

First let’s put 35 new drug approvals into context.  These new drug approvals were the results of decades of  hundreds of billions of dollars invested in research being done at or by more than a thousand Pharma and Biotech companies as well as thousands of government and university laboratories.  Pharma reminds us frequently of the hundreds of compounds in development for different diseases.   Yet, only 35 new products in 2012 … not very impressive.   Also interesting to note is that there are 31 different sponsors for the 35 New Drugs Approved.  So out of more than 1000 pharma and biotech companies out there, only 31 entities got a drug to the approval list.

The list better reflects the importance of smaller research organizations in discovering and developing new drugs.  Depending on how you define Big Pharma (e.g., are Gilead, Forest Labs, and Astellas Pharma considered Big Pharma?), you may end up with slightly different numbers but Genentech/Roche, Bayer, and Sanofi contributed 4 of the 12 “Priority Drug Approvals”.  More to the point, of the 35 drugs listed, if we’re generous in a definition of Big Pharma, only 13 of 35 drug approvals were “sponsored by” Big Pharma.

What’s more disheartening is the evidence of diminishing drug discovery at Big Pharma.  While celebrating the 35 approvals, a little investigating reveals that the overwhelming number of approved products were not discovered by or at Big Pharma.  You have to dig a little but even where Big Pharma is listed as the sponsor, many of the approved compounds were licensed in or acquired from collaborative work with smaller biotech companies.  Again, being generous in how you might define Big Pharma, only 6 or 7 products on the list were actually discovered at a Big Pharma.   And in this number, two were discovered by Genentech (acquired by Roche), one is an analog of a previously approved biotech product, one is a combination vaccine, and another is a combination of previously approved products.  So much for Big Pharma innovative “discovery research.”

So while the total number may be reassuring in the context of previously disappointing years of smaller numbers of drug approvals, a closer look reveals that drug discovery and drug development are no longer being driven by Big Pharma.  More importantly, despite the investments being made, new drug discoveries remain elusive and approvals are rare regardless of who is doing the work.

mike@pharmareform.com

Mindset Change Needed for Re-Engineering Pharmaceutical/Biotech R & D

In the previous post and in the book Pharmaplasia I suggest a need for a new approach to pharmaceutical R & D.  There are two essential elements to this proposal.

The first has to do with scientific and corporate integrity.  If the research scientists and corporate executives are not honest about what you have and what you have seen in drug development (if their priority is to protect the “potential“ of your compounds), you can’t make good decisions about your pipeline products.  Research scientists or corporate executives trying to preserve compound “potential” by strategically navigating around potentially damaging data, half-truth disclosures, or blatantly ignoring negative results and their implications can easily sabotage any corporate effort to produce a more robust pipeline with a higher probability of success.  The second essential element will be meaningless if this first element is ignored.

The second essential element:

Know more about your compounds before they go into Phase III clinical trials.

A more comprehensive basic science understanding of your compounds before they enter the clinic, especially expensive Phase III trials; can increase the probability of success.

For safety, this requires an aggressive exploratory preclinical program that “looks for toxicity” (going well beyond the regulatory requirements) and understanding what you find and what you see … not just being able to explain it away.

From an efficacy and safety perspective, if your compound affects one biologic system, what other systems does it affect? Have you really looked or have you been focused on “getting an indication?”   Do you have the basic science data to support the premise/hypothesis for efficacy (or comparative superiority)?  Have you scientifically challenged the premise with alternative outcome possibilities with data to support the different probabilities?   Or, is it still just a hypothesis you plan to prove in Phase III?  Are you sure you have the right endpoints for your Phase III trials?  Have they been sufficiently validated (high probability statistics) in Phase II studies?  By this I mean, have you done more than just a couple of regulatory required trials?  Have you looked at design alternatives that could affect outcomes for different endpoints options?

This strategy may take longer.

But, rather than how fast can you get how many compounds into patients and to the market, research teams and corporate executives need to shift their mindset to increasing the probability of success.  You can do this with a more comprehensive approach to preclinical research.  Thoroughly understanding how your products are going to perform in Phase III trials (or comparative trials) before the trials commence.  Eliminate the “surprises” with better, more comprehensive science around the products.  Be exhaustive in exploration, honest about the findings, inclusive of interpretations, and have better data to support the “go forward” premise or hypothesis.  This all may seem like a simplification but if you’re interested in a more elaborate discussion, I invite you to read about the need for pharmaceutical R & D change and recommendations for change in Pharmaplasia.

Sure there will still be failures, but they should be fewer.  But perhaps the biggest benefit from this approach is the potential for new discoveries of safer, more effective products.  Better diagnostics, more definitive efficacy benefits (maybe we are looking at the wrong endpoints), and advances in the understanding of human biology await this new Pharmaceutical R & D model.  Not to mention, less contentious regulatory reviews, renewed hope for patients with difficult to treat diseases, and more certainty for the investment community.  mike@pharmareform.com

Why so many Surprising Disappointments from Pharmaceutical R & D?

FDA rejections of new drug applications (insufficient efficacy or safety data), totally unexpected drug failures in Phase III trials,  bewildering “no significant differences” demonstrated in comparative trials, eye opening safety issues in late stage trials or raised by FDA Advisory Boards.  In many cases, negative results sufficient to delay approval if not “kill the drug.”

Along with these stories come the unscientific rationalizations of failures. “That’s drug development.”  “High risk, high reward.” “Biology is complicated.” “Diseases we are trying to treat today are far more complex.”

These are not new headlines for the pharmaceutical industry.  In fact, and unfortunately, they have almost become a cultural industry expectation. Patients ride the roller-coaster of hope and disappointment while investors, also frustrated, keep hoping for that occasional “big win” that makes it all worthwhile.

The pharmaceutical and biotech industries have to find a better R & D model before patients lose faith and investors no longer feel that the “drug discovery and development lottery” is worth playing.

How many more times can Big Pharma place big bets on “promising“ compounds with limited “proof of concept” only to find out they have been sold worthless technology that can’t even make it through a traditional development program to gain market approval?

How many Pharma pipelines boast the number of compounds in development merely to demonstrate that they have something worth investing in, while knowing full well most of the compounds have little or no chance of really making it to market or producing a profit?

How many compounds in these Pharma pipelines (or biotech compounds for that matter) have been strategically developed so as to embellish the efficacy “potential” without exposing or exploring the design flaws that might compromise this “potential?”  How many of these compounds have been carefully tested so as to avoid any suggestions of toxicity that might be difficult to explain or might raise concerns during a “Big Pharma due diligence” (for biotech) or worse, during a regulatory review?

But many of you might be thinking…well that’s just the way pharmaceutical and biotech R & D is.  Well, you’re right… it is and it has worked for decades when the benefits of drug treatment (versus no treatment) outweighed the risks and the market was far more receptive to paying for mediocre “follow-on” products?

Find a compound with biologic activity (remember “get a hit in high throughput screening?”), see if it causes any “apparent toxicity” (do the regulatory required testing but don’t look too hard beyond that) in a few animal models.  Do a quick Phase I trial to see if it causes any “apparent toxicity” in a few volunteers.  Your objective is to get into and out of Phase II (not to really understand what happens in Phase I or II).  Now, pick a dosage schedule and the easiest, fastest indication to establish a quick proof of concept. Then, if you’re a biotech company, find a Big Pharma to buy your compound and/or your company.  If no buyer, get more investment to start a Phase III trial.  If you’re a Big Pharma, push it into full-blown Phase III clinical trials as fast as possible on a timeline that shows investors your “quick to market” development strategy and then “hope for the best.”

The problem is that this historical Pharmaceutical/Biotech R & D model is no longer viable.  So what has to change?   mike@pharmareform.com

dowser

Divining the Future from JP Morgan Healthcare Conference Presentations

The J P Morgan Healthcare Conference is, among other things, an annual four days of back to back 30 minute presentations by Pharma, biotech, device companies, CROs, and a diversity of healthcare institutions.  C-level presenters, mostly CEOs, trying to persuade analysts and potential investors that they have the business model designed for increasing shareholder value, some bolstered by forward looking statement disclaimed historically based promises for product approvals, revenue and earnings growth,  dividends, and stock buy backs.

The conference is the premiere healthcare conference in the industry and has become “old home week” for industry executives to reconnect, schmooze, and initiate discussions for potential deals.  Getting an invitation is near impossible if you are not among the presenting companies or on the JP Morgan A-list.  I am neither, so I spent last week listening to all the webcasts that are available for the Pharma and biotech company presentations.

Perhaps the single most stunning, yet less obvious (non- investor perspective) “take away” for me was how rapidly Big Pharma is moving away from Primary Care.  With almost 75% of prescriptions now being filled with generic drugs, the trend may not be that surprising.  What is surprising is that the pace of proactive strategic abandonment of Primary Care is far more dramatic than what I believe most people in the industry would want to admit or even realize.

This trend really got my attention when companies with traditional Primary Care portfolios blatantly stated or clearly outlined that they have strategically refocused their pipelines and commercialization efforts to target specialty markets.  With very few exceptions, company presentations were absent references to products or commercial strategies targeting the Primary Care market.  Oncology, neurology, psychiatry, rheumatology, and dermatology seem to be the focus of attention unless you had a Hepatitis C compound in your pipeline.

Again, the interest in specialty products is not surprising.  They command higher prices, yielding higher margins with less onerous managed market intervention into prescribing practices.   From a commercial perspective, specialists represent a smaller, more easily targeted and sales force friendly customer base.   Specialty market physicians and their patients also seek out and are more receptive to disease and treatment information making promotional education a viable and efficient tactic.

The implications of this trend away from Primary Care are clear.  Fewer sales reps needed for calling on Primary Care.  Less need for expensive Primary Care sales and marketing support activities such as purchasing mass market prescription data, coordinating the complexities of territory management and sales reporting, and dealing with sales force related employee relations issues.  It also means fewer industry sponsored educational programs for Primary Care.  Fewer Primary Care clinical trials.   And,  fewer new Primary Care products means Primary Care physicians and their patients will have to be satisfied and content with the treatment options currently available to them.

The real message here is that while Primary Care has been at the foundation of Big Pharma growth and financial success in the past and there may well be exceptions in the future, the importance and interest of Primary Care to Big Pharma is diminishing quickly.  If your expertise or responsibilities include pharmaceutical sales and marketing to the Primary Care market, I believe your days are numbered and you probably have fewer days than you might think.  Specialty products and markets are where the action is and where the industry is headed and it is moving fast.   mike@pharmareform.com

New Job Requires Expertise: Electronic Health Records Clinical Researcher

The US government driven (CMS, Centers for Medicare & Medicaid Services) incentivized push for electronic health records (EHRs) has mostly focused on the business logistics of tracking healthcare delivery and associated costs.  And while the proposed Accountable Care Organization concept deepens the utility of EHRs to include quality and clinical outcome performance metrics they also have implicit goals for managing and controlling costs.  Under the guise of better healthcare at lower cost, my impression is that most healthcare systems are probably looking at this more in the context of making sure CMS or insurers are comprehensively billed and that they have a way to verify billing accuracy and any incentive payments have been rightfully earned.

I wonder if we will ever get to a point of exploiting the clinical information hidden in these electronic data files.   Could EHRs ever lead to better real world data to support evidence based medicine?  With millions of patients in the “real world” data sets over an extended period of time you would think that figuring out “best practice treatment guidelines” would be better served than by a couple of clinical trials with a few hundred or even a couple thousand carefully selected patients studied over a relatively short period of time.  Want comparative effectiveness?  You would think this could be determined with the electronic data on thousands if not millions of patients rather than a small statistically designed trial in a single institution or small number of sites.  EHRs could also be useful for identifying treatment trends or determining where companion diagnostics might be most helpful.

The challenges of HIPAA compliance, research regulations, and bioethical considerations are beyond my area of expertise but I feel it would be a unfortunate if they stood in the way of being able to use this valuable information.  There must be ways to design and execute this type of research without compromising patient confidentiality and ensuring patient safety.  I also appreciate the pitfalls, limitations, and scientific critiques of retrospectively data mining to assess and evaluate clinical data.

The value of EHRs goes well beyond the financial implications and benefits.  To realize their clinical potential the data must be accessible; it must be analyzed and accurately interpreted.  This will require a new breed of clinicians with specialization in the design, execution, and reporting of EHR clinical study data.  Clinical interpretation of the data will require therapeutic area expertise, an appreciation for statistics, and a comprehensive understanding of the data set and the nuances of the data limitations.  These are not part-time jobs but rather new job functions (staffed with expertise) that add cost to healthcare initially with cost benefits coming in the form of more cost effective, better treatment outcomes in the future.

The danger will be in executing poorly designed,  “quick and dirty” reviews and clinical assessments without expertise which can lead to misleading or wrong conclusions and potentially adverse or costly recommendations … purportedly supported by data.   mike@pharmareform.com

More Money Alone will not Increase Pharmaceutical Research Innovation?

While it is hard to argue that you don’t need money to discover innovative new treatments for all the complex diseases that continue to cause illness, disability, and even threaten life.  At the same time, Big Pharma has shown that merely throwing money at discovery research won’t necessarily deliver the results you might expect.

As evidenced by many academic researchers and their teams, it is possible to discovered relevant disease targets and disease altering compounds with far fewer research dollars than Big Pharma has been spending over the past three decades.  Big Pharma R & D budgets, however,  are a misleading indicator of investment in innovation.   In other words, when Pharma holds out the total amount they are spending on R & D ($68 billion), you have to know that only about 30% of that is for discovery and preclinical research.  Still billions of dollars for a disappointing drug discovery return on investment.

Here is another way to look at pharmaceutical innovation productivity.  Let’s say the average Big Pharma has a $1 billion per year to spend on drug discovery and preclinical research.  How do you think that compares to what academic labs (or start up biotechs for that matter) have to spend on discovery research?  Maybe a couple million dollars they have secured in government grants?  Yet, dollar for dollar, who’s delivering the innovation? And why?  An increasing number and percentage of innovative new drugs are being discovered in government or government funded public laboratories.

While they may have less money to work with, academic labs have three essential ingredients that increase the probability for innovative drug discoveries;  expertise, time, and a passionate focus for a comprehensive understanding of the science behind their work (e.g., disease, pathophysiology, biochemistry, and molecular biology).

This is not to say that all Big Pharma researchers lack these essential ingredients.  But even if they do have them, these attributes are mitigated by the distractions of organizational expectations, bureaucracy,  and time pressures to deliver compounds rather than understanding the science.  Perhaps most importantly, expertise in Big Pharma is often rewarded with more work (projects, administrative duties, or increased management responsibilities) that removes (mitigates) the expertise, or at least the focus of the expertise, from the day to day work of discovery research.

Sure, more money can facilitates innovative drug discovery but without expertise, time, and a passionate focus on the science, don’t expect to fill your pipeline.    mike@pharmareform.com

The Impact of Repealing Healthcare Reform on the Pharmaceutical Industry

There is plenty of discussion, debate, legal maneuvering by state governments, and media coverage dedicated to the potential repeal of healthcare reform legislation in the US.  While many feel comprehensive rejection of the bill is unlikely, others suggest there are several components of the reform legislation that should be redrafted or eliminated outright.

Without getting into all the nuances (e.g., implications of electronic health records or accountable care organizations) of the legislation, the major healthcare reform implications for the pharmaceutical industry include:

  • Commitment for fees, rebates, and discounts totaling over $100 billion over 10 years
  • Additional 30 million potential patients with insurance and drug coverage

Agreements and other negotiated benefits for the pharmaceutical industry:

  • 12 years of data exclusivity for biologics
  • No direct government negotiations on pricing
  • No reimportation of less expensive drugs from foreign countries

So, for pharmaceutical companies, does it really matter if the healthcare reform bill is repealed?

To answer this you have to look beyond the next couple of years and any politically driven tweaks to the legislation that might take effect as a result of trying to pacify special interest groups, including insurance companies, advocacy groups, and state governments.  Any near-term implications don’t and won’t change the fundamental realities of where the US and global healthcare markets are trending.  These realities include:

  • plenty of inexpensive generic drugs to treat many mass market diseases
  • an increasingly cost conscious managed market with direct or indirect (mandatory discounts and rebates) price control tactics
  • increasing market expectations for premium priced new products to deliver clinically meaningful benefits over other available therapeutic options (with sophisticated expert reviews of new treatment options)
  • increasing demands for definitive pharmacoeconomic data to support the relative value of premium priced new products

Any near-term changes, repeals, or tweaks to the US healthcare reform legislation will not impact these fundamental market expectations.  Interestingly, the more the US market moves to a single payer model with increasing government involvement, the more these expectations will drive the prescription drug market.

Regardless, I believe the implications of any repeal of healthcare reform will be inconsequential in the context of the long-term business model implications for the pharmaceutical industry.  Yet, it’s scary to think about the amount of lobbying money being spent right now by big drug companies and the industry to influence this legislation.

I’m sure there are also teams of people at pharmaceutical companies right now working diligently trying to forecast and model all the permutations of legislative repeal.  While a necessary exercise (don’t want to miss an opportunity or provide Wall Street with flawed financial guidance), a laborious review could be a huge distraction and probably a waste of time in the context of what needs to be done for the long-term.

The real focus for pharmaceutical companies should be on enhancing and bolstering their discovery research.  In the end, the pharmaceutical industry and drug company success will be determined by finding better more efficient ways to deliver products that satisfy a much more demanding market that has higher expectations for therapeutic benefits and value.  mike@pharmareform.com

The Single Biggest Reason we need Big Pharma Drug Discovery

“I’m sorry we have done everything we can do…there is nothing left to try.”

Nobody wants to hear these words, especially as it relates to our health or the health of a mother, father, son, daughter, close relative, or friend.  Most of us have had people in our lives who have heard these words.  From what should be simple to treat infectious diseases to the complexities of cancers and physically debilitating, if not lethal diseases like Alzheimer’s, there remains a huge medical need for effective and safe new treatments.  Too many people hear these helpless words today and even more may hear them in the future as the population ages with increasing life expectancy.

We have seen how financial rewards can drive  decision making and  behavior in the pharmaceutical industry but really… not wanting to hear these words should be the single biggest reason pharmaceutical companies continue to invest heavily in drug discovery research.

It starts with a mindset to discover truly innovative new drugs that are better than what we have available and that can treat diseases we can’t treat today.  It is frightening that the industry has spent so much in the last decade to deliver so little in terms of innovation.  Think about the billions of dollars spent on clinical trials just to get  “me-too” drugs to the market.  It is equally frightening to think of the cash being spent on mega-mergers and acquisitions to source near-term products to fill the depleted late stage pipelines.  Neither of these contributes to bringing innovative new products to the market that wouldn’t otherwise have come to market.  I’m also not sure how long biotech can support the drug discovery needs of Big Pharma before that well runs dry.

I believe the current “product driven mentality” of many Big Pharma company executives today (and Wall Street analysts) is blinding these companies to the long-term solutions to finding truly innovative new products.  I have said it before.  Drug discovery is hard work and getting harder. It is going to take a much deeper, multidisciplinary understanding of human biology, molecular biology, biochemistry, and pathophysiology of diseases that most companies only think about once they have a potential therapeutic target or drug candidate in hand (most likely acquired from outside the company in most recent history).

I believe this comprehensive approach to drug discovery is where Big Pharma should be making significant investments.  Choose a therapeutic area of interest.  Find, recruit, and collaborate with world class scientific and medical expertise in that therapeutic area.  Invest in an exhaustive understanding of the disease,  explore beyond the known,  and challenge common principles of disease management.  Don’t just look for a compound, look for a comprehensive approach to treating and possibly curing the disease.

Big Pharma is one of the few place with sufficient resources to fund, coordinate, and execute this comprehensive approach over a long period of time.  I am not suggesting universities and biotech companies won’t continue to be a great source of novel, innovative new drug candidates.  In fact, much of the necessary drug discovery expertise now resides in academia.   At the same time however, I am concerned with Big Pharma moving away from drug discovery and relying on universities and biotech as their primary sources of innovative new products.  Why?

Because… I don’t want to hear… “I’m sorry we have done everything we can do…there is nothing left to try.”

mike@pharmareform.com