Lawmakers push for speedy approvals of ‘breakthrough’ drugs

Posted under Blog, Companies, congress, Diagnostics, Drug approvals, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by John Carroll

New legislative initiatives aimed at speeding U.S. drug approvals are all the rage in Congress these days. Pharmalot has the story of a new proposal from a bipartisan group of lawmakers calling for accelerated approval paths for "breakthrough" drugs. A breakthrough, they explain, would be a new treatment that has preliminary clinical evidence that it can do a better job at treating a serious or life-threatening ailment than existing meds. "Time and again we've seen regulators in Washington fail to keep up with the industries they are tasked to oversee," says Sen. Orrin Hatch (R-UT), a sponsor. "This bipartisan bill ensures that when it comes to treating patients suffering with cancer or other devastating illnesses, science and patient care will not be slowed down by government red tape." Story

Senator launches campaign for speedier approvals of targeted drugs

Posted under Blog, Companies, congress, Diagnostics, Drug approvals, Funding, Kay Hagan, Medical Devices, Medical Supply, PDUFA, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

In an already politically charged year for the FDA, U.S. Senator Kay Hagan is spearheading legislation that would expedite reviews and approvals of drugs against serious medical conditions, her home state's North Carolina News Network reported.

The report says the legislation aims to give speedier reviews to drugs that deliver major benefits to patients, including targeted meds for patients with serious diseases and therapies that address underserved medical needs. The details of the North Carolina Democrat's plan were scant, and it's unclear exactly how quick she wants reviews to be. The FDA, of course, already has practices in place to speed new drugs to market if they offer superior benefits to patients, among other things.

“But for patients suffering today from some diseases for which there are no current treatments or for those patients who are clutching to the hope that a better treatment will be developed for their rare diseases, medical advances cannot come fast enough," said Hagan, as quoted by NCNN. She pointed to successful programs that sped new HIV treatments to patients in the 1990s to bolster her case.

Some lawmakers and biopharma backers have blasted the FDA for slowing the advance of innovative therapies to the market. FDA boss Margaret Hamburg has answered critics by emphasizing the agency hit a 7-year high in approvals last year, so the regulator can indeed act quickly to green light drugs when warranted. Take its recent speedy approval of Vertex's ($VRTX) cystic fibrosis drug Kalydeco, which the agency blessed last month ahead of schedule. Still, agency brass will need to gain support from U.S. lawmakers to reauthorize PDUFA this year, making 2012 a good time for members of Congress to voice their hopes and concerns about how the FDA conducts business.

- check out Hagan's release
- here’s the report from NCNN

Special Report: FDA approvals of 2011

Related Articles:
Increased U.S. aid, rare disease focus drives surge in drug approvals
Spike in approvals can't quell industry protests about the FDA

Let’s Get PDUFA V Approved, Fast

Posted under Blog, Companies, congress, Diagnostics, FDA, Food and Drug Administration, Funding, Health, Medical Devices, Medical Supply, National Institutes of Health, NIH, PDUFA, Pharmaceuticals, prescription drugs, Startups, u.s. politics, Universities, Videos by biotechnow@bio.org (Biotechnology Industry Organization)

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).

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.

BIO Supports SBIR Reauthorization in Senate

Posted under Blog, Companies, congress, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Public Policy, sbir, Senate, small business, Startups, Universities, Videos by biotechnow@bio.org (Biotechnology Industry Organization)

Last week, the Senate passed the National Defense Authorization Act (S. 1867), which included SBIR reauthorization through Senate Amendment 1115.

This is a critical step toward ensuring that all innovative companies can compete for SBIR grants – based on the promise of their science rather than the structure of their capital.  Reauthorizing the program to allow small companies that receive the majority of their financing from venture capital to once again be eligible to compete for SBIR grants is imperative.  This change will allow more small biotechnology start-ups to continue critical research and development of medical advancements and breakthroughs.

A compromise on a longer term reauthorization is critical. BIO will work with Congress to ultimately reauthorize the SBIR program to reflect the realities facing small companies in capital-intensive industries such as biotechnology. SBIR should be an aggressively competitive program that fulfills federal research and development goals of bringing breakthrough public health discoveries to the public.

Senate amendment 1115 was offered by Sens. Mary Landrieu (D-LA) and Olympia Snowe (R-ME), with the following co-sponsors: Sens. Jeanne Shaheen (D-NH), Scott Brown (R-MA), John Kerry (D-MA), Kelly Ayotte (R-NH), Christopher Coons (D-DE), Carl Levin (D-MI), Benjamin Cardin (D-MD), and Robert Casey (D-PA).

Recent news stories:

Senate OKs SBIR funding, but full passage remains a question mark, New Hampshire Business Review, December 6

BIO Applauds Small Business Innovation Measure in Senate Defense Bill – Inside Health Policy, December 2

Senate Passes SBIR Reauthorization – BioCentury, December 2

BIO Praises Senate Passage of SBIR Reauthorization – BioMedReports, December 2

Senate OKs Changes to High Tech Start Ups – Science, December 2