Some recent media coverage of the biopharmaceutical industry has painted something of a doomsday picture — patent cliffs, stagnant pipelines, the end of the blockbuster era, etc. In reality, the prospects of the biopharmaceutical sector — for patients in search of new medicines, for policy makers seeking value in the health care system and for American workers in need of high-quality jobs — are bright and promising.

Pharmaceutical Research and Manufacturers of America, PhRMA, John Castellani, biopharmaceutical industry, biopharmaceutical sector, biopharmaceutical research, biopharmaceuticals, pharmaceutical, Prescription Drug User Fee Act, PDUFA, PDUFA-V, pharmaceutical industry, health care, adherence to medicines

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Inside This Issue - Opinion

Outlook for biopharmaceutical sector is bright

January 2nd, 2012
by John Castellani

Some recent media coverage of the biopharmaceutical industry has painted something of a doomsday picture — patent cliffs, stagnant pipelines, the end of the blockbuster era, etc. In reality, the prospects of the biopharmaceutical sector — for patients in search of new medicines, for policy makers seeking value in the health care system and for American workers in need of high-quality jobs — are bright and promising.

JOHN CASTELLANI is president and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA).

While challenges lie ahead, the biopharmaceutical research sector remains one of the most innovative and productive sectors in America’s health care system and its economy.

Over the past decade the Food and Drug Administration approved more than 300 new medicines to help patients combat a variety of conditions. Today around 3,000 more are being developed by America’s biopharmaceutical research companies, including 300 for cardiovascular disease and stroke, nearly 900 for cancer and nearly 100 for Alzheimer’s disease.

Scientific advances and new technologies have dramatically changed how medicines are discovered. For instance, screening tools, which help researchers quickly sort through millions of compounds, have reduced the time and cost associated with discovering new medicines.

Other technologies are leading to more efficient delivery of drugs to the patient. And greater knowledge of how diseases work at the genetic and molecular level has enabled researchers to pursue new targets for therapy and better predict how certain biopharmaceuticals will affect specific groups of people.

This progress — and the companies that drive it — represents real hope for patients but only if we build and nurture an innovative ecosystem in America that better fosters new medical research and ­development.

Ultimately we all share the same goal: providing patients the medicines they need to become and stay well. In the coming year, the Pharmaceutical Research and Manufacturers of America (PhRMA) will continue working with a broad range of stakeholders to build upon the four key pillars I believe are needed to support a world-leading American biopharmaceutical research sector for decades to come.

Regulatory Framework to Spur Innovation

The first pillar is building a 21st century regulatory framework that efficiently enables the translation of innovations in science and technology into medical products that address unmet medical needs.

A modern regulatory framework will help assure that innovative therapies are brought to patients in a timely and predictable manner using only the best science to protect and promote public health. The state of science has advanced dramatically in recent years, and it’s imperative that the FDA has the resources and support it needs to keep up.

A 21st century regulatory framework will help provide patients with timely access to new medicines while continuing to maintain the highest of standards for safety, efficacy and quality. The patient must remain as the focus for medical product development, regulatory review and approval. Use of a more structured and transparent approach to benefit-risk assessment in regulatory decision-making will help ensure patients’ needs and wants are represented.

A more modern regulatory framework that applies only the best sciences and relies on a more collaborative and networked approach will also inspire and further incentivize greater investment in the research and development that leads to revolutionary new treatments.

A 21st century regulatory framework will also help enable a more streamlined drug development process with enhanced potential for increased success at bringing novel medicines to patients This also means a more predictable business environment for the biopharmaceutical sector — the type of environment venture capitalists and other investors have said is needed to continue to support the research in new, game-changing therapies.

Reauthorization of the Prescription Drug User Fee Act (PDUFA) is a great step in the right direction. If implemented as published, PDUFA-V will help ensure that the drug approval process reinforces science-based regulatory decision-making, optimizes the drug development and review process and — perhaps most importantly — enhances the public’s trust in the drug review process, the FDA and the biopharmaceutical industry.

The PDUFA-V agreement will provide the FDA with much-needed resources and management tools to support patient safety and promote innovation through increased transparency and predictability and the associated performance goals will make the process more efficient, reducing the time it takes for patients to access the medicines they need. Further, adoption of a structured benefit/risk framework, the first steps of which are included as part of PDUFA-V, will allow for fuller consideration of the benefits and risks of new treatments.

Public-Private Collaboration

The second pillar needed for a successful biopharmaceutical research sector future is a flourishing, collaborative scientific ecosystem that involves public and private sectors. Academic and government research organizations are critical partners for the biopharmaceutical research industry — and it is only through partnering that we will be able to tackle the unprecedented scientific challenges ahead.

Such partnerships must be based on an understanding of the importance of collaboration and information sharing among researchers and clinical experts in the interest of better patient care. Critical is the shared understanding that scientific discoveries are not made overnight; most successes are only possible because of the failures that have preceded them. In other words, perseverance and patience are paramount.

There is perhaps no greater example of the slow — but steady — progress made in biomedical discovery than research related to the HIV/AIDS virus. It has been three decades since HIV/AIDS was first mentioned in a medical journal. At that time, essentially nothing was known about a disease that was starting to take a frightening number of lives. We still need a cure to fight this brutal disease, but thanks to scientific discovery driven by industry in partnership with academia HIV has essentially become a chronic condition, not a death sentence.

The Business Dimension

Not only do we need a scientific environment committed to innovation we also need a business environment that equally embraces biomedical progress. That is the third ­pillar.

According to the Congressional Budget Office, “The pharmaceutical industry is one of the most research-intensive industries in the United States.” The biopharmaceutical sector invested $67.4 billion in research and development of new medicines in 2010. This is double the entire budget of the National Institutes of Health.

Our industry supports around 675,000 direct jobs and more than 4 million total jobs nationwide. The biopharmaceutical sector’s work is vital to our economy. For us to remain successful, policies that reflect the unique challenges inherent in biopharmaceutical discovery are essential.

Of every 10,000 compounds examined, only five will make it to clinical testing — and only one will gain FDA approval. And once medicines make it to market, only two in every 10 on average earn revenues large enough to offset average research and development costs.

With such a high level of risk, investment and resources, there must be appropriate incentives to continue innovating. For example, rigorous intellectual property protections are essential, as are R&D tax credits and, as discussed, a more modern and predictable regulatory review process.

Role in Health Care

The final pillar to help support America’s biopharmaceutical industry is a health care model that properly values the role medicines play in preventing disease.

Our nation faces exceptional economic and health care challenges, and it would be impossible to adequately solve them without a better appreciation for the value of medicine within the health care system. While prescription medicines represent only a little more than 10% of every health care dollar spent, they encompass roughly 40% of patients’ out-of-pocket expenses. This reflects a disconnect between health care payment models rooted in 20th century acute care and our current capabilities to prevent disease and avoid hospitalization through better use of medicines.

In an era when we have advanced therapies and increasing disease management capabilities, prevention of disease has to be a priority.

The evidence for this keeps coming. Two recent Harvard studies, for instance, highlight the value of medicines in limiting health care costs. The first, published in the Journal of the American Medical Association, found access and adherence to medicines driven by Part D saves Medicare about $1,200 per year in hospital, nursing home and other costs for each senior who previously lacked comprehensive prescription drug ­coverage. A follow-up study, published in the American Journal of Managed Care, reported that nearly 11 million senior citizens gained comprehensive prescription drug coverage through Medicare Part D. Based on the study published in JAMA, the authors of the second study concluded that the overall Medicare savings in 2007, the first year Part D was implemented, was equal to roughly $13.4 billion in 2007 — more than one quarter of Part D’s total cost that year.

Other studies have come to similar conclusions, showing that appropriate use of medicines leads to better health outcomes and savings on costly hospital and emergency care.

These four pillars are interdependent. If America is to retain its global leadership role in biopharmaceutical research, we need a modern, predictable regulatory framework, a collaborative ecosystem that leverages the unique strengths of the private and public sectors, robust incentives to encourage future innovation and a health care payment model that properly values prevention of ­disease.

We have the science to propel medical care to previously unfathomable levels of targeted care. The question is: Do we have the collective fortitude and commitment to fully realize this promise, or will we cede the advantage to other countries?