By Marc Boutin, JD, Executive Vice President and Chief Operating Officer, National Health Council

At this time of the year, it can be helpful to reflect on the past to guide us in the future. I was recently reading an editorial about the National Health Council (NHC) printed in January 1957 – 37 years after the organization’s creation. It spoke about the need for action with “an unprecedented degree of cooperation among health agencies and the people they serve.” Well, that time is now and the issue is reauthorization of the Prescription Drug User Fee Act (PDFUA).

Marc Boutin, JD, Executive Vice President and COO, National Health Council

About a year and a half ago, at a Food and Drug Administration (FDA) meeting with patient advocacy organizations, we talked about how it was patients, two decades earlier, who chained themselves to the gates outside the FDA and the National Institutes of Health demanding access to treatments that were still in clinical trials and under review. Patients were told that they couldn’t have access to those treatments because researchers didn’t know if the compounds were safe or effective. But the patient community pushed back; many were going to be dead in a year. Their actions resulted in the creation of the early access program and a new environment that led to the first PDUFA.

I want to thank the FDA and the pharmaceutical industry for again hearing the concerns of patients and for addressing our issues in a meaningful way. Today, we have a forward-thinking reauthorization agreement – PDUFA V.

Since the authorization of the first PDUFA, we have seen tremendous progress in the development of medicines for people with chronic diseases and disabilities. The time it took for the FDA to approve new drugs got shorter. However, there is now an emerging frustration among many people with chronic conditions who feel they still are not getting access to treatments as quickly as they would like. For many, such as patients with degenerative diseases like Alzheimer’s, clinical development moves slower than the progression of the disease.

For the past year and a half, the patient advocacy community has worked with the FDA and industry to get its three key priorities included in the proposed reauthorization agreement.

First, we need a qualitative, objective, framework for assessing the benefit-risk of new drugs. Benefit-risk is an important part of the FDA review process, but there is no consistently used framework or agreement among all stakeholders. How do we account for known and unknown risk or variation among subpopulations? Are benefits and risks appropriately placed in the therapeutic context?

No country in the world has articulated such a process, and there is increasing anxiety among patients who are denied access to new drugs based on a benefit/risk assessment they simply don’t understand.

The FDA and industry have agreed to include in PDUFA V the creation of a qualitative framework that would assess the level of certainty and variability of the benefits and risks, and that would provide for flexible scoring in the context of the therapeutic indication.

This benefit-risk framework will work for both patients and consumers. Both are stakeholders in this issue, but depending on where you fall on the health care needs spectrum, your perspective of benefit-risk can shift dramatically. For healthy consumers, the tolerance for risk, variability, and uncertainty for a new medicine to treat a condition such as hay fever is virtually zero. But if you are diagnosed with ALS (Lou Gehrig’s disease) and you have only two years to live, your tolerance for a risky new drug is dramatically different.

Second, we need greater use of patient-reported outcomes and biomarkers. Patients want more of a say in the drug review process, and they want the approval process to go faster than the progression of their disease. The patient community saw the reauthorization of PDUFA as an opportunity to develop metrics and tools to make the review process more effective, more efficient, and allow for the delivery of safe and effective medicines to people who need them.

Under PDUFA V, resources for patient-reported outcomes (PROs) will be increased, resulting in the patient perspective being incorporated earlier in the review process, dramatically reshaping drug research. It was psoriasis patients who taught us that it is not necessarily the size of their lesions that matters, but where the lesions are located on their body, such as on their faces or joints. This realization greatly altered the focus of psoriasis drug research.

PDUFA V would also increase resources to help the FDA speed up the approval of biomarkers in clinical trials. The use of these surrogate endpoints or clinical markers is greatly needed for people with chronic conditions. Polycystic kidney disease (PKD) is a genetic disorder of the kidneys. With PKD, fluid-filled cysts develop in the kidneys, which then can increase in both size and weight, sometimes weighing many pounds each. Currently, the recognized end point for PKD is kidney failure, which can occur as late as 40 years after diagnosis. No company would undertake a 40-year clinical trial, so qualifying a biomarker for a PKD drug is imperative. It is conceivable that if a new compound prevents a PKD patient’s kidney from getting larger, then something positive is happening.

Third, for the millions of people with rare diseases, we need new resources and greater flexibility in the regulatory review process. As proposed by FDA and industry, PDUFA V will utilize new regulatory science to speed the development of and access to new medicines for people who desperately need them.

The opportunity to engage in the PDUFA reauthorization process has been tremendously beneficial for the patient advocacy community, and I hope for the FDA and industry.

I say to those reading this column and to members of Congress, let’s get PDUFA V approved. Let’s get it done fast.

By Mary Woolley, President of Research!America

Research!America’s 12th edition of America Speaks, an annual summary of our public opinion polls, shows that Americans are deeply concerned about our country’s ability to create jobs and compete globally. In fact, 77 percent of those polled say that the U.S. is losing its competitive edge in science, technology and innovation and more than half of Americans (58 percent) do not believe we are making enough progress in medical research. Furthermore, the majority of Americans believe investing in health research (86 percent) is important to job creation and economic recovery.

Mary Woolley, President
Research!America

So much is at stake right now: our economic future, U.S. jobs and our global leadership. In this election year, we must make advancing health research a priority and part of the national dialogue.

As federal funding tightens, we are compromising discovery and the development of new products, which is important to patients and businesses alike. Incentives for industries to conduct research, including efforts to make the research and development tax credit permanent, are supported by many Americans. And most are willing to support higher taxes for research. Half of those surveyed said they would be willing to pay $1 per week more in taxes if they were certain their money would be spent for additional medical research.

Our public opinion polls indicate that Americans want viable solutions to improve our health, health care system and the economy. They see research as part the solution to rising health care costs and would like to see more of the health dollar dedicated toward research.

Similarly, careers in science, technology, engineering and math (STEM) continue to receive broad support among Americans who believe the federal government should place more emphasis on increasing the number of young Americans who pursue these fields.

Other findings in America Speaks show:

• 91 percent of Americans believe that research and development are important to their state’s economy;
• 84 percent of Americans think it is important to invest in regulatory science to ensure the safety and efficiency of the drug and device development process; and
• 87 percent of Americans think it is important that elected officials at all levels listen to advice from scientists and public health professionals;

These findings provide validation that Americans do not want to settle, and see their country fall behind in research and innovation. They want their country to remain resolute in its commitment toward advancing science and innovation.

As we face more budgetary challenges for health research in 2012, the community of stakeholders in research must continue to make the case that investing in health research will not only support the health of our nation, but also the prosperity of our country for years to come. Your voice from the front lines of science is essential in conveying what we could gain or lose with a robust or flat federal health budget and a sound and globally-competitive policy structure.

I invite you to take a look at our poll data summary, America Speaks, Volume 12 and to join the conversation about making research a priority this election season. Candidates who are running for office this year must hear from scientists and other stakeholders in the science enterprise; those who are elected without hearing from scientists are unlikely to become champions for science once they take office! Tell candidates why science is the key to better, more affordable health, and is key to driving the US economy. Please visit: http://www.researchamerica.org/uploads/AmericaSpeaksV12.pdf

Mary Woolley is the president of Research!America, the nation’s largest nonprofit public education and advocacy alliance working to make research to improve health a higher national priority.

By David Welch, President/Senior Producer at M2 MultiMedia

When the HIV/AIDS epidemic became widely known in the early 1980s I lived in San Francisco. I lost many dear friends during those years. That made working on “HIV/AIDS and Biotechnology” very personal to me.

Thirty years later and despite an all-too-common public perception that this terrible disease has been solved, the research to find a vaccine is actually more important than ever. The sad truth is that the cost in both human lives and economic treasure remains astronomical.

Producing this short film for BIO about the role of biotechnology in the on-going fight to save lives and dollars brought me face-to-face with three awesome individuals: a researcher, a patient, and an advocate. The researcher is Dr. David Asmuth, one of the world’s leading HIV experts. The patient, Brian Brown, received VACC-4X, a biotech vaccine produced by Bionor Pharma, in a clinical trial and went a full 18 months without anti-retroviral treatments. The advocate is Stephen Bailous, executive vice president of the National Association of People with AIDS (NAPWA) — he’s also an HIV survivor.

Together, these three men send a compelling message to all of us: If we are to conquer HIV/AIDS the necessity to fund sustained biotechnology research has never been more vital and more hopeful.

I wish to thank our entire team, especially our director Emily Deckelman, for their commitment to producing this important video.

David Welch is President/Senior Producer at M2 MultiMedia, a science oriented video production company in Rockville, MD.  His email is dwelch@m2inspired.com

Bits of technology from I.B.M.’s Watson are beginning to be used in actual products available to corporate clients. The cloud-based Strategic Intellectual Property Insight Platform “uses data mining, natural-language processing and analytics to pore through millions of patent filings and biomedical journals to look for chemical compounds used in drug discovery.” As a byproduct of this project, I.B.M. is contributing their research to the searchable NIH chemical database, which will ease access to information previously mired in paperwork.

Read more on Watson’s new job in the New York Times.

The Food and Drug Administration has given Canadian researchers approval to test a vaccine for HIV/AIDS on humans. Researchers from the University of Western Ontario are hopeful that with further tests, a vaccine could be on the market in about five years. Similar to the approaches used to develop vaccines for polio, influenza, rabies and hepatitis A, the vaccine is the first based on a genetically modified, killed whole virus and is the only HIV vaccine currently under development in Canada, and one of the only few in the world.

Beginning next month, a clinical trial on 40 HIV-positive volunteers will begin.  Following the trials, tests will begin on 6,600 HIV-negative but high-risk category volunteers. These tests will focus on immune responses and effectiveness of the vaccine in two more phases.

To read more about the University of Western Ontario’s AIDS vaccine, please follow this link.

By David Welch, President/Senior Producer at M2 MultiMedia

One of my closest childhood friends suffered from Type I Diabetes. Like millions of other children and adults his daily life was frequently interrupted with finger pokes, sugar level tests, and insulin shots. Having grown up with a friend afflicted with Diabetes and knowing dozens of others, throughout my life, with the same disease, it was very rewarding to produce a film on the topic. Although Bobby and I have lost touch over the years I hope he would be proud of our latest video, Diabetes and Biotechnology: A Shared History…A Great Future. In three minutes the video tells the pertinent story of how recombinant DNA technology was used to produce human – or synthetic – insulin in large amounts and how it became the first biotechnology treatment approved by the FDA.

I am thankful to Dr. Richard A. Insel, Chief Scientific Officer of the Juvenile Diabetes Research Foundation, for his incredible insight and for discussing in detail the historic connection between insulin and biotechnology. I am equally thankful to Kerry Morgan, a vivacious 17-year high school senior, who provides viewers with a very personal look at living with Diabetes. Kerry served as a JDRF Children’s Congress Delegate this past summer and actually spoke to a United States Senate committee.

Today, 30 years later, there is still no cure for Type I Diabetes. Recently published figures put the number of those afflicted with Diabetes at 350 million. By 2030, according to Dr. Insel, that number will climb to 500 million. As these cases approach astronomical levels it is essential we continue to support biotechnology research and hopefully one day reach an end to this very dangerous disease.

David Welch is President/Senior Producer at M2 MultiMedia, a science oriented video production company in Rockville, MD.  His email is dwelch@m2inspired.com

Holland & Knight’s Michael Werner talks to Dr. Russell Medford, President and CEO of Salutria Pharmaceuticals, a privately-held biotech company based in Alpharetta Georgia. Dr. Medford is also co-chair of BIO’s Board Standing Committee on Bioethics. Dr. Medford will be providing testimony on Wednesday November 16 to the Presidential Commission for the Study of Bioethical Issues on Principles Regarding Ethical Performance of International Clinical Trials.

By Dr. Russell Medford, Salutria Pharmaceuticals

Recent reports have raised concerns about how clinical trials are being performed in other countries, particularly in the developing world. Even prior to these reports, some have asserted that sponsors conduct clinical trials abroad to avoid red tape and avoid overly stringent and time consuming regulatory processes. However, research done by companies with the intent of seeking FDA approval and access to the U.S. market must still meet FDA standards regardless of where the research is conducted. FDA regulations require trial sponsors to undertake several quality assurance and audit activities, and failure to abide by these rules could delay or disqualify a product’s chances of getting marketing approval.  Companies develop numerous processes to ensure compliance and protect patient safety, including:

• Pharmacovigilance conducted by experts (oftentimes hundreds of individuals in one company) who collect, analyze, follow-up, and distribute safety-related information from new drug trials.
• Establishment of data and safety monitoring boards to impartially evaluate safety as studies progress.
• Regular sponsor monitoring of clinical trial sites to ensure quality, compliance, and safety of research participants.
• Quality assurance units that audit operations associated with clinical research to ensure adherence of company personnel to regulations, guidelines, and internal and external procedures, policies, and programs.

Unethical clinical trials, however rare, tarnish the reputation of all researchers and their work. BIO members are deeply concerned about any instance of unethical clinical research, and take seriously the ethical issues involved in conducting overseas clinical trials. BIO has long supported responsible and ethical testing, protection of individual privacy and genetic information, and regulatory systems that best serve humanity and advance research into new treatments for patients, regardless of where research is conducted. Toward that end, BIO supports appropriate oversight of clinical trials and medical research, whether conducted in the U.S. or elsewhere in the world. Performing important research and protecting research subjects are mutually attainable goals, and decisions regarding whether and how to use medical products and technologies must always be made with profound respect for the rights of patients. Appropriate regulation of biotech should be solidly rooted in values such as autonomy, privacy, beneficence, social justice, and intellectual freedom. Encouraging and enabling innovation through a timely and consistent regulatory process will ultimately result in medical treatments and cures for some of the most devastating and life-threatening diseases.

November 12 marks World Pneumonia Day, a campaign to educate the public about this disease and to advocate for global action to treat and prevent it. Pneumonia is the leading cause of death globally for children under the age of 5, claiming the lives of more than 1.5 million children annually. Pneumonia is also largely a vaccine-preventable disease. Since the introduction of pneumococcal vaccines in the U.S. in 2000, morbidity and mortality from pneumonia have decreased. Vaccines against haemophilus influenza type B (Hib) and measles have also reduced the incidence of pneumonia. However, a similar impact has not been felt in the developing world, where there is still limited access to these life-saving vaccines. The Pneumococcal Accelerated Development and Introduction Plan (PneumoADIP), financed by the GAVI Alliance, is a program to accelerate the evaluation of and access to pneumococcal vaccines in poorer parts of the world, with the goal of saving 5.4 million children from pneumonia by 2030.

While initiatives such as World Pneumonia Day and PneumoADIP serve the important purpose of addressing the impact of pneumonia on children, we must not forget about the impact of this disease on adults, particularly the elderly. In the U.S. each year, there are an estimated 175,000 hospitalized cases of pneumococcal pneumonia. According to the Centers for Disease Control and Prevention (CDC), invasive pneumococcal disease causes more than 6,000 deaths annually and in more than half of these cases, vaccination against pneumococcal disease was recommended. All adults over the age of 65 who lack evidence of immunity are recommended to receive pneumococcal vaccination by the CDC, as are younger adults with certain risk factors. Despite this recommendation and full coverage of the vaccine under Medicare Part B, only 60.6% of adults 65 years and older received the vaccine in 2009.

On November 12, World Pneumonia Day, each of us should consider vaccinations against pneumonia for our loved ones, young and old, and for ourselves, if appropriate and recommended. Vaccination is the best protection that we have against this serious disease.

Recently, the Foundation Fighting Blindness announced an $8.25 million investment in six new gene therapy research projects targeted to have treatments ready for clinical trials within three years. The projects include the following:

• The Institut de la Vision in Paris and the Friedrich Miescher Institute in Basel, Switzerland, are developing a gene therapy that may revive degenerating cones and potentially enable them to regain their ability to respond to light and provide vision. The treatment also has the potential to improve the health of cones and extend their lifespan significantly. This therapeutic approach holds the potential to benefit people affected by a range of conditions, because it works independently of the underlying disease-causing genetic defect. Resurrecting cones may be able to improve an affected individual’s well being, because these cells provide central, daytime and detailed vision that is critical for independent living.
• The Oklahoma University Health Sciences Center, which in collaboration with Copernicus Therapeutics, is developing a nanoparticle gene therapy delivery system. Nanoparticles are tiny manmade particles, 1/12,000^th the diameter of a human hair, which can potentially penetrate retinal cells, possibly making them effective for delivery of therapeutic genes. They may provide advantages in certain cases over viral gene delivery technologies currently used in retinal disease therapies. Perhaps most beneficial is their potential to deliver large genes — genes that exceed the capacity of viral delivery systems — for treating some diseases.
• AGTC, a clinical stage biotechnology company, funds will support researchers at Oregon Health & Science University’s Casey Eye Institute and the University of Florida in their pre-clinical work to evaluate a gene therapy treatment for X-linked retinoschisis, a blinding disease that affects over 35,000 patients in the U.S. and Europe.
• The Massachusetts Eye and Ear Infirmary and the University of Florida are investigating gene therapy for two different LCA-causing genes.
• The University of Pennsylvania is working on choroideremia gene therapy led by Dr. Jean Bennett, who is also one of the lead investigators on the landmark LCA gene therapy clinical trial that has restored vision in more than 40 patients.

The Foundation Fighting Blindness is a national nonprofit dedicated to advancing sight-saving research. The Foundation funds research toward preventions, treatments and cures for vision-robbing retinal degenerative diseases including macular degeneration, retinitis pigmentosa, Usher syndrome, and several other rare conditions. These newly-funded projects build on the success of landmark gene therapy clinical trials now underway that have restored some vision in children and young adults who were virtually blind.