Malaria-free mosquitoes created

Malaria is caused by a parasite called Plasmodium, which is transmitted via the bites of infected mosquitoes. The Anopheles species of mosquito is the only breed of mosquito that transmits the parasite to humans. According to the World Health Organization, about half of the world’s population is at risk. Every year, this leads to about 250 million malaria cases and nearly one million deaths

The strategies to reduce malaria transmission include mosquito-control measures, such as spraying insecticides inside houses and draining standing water where mosquitoes lay their eggs. Also, the mosquito bites can be prevented by using inexpensive mosquito nets and insect repellents. Unfortunately, these methods are becoming less effective as both mosquitoes and Plasmodium evolve ways to resist the toxic treatments. This means new ways of preventing malaria are sorely needed.

Now, new research has brought hope to all those living in regions affected by the disease. According to a study published in PLoS Pathogens, scientists led by Dr. Michael Riehle have managed to alter the mosquitoes’ genome in such a way that the Plasmodium parasite is no longer able to cause infection. The genetically-modified mosquitoes will still bite. However, they won’t leave behind Plasmodium, the malaria-causing parasite.

This advance could lead to the release of genetically-modified mosquitoes into malarial regions of the world to prevent the transmission of Plasmodium. But, to help with controlling the disease, the mosquito must first be proved safe for release into the wild and, second, must be given some advantage that renders it superior to natural populations so it can drive them out.

“The eradication scenario requires three things: A gene that disrupts the development of the parasite inside the mosquito, a genetic technique to bring that gene into the mosquito genome and a mechanism that gives the mosquito an edge over the natural populations so they can displace them over time,” said Dr. Riehle “The third requirement is going to be the most difficult of the three to realize. It would probably take at least a decade, if not more.”

For detailed analysis and comment of these news, please visit CheckOrphan website here.

The original study published on July 15 in PLoS Pathogens http://www.plospathogens.org is here.

Pfizer’s Viagra Faces FDA Review for Use in Children With Lung Condition

By Shannon Pettypiece and Molly Peterson – Jul 27, 2010

A form of Pfizer Inc.’s erection drug Viagra, sold as the blood-pressure treatment called Revatio in adults, may be used for children with a rare lung disorder if U.S. regulators can agree on how to test it.

The condition, called pulmonary arterial hypertension, affects only 600 children a year, New York-based Pfizer said. It causes high blood pressure in arteries in the lungs, making the right side of the heart work harder than normal and causing chest pain, dizziness and fatigue. Outside advisers to the Food and Drug Administration are set to meet July 29 to evaluate whether Pfizer’s study of Revatio is sufficient to determine its effectiveness in children, the agency said today.

If the New York-based company meets FDA requirements, the drug would get an extra six months on the market without generic competition. Patents on the drug, with 2009 sales of $1.89 billion as Viagra and $450 million as Revatio, are expected to expire in 2012. Some doctors are already using the treatment in kids with the lung condition.

“It’s a good option in pediatric patients because it is well-tolerated, in that it doesn’t have as many side effects as some of the other options,” said Chad Knoderer, a pediatric clinical pharmacist at Riley Hospital for Children in Indianapolis, who has used Viagra in kids with the disorder.

FDA Request

The FDA in 2001 asked Pfizer, the world’s biggest drugmaker, to study the medicine in children with the lung disorder. Sildenafil, the chemical name for both Viagra and Revatio, blocks an enzyme found in the lungs and penis that regulates blood flow. Pfizer is considering whether to seek approval for Revatio, a lower-dose form of sildenafil, in children, said Colin Ewen, an executive director at the company, in a July 23 telephone interview.

Pfizer shares rose 16 cents, or 1.1 percent, to $15.18 at 10:09 a.m. in New York Stock Exchange composite trading.

The FDA is asking its panel of advisers to decide what is the best measurement to use in determining if the drug is effective in children with pulmonary hypertension.

In adults, researchers typically use an exercise test to determine if the drug is having a benefit. Because that test was difficult to perform in young children, Pfizer has asked the FDA to consider an alternative test that measures blood flow by inserting a catheter through the arteries.

In Pfizer’s study of 234 children, the drug failed to show a benefit when the children’s exercise ability was measured. However, it did show a significant benefit when researchers used an alternative measure of blood flow.

NIH Takes On New Role in Fight Against Rare Diseases

July 24, 2010

By AMY DOCKSER MARCUS

A government program focusing on rare diseases has launched five pilot projects that are taking the National Institutes of Health in a new direction: developing drugs.

The NIH Therapeutics for Rare and Neglected Diseases (TRND) program was established last year with $24 million of funding. TRND will work together with scientists, advocates and others to do the required research and testing on drugs before a compound can be tried in humans in a clinical trial.

Promising new drugs discovered through basic research often flounder during this stage of the process, which is expensive, time-consuming and prone to failure.

The pilot projects, three of which were selected this spring, target drug development for sickle-cell disease; chronic lymphocytic leukemia; the fatal neurodegenerative disease Niemann-Pick Type C; the genetic muscle disorder hereditary inclusion body myopathy; and the parasitic diseases schistosomiasis and hookworm.

The projects, which are in various stages of development, were selected because they illustrate a range of problems and issues in the effort to drive drug development.

The problems include the high cost of studies in animals to determine if a drug is too toxic to give to humans, the challenges of meeting regulatory requirements before the Food and Drug Administration allows clinical trials to begin, and the sheer amount of coordination that goes into getting a new drug to market.

“Most of the problems we are addressing are not scientific problems,” said Christopher P. Austin, director of the NIH program. “They are operational issues.”

For most new drugs, these issues are handled by a pharmaceutical company. Rare diseases, which the NIH defines as diseases that affect fewer than 200,000 people in the U.S., represent a small market.

As a result of the small markets, many pharmaceutical companies are reluctant to take on the risks and expense of trying to develop new drugs for these conditions.

TRND is assigning project managers with experience in drug development to the pilot projects to help identify the necessary steps to get to clinical trials.

The sickle-cell disease project, for instance, involves AesRx LLC, a Newton, Mass., biotech company, and needs to complete toxicity studies and regulatory work to launch a trial.

“The alternative would be to raise outside capital from venture capitalists,” said Steve Seiler, chief executive of AesRx. Mr. Seiler said at this stage of the project it would have been difficult to get the financing. “Once we have human clinical data, it is much different,” he said.

With the muscle disorder hereditary inclusion body myopathy, William A. Gahl, clinical director of the National Human Genome Research Institute, said he had been unable to launch a clinical trial to test a promising compound for three years, before the project was taken up by the NIH program.

He said that before he could start a clinical trial with the compound, the FDA wanted toxicology studies conducted in animals, at an estimated cost of $500,000 to $1.5 million—money the small biotech company and patient advocacy group he is working with didn’t have available. “We were about to give up,” Dr. Gahl said.

TRND is getting toxicology studies done and has hired a regulatory consultant to help address any regulatory issues with the FDA to get permission to start a trial.

“TRND has smoothed the way enormously,” said Dr. Gahl, who said he hopes to launch the trial this year.

In the case of Niemann-Pick Type C, TRND’s Dr. Austin had previously worked with a group of scientists and parent funders called SOAR-NPC to screen already approved FDA compounds to see if they might be effective against the disease. A promising compound was identified, but extensive work will be required to determine whether the drug is safe and effective enough to be tried in patients, Dr. Austin said.

This kind of tinkering with a promising drug—testing it in animals and then going back to the lab for further tweaks—is both time-consuming and expensive, and can be out of the reach of a parent-funded organization like SOAR.

Last week, at the annual conference of the advocacy group Genetic Alliance, Dr. Austin, a parent funder in SOAR, and Steven U. Walkley, a professor at Albert Einstein College of Medicine who is a member of SOAR, gave a joint talk that addressed some of the pressures the NIH program faces.

“There is a lot of promise built in to TRND, but there is no guarantee that they will be able to make the science deliver a therapy for a disease,” Dr. Walkley said.

Dr. Austin said he recognized that “we have to succeed with these pilot projects, and if we don’t, the program won’t continue.”

Half of the program’s budget this year is going to fund the five pilot projects, he said. The other half of the budget is going to setting up TRND. Dr. Austin added that the program plans to solicit additional projects in September.

Link to article

Disability Pride Parade, a video.

Caught this video on Saturday in Chicago at the Disability Pride Parade, http://www.youtube.com/watch?v=-EBR_CJz6EU.

Just in time for the birthday of the Americans with Disabilities Act.

// Catherine Calhoun

First unified protocol to provide primary care to rare disease patients introduced in Spain

In the European Union, a disease or disorder is defined as rare in Europe when it affects less than 1 in 2,000 persons, according to EURORDIS www.eurordis.org. There are between 6,000 and 8,000 rare diseases, affecting between 6% and 8% of the European Union population. Rare diseases are characterised by a broad diversity of disorders and symptoms, which vary not only from disease to disease but also from patient to patient. Yet medical professionals lack a unified protocol that assists them with treating patients who require out-of-the-ordinary care.

To bridge this gap, the Spanish Society of Family and Community Medicine (Sociedad Española de Medicina Familiar y Comunitaria, semFYC), in cooperation with several other Spanish institutions, has recently released an on-line protocol – the DICE-APER – to improve the Primary Care for patients with Rare Diseases.

“The goal of this protocol is the improvement of diagnosis and information related to these conditions,” said Dr Miguel García Ribes, the coordinator of the Clinical Genetics and Rare Diseases Group, in a recent interview given to El Confidencial. “It also aims to facilitate coordination between the different specialist doctors and provide epidemiological data that allow better health planning, as well as to advance research.”

The DICE-APER protocol recommends a series of steps that a general practitioner or family doctor can follow once the patient has agreed to sign the informed consent . By using the DICE-APER protocol, the diagnosis of the rare disease can be further confirmed and catalogued. The search engine provided in the web site offers a number of links to more specialized pages, allowing a doctor to easily find more detailed information and treatments as well as patient organizations. The DICE-APER protocol also facilitates better coordination between the different medical professionals in contact with the patient. In addition to this, by ensuring a proper register of patients and epidemiology, it provides the health institutions with reliable data.

It is estimated that every Spanish family doctor examines every year between 10 to 15 patients affected by a rare disease. Following the DICE-APER protocol should not represent an additional effort, but provide another tool to help ensure that the care given to patients is correct. Also, medical professionals should explain to patients affected by rare diseases that they can join the registry and why they should do it. The growth of this database will contribute to establishing the real dimensions of this problem and at the same time support researchers and institutions.

To use the DICE-APER protocol, please visit this web site (in Spanish language)

To read more about the Rare Diseases Strategy of the Spanish National Health System, please click here (in English language)

A Great Win for Rare Diseases in U.S. Senate Appropriation Bill

New FDA funding and requirements for guidances will help give rare diseases access to the accelerated approval process.

July 15, 2010 (Novato, California) — Just 16 months after the Kakkis EveryLife Foundation kicked off the CureTheProcess Campaign, the Foundation, in association with the National Organization of Rare Diseases (NORD) and numerous other patient and physician organizations, have increased the support and commitment to improving rare disease regulatory policies.

A US Senate Appropriation bill has been submitted including language supporting two of the Campaign’s goals. Specifically, the Bill supports the creation of new guidances which could improve the scientifically sound use of surrogate endpoints and new clinical study designs and analysis. The Senate Bill also includes an appropriation for the Food and Drug Administration to hire new staff to fulfill these requirements.

“We are especially grateful to Senator Sam Brownback (R-KS) for his leadership on this issue and to Senator Herb Kohl (D-WI) for his support,” said Emil Kakkis, M.D. Ph.D., President of the Kakkis EveryLife Foundation. “We are very pleased to see so much progress made, in such a relatively short time.”

The Senate Appropriations Committee will now review the FY 2011 Agriculture, FDA, and Rural Development Appropriations Bill. The bill includes the first increase for the Orphan Product Development Grant program since FY 2005. The program is increased by $2,000,000 for a total grant level of $16,035,000. The Bill also includes specific funding for the Office of the Associate Director for Rare Diseases in the Center for Drug Evaluation and Research (CDER). Funding for this office is increased by $1,000,000 to hire additional staff with specific expertise in facilitating the development and review of products to treat rare diseases.

The manager’s package that should be adopted at Committee includes language, cleared by the FDA that builds on the Brownback/Brown Amendment language that was included in the FY 2010 Appropriations Bill (Section 740). The language requires the FDA Commissioner to “…develop updated guidance documents and review standards for the development of safe and effective products to treat rare diseases and neglected tropical diseases…”

Specifically, the Bill spells out requirements to:

• Maximize the use of accelerated approval where feasible and appropriate, including guidances on the use of surrogate endpoints that are reasonably likely to predict clinical benefit of drugs and biological products under the regulations under Subpart H
• Work with drug company sponsors to facilitate expanded access to investigational therapies
  Develop guidance on clinical development programs for rare diseases
• Increase coordination among individual drug, biological product, and device review divisions across FDA centers to support the development of safe and effective medical products for rare and neglected diseases

The FDA is required to implement these as a part of the FY 2010 Brownback/Brown Amendment reforms and report back to the Appropriation Committee on implementation of these items.

“The Senate Bill is a good step forward in improving the regulatory process for rare diseases. By creating a more predictable pathway for orphan treatments, we will shorten development timelines and reduce the financial risk associated with the development of rare disease therapeutics. The result will be a surge in development activity for even the most rare disorders, giving more patients with rare biochemical and genetic disorders earlier access to effective treatments.” said Dr. Kakkis.

The Foundation initiated the CureTheProcess Campaign to give even the rarest diseases access to the accelerated approval process and put treatments on the fast track. There are more than 7,000 rare disorders that together affect over 25 million Americans and their families; however less than 5% have treatments as few drug companies conduct research on rare diseases since it is currently difficult to recoup the costs of developing treatments for such small populations. More than 130 patient and physician organizations have endorsed the Campaign goals to:

1. Establish a new Office of Drug Evaluation for Genetic and Biochemical Diseases at the FDA, consolidating and expanding expertise to ensure safe, effective and timely patient access to needed treatment.
2. Create a new standard to qualify biomarker or surrogate measures of the effect for treatments of rare disorders, and allow these treatments full access to the accelerated approval pathway for life threatening diseases.
3. Devise new clinical study designs for rare diseases that account for disease complexity and patient variability to properly capture treatment effects on all aspects of the disease.

To learn more about the Kakkis EveryLife Foundation, please go to www.Kakkis.org.

European Conference on Rare Diseases 2010 – From Policies to Effective Services for Patients

The European Conference on Rare Diseases (ECRD), which takes place every two years, is a unique forum that sees patient representatives of all the rare diseases gather with health care professionals, academics, researchers, policy makers and industry representatives to discuss the most recent rare disease initiatives in the fields of research, health­care, information and social services. The 2010 event took place last May 13 to 15, in Krakow (Poland ). It was the largest to date, with 600 delegates coming from 43 countries from all over the world.

ECRD 2010, entitled From Policies to Effective Services for Patients, was the occasion to discuss strategic issues for the rare disease community. Amongst these, special attention was given to the National Plans for Rare Diseases, Centres of Expertise and European Reference Networks, Research and Treatments, Information and Specialised Services. The programme included presentations, debates, workshops, satellite workshops, a poster session, a welcome reception and plenty of opportunities for participants to network and meet informally. Overall, there were 25 different sessions covering eight themes, involving 73 speakers and 37 chairpersons. (For speakers, presentations and detailed program, please visit this site )

Also, the EU Committee of Experts on Rare Diseases was presented. The Committee will act as a sort of ‘Parliament’ of the rare disease community in order to follow up on the work initiated at the biennial Conference

“The ECRD 2010 Krakow has served to identify those areas that need better policies in order to fulfill the objectives of the Council Recommendation and to build momentum for national plans and strategies to be implemented across Europe,” declared EURORDIS CEO at the end of the conference,  Yann Le Cam.

The next ECRD conference will take place in 2012 in Brussels!

For more information, please visit these website:

_ The European Conference on Rare Diseases 2010

_Photos of the conference

_Video interviews about the conference

Picture © EURORDIS. All rights reserved

Ten years of Human Genome

Ten years ago, Peter Collins and Craig Venter,  leaders of the Human Genome Project anounced they had completed the first draft of the human genes sequence. Published later in 2001 in Nature, the initial decoding of the human genome’s 3 billion base pairs – about 22,000 genes – was considered a major scientific breakthrough. The expectations on future developments were set high. “With this profound new knowledge, humankind is on the verge of gaining immense, new power to heal. It will revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.”, said Bill Clinton, by then the United States President.

But, as a decade passed by, most of these promises remain unfulfilled. The reason for this is the extraordinary complexity of genes and gene expression. As it turned out, genetic sequences are not just a blueprint for an organism, as initially thought. From a genetic sequence, it is not possible to predict a structure or a behaviour. Ultimately, the end result is greatly influenced by its regulatory mechanisms, which in turn are conditioned by the environment. All in all, organisms are not simply the expression of the genome, but they are the result of a myriad of interactions between the environment and the genes.

Today, a small number of procedures based on the human genome have entered routine medical practice: some gene-specific treatments for certain cancers, some novel therapies for very specific types of traits and some strong genetic markers for assessing drug responsiveness, risk of disease, or risk of disease progression.

The initial Human Genome Project gave rise to other ventures. The HapMap project compared the genomes of several hundred people to produce a map of genomic hotspots where people would likely to have DNA differences. It was followed by  The 1000 Genomes Project, which aims the creation a detailed catalogue of human genetic variation .  The Encyclopedia of DNA Elements and the Roadmap Epigenomics Program will continue to identify the locations of genes and the patterns that determine whether genes are switched on or off, defining the ‘parts list’ of the human genome. Scientists hope these projects will help to understand the mechanisms behind common diseases leading to personalized medicine.

Both leaders of the Human Genome Project remain positive about the impact of genomics in healthcare and medicine. “The promise of a revolution in human health remains quite real,” wrote Collins, in the special issue of Nature dedicated to 10th anniversary of the human genome announcement. “The genome revolution is only just beginning”, concluded  Venter, who recently announced the creation of the first synthetic cell.

Sources: Nature 2010; 464 (7289): 649 – 804 , The Lancet 2010; 375:2194, The New England Journal of Medicine 2010 362:2028-2029,  Suite101.

Picture: Human genome (Source: revised from Image: Human genome to genes.png |Date=2007-08-03 |Author=Webridge |Permission=CC-BY-2.0. }})

FDA and Rare Disease Drug Development, from FDA Week

June 29, 2010 // [FDA Week, http://insidehealthpolicy.com/]

FDA is currently in the middle of a flurry of activity aimed at better focusing its regulation of rare disease treatments, but the agency disappointed some outside advocates by delaying the possibility of creating a special rare disease review division within FDA for at least a year. That news came to advocates from FDA staffers an hour before a Senate Appropriations subcommittee looking at federal regulation of these diseases last Wednesday (June 23), a source said. More surprising to the source were comments from National Organization for Rare Disorders leadership during the hearing that the establishment of the new agency division would be a mistake.

FDA has two working groups addressing the issue and a number of rare disease regulatory advocates are proposing improvements to the agency’s regulatory regime. Senate appropriators are also eying whether legislation is needed.

The concept of a rare disease review division has been championed by the Emil Kakkis and his foundation, which has long advocated for Congress to provide FDA $10 million to create the special division. Kakkis and other rare-disease treatment advocates argue that gathering FDA’s experts in the field into one group would help accelerate the regulatory process.

The move would have an “immediate impact on new treatments” and would signal to the biotechnology industry that the agency was “serious” about this effort, Kakkis told the Senate FDA appropriations subcommittee. A special division would encourage more investment and allow for products already in the pipeline to be more quickly acted on, Kakkis said.

FDA should “have a division that has particularly trained people in the diseases that are both chemical, muscular or some of the other genetic rare disorders that take specialized expertise,” Kakkis told the subcommittee. The best regulation would come from consolidating FDA’s experts who are “scattered throughout” the agency, adding staff and building a division where staffers can avoid “having their expertise diffused and not leveraged over time,” Kakkis said.

The problem without a dedicated division also comes with a lack of centralized responsibility for reviewers, Kakkis added. “There are definitely people [in FDA] with expertise who can review these, but often they are called on to do other tasks,” Kakkis said. “This is a way to get better decision-making.”

But a Kakkis foundation staffer told FDA Week that meetings between advocates and FDA staffers prior to the hearing indicated that, while conceptually supportive of the idea of specialization, the timing did not work for the division to be created this year. That impression became formal an hour before the hearing.

The source said his reading of the situation was that FDA was possibly waiting until after drug user fees are reauthorized in 2012 to create the office.

The “biggest shock” from the day, the source said, came from Diane Dorman, the National Organization for Rare Disorders’ vice president for public policy, who said NORD flat-out disagreed with the concept. Dorman said NORD was “somewhat concerned” because of the worry that a separate division would create “narrow silos” within FDA.

By way of example, Dorman described the National Institutes of Health’s office on rare diseases as a nimble group with which the office’s director can leverage opportunities across all NIH offices and institutes without barriers. There’s a possibility to create an ad hoc group, “but creating an additional silo [within FDA] in our opinion may cause problems to access,” Dorman told the subcommittee.

In his response, Kakkis said without the review division FDA “cannot alter the review process for drugs in a way that is fundamental.” Creation of the division would let staffers be “personally responsible to make decisions,” he added.

“I think you’d want your drugs to be reviewed by people who are trained in the area, that specialized in it, and really understand it,” Kakkis said.

FDA Deputy Commissioner for Science and Public Health Jesse Goodman said the agency is aggressively addressing orphan drug issues, pointing to two new expert working groups FDA established in March, the Rare Disease Review Group and the Neglected Disease Review Group.

Those workgroups “will look at all the ideas we’ve heard here” before issuing a report to Congress, Goodman said. The two groups were created to help implement language in the FY 2010 Appropriation Act, which directed FDA to establish the internal review groups to address rare and neglected diseases, and to report to Congress one year after establishing the groups before issuing guidance.

That language came at a request from Sen. Sam Brownback (R-KS), the ranking member on the subcommittee.

Other work recently undertaken by FDA include the agency’s Office of Orphan Products Development starting use of a new tool, the Rare Disease Repurposing Database, which identifies drugs deemed promising for rare illnesses and are already approved by FDA for another disease (see related story).

A “novel feature and major advantage of this database” is its focus on drugs that have already gone through the FDA approval process, Goodman noted in its written testimony. “Thus, repurposing of these drugs for a new rare disease indication might be attainable quickly, relatively inexpensively, and at great benefit to the patients involved.”

In February, FDA created a position of associate director for rare diseases in the Center for Drug Evaluation and Research, which serves as the center’s focal point to the orphan drug development community “in navigating the complex regulatory requirements for bringing safe and effective treatments to patients,” Goodman wrote.

The agency is also collaborating with other entities to help ramp up its rare-disease treatment efforts.

In October 2010, FDA will co-sponsor its first rare disease investigator training course, in collaboration with NORD and NIH. FDA is also planning a series of scientific workshops to address difficult rare disease research issues. And FDA and NIH are co-sponsoring an Institute of Medicine study to review national policy for rare disease research and related medical product regulation, with recommendations due this September.

Experts suggest that there are roughly 200 FDA-approved treatments for the nearly 7,000 rare diseases that exist globally. So “while there have been many successes, the unmet needs are huge,” Goodman told the subcommittee. Rare diseases are defined as diseases affecting fewer than 200,000 people in the United States.

The FDA estimates that a billion people worldwide are affected by at least one neglected disease like tuberculosis, malaria and leprosy.

These issues are FDA’s concern because “infectious diseases know no boundaries,” Goodman said. “Threats to health anywhere are threats to everyone.”

The agency also is preparing for public hearings on June 29-30 focusing on how it approves and encourages medical products for rare diseases. — Seth Freedland (sfreedland@iwpnews.com)

Regulatory Flexibility

Patient advocates call on FDA to develop policy that would speed development of new therapies for rare disease.

Rare disease patient advocates called on the U.S. Food and Drug Administration to remove regulatory uncertainty and allow for greater flexibility in how the agency reviews drugs for diseases afflicting small populations of patients. The call came during a two-day public hearing held by the agency as it seeks ways to spur drug development around diseases where markets may be too small to entice drugmakers to make the large investment needed to bring drugs to market for these conditions.

“No one is arguing against rigorous standards, it’s just the standards for rare diseases need to take into account the size of the patient population and the devastating nature of these diseases,” says Jonathan Jacoby, CEO of the RARE Project, a group that advocates for accelerated rare disease research.

There are about 7,000 diseases that are considered rare and treatments for only about 200 of them. A disease is considered to be rare if it afflicts 200,000 people or less. Though individually each of these diseases is rare, about 10 percent of the population suffers from a rare disease.

The FDA’s 2010 appropriations bill required the agency to convene an expert committee to review the ways the agency deals with therapies to treat people with rare diseases. As part of that effort, the expert committee held the hearings as a way to reach out to patients, their families, patient advocates, drugmakers and researchers for their input.

Frank Sasinowski, chairman of the National Organization for Rare Diseases, said in a statement at the hearing that the FDA in the 1980s put into place regulations in response to the AIDS crisis that provided for flexibility in applying standards to drugs in development to treat life-threatening and debilitating illnesses in order to speed the development, evaluation, and availability of new drugs.

“When these trials are conducted, sometimes with designs with which all parties may not be in full concurrence, including the FDA, great deference should be afforded the design of these trials and flexibility applied in the interpretation of their results,” says Sasinowski. “If such a principle were to be addressed and accepted by the FDA, much good would come of it.”

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