II 

110TH CONGRESS 

2D SESSION S. 3046 

To amend the Federal Food, Drug, and Cosmetic Act to create a new 

conditional approval system for drugs, biological products, and devices 

that is responsive to the needs of seriously ill patients, and for other 

purposes. 

IN THE SENATE OF THE UNITED STATES 

MAY 21, 2008 

Mr. BROWNBACK (for himself, Mr. CASEY, Mr. COLEMAN, Mr. SPECTER, and 

Mr. INHOFE) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions 

A BILL 

To amend the Federal Food, Drug, and Cosmetic Act to 

create a new conditional approval system for drugs, biological products, and devices that is responsive to the 

needs of seriously ill patients, and for other purposes. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Access, Compassion, 

5 Care, and Ethics for Seriously Ill Patients Act’’ or the 

6 ‘‘ACCESS Act’’. 


sroberts on PROD1PC70 with BILLS
2 

•S 3046 IS

1 SEC. 2. FINDINGS. 

2 Congress finds the following: 

3 (1) As of 2008, the standards of the Food and 

4 Drug Administration for approval of drugs, biologi5 cal products, and devices may deny the benefits of 

6 medical progress to seriously ill patients who face 

7 morbidity or death from their disease. 

8 (2) Seriously ill patients have a right to take 

9 actions to preserve their life by accessing available 

10 investigational drugs, biological products, and de11 vices. 

12 (3) The emphasis on statistical analysis of clin13 ical information needs to be balanced by a reliance 

14 on clinical evaluation and patient-reported outcomes 

15 and considered with an understanding of the risks to 

16 patients from their disease, with the goal of pro17 viding additional treatment options for patients and 

18 their physicians to consider. 

19 (4) Food and Drug Administration advisory 

20 committees should have greater representation of 

21 medical clinicians and patient advocates who rep22 resent the interests of seriously ill patients in early 

23 access to promising investigational therapies. 

24 (5) The use of available investigational products 

25 for treatment is the responsibility of the physician 

26 and the seriously ill patient. 


sroberts on PROD1PC70 with BILLS
3 

•S 3046 IS

1 (6) The use of combinations of available inves2 tigational and approved products for treatment is 

3 the responsibility of the physician and the seriously 

4 ill patient. 

5 (7) The Food and Drug Administration should 

6 have the expertise and flexibility to address the 

7 growing needs of seriously ill patients for individual8 ized or personalized therapies. 

9 SEC. 3. COMPASSIONATE INVESTIGATIONAL ACCESS AP10 PROVAL SYSTEM FOR DRUGS, BIOLOGICAL 

11 PRODUCTS, AND DEVICES. 

12 (a) COMPASSIONATE INVESTIGATIONAL ACCESS.— 

13 Section 561 of the Federal Food, Drug, and Cosmetic Act 

14 (21 U.S.C. 360bbb) is amended— 

15 (1) by redesignating subsections (d) and (e) as 

16 subsections (e) and (f), respectively; and 

17 (2) by inserting after subsection (c) the fol18 lowing: 

19 ‘‘(d) COMPASSIONATE INVESTIGATIONAL ACCESS.— 

20 ‘‘(1) PURPOSE.—The purpose of this subsection 

21 is to facilitate the availability of promising new 

22 drugs to seriously ill patients as early in the drug 

23 development process as possible, before general mar24 keting begins. 


sroberts on PROD1PC70 with BILLS
4 

•S 3046 IS

1 ‘‘(2) ACCESS.—Notwithstanding any other pro2 vision of law, upon submission by a sponsor of an 

3 application intended to provide widespread access to 

4 an investigational drug, biological product, or device 

5 for eligible patients (referred to in this subsection as 

6 ‘Compassionate Investigational Access’), the Sec7 retary shall permit such investigational drug, biologi8 cal product, or device, to be made available for ex9 panded access under a treatment investigational new 

10 drug application or treatment investigational device 

11 exemption if the Secretary determines that the re12 quirements of this section are met with respect to 

13 Compassionate Investigational Access. 

14 ‘‘(3) COMPASSIONATE INVESTIGATIONAL AC15 CESS.—Notwithstanding any other provision of law, 

16 an investigational drug, biological product, or device 

17 that receives approval for Compassionate Investiga18 tional Access under this subsection shall be subject 

19 to the provisions of section 505(i) or 520(g), as ap20 plicable, and regulations promulgated by the Sec21 retary pursuant to this Act. The Secretary and the 

22 sponsor may inform national, State, and local med23 ical associations and societies, voluntary health asso24 ciations, and other appropriate persons about the 

25 availability of an investigational drug or investigaVerDate Aug 31 2005 01:55 May 24, 2008 Jkt 069200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S3046.IS S3046

sroberts on PROD1PC70 with BILLS
5 

•S 3046 IS

1 tional device under Compassionate Investigational 

2 Access as approved under this subsection. The infor3 mation submitted by the Secretary, in accordance 

4 with the preceding sentence, shall be the same type 

5 of information that is required by section 402(i)(3) 

6 of the Public Health Service Act. 

7 ‘‘(4) SUBMISSION OF APPLICATION.— 

8 ‘‘(A) APPLICATION CONTENT.—A sponsor 

9 of an investigational drug, biological product, or 

10 device applying for Compassionate Investiga11 tional Access approval of the product shall sub12 mit to the Secretary a notice of claimed exemp13 tion under section 505(i) or 520(g), as applica14 ble, (referred to in this subsection as an ‘appli15 cation for Compassionate Investigational Ac16 cess’), which shall contain— 

17 ‘‘(i) data and information from com18 pleted Phase I clinical investigations and 

19 any other nonclinical or clinical investiga20 tions; 

21 ‘‘(ii) preliminary evidence that the 

22 product may be effective in humans 

23 against a serious or life-threatening condi24 tion or disease, which evidence may be 

25 based on uncontrolled data such as case 


sroberts on PROD1PC70 with BILLS
6 

•S 3046 IS

1 histories, information about the pharma2 cological mechanism of action, data from 

3 animal and computer models, comparison 

4 with historical data, or other preliminary 

5 information, and may be based on a small 

6 number of patients or a subset of the pa7 tient population; 

8 ‘‘(iii) evidence that the product is safe 

9 at the dose and duration proposed, consid10 ering whether the potential risk to a pa11 tient of the condition or disease outweighs 

12 the potential risk to a patient of the pro13 posed dose and duration of treatment with 

14 the product, consistent with the level of in15 formation needed to initiate a Phase II 

16 clinical trial; and 

17 ‘‘(iv) a statement that the sponsor is 

18 actively pursuing marketing approval with 

19 due diligence. 

20 ‘‘(B) LIMITATION.—Compassionate Inves21 tigational Access approval shall be based upon 

22 multiple considerations that shall include clin23 ical evaluation and unmet patient needs. 

24 ‘‘(5) DETERMINATION BY SECRETARY.— 


sroberts on PROD1PC70 with BILLS
7 

•S 3046 IS

1 ‘‘(A) IN GENERAL.—Not later than 30 

2 days after the receipt of an application for 

3 Compassionate Investigational Access approval, 

4 the Secretary shall either— 

5 ‘‘(i) provide Compassionate Investiga6 tional Access approval of the application; 

7 or 

8 ‘‘(ii) refer the application to the Accel9 erated Approval Advisory Committee. 

10 ‘‘(B) RECOMMENDATION.—Not later than 

11 90 days after receipt of an application for Com12 passionate Investigational Access approval, the 

13 Accelerated Approval Advisory Committee shall 

14 issue a recommendation to the Secretary on 

15 whether the Secretary shall provide Compas16 sionate Investigational Access approval of the 

17 application. 

18 ‘‘(C) FINAL DECISION.—Not later than 30 

19 days after receipt of the recommendation from 

20 the Accelerated Approval Advisory Committee, 

21 the Secretary shall either provide Compas22 sionate Investigational Access approval of the 

23 application or shall issue an order setting forth 

24 a detailed explanation of the reasons why the 

25 application was not so approved and the specific 


sroberts on PROD1PC70 with BILLS
8 

•S 3046 IS

1 data that the sponsor must provide so that the 

2 application may be so approved. 

3 ‘‘(6) APPEAL.—If the Secretary does not pro4 vide Compassionate Investigational Access approval 

5 of an application, the sponsor of the application 

6 shall have the right to appeal the decision to the 

7 Secretary. The Secretary shall provide the sponsor 

8 with a hearing not later than 30 days following the 

9 nonapproval under this subsection of the application 

10 and shall issue an order not later than 30 days fol11 lowing the hearing either concurring in the non12 approval or so approving the application. The Sec13 retary shall not delegate the responsibility described 

14 in this paragraph to any other person. 

15 ‘‘(7) CRITERIA.—In making a determination 

16 under paragraph (5), the Secretary shall consider 

17 whether the totality of the information available to 

18 the Secretary regarding the safety and effectiveness 

19 of an investigational drug, biological product, or de20 vice, as compared to the risk of morbidity or death 

21 from a condition or disease, indicates that a patient 

22 (who may be representative of a small patient sub23 population) may obtain more benefit than risk if 

24 treated with the drug, biological product, or device. 

25 If the potential risk to a patient of the condition or 


sroberts on PROD1PC70 with BILLS
9 

•S 3046 IS

1 disease outweighs the potential risk of the product, 

2 and the product may possibly provide benefit to the 

3 patient, the Secretary shall provide Compassionate 

4 Investigational Access approval of the application. 

5 ‘‘(8) PATIENT ELIGIBILITY FOR COMPAS6 SIONATE ACCESS.—In order for a patient to access 

7 a product available through Compassionate Inves8 tigational Access, the physician must document in 

9 writing that the patient— 

10 ‘‘(A) is seriously ill; 

11 ‘‘(B) has exhausted all treatment options 

12 approved by the Secretary for the condition or 

13 disease for which the patient is a reasonable 

14 candidate; and 

15 ‘‘(C) has unsuccessfully sought treatment 

16 or obtained treatment that was not effective, 

17 with an investigational drug, biological product, 

18 or device for which such individual is a reason19 able candidate, which shall include consider20 ation of a patient’s ineligibility for participation 

21 in clinical trials, the lack of source of supply 

22 and geographic factors. 

23 ‘‘(9) PRODUCT LABELING.—To receive Compas24 sionate Investigational Access approval under this 

25 subsection, the sponsor of the product shall provide 


sroberts on PROD1PC70 with BILLS
10 

•S 3046 IS

1 labeling approved by the Secretary for the drug, bio2 logical product, or device that— 

3 ‘‘(A) states that the product is intended 

4 for use by a patient whose physician has docu5 mented in writing that the patient has— 

6 ‘‘(i) exhausted all treatment options 

7 approved by Secretary for the condition or 

8 disease for which the patient is a reason9 able candidate; and 

10 ‘‘(ii) unsuccessfully sought treatment, 

11 or obtained treatment that was not effec12 tive with an investigational drug, biological 

13 product, or device for which such indi14 vidual is a reasonable candidate, which 

15 shall include a patient’s ineligibility for 

16 participation in clinical trials, the lack of 

17 source of supply and geographic factors; 

18 and 

19 ‘‘(B) states that every patient to whom the 

20 product is administered shall, as a mandatory 

21 condition of receiving the product, provide— 

22 ‘‘(i) written informed consent, as de23 scribed under part 50 of title 21, Code of 

24 Federal Regulations (or any successor reg25 ulations); and 


sroberts on PROD1PC70 with BILLS
11 

•S 3046 IS

1 ‘‘(ii) consent for the manufacturer of 

2 the product to obtain data and information 

3 about the patient and the patient’s use of 

4 the product that may be used to support 

5 an application for Accelerated Approval or 

6 final approval. 

7 ‘‘(10) CHARGING FOR COMPASSIONATE INVES8 TIGATIONAL ACCESS.—A sponsor or investigator 

9 may charge for a Compassionate Investigational Ac10 cess drug without notifying the Secretary or seeking 

11 or obtaining prior approval of the amount charged, 

12 provided the sponsor of the drug is actively pursuing 

13 marketing approval with due diligence. 

14 ‘‘(11) COMMENCEMENT OF REVIEW.—If the 

15 Secretary determines, after preliminary evaluation of 

16 the data and information submitted by the sponsor, 

17 that the product may be effective, the Secretary 

18 shall evaluate for filing, and may commence review 

19 of portions of, an application under this subsection 

20 before the sponsor submits a complete application. 

21 The Secretary shall commence such review only if 

22 the applicant provides a schedule for submission of 

23 information necessary to make the application com24 plete. 

25 ‘‘(12) IMMUNITY.— 


sroberts on PROD1PC70 with BILLS
12 

•S 3046 IS

1 ‘‘(A) IN GENERAL.—A manufacturer, dis2 tributor, administrator, sponsor, or physician 

3 who manufactures, supplies, distributes or pre4 scribes a product approved under an application 

5 for Compassionate Investigational Access shall 

6 be immune from suit or liability caused by, aris7 ing out of, or relating to the design, develop8 ment, clinical testing and investigation, manu9 facture, labeling, distribution, sale, purchase, 

10 donation, dispensing, prescribing, administra11 tion, efficacy, or use of a drug, biological prod12 uct, or device subject to an approved Compas13 sionate Investigational Access application. 

14 ‘‘(B) CLAIMS.—No claim or cause of ac15 tion against a manufacturer, distributor, ad16 ministrator, sponsor, or physician who manu17 factures, supplies, distributes or prescribes a 

18 product subject to an approved Compassionate 

19 Investigational Access application shall exist in 

20 any Federal or State court for claims of prop21 erty, personal injury, or death caused by, aris22 ing out of, or relating to the design, develop23 ment, clinical testing and investigation, manu24 facture, labeling, distribution, sale, purchase, 

25 donation, dispensing, prescribing, administraVerDate Aug 31 2005 01:55 May 24, 2008 Jkt 069200 PO 00000 Frm 00012 Fmt 6652 Sfmt 6201 E:\BILLS\S3046.IS S3046

sroberts on PROD1PC70 with BILLS
13 

•S 3046 IS

1 tion, efficacy, or use of a drug, biological prod2 uct, or device subject to an approved Compas3 sionate Investigational Access application. Any 

4 such claim or cause of action that is filed in 

5 Federal or State court shall be immediately dis6 missed. 

7 ‘‘(13) FINAL APPROVAL.—For purposes of this 

8 Act, the term ‘final approval’ means— 

9 ‘‘(A) with respect to a new drug or new bi10 ological product, approval of such drug or prod11 uct under section 505(b)(1) or 505(b)(2) or 

12 section 351 of the Public Health Service Act, as 

13 the case may be; and 

14 ‘‘(B) with respect to a new device, clear15 ance of such device under section 510(k) or ap16 proval of such device under section 515(c)(1).’’. 

17 (b) ACCELERATED APPROVAL.—Chapter V of the 

18 Federal, Food, Drug, and Cosmetic Act (21 U.S.C. 351 

19 et seq.) is amended by inserting after section 561 the fol20 lowing: 

21 ‘‘SEC. 561A. ACCELERATED APPROVAL. 

22 ‘‘(a) IN GENERAL.— 

23 ‘‘(1) IN GENERAL.—As soon as practicable 

24 after the date of enactment of the Access, Compas25 sion, Care, and Ethics for Seriously Ill Patients Act, 


sroberts on PROD1PC70 with BILLS
14 

•S 3046 IS

1 the Secretary shall promulgate regulations to pro2 vide for the treatment of an investigational drug, bi3 ological product, or device that receives Accelerated 

4 Approval under this section. This section shall be 

5 carried out in accordance with such regulations (and 

6 any successor regulations). 

7 ‘‘(2) APPLICATION.—A sponsor of an investiga8 tional drug, biological product, or device applying for 

9 Accelerated Approval shall submit to the Secretary 

10 an application as described under section 505(b)(1) 

11 or 505(b)(2), or section 510(k) or 515(c)(1) of this 

12 Act, or section 351(a) of the Public Health Service 

13 Act, as applicable, which shall contain— 

14 ‘‘(A) data and information that the drug, 

15 biological product, or device has an effect on a 

16 clinical endpoint or on a surrogate endpoint or 

17 biomarker that is reasonably likely to predict 

18 clinical benefit to a patient (who may be rep19 resentative of a small patient subpopulation) 

20 suffering from a serious or life-threatening con21 dition or disease; and 

22 ‘‘(B) a statement that the sponsor is ac23 tively pursuing marketing approval with due 

24 diligence. 

25 ‘‘(3) DETERMINATION BY SECRETARY.— 


sroberts on PROD1PC70 with BILLS
15 

•S 3046 IS

1 ‘‘(A) IN GENERAL.—Not later than 120 

2 days after the receipt of an application for Ac3 celerated Approval, the Secretary shall either— 

4 ‘‘(i) provide Accelerated Approval of 

5 the application; or 

6 ‘‘(ii) refer the application to the Accel7 erated Approval Advisory Committee. 

8 ‘‘(B) RECOMMENDATION.—Not later than 

9 90 days after receipt of an application for Ac10 celerated Approval, the Accelerated Approval 

11 Advisory Committee shall issue a recommenda12 tion to the Secretary on whether the Secretary 

13 should provide Accelerated Approval of the ap14 plication. 

15 ‘‘(C) LIMITATION.—The Accelerated Ap16 proval Advisory Committee shall not consider 

17 off-label uses of drugs, biological products, and 

18 devices as existing or available therapies, to the 

19 extent that the Accelerated Approval Advisory 

20 Committee weighs existing or available thera21 pies in determination of whether an investiga22 tional drug provides an improvement over treat23 ments that are already available. 

24 ‘‘(D) FINAL DECISION.—Not later than 30 

25 days after receipt of the recommendation from 


sroberts on PROD1PC70 with BILLS
16 

•S 3046 IS

1 the Accelerated Approval Advisory Committee, 

2 the Secretary shall either provide Accelerated 

3 Approval of the application or issue an order 

4 setting forth a detailed explanation of the rea5 sons why the application was not so approved 

6 and the specific data that the sponsor must 

7 provide so that the application may be so ap8 proved. 

9 ‘‘(4) APPEAL.—If the Secretary does not pro10 vide Accelerated Approval of an application, the 

11 sponsor of the application shall have the right to ap12 peal the decision to the Secretary. The Secretary 

13 shall provide the sponsor with a hearing not later 

14 than 30 days following the nonapproval under this 

15 subsection of the application and shall issue an order 

16 not later than 30 days following the hearing either 

17 concurring in such nonapproval or so approving the 

18 application. The Secretary shall not delegate the re19 sponsibility described in this paragraph to any other 

20 person. 

21 ‘‘(A) with respect to a new drug or new bi22 ological product, approval of such drug or prod23 uct under section 505(b)(1) or 505(b)(2) or 

24 section 351 of the Public Health Service Act, as 

25 the case may be; and 


sroberts on PROD1PC70 with BILLS
17 

•S 3046 IS

1 ‘‘(B) with respect to a new device, clear2 ance of such device under section 510(k) or ap3 proval of such device under section 515(c)(1). 

4 ‘‘(b) ACCELERATED APPROVAL ADVISORY COM5 MITTEE.— 

6 ‘‘(1) IN GENERAL.—In order to facilitate the 

7 development and expedite the review of drugs, bio8 logical products, and devices intended to treat seri9 ous or life threatening conditions, the Secretary shall 

10 establish the Accelerated Approval Advisory Com11 mittee (referred to in this subsection as the ‘Com12 mittee’). 

13 ‘‘(2) DELEGATION.—The Secretary may dele14 gate decisionmaking authority for the Accelerated 

15 Approval Advisory Committee to the Office of the 

16 Commissioner of Food and Drugs. Such authority 

17 shall not be further delegated. 

18 ‘‘(3) COMPOSITION.— 

19 ‘‘(A) IN GENERAL.—The Committee shall 

20 be composed of 11 voting members, including 1 

21 chairperson and 5 permanent members each of 

22 whom shall serve a term of 3 years and may be 

23 reappointed for a second 3-year term, and 5 

24 nonpermanent members who shall be appointed 

25 to the Committee for a specific meeting, or part 


sroberts on PROD1PC70 with BILLS
18 

•S 3046 IS

1 of a meeting, in order to provide adequate ex2 pertise in the subject being reviewed. The Com3 mittee shall include as voting members no less 

4 than 2 representatives of patient interests, of 

5 which 1 shall be a permanent member of the 

6 Committee. The Committee shall include as 

7 nonvoting members a representative of interests 

8 of the drug, biological product, and device in9 dustry. 

10 ‘‘(B) APPOINTMENTS.—The Secretary 

11 shall appoint to the Committee persons who are 

12 qualified by training and experience to evaluate 

13 the safety and effectiveness of the types of 

14 products to be referred to the Committee and 

15 who, to the extent feasible, possess skill in the 

16 use of, or experience in the development, manu17 facture, or utilization of, such products. The 

18 Secretary shall make appointments to the Com19 mittee so that the Committee shall consist of 

20 members with adequately diversified expertise 

21 and practical experience in such fields as clin22 ical medicine, biological and physical sciences, 

23 and other related professions. Scientific, indus24 try, and consumer organizations and members 

25 of the public shall be afforded an opportunity to 


sroberts on PROD1PC70 with BILLS
19 

•S 3046 IS

1 nominate individuals for appointment to the 

2 Committee. No individual who is in the regular 

3 full-time employ of the United States and en4 gaged in the administration of this chapter may 

5 be a member of the Committee. 

6 ‘‘(4) COMPENSATION.—Committee members, 

7 while attending meetings or conferences of the Com8 mittee or otherwise engaged in its business, shall be 

9 entitled to receive compensation at rates to be fixed 

10 by the Secretary, but not at rates exceeding the 

11 daily equivalent of the rate in effect for grade GS– 

12 18 of the General Schedule, for each day so en13 gaged, including traveltime, and while so serving 

14 away from their homes or regular places of business 

15 each member may be allowed travel expenses (in16 cluding per diem in lieu of subsistence) as author17 ized by section 5703 of title 5, for persons in the 

18 Government service employed intermittently. 

19 ‘‘(5) ASSISTANCE.—The Secretary shall furnish 

20 the Committee with adequate clerical and other nec21 essary assistance. 

22 ‘‘(6) ANNUAL TRAINING.—The Secretary shall 

23 employ nongovernmental experts to provide annual 

24 training to the Committee on the statutory and reg25 ulatory standards for product approval. 


sroberts on PROD1PC70 with BILLS
20 

•S 3046 IS

1 ‘‘(7) TIMELINE.—The Committee shall be 

2 scheduled to meet at such times as may be appro3 priate for the Secretary to meet applicable statutory 

4 deadlines. 

5 ‘‘(8) MEETINGS.— 

6 ‘‘(A) OPPORTUNITIES FOR INTERESTED

7 PERSONS.—Any person whose product is spe8 cifically the subject of review by the Committee 

9 shall have— 

10 ‘‘(i) the same access to data and in11 formation submitted to the Committee as 

12 the Secretary; 

13 ‘‘(ii) the opportunity to submit, for re14 view by the Committee, data or informa15 tion, which shall be submitted to the Sec16 retary for prompt transmittal to the Com17 mittee; 

18 ‘‘(iii) the same opportunity as the 

19 Secretary to participate in meetings of the 

20 Committee; and 

21 ‘‘(iv) consent for the manufacturer of 

22 the product to obtain data about adverse 

23 events relating to the patient’s use of the 

24 product. 


sroberts on PROD1PC70 with BILLS
21 

•S 3046 IS

1 ‘‘(B) ADEQUATE TIME; FREE AND OPEN

2 PARTICIPATION.—Any meetings of the Com3 mittee shall provide adequate time for initial 

4 presentations and for response to any differing 

5 views by persons whose products are specifically 

6 the subject of the Committee review. 

7 ‘‘(C) SUMMARIES.—At all meetings of the 

8 Committee, the Secretary shall provide a sum9 mary to the Committee of all applications sub10 mitted under this subsection and section 561(d) 

11 that the Committee did not consider that were 

12 approved by the Secretary since the last meet13 ing of the Committee. 

14 ‘‘(c) FINAL APPROVAL.—For purposes of this Act, 

15 the term ‘final approval’ means— 

16 ‘‘(1) with respect to a new drug or new biologi17 cal product, approval of such drug or product under 

18 section 505(b)(1) or 505(b)(2) or section 351 of the 

19 Public Health Service Act, as the case may be; and 

20 ‘‘(2) with respect to a new device, clearance of 

21 such device under section 510(k) or approval of such 

22 device under section 515(c)(1).’’. 

23 (c) REGULATIONS.—The Secretary of Health and 

24 Human Services shall promulgate regulations that define 

25 the terms ‘‘seriously ill’’ and ‘‘serious or life-threatening’’ 


sroberts on PROD1PC70 with BILLS
22 

•S 3046 IS

1 for purposes of the amendments made by this Act, consid2 ering either— 

3 (1) the medical prognosis for an individual’s life 

4 expectancy from a disease or condition; or 

5 (2) the prospect of irreversible disability from a 

6 disease or condition. 

7 SEC. 4. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS 

8 AND DEVICES. 

9 Chapter V of the Federal Food, Drug, and Cosmetic 

10 Act (21 U.S.C. 351 et seq.) is amended by inserting after 

11 section 561A, as added by section 3, the following: 

12 ‘‘SEC. 561B. EXPANDED ACCESS TO INVESTIGATIONAL 

13 DRUGS AND DEVICES. 

14 ‘‘(a) EXPANDED ACCESS PROGRAM.—The Secretary 

15 shall establish a new program to expand access to inves16 tigational treatments for individuals with serious or life 

17 threatening conditions and diseases. In carrying out this 

18 expanded access program, the Secretary shall publish and 

19 broadly disseminate written guidance that— 

20 ‘‘(1) describes such expanded access programs 

21 for investigational drugs, biological products, and de22 vices intended to treat serious or life-threatening 

23 conditions or diseases; 


sroberts on PROD1PC70 with BILLS
23 

•S 3046 IS

1 ‘‘(2) encourages and facilitates submission of 

2 applications and approvals under section 561(d) and 

3 561A; and 

4 ‘‘(3) facilitates the provision of investigational 

5 drugs, biological products, and devices to seriously ill 

6 individuals without unreasonable delay by recog7 nizing that the use of available investigational prod8 ucts for treatment is the responsibility of the physi9 cian and the patient, and also by recognizing the 

10 goal of providing additional treatment options for 

11 patients and their physicians to consider. 

12 ‘‘(b) IMPLEMENTATION OF EXPANDED ACCESS PRO13 GRAMS.— 

14 ‘‘(1) TRAINING OF PERSONNEL.—Not later 

15 than 90 days after the date of enactment of this sec16 tion, the Secretary shall implement training pro17 grams at the Food and Drug Administration with 

18 respect to the expanded access programs established 

19 under this section. 

20 ‘‘(2) POLICIES, REGULATIONS, AND GUID21 ANCE.—The Secretary shall establish policies, regu22 lations, and guidance designed to most directly ben23 efit seriously ill patients. 

24 ‘‘(c) DEVELOPMENT OF SURROGATE ENDPOINTS

25 AND BIOMARKERS.— 


sroberts on PROD1PC70 with BILLS
24 

•S 3046 IS

1 ‘‘(1) IN GENERAL.—The Secretary shall— 

2 ‘‘(A) establish a program or expand upon 

3 an existing program to encourage the develop4 ment of surrogate endpoints and biomarkers 

5 that are reasonably likely to predict clinical 

6 benefit for serious or life-threatening conditions 

7 for which there exist significant unmet patient 

8 needs; 

9 ‘‘(B) request the Institute of Medicine to 

10 undertake a study to identify validated surro11 gate endpoints and biomarkers, and recommend 

12 research to validate surrogate endpoints and 

13 biomarkers, that may support approvals for 

14 products intended for the treatment of serious 

15 or life-threatening conditions or diseases; and 

16 ‘‘(C) make widely available to the public a 

17 list of drugs, biological products, and devices 

18 that are being investigated for serious or life19 threatening conditions or diseases and that 

20 have not yet received approval under section 

21 561(d) or 561A for marketing. 

22 ‘‘(2) STUDY CONTENT.—The study under para23 graph (1)(B) shall include endpoints and biomarkers 

24 that address the unmet medical needs of subpopula25 tions of patients and that facilitate the development 


sroberts on PROD1PC70 with BILLS
25 

•S 3046 IS

1 of individualized treatment approaches for patients 

2 with serious or life-threatening conditions or dis3 eases.’’. 

4 SEC. 5. DEMONSTRATION PROJECT FOR COVERAGE OF 

5 CERTAIN DRUGS, BIOLOGICAL PRODUCTS, 

6 AND DEVICES UNDER THE MEDICARE PRO7 GRAM. 

8 (a) IN GENERAL.—The Secretary of Health and 

9 Human Services (in this section referred to as the ‘‘Sec10 retary’’) shall establish a demonstration project under the 

11 Medicare program under title XVIII of the Social Security 

12 Act (42 U.S.C. 1395 et seq.) under which payment is 

13 made for drugs, biological products, and devices approved 

14 for Compassionate Investigational Access under section 

15 561(d) of the Federal Food, Drug, and Cosmetic Act in 

16 the case where the drug, biological product, or device is 

17 not otherwise covered under the Medicare program or by 

18 any other organization or entity (including a public assist19 ance program or the sponsor of the application for such 

20 drug, biological product, or device). 

21 (b) DURATION.—The demonstration project under 

22 this section shall be conducted for a 5-year period. 

23 (c) FUNDING.— 

24 (1) IN GENERAL.—The Secretary shall provide 

25 for the transfer from the Federal Hospital Insurance 


sroberts on PROD1PC70 with BILLS
26 

•S 3046 IS

1 Trust Fund under section 1817 of the Social Secu2 rity Act (42 U.S.C. 1395i) and the Federal Supple3 mentary Medical Insurance Trust Fund under sec4 tion 1841 of such Act (42 U.S.C. 1395t), in such 

5 proportion as the Secretary determines to be appro6 priate, of such sums as are necessary for the costs 

7 of carrying out the demonstration project under this 

8 section. 

9 (2) BUDGET NEUTRALITY.—In conducting the 

10 demonstration project under this section, the Sec11 retary shall ensure that the aggregate payments 

12 made by the Secretary do not exceed the amount 

13 which the Secretary estimates would have been paid 

14 if the demonstration project under this section was 

15 not implemented. 

16 (d) WAIVER AUTHORITY.—The Secretary may waive 

17 such requirements of title XVIII of the Social Security Act 

18 (42 U.S.C. 1395 et seq.) as may be necessary for the pur19 pose of carrying out the demonstration project under this 

20 section. 

21 (e) REPORT.—Not later than 90 days after the last 

22 day of the 5-year period of the demonstration project 

23 under this section, the Secretary shall submit to Congress 

24 a report describing the rates of utilization by Medicare 

25 beneficiaries of drugs, biological products, and devices apVerDate Aug 31 2005 01:55 May 24, 2008 Jkt 069200 PO 00000 Frm 00026 Fmt 6652 Sfmt 6201 E:\BILLS\S3046.IS S3046

sroberts on PROD1PC70 with BILLS
27 

•S 3046 IS

1 proved for Compassionate Investigational Access and the 

2 total cost of payments made under the Medicare program 

3 resulting from the demonstration project. The report shall 

4 describe recommendations for legislation or administrative 

5 action as the Secretary deems appropriate. 

6 (f) TERMINATION.—The Secretary shall terminate 

7 payments under this section on the day after the last day 

8 of the 5-year period of the demonstration project under 

9 this section. 

10 SEC. 6. USE OF PART B DEFINITION OF MEDICALLY AC11 CEPTED INDICATION FOR PART D DRUGS. 

12 (a) IN GENERAL.—Section 1860D–2(e) of the Social 

13 Security Act (42 U.S.C. 1395w–102(e)) is amended— 

14 (1) in paragraph (1), in the matter following 

15 subparagraph (B), by striking ‘‘(as defined in sec16 tion 1927(k)(6))’’ and inserting ‘‘(as defined in 

17 paragraph (4))’’; and 

18 (2) by adding at the end the following new 

19 paragraph: 

20 ‘‘(4) MEDICALLY ACCEPTED INDICATION DE21 FINED.— 

22 ‘‘(A) IN GENERAL.—Subject to subpara23 graph (B), for purposes of paragraph (1), the 

24 term ‘medically accepted indication’ has the 

25 meaning given that term— 


sroberts on PROD1PC70 with BILLS
28 

•S 3046 IS

1 ‘‘(i) in the case of a covered part D 

2 drug used in an anticancer 

3 chemotherapeutic regimen, in section 

4 1861(t)(2)(B), except that in applying 

5 such section, ‘PDP sponsor or MA–PD 

6 sponsor’ shall be substituted for ‘carrier’ 

7 each place it appears and the compendia 

8 identified in section 1927(g)(1)(B)(i)(III) 

9 shall be deemed to be included in the com10 pendia described in section 

11 1861(t)(2)(B)(ii)(I); and 

12 ‘‘(ii) in the case of any other covered 

13 part D drug, in section 1927(k)(6). 

14 ‘‘(B) CONSIDERATION OF OTHER CRITERIA

15 ON A CASE-BY-CASE BASIS.—Nothing in this 

16 subsection shall preclude a PDP sponsor offer17 ing a prescription drug plan or an MA organi18 zation offering an MA–PD plan from, after a 

19 request by an individual enrolled in the plan for 

20 a coverage determination under section 1860D– 

21 4(g)(1), providing coverage of a covered part D 

22 drug for the individual in the case where the 

23 sponsor or organization determines, based on 

24 guidance provided by the Secretary for deter25 mining whether the use of a covered part D 


sroberts on PROD1PC70 with BILLS
29 

•S 3046 IS

1 drug is for a medically accepted indication and 

2 supportive clinical evidence in peer reviewed 

3 medical literature, that the use of the covered 

4 part D drug is for a medically accepted indica5 tion.’’. 

6 (b) EFFECTIVE DATE.—The amendments made by 

7 this section shall apply to plan years beginning on or after 

8 January 1, 2010. 

9 SEC. 7. MODERNIZATION OF THE FOOD AND DRUG ADMIN10 ISTRATION. 

11 Subchapter E of chapter V of the Federal Food, 

12 Drug, and Cosmetic Act (21 U.S.C. 360bbb et seq.) is 

13 amended by adding at the end the following: 

14 ‘‘SEC. 568. POLICIES RELATED TO STUDY EVALUATION IN15 FORMATION. 

16 ‘‘(a) IN GENERAL.— 

17 ‘‘(1) NONSTATISTICAL MEASURES.—The Sec18 retary shall give consideration to clinical judgment 

19 and risks to the patient from the disease or condi20 tion involved in the evaluation of the safety and ef21 fectiveness of drugs, biological products, and devices 

22 that treat serious or life-threatening diseases or con23 ditions. This policy shall apply— 

24 ‘‘(A) in evaluating clinical study designs 

25 and endpoints; and 


sroberts on PROD1PC70 with BILLS
30 

•S 3046 IS

1 ‘‘(B) in making decisions with respect to 

2 product applications for approval under section 

3 561(d) or 561A. 

4 ‘‘(2) TYPES OF NONSTATISTICAL MEASURES.— 

5 The policy established under paragraph (1), for the 

6 purposes described in such paragraph— 

7 ‘‘(A) shall include such nonstatistical infor8 mation as— 

9 ‘‘(i) clinical evaluation information, 

10 such as case history reports; 

11 ‘‘(ii) scientific and clinical studies de12 signed to measure or define mechanisms of 

13 action or molecular targeting; 

14 ‘‘(iii) data from animal and computer 

15 models; and 

16 ‘‘(iv) comparison with historical data; 

17 and 

18 ‘‘(B) shall incorporate the use of— 

19 ‘‘(i) evaluations of the adverse effect 

20 of delaying the availability of an investiga21 tional drug to even a small subpopulation 

22 of seriously ill patients; and 

23 ‘‘(ii) scientific, observational, or clin24 ical studies designed and conducted to col25 lect well-documented information. 


sroberts on PROD1PC70 with BILLS
31 

•S 3046 IS

1 ‘‘(b) MEETINGS.—A meeting to address any pending 

2 scientific, medical, regulatory, or other issue relating to 

3 the development, investigation, review, or other aspect of 

4 a drug, biological product, or device shall ordinarily be 

5 held not later than 15 days of the receipt of a written 

6 request for the meeting by the sponsor of the product, 

7 which may be extended to 30 days for good cause. Such 

8 meetings shall ordinarily be conducted in person, but may 

9 be conducted by telephone or other form of communication 

10 if both parties agree. In order to reduce the burden of 

11 meetings, only those Food and Drug Administration em12 ployees who are intended to actively participate in the dis13 cussion shall attend a meeting. Minutes of a meeting shall 

14 be promptly prepared and exchanged by both parties im15 mediately following the meeting and shall accurately sum16 marize what occurred at the meeting. 

17 ‘‘(c) RULE OF CONSTRUCTION.—The provisions of 

18 this chapter and section 351 of the Public Health Service 

19 Act shall be construed to incorporate the policy established 

20 in this section.’’. 

21 SEC. 8. MEMBERSHIP OF ONCOLOGY DRUGS ADVISORY 

22 COMMITTEE AND THE CELLULAR, TISSUE, 

23 AND GENE THERAPY ADVISORY COMMITTEE. 

24 Notwithstanding any other provision of law, member25 ship of the Oncology Drugs Advisory Committee, the CelVerDate Aug 31 2005 01:55 May 24, 2008 Jkt 069200 PO 00000 Frm 00031 Fmt 6652 Sfmt 6201 E:\BILLS\S3046.IS S3046

sroberts on PROD1PC70 with BILLS
32 

•S 3046 IS

1 lular, Tissue, and Gene Therapy Advisory Committee of 

2 the Food and Drug Administration, and any other com3 mittee created by such Administration to evaluate or ad4 vise with respect to applications submitted under section 

5 561(d) or 561A of the Federal Food, Drug, and Cosmetic 

6 Act (as added by this Act), shall consist of no less than 

7 2 patient representatives who are voting members of the 

8 committee. 

Æ 


sroberts on PROD1PC70 with BILLS