The process of clinical research and new drug development is getting more complicated and time consuming despite the advances in communication and digital technology. A reform is more urgent than ever now, since the trends in the government seem to be to discourage the use of expensive new drugs and medical devices. Such policy is not a good incentive for the small and large biotechnology companies to come up with new agents needed for the treatment of chronic diseases. Likewise, there would be fewer funds available to be spent on basic research, since the prospect of future financial gains from the applications of such research would be minimal.

Predictably, federal funding for all basic and translational research will be curtailed. Although this is not spelled out clearly by the new administration, it can be safely inferred. Likewise, the private sector, including the venture capitalists, will be left with little reason to invest dollars when the prospect of return is not there. Ultimately, the critical question of why are we at this crucial crossroad arises and begs for an answer.

Undoubtedly, the cost of new drugs and gadgets is draining the health care budgets of Medicare, Medicaid, and the private insurers, so much so, that necessary cuts are made in the critical services especially those to do with access to physicians' care. The pharmaceutical industry cites the cost of new drug development as the main culprit for the high cost of new drugs and there is some partial truth to that. But the cost of marketing new drugs consumes more than 40% of the average drug company budget, while the fund allocated to research and development has plummeted recently to as low as 17% of total pharmaceutical companies’ expenditure.

In these critical and difficult times a give-and-take strategy can only help our patients and all parties involved. To demand change and adjustment from one side would not only be unfair, but rather counterproductive. Let’s look at each entity and propose changes that can be fair and equitable.

Economists (my son, Paul, included) would suggest that during down times companies should spend more money on marketing and advertisement. Yet, drug companies are usually quick to cut back on their sales force and advertisements when their bottom line suffers. This indicates that they are willing and able to take such measures when needed; they just need to be enticed to do so.

The new drug development process is not only expensive, but is infested with redundancies and fat bureaucracy--so fat that bariatric surgery is needed. It takes more than 8 years of research and in excess of $900 million to get one single drug to the market. The "bean counters" in the pharmaceutical industry know that time is "gold" and each extra day spent in developing a new drug is money lost. Factors that slow down the process logically translate into more money spent in the process, and that money has to be retrieved when the drug is approved. That is logical and fair.

Take for example the process of recruiting patients to clinical trials; not an easy process since less than 13% of U.S. physicians are involved in clinical research; moreover, less than 3% of all patients are enrolled in clinical trials. That says a lot about the process and sheds light on how to improve the process to the benefit of all. Most physicians cite excessive cumbersome paperwork, accountability, and regulatory demands as the reason for shying away from the crucial process--a process that is critical in present and future patient care. Our cancer center has been heavily involved in clinical research for the last 10 years and I attest to the validity of those reasons.

Anecdotes can be useful. They are like a picture that is worth a thousand words. A year ago we decided to participate in the Cougar clinical trial where Abiraterone is used in a placebo controlled trial for patients with a castration refractory stage 4 prostate cancer who have failed prior chemotherapy. This is a dire situation indeed, where there is nothing else to offer. Accrual to the trial nationwide had been slow at best. A few months ago, NBC decided to air the subject with an interview with some patients and one of the principle investigators in the Cougar trial. The result was beyond imagination. Accrual rate quadrupled with patients calling and traveling more than 250 miles seeking enrollment in the trial. Patients are happy and so is the Cougar team.

The NBC piece cited the initial phase II trial done in the U.K. for chemotherapy naïve prostate cancer patients with an overall response rate in excess of 80%. This is remarkable indeed, but regulatory agencies were not happy with the NBC piece because it hinted to “unrealistic hope” and the principle investigator was in hot water because he was part of the program.

An anecdote spurred my thoughts and initiated this writing. A patient who saw the NBC program called his oncologist and inquired if he would be a candidate for the trial. He was then referred to us. He was enrolled and he is happy that his PSA has dropped from 76 to 0.54. In addition, his repeat CT scan showed a remarkable partial response with absolute pain relief.

The patient’s son who is a high school principal confronted me with logical questions such as, “Why should we rely on the media who scarcely air stories like this? Why didn’t the treating oncologist tell the patient about this trial which seemingly is helping the patients? And, why should the FDA and other regulatory agencies be against such publicity?” His dad had no hope of anything else working, since he had received both Taxotere and Mitoxantrone, the only two agents known to be effective for his disease.

In dire situations like this, any hope is better than nothing and to claim that publicizing such trial is potentially harmful to patients is a far-fetched proposal. It is predicted that the trial will be completed ahead of schedule. If positive, tens of thousands of patients will benefit. If negative, we move on to the next trial or drug under development.

At a time when clinical trialists and pharmaceutical sponsors are frustrated with the slow enrollment, we seem to continue to adhere to antiquated principles that are hardly relevant to patient’s safety or psyche. The Geneva Convention and the Helsinki accord were well meaning at the time when human experimentation during World War II were not only extreme, but criminal. We are in a different era now. We have elaborate consent forms, a comprehensive consenting process, and ethically sound institutional review boards where patient’s safety is well-guarded and alternative therapies are explained thoroughly to patients in the consenting process.

In fact, I can safely argue that the regulations designed and implemented to safeguard patients are hurting those same patients through limited access and obstruction to inform them about the hopeful options they have available to them. While only a small minority of physicians is willing to take on the task of clinical research, the majority don’t. Sometimes they are even obstructive to the process fearing that they will “lose” their patients.

Although patients and families are increasingly becoming internet savvy, and although www.clinicaltrials.gov lists all the FDA approved trials, this doesn’t seem to be enough based on our experience and the national statistics alluded to earlier.

Many (or rather, most) pharmaceutical sponsors are resorting to clinical research offshore. This can and does pose serious quality issues, since the FDA access to many countries is limited which can potentially cast shadow on the conduct of clinical research results. Moreover, this outsourcing does have economic deleterious effects to the U.S. economy. More serious, it is limiting access to our patients who, in my opinion, deserve the best health care, including their access to promising cost-saving treatments through the clinical trial programs.

We all agree to that our patients have the right to know their options, and it is incumbent on all of us that information about treatment options--including the option to participate in clinical trials--is readily available to them through more liberal advertisements of those trials with less restrictions than what we have now. In fact, I propose that national media should be persuaded to allocate regular ads promoting clinical trials at no charge. Alternatively, we should allow pharmaceutical companies to sponsor such ads, commercially, with reasonable restrictive guidelines.