Life Science Compliance Update

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February 22, 2011

The Difficulties and Challenges of Biomedical Research and Health Advances

Bio Medical Research Lab HGS2 
The breakthroughs in medicine and advances in health care over the past several decades have been miraculous. The biomedical research enterprise in the United States has become the envy of other nations, as well as the primary source of the world's new drugs and medical devices. Many of America’s advances in vaccinations, antibiotics, antipsychotic agents, surgery, and medical devices have contributed to this success and have helped turn uniformly fatal diseases to chronic conditions. This created an expectation that similar advances would occur for other devastating diseases.

As a recent article in the New England Journal of Medicine (NEJM) explained, “these accomplishments led to mutual trust among supporters in Congress, disease advocacy groups, universities, and companies.” In the article, entitled “Biomedical Research and Health Advances,” the authors discuss how the progress and clinical advances that came from these accomplishments “was based on an underlying faith that new technology was valuable and that it would produce effective preventive measures and treatments as long as the translation of basic science to clinical application was sustained.”

However, the authors point out that today, “the primacy of biomedical research and technology development is being challenged.” Specifically, “patients, physicians, insurers, and policymakers are all questioning the slow pace of advance, escalating cost, dubious clinical value, inappropriate commercial exploitation, and lure of false hope for patients with serious diseases.”

In particular, the authors assert that “the mutual trust among patients, clinicians, and researchers is in danger of forfeiture … due to suspicion that economic self-interest is disrupting medicine's compact with patients.” The authors cite “high-profile ethical lapses in the protection of human research subjects and the involvement of physicians in the marketing activities of companies” as examples of this behavior.

The author completely misses the fact that biomedical advances are for the most part incremental.   The criticism that he lobs on physicians involved in marketing misses an important economic point that one must promote a product to make it profitable and thus have enough funds to support future research endeavors.

Biomedical Research in 2011

Over the past two decades, the number of new drugs entering human trials has fallen, especially for new molecular entities and entirely new classes of drugs. To address the challenges drug discovery is currently facing in America, the authors recognize a need for the US to reexamine “funding priorities, open interactions among researchers, and more effective relations among companies, government, foundations, and universities.” They also assert that the American biomedical community must take “stock and recommit its efforts to diseases that have a major effect on the population.”  

For example, while biomedical research in the U.S. is a $100 billion enterprise—65% supported by industry, 30% by government (predominately the NIH), and 5% by charities, foundations, or individual donors—only 4.5% of America’s total health expenditures is on biomedical research. “In contrast, only 0.1% supports research in health services, comparative effectiveness, new care models, best practices, and quality, outcome, or service innovations. This funding will increase to approximately 0.3% from appropriations in 2010 health legislation.”

Private-Public Collaboration is Crucial

As the authors recognize, “research is costly, capital-intensive, and collaborative. Researchers in both academic and industrial settings require access to much the same information, samples and tissue, instrumentation, and specialized technical skills. They also depend on one another as a source of new ideas.” So why are we seeing a decrease in new drug discovery and development?

America has been going through a “decade of growing scrutiny of ties between academic institutions and companies,” which has reduced the productivity of both parties. While sponsors have sought to improve their research productivity through the NIH Roadmap initiative (especially Clinical and Translational Science Awards) that encourage alliances between companies and universities, the authors note that some early models did “not accelerate the pace of either discovery or clinical application.”

One of the main reasons the authors identify for lack of success in public-private partnership is the “failure at inception to agree on intellectual-property provisions, excessive secrecy, and disagreements over research aims.” Moreover, the authors assert that “the most salient reason for failure [in public-private partnerships] is the centralization of authority within large, inherently cautious bureaucracies in government, universities, foundations, and companies. Collectively, such factors inhibit scientists' creativity by disregarding the pluralism of ideas and the diversity of approaches that are necessary for innovation.”

As a result, the authors make seven recommendations to “reestablish public confidence in researchers and clinicians, along with their institutions.” Recommendations that will help establish new models to foster productivity and “go beyond those recommended by the Institute of Medicine, the Council of Medical Specialty Societies, and the NIH,” 

Recommendations

Improve Data on Clinical Value: Develop and apply better objective information about clinical value. This goal implies a higher standard for adopting new devices (including clinical trials similar to those for drugs) and better information on the effectiveness of existing drugs and devices, especially data that are available only from proprietary insurance databases. Also, new incentives for private and government insurers to disclose clinical data to researchers.

Change the Role of Teaching Hospitals: Modify the roles of academic health centers and teaching hospitals to improve their ability to conduct early-stage (proof-of-concept) clinical trials. Two very different approaches should be tried: creating patent pools involving multiple companies and universities and a renunciation of patenting in return for more latitude to conduct high-risk laboratory experimentation and initial clinical trials. 

Develop New Models for Collaboration and Financing: Consider new models of collaboration and cooperation that would allow the NIH to concentrate on basic biomedical science and large, multi-institutional projects, where its scale can be most valuable, while providing offset to industry's declining investment in research. These models might include the following:

-       Establish Biomedical Innovation Trusts: A new nonprofit, public–private partnerships — biomedical innovation trusts — could enable individuals and corporations to receive immediate federal tax credits (not deductions) for contributions to support research in high-priority diseases. Similar tax incentives have been used historically to preserve land, create parks, and build factories.

-       Create a New Class of Bonds: States and the federal government might issue bonds to support innovation in biomedical science and health services, with preference given to high-risk research and diseases important to public health. Such bonds have long been used to support athletic facilities, airports, and roads. They provide a mechanism for private investment to meet public needs.

-       Use Incentives to Promote Pluralism: Give preference in funding to new research institutes or entities, rather than existing universities or companies.

-       Defer Patents to Later in the Discovery Chain: In return for new sources of funding and greater latitude to conduct high-risk research, the new entities would forgo claims to patents or other intellectual property and place positive and negative findings immediately in the public domain.

Renew Professional Commitments: All physicians must renew their commitment to professionalism and their duty to their patients by affirming objectivity in medicine, with open communication and an absence of bias, and a blanket proscription of product promotion in any guise.

Focus on Cost-Effective Targets: Recognize that new technology creates value to the general economy and has many clinical benefits. This requires incentives for researchers to focus on diseases that are common, cannot currently be prevented or effectively treated, are expensive, and have a major effect on the patients' health.

Adopt Realistic Research Goals: Embrace a new realism about the difficulty of the scientific process and what can (and cannot) be expected from it.

Redefine the Terms of Conflict: Inevitably, we face growing conflict over individual choice, access to the latest drug or device, the true cost of technology over a lifetime, perceptions of value, and preferences for competition versus regulation. Not everyone believes biomedical research is essential.

Discussion

There are a number of concerns with some of the recommendations. First, to recommend adopting higher standards, while fundamentally sensible, ignores the common pathway by which breakthroughs typically improve health.

Major discoveries are at first generally dismal in performance and outcome. Insulin and transplantation are two examples. The ability to accumulate large clinical cases and adjust from these is what typically leads to rapid improvement of novel and imperfect ideas. If the standard for early adoption becomes too high, early failure and ultimate success will be lost.

Second a “blanket proscription of product promotion in any guise,” if taken literally,
prohibits promotion of products/interventions/cures that have met the scientific burden of proof and have been validated as superior therapeutic options. Not to recommend the details of better medical practice, in any forum, insults the very notion of professionalism.

Finally, under the “focus on cost-effective targets” recommendation, the authors should realize that technological advances and breakthroughs are only marginally more expensive in the early stages, especially while under patent, but in relatively short periods, prices plummet and utilization skyrockets, delivering extreme efficiency and cost-effectiveness. Advances such as the X-ray, CT scan, and vaccinations were at first rarely used, but today they are examples of basic level of care for virtually all patients. Thus, technological advances will “ensure a basic level of care for all citizens and a reduction in the rate of increase of aggregate health care spending” that the authors discuss. 

It is interesting to note that the author completely ignores the role of regulatory agencies in drug development.  If the bar is too high for FDA approval in a therapeutic area or patient law suits are too expensive to litigate, companies will not develop products in those high risk areas.  A good example in the area of antibiotics where because of the limited return on investment and regulatory problems associated with antibiotics, research for the next generation is seriously limited.   

Conclusion

Governments need to seriously consider how to get out of the way of innovation; all the government funds in the world won’t replace private sector ingenuity.  It is certainly crucial that public-private partnerships are encouraged to reinvigorate the discovery and development of new drugs and devices to treat diseases that affect millions of Americans.

Those recommendations that encourage public-private partnerships are important to consider, and policymakers and federal health officials should begin discussing these proposed incentives to recharge the funding and interest in biomedical research before companies, tax dollars, and budgets continue to flow offshore to other companies and foreign citizens are the first to benefit from medical breakthroughs and discoveries.

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